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101. Final overall survival results of frontline bortezomib plus rituximab, cyclophosphamide, doxorubicin, and prednisone (VR-CAP) vs R-CHOP in transplantation-ineligible patients (pts) with newly diagnosed mantle-cell lymphoma (MCL): A randomized, open-label, phase III (LYM-3002) study

Author

Cavalli, F., primary, Jin, J., additional, Pylypenko, H., additional, Verhoef, G., additional, Siritanaratkul, N., additional, Drach, J., additional, Raderer, M., additional, Mayer, J., additional, Pereira, J., additional, Tumyan, G., additional, Okamoto, R., additional, Nakahara, S., additional, Hu, P., additional, Appiani, C., additional, Nemat, S., additional, and Robak, T., additional

Published
2018
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102. Safety and Efficacy of Acalabrutinib, Bendamustine, and Rituximab in Patients with Treatment‐Naive or Relapsed/Refractory Mantle Cell Lymphoma: Phase Ib Trial.

Author

Phillips, T., Wang, M., Robak, T., Gallinson, D., Stevens, D., Patel, K., Ramadan, S., Wun, C., Jurczak, W., and Smith, S. D.

Subjects
MANTLE cell lymphoma, DISEASE relapse, RITUXIMAB, BRUTON tyrosine kinase
Abstract

Safety and Efficacy of Acalabrutinib, Bendamustine, and Rituximab in Patients with Treatment-Naive or Relapsed/Refractory Mantle Cell Lymphoma: Phase Ib Trial Grade 3-4 AEs occurred in 13 (72.2%) TN pts and 17 (85.0%) R/R pts, most commonly neutropenia ( I n i = 7, 38.9% [TN]; I n i = 10, 50.0% [R/R]). B Background: b Acalabrutinib (A) is a next-generation Bruton tyrosine kinase inhibitor approved for relapsed/refractory (R/R) mantle cell lymphoma (MCL). [Extracted from the article]

Published
2023
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103. Characterization of Mechanisms of Resistance in Previously Treated Chronic Lymphocytic Leukemia (CLL) From a Head‐to‐Head Trial of Acalabrutinib Versus Ibrutinib.

Author

Woyach, J. A., Jones, D., Jurczak, W., Robak, T., Illés, A., Kater, A. P., Ghia, P., Byrd, J. C., Seymour, J. F., Long, S., Mohamed, N., De Jesus, G., Lai, R., de Bruin, G., Butturini, A., Rule, S., and Munugalavadla, V.

Subjects
CHRONIC lymphocytic leukemia, BRUTON tyrosine kinase
Abstract

In ELEVATE-RR (NCT02477696) at a median follow-up of 41 mo, Acala demonstrated noninferior progression-free survival with fewer cardiovascular adverse events versus ibrutinib (Ibr) in patients (pts) with relapsed/refractory (R/R) CLL. Characterization of Mechanisms of Resistance in Previously Treated Chronic Lymphocytic Leukemia (CLL) From a Head-to-Head Trial of Acalabrutinib Versus Ibrutinib B Introduction: b Acalabrutinib (Acala) is a highly selective, next-generation covalent Bruton tyrosine kinase inhibitor (BTKi) approved for CLL. [Extracted from the article]

Published
2023
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104. Analysis of Prognostic Factors in Acute Leukemias in Adults

Author

Krykowski, E., Polkowska-Kulesza, E., Robak, T., Matusewicz, W., Urbańska-Rys, H., Hołub, A., Heimpel, H., editor, Huhn, D., editor, Mueller-Eckhardt, C., editor, Ruhenstroth-Bauer, G., editor, Büchner, T., editor, Schellong, G., editor, Hiddemann, W., editor, Urbanitz, D., editor, and Ritter, J., editor

Published
1987
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105. The european hematology association roadmap for european hematology research: A consensus document

Author

Engert, A. Balduini, C. Brand, A. Coiffier, B. Cordonnier, C. Döhner, H. De Wit, T.D. Eichinger, S. Fibbe, W. Green, T. De Haas, F. Iolascon, A. Jaffredo, T. Rodeghiero, F. Sall Es, G. Schuringa, J.J. André, M. Andre-Schmutz, I. Bacigalupo, A. Bochud, P.-Y. Den Boer, M. Bonini, C. Camaschella, C. Cant, A. Cappellini, M.D. Cazzola, M. Celso, C.L. Dimopoulos, M. Douay, L. Dzierzak, E. Einsele, H. Ferreri, A. De Franceschi, L. Gaulard, P. Gottgens, B. Greinacher, A. Gresele, P. Gribben, J. De Haan, G. Hansen, J.-B. Hochhaus, A. Kadir, R. Kaveri, S. Kouskoff, V. Kühne, T. Kyrle, P. Ljungman, P. Maschmeyer, G. Méndez-Ferrer, S. Milsom, M. Mummery, C. Ossenkoppele, G. Pecci, A. Peyvandi, F. Philipsen, S. Reitsma, P. Ribera, J.M. Risitano, A. Rivella, S. Ruf, W. Schroeder, T. Scully, M. Socie, G. Staal, F. Stanworth, S. Stauder, R. Stilgenbauer, S. Tamary, H. Theilgaard-Mönch, K. Thein, S.L. Tilly, H. Trneny, M. Vainchenker, W. Vannucchi, A.M. Viscoli, C. Vrielink, H. Zaaijer, H. Zanella, A. Zolla, L. Zwaginga, J.J. Martinez, P.A. Van Den Akker, E. Allard, S. Anagnou, N. Andolfo, I. Andrau, J.-C. Angelucci, E. Anstee, D. Aurer, I. Avet-Loiseau, H. Aydinok, Y. Bakchoul, T. Balduini, A. Barcellini, W. Baruch, D. Baruchel, A. Bayry, J. Bento, C. Van Den Berg, A. Bernardi, R. Bianchi, P. Bigas, A. Biondi, A. Bohonek, M. Bonnet, D. Borchmann, P. Borregaard, N. Brækkan, S. Van Den Brink, M. Brodin, E. Bullinger, L. Buske, C. Butzeck, B. Cammenga, J. Campo, E. Carbone, A. Cervantes, F. Cesaro, S. Charbord, P. Claas, F. Cohen, H. Conard, J. Coppo, P. Vives Corron, J.-L. Da Costa, L. Davi, F. Delwel, R. Dianzani, I. Domanović, D. Donnelly, P. Drnovšek, T.D. Dreyling, M. Du, M.-Q. Dufour, C. Durand, C. Efremov, D. Eleftheriou, A. Elion, J. Emonts, M. Engelhardt, M. Ezine, S. Falkenburg, F. Favier, R. Federico, M. Fenaux, P. Fitzgibbon, J. Flygare, J. Foà, R. Forrester, L. Galacteros, F. Garagiola, I. Gardiner, C. Garraud, O. Van Geet, C. Geiger, H. Geissler, J. Germing, U. Ghevaert, C. Girelli, D. Godeau, B. Gökbuget, N. Goldschmidt, H. Goodeve, A. Graf, T. Graziadei, G. Griesshammer, M. Gruel, Y. Guilhot, F. Von Gunten, S. Gyssens, I. Halter, J. Harrison, C. Harteveld, C. Hellström-Lindberg, E. Hermine, O. Higgs, D. Hillmen, P. Hirsch, H. Hoskin, P. Huls, G. Inati, A. Johnson, P. Kattamis, A. Kiefel, V. Kleanthous, M. Klump, H. Krause, D. Hovinga, J.K. Lacaud, G. Lacroix-Desmazes, S. Landman-Parker, J. Legouill, S. Lenz, G. Von Lilienfeld-Toal, M. Von Lindern, M. Lopez-Guillermo, A. Lopriore, E. Lozano, M. Macintyre, E. Makris, M. Mannhalter, C. Martens, J. Mathas, S. Matzdorff, A. Medvinsky, A. Menendez, P. Migliaccio, A.R. Miharada, K. Mikulska, M. Minard, V. Montalbán, C. De Montalembert, M. Montserrat, E. Morange, P.-E. Mountford, J. Muckenthaler, M. Müller-Tidow, C. Mumford, A. Nadel, B. Navarro, J.-T. El Nemer, W. Noizat-Pirenne, F. O’Mahony, B. Oldenburg, J. Olsson, M. Oostendorp, R. Palumbo, A. Passamonti, F. Patient, R. De Latour, R.P. Pflumio, F. Pierelli, L. Piga, A. Pollard, D. Raaijmakers, M. Radford, J. Rambach, R. Koneti Rao, A. Raslova, H. Rebulla, P. Rees, D. Ribrag, V. Rijneveld, A. Rinalducci, S. Robak, T. Roberts, I. Rodrigues, C. Rosendaal, F. Rosenwald, A. Rule, S. Russo, R. Saglio, G. Sanchez, M. Scharf, R.E. Schlenke, P. Semple, J. Sierra, J. So-Osman, C. Soria, J.M. Stamatopoulos, K. Stegmayr, B. Stunnenberg, H. Swinkels, D. Barata, J.P.T. Taghon, T. Taher, A. Terpos, E. Thachil, J. Tissot, J.D. Touw, I. Toye, A. Trappe, R. Traverse-Glehen, A. Unal, S. Vaulont, S. Viprakasit, V. Vitolo, U. Van Wijk, R. Wójtowicz, A. Zeerleder, S. Zieger, B. EHA Roadmap for European Hematology Research

Abstract

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at ∈ European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better fu treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine ‘sections’ in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients. © 2016 Ferrata Storti Foundation.

Published
2016

106. Randomized phase 3 study of lenalidomide versus chlorambucil as first-line therapy for older patients with chronic lymphocytic leukemia (the ORIGIN trial)

Author

Chanan-Khan, A, primary, Egyed, M, additional, Robak, T, additional, Martinelli de Oliveira, F A, additional, Echeveste, M A, additional, Dolan, S, additional, Desjardins, P, additional, Blonski, J Z, additional, Mei, J, additional, Golany, N, additional, Zhang, J, additional, and Gribben, J G, additional

Published
2017
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107. Chlorambucil plus ofatumumab versus chlorambucil alone in previously untreated patients with chronic lymphocytic leukaemia (COMPLEMENT 1): A randomised, multicentre, open-label phase 3 trial

Author

Hillmen, P. Robak, T. Janssens, A. Babu, K.G. Kloczko, J. Grosicki, S. Doubek, M. Panagiotidis, P. Kimby, E. Schuh, A. Pettitt, A.R. Boyd, T. Montillo, M. Gupta, I.V. Wright, O. Dixon, I. Carey, J.L. Chang, C.-N. Lisby, S. McKeown, A. Offner, F.

Subjects
hemic and lymphatic diseases
Abstract

Background Treatment for patients with chronic lymphocytic leukaemia who are elderly or who have comorbidities is challenging because fludarabine-based chemoimmunotherapies are mostly not suitable. Chlorambucil remains the standard of care in many countries. We aimed to investigate whether the addition of ofatumumab to chlorambucil could lead to better clinical outcomes than does treatment with chlorambucil alone, while also being tolerable for patients who have few treatment options. Methods We carried out a randomised, open-label, phase 3 trial for treatment-naive patients with chronic lymphocytic leukaemia in 109 centres in 16 countries. We included patients who had active disease needing treatment, but in whom fludarabine-based treatment was not possible. We randomly assigned patients (1:1) to receive oral chlorambucil (10 mg/m2) on days 1-7 of a 28 day treatment course or to receive chlorambucil by this schedule plus intravenous ofatumumab (cycle 1: 300 mg on day 1 and 1000 mg on day 8; subsequent cycles: 1000 mg on day 1) for three to 12 cycles. Assignment was done with a randomisation list that was computer generated at GlaxoSmithKline, and was stratified, in a block size of two, by age, disease stage, and performance status. The primary endpoint was progression-free survival in the intention-to-treat population and assessment was done by an independent review committee that was masked to group assignment. The study is registered with ClinicalTrials.gov, number NCT00748189. Findings We enrolled 447 patients, median age 69 years (range 35-92). Between Dec 22, 2008, and May 26, 2011, we randomly assigned 221 patients to chlorambucil plus ofatumumab and 226 patients to chlorambucil alone. Median progression-free survival was 22·4 months (95% CI 19·0-25·2) in the group assigned to chlorambucil plus ofatumumab compared with 13·1 months (10·6-13·8) in the group assigned to chlorambucil only (hazard ratio 0·57, 95% CI 0·45-0·72; p

Published
2015

109. A phase 2, randomized, double-blind, placebo-controlled study of siltuximab (anti-IL-6 mAb) and bortezomib versus bortezomib alone in patients with relapsed or refractory multiple myeloma

Author

Orlowski, R.Z. Gercheva, L. Williams, C. Sutherland, H. Robak, T. Masszi, T. Goranova-Marinova, V. Dimopoulos, M.A. Cavenagh, J.D. Špička, I. Maiolino, A. Suvorov, A. Bladé, J. Samoylova, O. Puchalski, T.A. Reddy, M. Bandekar, R. van de Velde, H. Xie, H. Rossi, J.-F.

Abstract

We compared the safety and efficacy of siltuximab (S), an anti-interleukin-6 chimeric monoclonal antibody, plus bortezomib (B) with placebo (plc) + B in patients with relapsed/refractory multiple myeloma in a randomized phase 2 study. Siltuximab was given by 6 mg/kg IV every 2 weeks. On progression, B was discontinued and high-dose dexamethasone could be added to S/plc. Response and progression-free survival (PFS) were analyzed pre-dexamethasone by European Group for Blood and Marrow Transplantation (EBMT) criteria. For the 281 randomized patients, median PFS for S + B and plc + B was 8.0 and 7.6 months (HR 0.869, P = 0.345), overall response rate was 55 versus 47% (P = 0.213), complete response rate was 11 versus 7%, and median overall survival (OS) was 30.8 versus 36.8 months (HR 1.353, P = 0.103). Sustained suppression of C-reactive protein, a marker reflective of inhibition of interleukin-6 activity, was seen with S + B. Siltuximab did not affect B pharmacokinetics. Siltuximab/placebo discontinuation (75 versus 66%), grade ≥3 neutropenia (49 versus 29%), thrombocytopenia (48 versus 34%), and all-grade infections (62 versus 49%) occurred more frequently with S + B. The addition of siltuximab to bortezomib did not appear to improve PFS or OS despite a numerical increase in response rate in patients with relapsed or refractory multiple myeloma. © 2014 Wiley Periodicals, Inc.

Published
2015

111. Systematic review of purine analog treatment for chronic lymphocytic leukemia: lessons for future trials

Author

Bauduer, M., Gribben, J., Herrmann, R., Thiel, E., Rai, K., Larson, R., Ferrara, F., Barnard, J., Pearce, H., Taylor, C., Brillant, B., Steurer, M., Weingart, O., Flinn, W., Funkhouser, A., Nallman, M., Sun, Z., Jakšić, Branimir, Suciou, S., Chevret, S., Dighiero, G., Leporrier, M., Frankel, S.R., Sirard, C., Hillmen, P., Trehu, B., Felder, M., Busch, R., Eichorst, B., Mallek, M., Stilgenbauer, S., Pangalis, G., Bezares, R., van Oers, M.H.J., van Putten, W., Gobbi, M., Spriano, M., Mabed, M., Catovsky, D., Richards, S., Wade, R., Abdelhamid, T., Dearden, C., Knauf, W., Blonski, J., Jamroziak, K., Robak, T., Mauro, F., Hiddeman, W., Johnson, S.A., Longthorne, G., Rummel, M.J., Juliusson, G., Pulluqui, P., Zinzani, P.L., Pozzato, G., Reynolds, C, Furman, R.R., Durrant, J., Elphinstone, P., Evans, V., Gettins, .L, Hicks, C., James, S., Clarke, M., MacKinnon, L., McHugh, T.M., Morris, P., Read, S., Gregory, C., Pozzato, Gabriele, Bauduer M, Gribben J, Herrmann R, Thiel E, Rai K, Larson R, Ferrara F, Barnard J, Pearce H, Taylor C, Brillant C, Steurer M, Weingart O, Flinn IW, Funkhouser A, Tallman M, Sun Z, Jaksic B, Suciu S, Chevret S, Dighiero G, Leporrier M, Frankel SR, Sirard C, Hillmen P, Trehu B, Felder M, Busch R, Eichhorst B, Hallek M, Stilgenbauer S, Pangalis G, Bezares R, van Oers MH, van Putten W, Gobbi M, Spriano M, Mabed M, Catovsky D, Richards S, Wade R, Abdelhamid T, Dearden C, Knauf W, Blonski J, Jamroziak K, Robak T, Mauro F, Hiddeman W, Johnson SA, Longthorne G, Juliusson G, Pulluqi P, Zinzani PL, Pozzato G, Oncology US, Reynolds C, Furman RR, Durrant J, Elphinstone P, Evans V, Gettins L, Hicks C, James S, Clarke M, MacKinnon L, McHugh TM, Morris P, Read S, and Gregory C. CLL Trialists’ Collaborative Group

Subjects
Oncology, medicine.medical_specialty, review, Purine analogue, chronic lymphocytic leukemia, fludarabine, clinical trial, Pharmacology, Disease-Free Survival, combination therapy, purine analog, Internal medicine, Antineoplastic Combined Chemotherapy Protocols, Medicine, Humans, Progression-free survival, Cladribine, Antineoplastic Agents, Alkylating, Chlorambucil, business.industry, Hematology, Odds ratio, Leukemia, Lymphocytic, Chronic, B-Cell, Fludarabine, Clinical trial, Treatment Outcome, CLL, cyclophosphamide, Purines, Original Articles and Brief Reports, business, Untreated Chronic Lymphocytic Leukemia, medicine.drug
Abstract

Background. A systematic review of purine analogs revealed heterogeneity between trials in treatment effects on response and progression free survival, but not survival, perhaps partly due to variations in analysis methods. In addition, combination treatments required evaluation. Design and Methods. Individual patient data were sought for all randomized trials in untreated chronic lymphocytic leukemia which involved a purine analog, but not including antibody therapies. Results. Sixteen trials were found, addressing seven comparisons. Eight trials, with 2753 patients, showed that single agent purine analog improved progression free survival (Odds ratio = 0.71; 95% confidence interval =0.63-0.79). Heterogeneity remained substantial. Three trials, with 1403 patients, showed that progression free survival was further improved by the addition of cyclophosphamide (Odds ratio = 0.54; 0.47-0.62). Fewer data were available on the addition of other drugs to purine analog, and none showed clear benefit. Two trials, with 544 patients, suggested cladribine improved progression free survival compared to fludarabine (Odds ratio = 0.77; 0.63-0.95). No differences were seen in overall survival for any comparisons. Conclusions. Purine analogs, particularly combined with cyclophosphamide, significantly improve progression free survival but not survival. Some groups, such as the elderly, may not see the same benefits and maximising doses may be important for all treatments, including chlorambucil. Longer follow-up, consistent definitions and detailed reporting of trials should be encouraged.

Published
2012

112. Efficacy and safety of prophylaxis with once-weekly BAY 79-4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients: A randomised, active-controlled, double-blind study

Author

Powell, Jerry, Martinowitz, Uri, Windyga, Jerzy, Hellmann, Andrzej, Pabinger, Ingrid, Enriquez, Monika Maas, Schwartz, Lawrence, Ingerslev, Jørgen, Group Author: Pabinger Fasching I, Hermans C, Poon MC, Ritchie B, Tinmouth A, Teitel J, Zupancic Salek S, Ingerslev J, Chambos H, Schved JF, Guillet B, Oldenburg J, Klamroth R, Martinowitz U, Santagostino E, Schinco P, Musso R, Morfini M, Rocin A, Meijer K, Larosvan B, Holme P, Windyga J, Skotnicki A, Hellmann A, Zawilska K, Robak T, Soto I, Haya S, Kavakli K, Antmen B, Yesilipek MA, Hay C, Wilde J, Rangarajan S, Bernstein J, Damon L, Gill J, Gruppo R, Cuker A, Kuriakose P, Lin J, Manco Johnson M, Mathew P, Neufeld E, Soni A, Powell J, Kulkarni R, Wicklund B., DI MINNO, GIOVANNI, DI MINNO, MATTEO, Powell, Jerry, Martinowitz, Uri, Windyga, Jerzy, DI MINNO, Giovanni, Hellmann, Andrzej, Pabinger, Ingrid, Enriquez, Monika Maa, Schwartz, Lawrence, Ingerslev, Jørgen, Group Author: Pabinger Fasching, I, Hermans, C, Poon, Mc, Ritchie, B, Tinmouth, A, Teitel, J, Zupancic Salek, S, Ingerslev, J, Chambos, H, Schved, Jf, Guillet, B, Oldenburg, J, Klamroth, R, Martinowitz, U, DI MINNO, Matteo, Santagostino, E, Schinco, P, Musso, R, Morfini, M, Rocin, A, Meijer, K, Larosvan, B, Holme, P, Windyga, J, Skotnicki, A, Hellmann, A, Zawilska, K, Robak, T, Soto, I, Haya, S, Kavakli, K, Antmen, B, Yesilipek, Ma, Hay, C, Wilde, J, Rangarajan, S, Bernstein, J, Damon, L, Gill, J, Gruppo, R, Cuker, A, Kuriakose, P, Lin, J, Manco Johnson, M, Mathew, P, Neufeld, E, Soni, A, Powell, J, Kulkarni, R, and Wicklund, B.

Subjects
0301 basic medicine, Male, Sucrose, rFVIII-FS, Chemistry, Pharmaceutical, 030204 cardiovascular system & hematology, Polyethylene Glycol, Polyethylene Glycols, law.invention, 0302 clinical medicine, Randomized controlled trial, law, Delayed-Action Preparation, Clinical endpoint, Medicine, Prophylaxi, Hematology, Middle Aged, Liposome, PEGylated liposome, Treatment Outcome, Tolerability, Solvent, Human, Adult, medicine.medical_specialty, Haemophilia, Adolescent, Haemophilia A, Hemorrhage, Hemophilia A, Drug Administration Schedule, 03 medical and health sciences, Young Adult, Double-Blind Method, Internal medicine, Humans, Dosing, Factor VIII, business.industry, medicine.disease, Surgery, Regimen, 030104 developmental biology, Delayed-Action Preparations, Liposomes, Solvents, business, Bay
Abstract

SummaryThe benefits of prophylaxis of haemophilia A patients regarding joint health and quality-of-life are well established. However, adherence to an up to every-other-day infusion regimen is a barrier to widespread adoption of prophylaxis. BAY 79–4980 is an investigational drug consisting of rFVIII-FS (sucrose-formulated recombinant FVIII) reconstituted with liposome solvent. Previous clinical studies showed extended protection from bleeding after a single injection of BAY 79–4980 (13.3 ± 6.2 days) compared with rFVIII-FS (7.2 ± 1.7 days). The effect of once-a-week prophylaxis with BAY 79–4980 (35 IU/kg) compared with three times-per-week rFVIII-FS (25 IU/kg) in previously treated, severe haemophilia A patients was evaluated in a 52-week, double-blind, two-arm, randomised, controlled study. The primary and secondary endpoints were protection from total bleeds and joint bleeds, respectively. Short- and long-term safety and tolerability of BAY 79–4980 including effects on lipid levels were assessed. A total of 139 and 131 subjects were evaluable for safety and efficacy analyses, respectively. A large difference in efficacy between treatment groups was observed with 72.1% (49/68) in the rFVIII-FS control group demonstrating

Published
2012

113. The European Hematology Association Roadmap for European Hematology Research: a consensus document.

Author

EHA Roadmap for European Hematology, Research, Engert, A., Balduini, C., Brand, A., Coiffier, B., Cordonnier, C., Döhner, H., de Wit TD., Eichinger, S., Fibbe, W., Green, T., de Haas, F., Iolascon, A., Jaffredo, T., Rodeghiero, F., Salles, G., Schuringa, JJ., André, M., Andre-Schmutz, I., Bacigalupo, A., Bochud, PY., Boer, Md., Bonini, C., Camaschella, C., Cant, A., Cappellini, MD., Cazzola, M., Celso, CL., Dimopoulos, M., Douay, L., Dzierzak, E., Einsele, H., Ferreri, A., De Franceschi, L., Gaulard, P., Gottgens, B., Greinacher, A., Gresele, P., Gribben, J., de Haan, G., Hansen, JB., Hochhaus, A., Kadir, R., Kaveri, S., Kouskoff, V., Kühne, T., Kyrle, P., Ljungman, P., Maschmeyer, G., Méndez-Ferrer£££Simón£££ S., Milsom, M., Mummery, C., Ossenkoppele, G., Pecci, A., Peyvandi, F., Philipsen, S., Reitsma, P., Ribera, JM., Risitano, A., Rivella, S., Ruf, W., Schroeder, T., Scully, M., Socie, G., Staal, F., Stanworth, S., Stauder, R., Stilgenbauer, S., Tamary, H., Theilgaard-Mönch, K., Thein, SL., Tilly, H., Trneny, M., Vainchenker, W., Vannucchi, AM., Viscoli, C., Vrielink, H., Zaaijer, H., Zanella, A., Zolla, L., Zwaginga, JJ., Martinez, PA., van den Akker, E., Allard, S., Anagnou, N., Andolfo, I., Andrau, JC., Angelucci, E., Anstee, D., Aurer, I., Avet-Loiseau, H., Aydinok, Y., Bakchoul, T., Balduini, A., Barcellini, W., Baruch, D., Baruchel, A., Bayry, J., Bento, C., van den Berg, A., Bernardi, R., Bianchi, P., Bigas, A., Biondi, A., Bohonek, M., Bonnet, D., Borchmann, P., Borregaard, N., Brækkan, S., van den Brink, M., Brodin, E., Bullinger, L., Buske, C., Butzeck, B., Cammenga, J., Campo, E., Carbone, A., Cervantes, F., Cesaro, S., Charbord, P., Claas, F., Cohen, H., Conard, J., Coppo, P., Corrons, JL., Costa, Ld., Davi, F., Delwel, R., Dianzani, I., Domanović, D., Donnelly, P., Drnov?ek£££Tadeja Dovč£££ TD., Dreyling, M., Du, MQ., Dufour, C., Durand, C., Efremov, D., Eleftheriou, A., Elion, J., Emonts, M., Engelhardt, M., Ezine, S., Falkenburg, F., Favier, R., Federico, M., Fenaux, P., Fitzgibbon, J., Flygare, J., Foà, R., Forrester, L., Galacteros, F., Garagiola, I., Gardiner, C., Garraud, O., van Geet, C., Geiger, H., Geissler, J., Germing, U., Ghevaert, C., Girelli, D., Godeau, B., Gökbuget, N., Goldschmidt, H., Goodeve, A., Graf, T., Graziadei, G., Griesshammer, M., Gruel, Y., Guilhot, F., von Gunten, S., Gyssens, I., Halter, J., Harrison, C., Harteveld, C., Hellström-Lindberg, E., Hermine, O., Higgs, D., Hillmen, P., Hirsch, H., Hoskin, P., Huls, G., Inati, A., Johnson, P., Kattamis, A., Kiefel, V., Kleanthous, M., Klump, H., Krause, D., Hovinga, JK., Lacaud, G., Lacroix-Desmazes, S., Landman-Parker, J., LeGouill, S., Lenz, G., von Lilienfeld-Toal, M., von Lindern, M., Lopez-Guillermo, A., Lopriore, E., Lozano, M., MacIntyre, E., Makris, M., Mannhalter, C., Martens, J., Mathas, S., Matzdorff, A., Medvinsky, A., Menendez, P., Migliaccio, AR., Miharada, K., Mikulska, M., Minard, V., Montalbán, C., de Montalembert, M., Montserrat, E., Morange, PE., Mountford, J., Muckenthaler, M., Müller-Tidow, C., Mumford, A., Nadel, B., Navarro, JT., Nemer, We., Noizat-Pirenne, F., O'Mahony, B., Oldenburg, J., Olsson, M., Oostendorp, R., Palumbo, A., Passamonti, F., Patient, R., Peffault, R., Pflumio, F., Pierelli, L., Piga, A., Pollard, D., Raaijmakers, M., Radford, J., Rambach, R., Rao, AK., Raslova, H., Rebulla, P., Rees, D., Ribrag, V., Rijneveld, A., Rinalducci, S., Robak, T., Roberts, I., Rodrigues, C., Rosendaal, F., Rosenwald, A., Rule, S., Russo, R., Saglio, G., Sanchez, M., Scharf, RE., Schlenke, P., Semple, J., Sierra, J., So-Osman, C., Soria, JM., Stamatopoulos, K., Stegmayr, B., Stunnenberg, H., Swinkels, D., Barata£££João Pedro Taborda£££ JP., Taghon, T., Taher, A., Terpos, E., Thachil, J., Tissot, JD., Touw, I., Toye, A., Trappe, R., Traverse-Glehen, A., Unal, S., Vaulont, S., Viprakasit, V., Vitolo, U., van Wijk, R., Wójtowicz, A., Zeerleder, S., Zieger, B., de Wit, T.D., Schuringa, J.J., EHA Roadmap for European Hematology, Research, Engert, A., Balduini, C., Brand, A., Coiffier, B., Cordonnier, C., Döhner, H., de Wit TD., Eichinger, S., Fibbe, W., Green, T., de Haas, F., Iolascon, A., Jaffredo, T., Rodeghiero, F., Salles, G., Schuringa, JJ., André, M., Andre-Schmutz, I., Bacigalupo, A., Bochud, PY., Boer, Md., Bonini, C., Camaschella, C., Cant, A., Cappellini, MD., Cazzola, M., Celso, CL., Dimopoulos, M., Douay, L., Dzierzak, E., Einsele, H., Ferreri, A., De Franceschi, L., Gaulard, P., Gottgens, B., Greinacher, A., Gresele, P., Gribben, J., de Haan, G., Hansen, JB., Hochhaus, A., Kadir, R., Kaveri, S., Kouskoff, V., Kühne, T., Kyrle, P., Ljungman, P., Maschmeyer, G., Méndez-Ferrer£££Simón£££ S., Milsom, M., Mummery, C., Ossenkoppele, G., Pecci, A., Peyvandi, F., Philipsen, S., Reitsma, P., Ribera, JM., Risitano, A., Rivella, S., Ruf, W., Schroeder, T., Scully, M., Socie, G., Staal, F., Stanworth, S., Stauder, R., Stilgenbauer, S., Tamary, H., Theilgaard-Mönch, K., Thein, SL., Tilly, H., Trneny, M., Vainchenker, W., Vannucchi, AM., Viscoli, C., Vrielink, H., Zaaijer, H., Zanella, A., Zolla, L., Zwaginga, JJ., Martinez, PA., van den Akker, E., Allard, S., Anagnou, N., Andolfo, I., Andrau, JC., Angelucci, E., Anstee, D., Aurer, I., Avet-Loiseau, H., Aydinok, Y., Bakchoul, T., Balduini, A., Barcellini, W., Baruch, D., Baruchel, A., Bayry, J., Bento, C., van den Berg, A., Bernardi, R., Bianchi, P., Bigas, A., Biondi, A., Bohonek, M., Bonnet, D., Borchmann, P., Borregaard, N., Brækkan, S., van den Brink, M., Brodin, E., Bullinger, L., Buske, C., Butzeck, B., Cammenga, J., Campo, E., Carbone, A., Cervantes, F., Cesaro, S., Charbord, P., Claas, F., Cohen, H., Conard, J., Coppo, P., Corrons, JL., Costa, Ld., Davi, F., Delwel, R., Dianzani, I., Domanović, D., Donnelly, P., Drnov?ek£££Tadeja Dovč£££ TD., Dreyling, M., Du, MQ., Dufour, C., Durand, C., Efremov, D., Eleftheriou, A., Elion, J., Emonts, M., Engelhardt, M., Ezine, S., Falkenburg, F., Favier, R., Federico, M., Fenaux, P., Fitzgibbon, J., Flygare, J., Foà, R., Forrester, L., Galacteros, F., Garagiola, I., Gardiner, C., Garraud, O., van Geet, C., Geiger, H., Geissler, J., Germing, U., Ghevaert, C., Girelli, D., Godeau, B., Gökbuget, N., Goldschmidt, H., Goodeve, A., Graf, T., Graziadei, G., Griesshammer, M., Gruel, Y., Guilhot, F., von Gunten, S., Gyssens, I., Halter, J., Harrison, C., Harteveld, C., Hellström-Lindberg, E., Hermine, O., Higgs, D., Hillmen, P., Hirsch, H., Hoskin, P., Huls, G., Inati, A., Johnson, P., Kattamis, A., Kiefel, V., Kleanthous, M., Klump, H., Krause, D., Hovinga, JK., Lacaud, G., Lacroix-Desmazes, S., Landman-Parker, J., LeGouill, S., Lenz, G., von Lilienfeld-Toal, M., von Lindern, M., Lopez-Guillermo, A., Lopriore, E., Lozano, M., MacIntyre, E., Makris, M., Mannhalter, C., Martens, J., Mathas, S., Matzdorff, A., Medvinsky, A., Menendez, P., Migliaccio, AR., Miharada, K., Mikulska, M., Minard, V., Montalbán, C., de Montalembert, M., Montserrat, E., Morange, PE., Mountford, J., Muckenthaler, M., Müller-Tidow, C., Mumford, A., Nadel, B., Navarro, JT., Nemer, We., Noizat-Pirenne, F., O'Mahony, B., Oldenburg, J., Olsson, M., Oostendorp, R., Palumbo, A., Passamonti, F., Patient, R., Peffault, R., Pflumio, F., Pierelli, L., Piga, A., Pollard, D., Raaijmakers, M., Radford, J., Rambach, R., Rao, AK., Raslova, H., Rebulla, P., Rees, D., Ribrag, V., Rijneveld, A., Rinalducci, S., Robak, T., Roberts, I., Rodrigues, C., Rosendaal, F., Rosenwald, A., Rule, S., Russo, R., Saglio, G., Sanchez, M., Scharf, RE., Schlenke, P., Semple, J., Sierra, J., So-Osman, C., Soria, JM., Stamatopoulos, K., Stegmayr, B., Stunnenberg, H., Swinkels, D., Barata£££João Pedro Taborda£££ JP., Taghon, T., Taher, A., Terpos, E., Thachil, J., Tissot, JD., Touw, I., Toye, A., Trappe, R., Traverse-Glehen, A., Unal, S., Vaulont, S., Viprakasit, V., Vitolo, U., van Wijk, R., Wójtowicz, A., Zeerleder, S., Zieger, B., de Wit, T.D., and Schuringa, J.J.

Abstract

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at euro23 billion per year, a level of cost that is not matched in current European hematology research funding. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diagnostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap.The EHA Roadmap identifies nine 'sections' in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders.The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

Published
2016

114. The european hematology association roadmap for european hematology research: A consensus document

Author

Engert, A, Balduini, C, Brand, A, Coiffier, B, Cordonnier, C, Döhner, H, De Wit, T, Eichinger, S, Fibbe, W, Green, T, De Haas, F, Iolascon, A, Jaffredo, T, Rodeghiero, F, Sall Es, G, Schuringa, J, André, M, Andre Schmutz, I, Bacigalupo, A, Bochud, P, Den Boer, M, Bonini, C, Camaschella, C, Cant, A, Cappellini, M, Cazzola, M, Celso, C, Dimopoulos, M, Douay, L, Dzierzak, E, Einsele, H, Ferreri, A, De Franceschi, L, Gaulard, P, Gottgens, B, Greinacher, A, Gresele, P, Gribben, J, De Haan, G, Hansen, J, Hochhaus, A, Kadir, R, Kaveri, S, Kouskoff, V, Kühne, T, Kyrle, P, Ljungman, P, Maschmeyer, G, Méndez Ferrer, S, Milsom, M, Mummery, C, Ossenkoppele, G, Pecci, A, Peyvandi, F, Philipsen, S, Reitsma, P, Ribera, J, Risitano, A, Rivella, S, Ruf, W, Schroeder, T, Scully, M, Socie, G, Staal, F, Stanworth, S, Stauder, R, Stilgenbauer, S, Tamary, H, Theilgaard Mönch, K, Thein, S, Tilly, H, Trneny, M, Vainchenker, W, Vannucchi, A, Viscoli, C, Vrielink, H, Zaaijer, H, Zanella, A, Zolla, L, Zwaginga, J, Martinez, P, Van Den Akker, E, Allard, S, Anagnou, N, Andolfo, I, Andrau, J, Angelucci, E, Anstee, D, Aurer, I, Avet Loiseau, H, Aydinok, Y, Bakchoul, T, Balduini, A, Barcellini, W, Baruch, D, Baruchel, A, Bayry, J, Bento, C, Van Den Berg, A, Bernardi, R, Bianchi, P, Bigas, A, Biondi, A, Bohonek, M, Bonnet, D, Borchmann, P, Borregaard, N, Brækkan, S, Van Den Brink, M, Brodin, E, Bullinger, L, Buske, C, Butzeck, B, Cammenga, J, Campo, E, Carbone, A, Cervantes, F, Cesaro, S, Charbord, P, Claas, F, Cohen, H, Conard, J, Coppo, P, Vives Corron, J, Da Costa, L, Davi, F, Delwel, R, Dianzani, I, Domanović, D, Donnelly, P, Drnovšek, T, Dreyling, M, Du, M, Dufour, C, Durand, C, Efremov, D, Eleftheriou, A, Elion, J, Emonts, M, Engelhardt, M, Ezine, S, Falkenburg, F, Favier, R, Federico, M, Fenaux, P, Fitzgibbon, J, Flygare, J, Foà, R, Forrester, L, Galacteros, F, Garagiola, I, Gardiner, C, Garraud, O, Van Geet, C, Geiger, H, Geissler, J, Germing, U, Ghevaert, C, Girelli, D, Godeau, B, Gökbuget, N, Goldschmidt, H, Goodeve, A, Graf, T, Graziadei, G, Griesshammer, M, Gruel, Y, Guilhot, F, Von Gunten, S, Gyssens, I, Halter, J, Harrison, C, Harteveld, C, Hellström Lindberg, E, Hermine, O, Higgs, D, Hillmen, P, Hirsch, H, Hoskin, P, Huls, G, Inati, A, Johnson, P, Kattamis, A, Kiefel, V, Kleanthous, M, Klump, H, Krause, D, Hovinga, J, Lacaud, G, Lacroix Desmazes, S, Landman Parker, J, Legouill, S, Lenz, G, Von Lilienfeld Toal, M, Von Lindern, M, Lopez Guillermo, A, Lopriore, E, Lozano, M, Macintyre, E, Makris, M, Mannhalter, C, Martens, J, Mathas, S, Matzdorff, A, Medvinsky, A, Menendez, P, Migliaccio, A, Miharada, K, Mikulska, M, Minard, V, Montalbán, C, De Montalembert, M, Montserrat, E, Morange, P, Mountford, J, Muckenthaler, M, Müller Tidow, C, Mumford, A, Nadel, B, Navarro, J, El Nemer, W, Noizat Pirenne, F, O’Mahony, B, Oldenburg, J, Olsson, M, Oostendorp, R, Palumbo, A, Passamonti, F, Patient, R, De Latour, R, Pflumio, F, Pierelli, L, Piga, A, Pollard, D, Raaijmakers, M, Radford, J, Rambach, R, Koneti Rao, A, Raslova, H, Rebulla, P, Rees, D, Ribrag, V, Rijneveld, A, Rinalducci, S, Robak, T, Roberts, I, Rodrigues, C, Rosendaal, F, Rosenwald, A, Rule, S, Russo, R, Saglio, G, Sanchez, M, Scharf, R, Schlenke, P, Semple, J, Sierra, J, So Osman, C, Soria, J, Stamatopoulos, K, Stegmayr, B, Stunnenberg, H, Swinkels, D, Barata, J, Taghon, T, Taher, A, Terpos, E, Thachil, J, Tissot, J, Touw, I, Toye, A, Trappe, R, Traverse Glehen, A, Unal, S, Vaulont, S, Viprakasit, V, Vitolo, U, Van Wijk, R, Wójtowicz, A, Zeerleder, S, Zieger, B, Zieger, B., ZANELLA, ALBERTO, BIONDI, ANDREA, Engert, A, Balduini, C, Brand, A, Coiffier, B, Cordonnier, C, Döhner, H, De Wit, T, Eichinger, S, Fibbe, W, Green, T, De Haas, F, Iolascon, A, Jaffredo, T, Rodeghiero, F, Sall Es, G, Schuringa, J, André, M, Andre Schmutz, I, Bacigalupo, A, Bochud, P, Den Boer, M, Bonini, C, Camaschella, C, Cant, A, Cappellini, M, Cazzola, M, Celso, C, Dimopoulos, M, Douay, L, Dzierzak, E, Einsele, H, Ferreri, A, De Franceschi, L, Gaulard, P, Gottgens, B, Greinacher, A, Gresele, P, Gribben, J, De Haan, G, Hansen, J, Hochhaus, A, Kadir, R, Kaveri, S, Kouskoff, V, Kühne, T, Kyrle, P, Ljungman, P, Maschmeyer, G, Méndez Ferrer, S, Milsom, M, Mummery, C, Ossenkoppele, G, Pecci, A, Peyvandi, F, Philipsen, S, Reitsma, P, Ribera, J, Risitano, A, Rivella, S, Ruf, W, Schroeder, T, Scully, M, Socie, G, Staal, F, Stanworth, S, Stauder, R, Stilgenbauer, S, Tamary, H, Theilgaard Mönch, K, Thein, S, Tilly, H, Trneny, M, Vainchenker, W, Vannucchi, A, Viscoli, C, Vrielink, H, Zaaijer, H, Zanella, A, Zolla, L, Zwaginga, J, Martinez, P, Van Den Akker, E, Allard, S, Anagnou, N, Andolfo, I, Andrau, J, Angelucci, E, Anstee, D, Aurer, I, Avet Loiseau, H, Aydinok, Y, Bakchoul, T, Balduini, A, Barcellini, W, Baruch, D, Baruchel, A, Bayry, J, Bento, C, Van Den Berg, A, Bernardi, R, Bianchi, P, Bigas, A, Biondi, A, Bohonek, M, Bonnet, D, Borchmann, P, Borregaard, N, Brækkan, S, Van Den Brink, M, Brodin, E, Bullinger, L, Buske, C, Butzeck, B, Cammenga, J, Campo, E, Carbone, A, Cervantes, F, Cesaro, S, Charbord, P, Claas, F, Cohen, H, Conard, J, Coppo, P, Vives Corron, J, Da Costa, L, Davi, F, Delwel, R, Dianzani, I, Domanović, D, Donnelly, P, Drnovšek, T, Dreyling, M, Du, M, Dufour, C, Durand, C, Efremov, D, Eleftheriou, A, Elion, J, Emonts, M, Engelhardt, M, Ezine, S, Falkenburg, F, Favier, R, Federico, M, Fenaux, P, Fitzgibbon, J, Flygare, J, Foà, R, Forrester, L, Galacteros, F, Garagiola, I, Gardiner, C, Garraud, O, Van Geet, C, Geiger, H, Geissler, J, Germing, U, Ghevaert, C, Girelli, D, Godeau, B, Gökbuget, N, Goldschmidt, H, Goodeve, A, Graf, T, Graziadei, G, Griesshammer, M, Gruel, Y, Guilhot, F, Von Gunten, S, Gyssens, I, Halter, J, Harrison, C, Harteveld, C, Hellström Lindberg, E, Hermine, O, Higgs, D, Hillmen, P, Hirsch, H, Hoskin, P, Huls, G, Inati, A, Johnson, P, Kattamis, A, Kiefel, V, Kleanthous, M, Klump, H, Krause, D, Hovinga, J, Lacaud, G, Lacroix Desmazes, S, Landman Parker, J, Legouill, S, Lenz, G, Von Lilienfeld Toal, M, Von Lindern, M, Lopez Guillermo, A, Lopriore, E, Lozano, M, Macintyre, E, Makris, M, Mannhalter, C, Martens, J, Mathas, S, Matzdorff, A, Medvinsky, A, Menendez, P, Migliaccio, A, Miharada, K, Mikulska, M, Minard, V, Montalbán, C, De Montalembert, M, Montserrat, E, Morange, P, Mountford, J, Muckenthaler, M, Müller Tidow, C, Mumford, A, Nadel, B, Navarro, J, El Nemer, W, Noizat Pirenne, F, O’Mahony, B, Oldenburg, J, Olsson, M, Oostendorp, R, Palumbo, A, Passamonti, F, Patient, R, De Latour, R, Pflumio, F, Pierelli, L, Piga, A, Pollard, D, Raaijmakers, M, Radford, J, Rambach, R, Koneti Rao, A, Raslova, H, Rebulla, P, Rees, D, Ribrag, V, Rijneveld, A, Rinalducci, S, Robak, T, Roberts, I, Rodrigues, C, Rosendaal, F, Rosenwald, A, Rule, S, Russo, R, Saglio, G, Sanchez, M, Scharf, R, Schlenke, P, Semple, J, Sierra, J, So Osman, C, Soria, J, Stamatopoulos, K, Stegmayr, B, Stunnenberg, H, Swinkels, D, Barata, J, Taghon, T, Taher, A, Terpos, E, Thachil, J, Tissot, J, Touw, I, Toye, A, Trappe, R, Traverse Glehen, A, Unal, S, Vaulont, S, Viprakasit, V, Vitolo, U, Van Wijk, R, Wójtowicz, A, Zeerleder, S, Zieger, B, Zieger, B., ZANELLA, ALBERTO, and BIONDI, ANDREA

Abstract

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at ∈ European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better fu treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine ‘sections’ in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

Published
2016

115. Ibrutinib versus ofatumumab in previously treated chronic lymphoid leukemia

Author

Byrd, Jc, Brown, Jr, O'Brien, S, Barrientos, Jc, Kay, Ne, Reddy, Nm, Coutre, S, Tam, Cs, Mulligan, Sp, Jaeger, U, Devereux, S, Barr, Pm, Furman, Rr, Kipps, Tj, Cymbalista, F, Pocock, C, Thornton, P, Caligaris Cappio, F, Robak, T, Delgado, J, Schuster, Sj, Montillo, M, Schuh, A, de Vos, S, Gill, D, Bloor, A, Dearden, C, Moreno, C, Jones, Jj, Chu, Ad, Fardis, M, Mcgreivy, J, Clow, F, James, Df, Hillmen, P, and Semenzato, GIANPIETRO CARLO

Subjects
Male, Oncology, Lymphoma, Medical and Health Sciences, chemistry.chemical_compound, Piperidines, Recurrence, Obinutuzumab, Monoclonal, 80 and over, Agammaglobulinaemia Tyrosine Kinase, Chronic, Humanized, Fatigue, Cancer, Aged, 80 and over, Leukemia, Hazard ratio, Antibodies, Monoclonal, Hematology, General Medicine, Middle Aged, Protein-Tyrosine Kinases, Duvelisib, Lymphocytic, Survival Rate, 6.1 Pharmaceuticals, Ibrutinib, Acalabrutinib, Female, Diarrhea, Adult, medicine.medical_specialty, Antibodies, Monoclonal, Humanized, Ofatumumab, Antibodies, Disease-Free Survival, Rare Diseases, Clinical Research, General & Internal Medicine, Internal medicine, medicine, Humans, Survival rate, Aged, Venetoclax, business.industry, Adenine, B-Cell, Evaluation of treatments and therapeutic interventions, Leukemia, Lymphocytic, Chronic, B-Cell, Pyrimidines, Orphan Drug, Cough, chemistry, Immunology, Pyrazoles, business, RESONATE Investigators, Follow-Up Studies
Abstract

Background In patients with chronic lymphoid leukemia (CLL) or small lymphocytic lymphoma (SLL), a short duration of response to therapy or adverse cytogenetic abnormalities are associated with a poor outcome. We evaluated the efficacy of ibrutinib, a covalent inhibitor of Bruton's tyrosine kinase, in patients at risk for a poor outcome. Methods In this multicenter, open-label, phase 3 study, we randomly assigned 391 patients with relapsed or refractory CLL or SLL to receive daily ibrutinib or the anti-CD20 antibody ofatumumab. The primary end point was the duration of progression-free survival, with the duration of overall survival and the overall response rate as secondary end points. Results At a median follow-up of 9.4 months, ibrutinib significantly improved progression-free survival; the median duration was not reached in the ibrutinib group (with a rate of progression-free survival of 88% at 6 months), as compared with a median of 8.1 months in the ofatumumab group (hazard ratio for progression or death in the ibrutinib group, 0.22; P

Published
2014

116. Preface

Author

Giacomello, Alessandro, Peters, G. J., Eriksson, Staffan, De Abreu, Ronney, Kristensen, T., Munch-Petersen, B., Vincenzetti, S., Cambi, A., Neuhard, J., Garattini, E., Vita, A., Oka, J., Matsumoto, A., Hosokawa, Y., Inoue, S., Allegrini, S., Johnson, R. B., Fiol, C. J., Eriksson, S., Fabianowska-Majewska, K., Wasiak, T., Duley, J., Simmonds, A., Bretner, M., Felczak, K., Poznański, J., Dzik, J. M., Golos, B., Jarmuła, A., Rode, W., Kulikowski, T., Codacci-Pisanelli, G., Pinedo, H. M., Noordhuis, P., van Groeningen, C. J., van der Wilt, C. L., Franchi, F., Hatse, S., Balzarini, J., De Clercq, E., Marinello, E., Rosi, F., Dispensa, E., Mangiavacchi, P., Riario-Sforza, G., Agostinho, A. B., Smolenski, R. T., Müller, Mathias M., Roch-Ramel, F., Guisan, B., Diezi, J., Tavenier, M., Skladanowski, A. C., de Abreu, R. A., de Jong, J. W., Åmellem, Øystein, Löffler, Monika, Pettersen, Erik O., Boulieu, R., Lenoir, A., Bertocchi, M., Mornex, J. F., Makarewicz, W., Spychala J., Mitchell B. S., Barankiewcz J., Góra-Tybor, Joanna, Robak, Tadeusz, Spasokukotskaja T., Sasvári-Székely M., Piróth Zs., Kazimierczuk Z., Staub M., Keuzenkamp-Jansen, C W, De Abreu, R A, Bökkerink, J P M, Trijbels, J M F, Eriksson S., Warzocha, K., Krykowski, E., Góra-Tybor, J., Fronczak, A., Robak, T., Minelli, A., Moroni, M., Monacelli, N., Mezzasoma, I., Amici, A., Emanuelli, M., Raffaelli, N., Ruggieri, S., Magni, G., Carta, M. C., Mattana, A., Poddie, F., Sgarrella, F., Tozzi, M. G., Veerman, G., Ruiz van Haperen, V. W. T., van Moorsel, C. J. A., Pesi, R., Baiocchi, C., Camici, M., Ipata, P. L., Kozłowska, M., Świerczyński, J., Smoleński, R. T., Jastorff, B., Messina, E., Savini, F., Procopio, A., Giacomello, A., Wielgus-Kutrowska, B., Kulikowska, E., Wierzchowski, J., Bzowska, A., Shugar, D., Fairbanks, Lynette D, Ruckemann, Katarzyna, Simmonds, H Anne, Kaletha, K., Szymańska, G., Thebault, M., Raffin, J. P., Le Gal, Y., Griesmacher, Andrea, De Abreu, Ronney A., Zych, M., Ruckemann, K., Jagodzinski, P., Kochan, Z., Stolk, J., Boerbooms, A., De Abreu, R., de Koning, D., van de Putte, L., Fiorini, M., Bazzichi, L., Bertolini, G., Martini, C., Ciompi, M. L., Lucacchini, A., Pizzichini, M., Terzuoli, L., Arezzini, L., Fe, L., Pagani, R., Miscetti, P., Allegrucci, C., Sebesta, I., Duley, J. A., Simmonds, H. A., Gross, M., Salerno, C., Stone, T. W., Van den Berghe, G., Valik, Dalibor, Jones, James D., Guerranti, R., Fè, L., Sforza, G. Riario, Knecht, Wolfgang, Grein, Klaus, Lodi, R., Iotti, S., Barbiroli, B., Bonin, B., Chantin, C., Bory, C., Micheli, V., Jacomelli, G., Morozzi, G., Fioravanti, A., Marcolongo, R., Pompucci, G., Peters G J, Noordhuis P, Komissarov A, Holwerda U, Kok R M, Van Laar J A M, Van der Wilt C L, Van Groeningen C J, Pinedo H M, Perrett, David, Jacobsson, Bengt, Sisto A., Iezzi A., Di Carlo M., Pizzigallo E., Akhondzadeh, S., MacGregor, D. G., Ogilvy, H. V., Zoref-Shani, E., Brosh, S., Sidi, Y., Bromberg, Y., Sperling, O., van Gennip, A. H., Abeling, N. G. G. M., Stroomer, A. E. M., van Lenthe, H., Bakker, H. D., van Kuilenburg, A. B. P., Connolly, G. P., Abbott, N. J., Lilling, G., Gozes, I., Vreken, P., Meinsma, R., de Ahreu, R. A., Diasio, R. B., Albin, N., Johnson, M. R., Shahinian, H., Wang, K., Gathof, B. S., Rocchigiani, M., Puig, J. G., Mateos, F., Sestini, S., Krijt, J., Shin, Y., Gresser, U., Costa, A., Maximova, N., Andolina, M., Paci, M., Carrozzi, M., Osbich, A., Durighello, M., Cavalli, F., Geatti, O., Zammarchi, E., Morgan, Gareth, Webster, A. D. B., Slavin, S., Naparstek, E., Nagler, A., Acker, M., Cividalli, G., Kapellushnik, Y., Varadi, G., Ben-Yoseph, R., Or, R., Parfenov, V. V., Ignatenko, M. A., Amchenkova, A. M., Narovlyansky, A. N., Spoto, G., Mastropasqua, L., Gizzi, F., Arduini, A., Del Gallo, P., Ciancaglini, M., Gallenga, P. E., Šebesta, I., Zeman, J., Crifò, C., Di Vito, M., Lomonte, A., Gerber, G., Carlucci, F., Tabucchi, A., Vannoni P., Di Pietro M. C., Vincent, M. F., Bontemps, F., Boer, P., Rötzer, E., Ehrmann, D., Empl, W., Bride, M. B. Mc, Ogg, C. S., Cameron, J. S., Moro, F., Rigden, S., Rees, L., Hoff, W. Van't, Raman, V., Palmieri, P., Mastropierro, G., Albertazzi, A., Rucci, C., Darlington, L. G., Cotton, S. R., de Gorter, J. J., Lawrence, E. S., Petrie, A., Sarsam, R. P., Semple, M. J., Warburton, E. A., Quaratino, C. P., Talone, L., Di Sciascio, N., Hrebíček, M. H., Poupětová, H., Ledvinová, J., Elleder, M., Vondrák, K., Rees, P. C., Wonke, B., Thein, S. L., Clegg, J. B., Marlewski, M., Pennelli, A., Di Marzio, M., Angelini, G., Sabatino, G., de Koning, P., Kerstens, P., de Graaf, R., Hayek, G., and Cardona, F.

Published
1995
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118. THE INTERNATIONAL PROGNOSTIC INDEX FOR PATIENTS WITH CHRONIC LYMPHOCYTIC LEUKEMIA (CLL-IPI)-AN INTERNATIONAL META-ANALYSIS

Author

Bahlo, J., Kutsch, N., Bergmann, M., Byrd, J., Doehner, H., Eichhorst, B., Else, M., Geisler, C., Grever, M., Lepretre, S., Neuberg, D., Oscier, D., Robak, T., Rosenquist, Richard Brandell, Shanafelt, T., Stilgenbauer, S., Hallek, M., Bahlo, J., Kutsch, N., Bergmann, M., Byrd, J., Doehner, H., Eichhorst, B., Else, M., Geisler, C., Grever, M., Lepretre, S., Neuberg, D., Oscier, D., Robak, T., Rosenquist, Richard Brandell, Shanafelt, T., Stilgenbauer, S., and Hallek, M.

Published
2015

119. A randomized, open-label, multicentre, phase 2/3 study to evaluate the safety and efficacy of lumiliximab in combination with fludarabine, cyclophosphamide and rituximab versus fludarabine, cyclophosphamide and rituximab alone in subjects with relapsed chronic lymphocytic leukaemia.

Author

Deshmukh C.D., Offner F., Van Den Neste E.W., Wu K.L., Van Hoof A., Maiolino A., Pinczowski H., Zanichelli M.A., Pereira J., Larratt L., Spaner D., Howson-Jan K., Chen C.I., Cantin G., Fernandez L.A., Fraser G., Mayer J., Trneny M., Jebavy L., Bordessoule D., Lamy T., Milpied N., Truchan-Graczyk M., Eghbali H., Karsenti J.-M., Celigny P.S., Mans L., Cazin B., Gyan E., Lepretre S., Bergmann L., Tsionos K., Lokeshwar N.M., Agarwal M.B., Ross C.R., Narayanan G., Raina V., Bondarde S.A., Shah B.A., Bairey O., Tikva P., Shvidel L., Ambrosetti A., Rossi P.G.B., Angelucci E., Carella A.M., Massaia M., Zinzani P.L., Caligaris-Cappio F., Foa R., Gaidano G., della Carita A.O.M., Leone G., Santoro A., Griskevicius L., Jurgutis R., Baker B.W., Hawkins T., Corbett G.M., Ganly P., D'Souza A.B., Deptala A., Holowiecki J., Kloczko J., Skotnicki A., Zdziarska B., Kyrcz-Krzemien S., Dmoszynska A., Moreira I., Pereira A.P., Colita A., Moicean A.D., Vasilica M., Danaila C., Gheorghita E., Pavlov V.V., Rossiev V.A., Konstantinova T., Samoilova O.S., Novgorod N., Shelekhova T., Zaritsky A.Y., Abdulkadyrov K.M., Zyuzgin I.S., Pristupa A.S., Loscertales J., Vidal J.B., de Mallorca P., Gonzalez M., Ortuno F., Giraldo P., Nathwani A., Agrawal S.G., Rule S., Dearden C.E., Bloor A.J., Haynes A., Singer C., Boclek R.G., Bosserman L.D., Chan D., Davidson S.J., Dichmann R.A., Farber C., Hart L., Hermann R., Hu E., Janakiraman N., Jonas W., Liem K.D., Mcintyre R.E., O'Brien S., Patel G., Rado T., Schilder R., Smith S.E., Stock W., Turturro F., Venugopal P., Anderson T.C., Berry W., Boyd T.E., Byrd J., Cooper M., Flinn I., Gersh R., Gordon D., Guzley G.J., Wilks S.T., Klein A., Krauss J.C., Lister J., Mandell L., Molina A., Cooper B., Pendergrass K.B., Reeder C., Savin M.A., Spitzer G., Tuscano J.M., vanDeventer H., Eradat H.A., Masood A., Mena R., Awan F.T., Hillmen P., Hellmann A., Robak T., Hughes S.G., Trone D., Shannon M., Flinn I.W., Byrd J.C., Riveros D., Pavlovsky S., Iastrebner C.M., Carney D.A., Deveridge S., Durrant S., Hahn U.H., Hertzberg M., Leahy M.F., Ma D., Marlton P., Mulligan S., Opat S.S., Tiley C., Wickham N.W., Cannell P., Gatalano J., Catalano J., Cull G., To L.B., Hopfinger G., Jager U., Linkesch W., Petzer A., Schwarzmeier J., Steurer M., Greil R., Bememan Z., Bosly A., Bron D., Janssens A., Deshmukh C.D., Offner F., Van Den Neste E.W., Wu K.L., Van Hoof A., Maiolino A., Pinczowski H., Zanichelli M.A., Pereira J., Larratt L., Spaner D., Howson-Jan K., Chen C.I., Cantin G., Fernandez L.A., Fraser G., Mayer J., Trneny M., Jebavy L., Bordessoule D., Lamy T., Milpied N., Truchan-Graczyk M., Eghbali H., Karsenti J.-M., Celigny P.S., Mans L., Cazin B., Gyan E., Lepretre S., Bergmann L., Tsionos K., Lokeshwar N.M., Agarwal M.B., Ross C.R., Narayanan G., Raina V., Bondarde S.A., Shah B.A., Bairey O., Tikva P., Shvidel L., Ambrosetti A., Rossi P.G.B., Angelucci E., Carella A.M., Massaia M., Zinzani P.L., Caligaris-Cappio F., Foa R., Gaidano G., della Carita A.O.M., Leone G., Santoro A., Griskevicius L., Jurgutis R., Baker B.W., Hawkins T., Corbett G.M., Ganly P., D'Souza A.B., Deptala A., Holowiecki J., Kloczko J., Skotnicki A., Zdziarska B., Kyrcz-Krzemien S., Dmoszynska A., Moreira I., Pereira A.P., Colita A., Moicean A.D., Vasilica M., Danaila C., Gheorghita E., Pavlov V.V., Rossiev V.A., Konstantinova T., Samoilova O.S., Novgorod N., Shelekhova T., Zaritsky A.Y., Abdulkadyrov K.M., Zyuzgin I.S., Pristupa A.S., Loscertales J., Vidal J.B., de Mallorca P., Gonzalez M., Ortuno F., Giraldo P., Nathwani A., Agrawal S.G., Rule S., Dearden C.E., Bloor A.J., Haynes A., Singer C., Boclek R.G., Bosserman L.D., Chan D., Davidson S.J., Dichmann R.A., Farber C., Hart L., Hermann R., Hu E., Janakiraman N., Jonas W., Liem K.D., Mcintyre R.E., O'Brien S., Patel G., Rado T., Schilder R., Smith S.E., Stock W., Turturro F., Venugopal P., Anderson T.C., Berry W., Boyd T.E., Byrd J., Cooper M., Flinn I., Gersh R., Gordon D., Guzley G.J., Wilks S.T., Klein A., Krauss J.C., Lister J., Mandell L., Molina A., Cooper B., Pendergrass K.B., Reeder C., Savin M.A., Spitzer G., Tuscano J.M., vanDeventer H., Eradat H.A., Masood A., Mena R., Awan F.T., Hillmen P., Hellmann A., Robak T., Hughes S.G., Trone D., Shannon M., Flinn I.W., Byrd J.C., Riveros D., Pavlovsky S., Iastrebner C.M., Carney D.A., Deveridge S., Durrant S., Hahn U.H., Hertzberg M., Leahy M.F., Ma D., Marlton P., Mulligan S., Opat S.S., Tiley C., Wickham N.W., Cannell P., Gatalano J., Catalano J., Cull G., To L.B., Hopfinger G., Jager U., Linkesch W., Petzer A., Schwarzmeier J., Steurer M., Greil R., Bememan Z., Bosly A., Bron D., and Janssens A.

Abstract

Summary: Lumiliximab is a chimeric monoclonal antibody that targets CD23 on the surface of chronic lymphocytic leukaemia (CLL) B-cells. Early phase clinical studies with lumiliximab alone and in combination with fludarabine, cyclophosphamide and rituximab (FCR) established its potential efficacy and tolerability. The 152CL201 trial [Lumiliximab with fludarabine, cyclophosphamide and rituximab (FCR) versus FCR alone in subjects with relapsed CLL; LUCID] was a phase 2/3, randomized (1:1), open-label, multicentre study of lumiliximab in combination with FCR versus FCR alone in patients with relapsed CLL. Six hundred and twenty-seven patients were randomized to either arm. Overall the combination of lumiliximab with FCR was not significantly better than FCR alone (overall response rate 71% vs. 72%, complete response rate 16% vs. 15%, median progression-free survival 24.6 vs. 23.9 months respectively, for FCR with and without lumiliximab). There was a slightly increased incidence of adverse events with lumiliximab but these increases did not appear to lead to differences in eventual outcomes. An interim analysis failed to show sufficient efficacy of the combination of lumiliximab with FCR. The study was therefore stopped early for lack of efficacy. Despite the eventual outcome, the LUCID trial is one of the largest studies that provides valuable insight into the efficacy and tolerability of FCR as a therapeutic option for patients with relapsed CLL.Copyright © 2014 John Wiley & Sons Ltd.

Published
2015

120. 1004O - Final overall survival results of frontline bortezomib plus rituximab, cyclophosphamide, doxorubicin, and prednisone (VR-CAP) vs R-CHOP in transplantation-ineligible patients (pts) with newly diagnosed mantle-cell lymphoma (MCL): A randomized, open-label, phase III (LYM-3002) study

Author

Cavalli, F., Jin, J., Pylypenko, H., Verhoef, G., Siritanaratkul, N., Drach, J., Raderer, M., Mayer, J., Pereira, J., Tumyan, G., Okamoto, R., Nakahara, S., Hu, P., Appiani, C., Nemat, S., and Robak, T.

Published
2018
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121. Bortezomib plus rituximab versus rituximab alone in patients with relapsed, rituximab-naive or rituximab-sensitive, follicular lymphoma: a randomised phase 3 trial

Author

Coiffier, B, Osmanov, E, Hong, X, Scheliga, A, Mayer, J, Offner, F, Rule, S, Teixeira, A, Walewski, J, de Vos, S, Crump, M, Shpilberg, O, Esseltine, D, Zhu, E, Enny, C, Theocharous, P, van de Velde, H, Elsayed, Y, Zinzani, P, Abdulkadyrov, K, Afanasiev, B, Aguayo Gonzalez, A, Andre, M, Belada, D, Ben Yehuda, D, Bezares, R, Biakhov, M, Bolam, S, Borbenyi, Z, Bron, D, Buckstein, R, Bumbea, H, Caballero Barrigon, M, Campos, L, Cantonetti, M, Capra Zanella, M, Christiansen, N, Cohen, G, Colita, N, Cosgriff, T, Culligan, D, Del Giglio, A, Dichmann, R, Dietzfelbinger, H, Digumarti, R, Dmoszynska, A, Domnikova, N, Dubinsky, P, Dunaev, Y, Easow, J, Eberwine, S, Economopoulos, T, Egyed, M, Ellerton, J, Eom, H, Farmer, L, Fenske, T, Fields, P, Fillet, G, Frank, R, Gaisarova, G, Garicochea, B, Gasztonyi, Z, Gavish, I, Gheorghita, E, Gladkov, O, Goldberg, V, Golenkov, A, Gomez Almaguer, D, Gonzalez Barca, E, Guan, Z, Gupta, S, Hellmann, A, Hermann, R, Honkanen, T, Hu, E, Huang, X, Hudecek, J, Illes, A, Intragumtornchai, T, Jedrzejczak, W, Jones, L, Jootar, S, Kahanic, S, Karamanesht, E, Ke, X, Khuageva, N, Kim, W, Kimby, E, Komisarenko, V, Kouroukis, T, Kuliczkowski, K, Kuzina, L, Kyselyova, M, Labanca, V, Lange, A, Le Gouill, S, Leahy, M, Liberati, A, Linden, O, Liu, T, Lubennikov, V, Lundin, J, Lysa, T, Lysenko, I, Lytvyn, I, Makhson, A, Manikhas, G, Masliak, Z, Mcintyre, R, Medvedeva, N, Mena, R, Merkulov, V, Mesters, R, Milpied, N, Min, Y, Moezi, M, Mohrbacher, A, Mollee, P, Morgan, D, Morschhauser, F, Mysanikov, A, Nagler, A, Nair, S, Naparstek, E, Nawarawong, W, Noga, S, Oliveira, I, Okada, C, Oriol Rocafiguera, A, Page, R, Papajik, T, Pasquini, R, Patel, M, Patel, R, Paton, E, Pavlov, V, Pospelova, T, Prasad, S, Pylypenko, H, Raposo, J, Rekhtman, G, Rivas, S, Robak, T, Saba, S, Salles, G, Saltzman, M, Samoilova, O, Samuels, B, Sanani, S, Sebban, C, Silva da Gomes, M, Shen, Z, Shi, Y, Shtalrid, M, Siritanaratkul, N, Skotnicki, A, Solal Celigny, P, Soubeyran, P, Spencer, A, Stevens, D, Suh, C, Sulek, K, Suvorov, A, Szer, J, Theunissen, K, To Bik, L, Tothova, E, Trneny, M, Van De Velde, A, Van Hoof, A, Van Steenweghen, S, Vanhatalo, S, Varma, S, Vidyasagar, M, Vilchevskaya, K, Vitolo, U, Wang, H, Warzocha, K, Wild, A, Zachee, P, Zanichelli, M, Zhang, W, Zoppegno, L, Zoumbos, N, Coiffier B., Osmanov E.A., Hong X., Scheliga A., Mayer J., Offner F., Rule S., Teixeira A., Walewski J., de Vos S., Crump M., Shpilberg O., Esseltine D.L., Zhu E., Enny C., Theocharous P., van de Velde H., Elsayed Y.A., Zinzani P.L., and LYM-3001 study investigators

Subjects
Oncology, Male, Lymphoma, Settore MED/06 - Oncologia Medica, Follicular lymphoma, Bortezomib, Antibodies, Monoclonal, Murine-Derived, 0302 clinical medicine, Maintenance therapy, Prednisone, immune system diseases, Recurrence, hemic and lymphatic diseases, Monoclonal, Antineoplastic Combined Chemotherapy Protocols, 80 and over, Lymphoma, Follicular, Multiple myeloma, Infusion Pumps, Aged, 80 and over, Middle Aged, Boronic Acids, 3. Good health, 030220 oncology & carcinogenesis, Pyrazines, Rituximab, Female, medicine.drug, Murine-Derived, Adult, medicine.medical_specialty, rituximab-naive, Antineoplastic Agents, Antibodies, Disease-Free Survival, 03 medical and health sciences, Young Adult, follicular lymphoma, Internal medicine, Neoplasm Staging, Humans, Aged, medicine, business.industry, Follicular, medicine.disease, Clinical trial, rituximab-sensitive, Immunology, business, Settore MED/15 - Malattie del Sangue, 030215 immunology
Abstract

BACKGROUND: Bortezomib and rituximab have shown additive activity in preclinical models of lymphoma, and have been shown to be active and generally well tolerated in a randomised phase 2 study in patients with follicular and marginal zone lymphoma. We compared the efficacy and safety of rituximab alone or combined with bortezomib in patients with relapsed or refractory follicular lymphoma in a phase 3 setting. METHODS: In this multicentre phase 3 trial, rituximab-naive or rituximab-sensitive patients aged 18 years or older with relapsed grade 1 or 2 follicular lymphoma were randomly assigned (1:1) to receive five 35-day cycles consisting of intravenous infusions of rituximab 375 mg/m(2) on days 1, 8, 15, and 22 of cycle 1, and on day 1 of cycles 2-5, either alone or with bortezomib 1·6 mg/m(2), administered by intravenous injection on days 1, 8, 15, and 22 of all cycles. Randomisation was stratified by FLIPI score, previous use of rituximab, time since last therapy, and region. Treatment assignment was based on a computer-generated randomisation schedule prepared by the sponsor. Patients and treating physicians were not masked to treatment allocation. The primary endpoint was progression-free survival analysed by intention to treat. This trial has been completed and is registered with ClinicalTrials.gov, number NCT00312845. FINDINGS: Between April 10, 2006, and Aug 12, 2008, 676 patients were randomised to receive rituximab (n=340) or bortezomib plus rituximab (n=336). After a median follow-up of 33·9 months (IQR 26·4-39·7), median progression-free survival was 11·0 months (95% CI 9·1-12·0) in the rituximab group and 12·8 months (11·5-15·0) in the bortezomib plus rituximab group (hazard ratio 0·82, 95% CI 0·68-0·99; p=0·039). The magnitude of clinical benefit was not as large as the anticipated prespecified improvement of 33% in progression-free survival. Patients in both groups received a median of five treatment cycles (range 1-5); 245 of 339 (72%) and 237 of 334 (71%) patients in the rituximab and bortezomib plus rituximab groups, respectively, completed five cycles. Of patients who did not complete five cycles, most discontinued early because of disease progression (77 [23%] patients in the rituximab group, and 56 [17%] patients in the bortezomib plus rituximab group). Rates of adverse events of grade 3 or higher (70 [21%] of 339 rituximab-treated patients vs 152 [46%] of 334 bortezomib plus rituximab treated patients), and serious adverse events (37 [11%] patients vs 59 [18%] patients) were lower in the rituximab group than in the combination group. The most common adverse events of grade 3 or higher were neutropenia (15 [4%] patients in the rituximab group and 37 [11%] patients in the bortezomib plus rituximab group), infection (15 [4%] patients and 36 [11%] patients, respectively), diarrhoea (no patients and 25 [7%] patients, respectively), herpes zoster (one [

Published
2011

122. Efficacy and safety of a nanofiltered liquid intravenous immunoglobulin product in patients with primary immunodeficiency and idiopathic thrombocytopenic purpura

Author

Meer, J.W.M. van der, Beem, R.T. van, Robak, T., Deptala, A., and Strengers, P.F.W.

Subjects
hemic and lymphatic diseases, Invasive mycoses and compromised host Infection and autoimmunity [N4i 2]
Abstract

Item does not contain fulltext BACKGROUND AND OBJECTIVES: In the production process of a new 5% liquid intravenous immunoglobulin (IVIG-L) product (Nanogam((R)) ), a combined pepsin/pH 4.4 treatment/15-nm filtration (pH 4.4/15NF) step and a solvent-detergent (SD) treatment step were incorporated to improve the virus inactivating/reducing capacity of the manufacturing process. Two prospective uncontrolled multicentre studies were performed to evaluate the safety and efficacy of this product. MATERIALS AND METHODS: Efficacy, including pharmacokinetics, of IVIG-L was studied for 6 months in 18 primary immunodeficiency (PID) patients, succeeded by a long-term follow-up study (mean 2.2 years, n=17). Second, in 24 patients with idiopathic thrombocytopenic purpura (ITP), IVIG-L was studied for efficacy for 14 days. In both studies, adverse events and vital signs were recorded to study safety. RESULTS: In PID patients treated with IVIG-L, 0.60 and 0.38 severe infections per patient per year were reported during, respectively, the short-term and long-term follow-up. Pharmacokinetic studies resulted in an IgG half-life of 30.9 +/- 11.3 days and a mean IgG trough level of 6.8 +/- 1.2 g/l. In the ITP study, all patients showed an increase in platelet counts after infusion with IVIG-L, and 20/24 patients responded with a platelet count >50 x 10(9) /l (83.3%) within 1 week. IVIG-L infusions did not cause clinical relevant changes in laboratory parameters or vital signs. CONCLUSIONS: In clinical studies, IVIG-L (Nanogam(R)) demonstrated to be efficacious, well tolerated and safe.

Published
2011

126. Ofatumumab monotherapy in fludarabine-refractory chronic lymphocytic leukemia: final results from a pivotal study

Author

Osterborg, A., primary, Jewell, R. C., additional, Padmanabhan-Iyer, S., additional, Kipps, T. J., additional, Mayer, J., additional, Stilgenbauer, S., additional, Williams, C. D., additional, Hellmann, A., additional, Furman, R. R., additional, Robak, T., additional, Hillmenn, P., additional, Trn ny, M., additional, Dyer, M. J. S., additional, Piotrowska, M., additional, Kozak, T., additional, Gupta, I. V., additional, Phillips, J. L., additional, Goldstein, N., additional, Struemper, H., additional, Losic, N., additional, Lisby, S., additional, and Wierda, W. G., additional

Published
2015
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127. A randomized, open-label, multicentre, phase 2/3 study to evaluate the safety and efficacy of lumiliximab in combination with fludarabine, cyclophosphamide and rituximab versus fludarabine, cyclophosphamide and rituximab alone in subjects with relapsed chronic lymphocytic leukaemia

Author

Awan, F.T., Hillmen, P., Hellmann, A., Robak, T., Hughes, S.G., Trone, D., Shannon, M., Flinn, I.W., Byrd, J.C., Liem, K.D., et al., Awan, F.T., Hillmen, P., Hellmann, A., Robak, T., Hughes, S.G., Trone, D., Shannon, M., Flinn, I.W., Byrd, J.C., Liem, K.D., and et al.

Abstract

Item does not contain fulltext, Lumiliximab is a chimeric monoclonal antibody that targets CD23 on the surface of chronic lymphocytic leukaemia (CLL) B-cells. Early phase clinical studies with lumiliximab alone and in combination with fludarabine, cyclophosphamide and rituximab (FCR) established its potential efficacy and tolerability. The 152CL201 trial [Lumiliximab with fludarabine, cyclophosphamide and rituximab (FCR) versus FCR alone in subjects with relapsed CLL; LUCID] was a phase 2/3, randomized (1:1), open-label, multicentre study of lumiliximab in combination with FCR versus FCR alone in patients with relapsed CLL. Six hundred and twenty-seven patients were randomized to either arm. Overall the combination of lumiliximab with FCR was not significantly better than FCR alone (overall response rate 71% vs. 72%, complete response rate 16% vs. 15%, median progression-free survival 24.6 vs. 23.9 months respectively, for FCR with and without lumiliximab). There was a slightly increased incidence of adverse events with lumiliximab but these increases did not appear to lead to differences in eventual outcomes. An interim analysis failed to show sufficient efficacy of the combination of lumiliximab with FCR. The study was therefore stopped early for lack of efficacy. Despite the eventual outcome, the LUCID trial is one of the largest studies that provides valuable insight into the efficacy and tolerability of FCR as a therapeutic option for patients with relapsed CLL.

Published
2014

128. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study

Author

Fenaux, P, Mufti, Gj, Hellstrom Lindberg, E, Santini, V, Finelli, C, Giagounidis, A, Schoch, R, Gattermann, N, Sanz, G, List, A, Gore, Sd, Seymour, Jf, Bennett, Jm, Byrd, J, Backstrom, J, Zimmerman, L, Mckenzie, D, Beach, C, Silverman, Lr, Durrant, S, Enno, A, Herrmann, R, Horvath, N, Mills, A, Spencer, A, Szer, J, Gallo, J, Dunlop, L, Arthur, C, Goranov, S, Peytchev, D, Gercheva, L, Cermak, J, Voglova, J, Vey, N, Dreyfus, F, Laurent, G, Quesnel, B, Dombret, H, Stamatoullas, A, Wattel, E, Hunault Berger, M, Aul, C, Duhrsen, U, Platzbecker, U, Schmid, M, Hanel, M, Haase, D, Fiedler, W, Schmitz, N, Hofmann, W, Horst, H, Anagnostopoulos, N, Pappa, V, Papadaki, E, Zoumbos, N, Borbenyi, Z, Masszi, T, Baccarani, M, Bacigalupo, A, Corradini, P, Leone, G, Sacchi, Stefano, Bosi, A, Musto, P, Muus, P, Dmoszynska, A, Robak, T, Sulek, K, Kuliczkowski, K, Jedrzejczak, W, Zaritsky, A, Abdulkadyrov, K, Podoltseva, E, Afanasiev, B, Bargay, J, Brunet, S, Del Canizo, C, Ribera, J, Figuera Alvarez, A, Diaz Mediavilla, J, Canales, M, Ortega F, Ramos y., Nilsson, L, Olsson, A, Cavenagh, J, Parker, J, Killick, S, Kruger, A, Vyas, P, Dennis, M, Cripe, L, Dipersio, J, and Emanuel, P.

Subjects
Adult, Male, medicine.medical_specialty, azacitidine, Antimetabolites, Antineoplastic, Azacitidine, Decitabine, Article, law.invention, Randomized controlled trial, law, Internal medicine, medicine, Clinical endpoint, Humans, Intensive care medicine, Aged, Proportional Hazards Models, Aged, 80 and over, Intention-to-treat analysis, business.industry, Hazard ratio, Middle Aged, Oncology, Hypomethylating agent, International Prognostic Scoring System, Myelodysplastic Syndromes, Female, business, medicine.drug
Abstract

Drug treatments for patients with high-risk myelodysplastic syndromes provide no survival advantage. In this trial, we aimed to assess the effect of azacitidine on overall survival compared with the three commonest conventional care regimens.In a phase III, international, multicentre, controlled, parallel-group, open-label trial, patients with higher-risk myelodysplastic syndromes were randomly assigned one-to-one to receive azacitidine (75 mg/m(2) per day for 7 days every 28 days) or conventional care (best supportive care, low-dose cytarabine, or intensive chemotherapy as selected by investigators before randomisation). Patients were stratified by French-American-British and international prognostic scoring system classifications; randomisation was done with a block size of four. The primary endpoint was overall survival. Efficacy analyses were by intention to treat for all patients assigned to receive treatment. This study is registered with ClinicalTrials.gov, number NCT00071799.Between Feb 13, 2004, and Aug 7, 2006, 358 patients were randomly assigned to receive azacitidine (n=179) or conventional care regimens (n=179). Four patients in the azacitidine and 14 in the conventional care groups received no study drugs but were included in the intention-to-treat efficacy analysis. After a median follow-up of 21.1 months (IQR 15.1-26.9), median overall survival was 24.5 months (9.9-not reached) for the azacitidine group versus 15.0 months (5.6-24.1) for the conventional care group (hazard ratio 0.58; 95% CI 0.43-0.77; stratified log-rank p=0.0001). At last follow-up, 82 patients in the azacitidine group had died compared with 113 in the conventional care group. At 2 years, on the basis of Kaplan-Meier estimates, 50.8% (95% CI 42.1-58.8) of patients in the azacitidine group were alive compared with 26.2% (18.7-34.3) in the conventional care group (p0.0001). Peripheral cytopenias were the most common grade 3-4 adverse events for all treatments.Treatment with azacitidine increases overall survival in patients with higher-risk myelodysplastic syndromes relative to conventional care.

Published
2009

135. Marrow versus peripheral blood for geno-identical allogeneic stem cell transplantation in acute myelocytic leukemia: Influence of dose and stem cell source shows better outcome with rich marrow

Author

Gorin N.C., Labopin M., Rocha V., Arcese W., Beksac M., Gluckman E., Ringden O., Ruutu T., Reiffers J., Bandini G., Falda M., Zikos P., Willemze R., Frassoni F., Abecasis M., Abráhamová J., Afanassiev B.V., Aglietta M., Alabdulaaly A., Aleinikova O., Paolo Alessandrino E., Al Shemmari S.H., Amadori D., Amadori S., Amos T., Andolina M., Andreesen R., Angelucci E., Anhuf J., Arnold R., Arpaci F., Attal M., Azevedo W., Azim H.A., Baccarani M., Bacigalupo A., Barbui T., Bargetzi M., Barnard D.L., Bartsch H.H., Baruchel A., Battista C., Bay J.-O., Bayik M., Bazarbachi A., Beguin Y., López J.L.B., Benedek I., Benedetti F., Bengala C., Berrebi A., Besalduch J., Biesma D., Biron P., Björkholm M., Blaise D., Blesing N.E., Boasson M., Bobev D., Boccadoro M., Bolaman Z., Boogaerts M.A., Bordessoule D., Bosi A., Aida B.S., Bourhis J.H., Bourikas G., Bowen D.T., Bregni M., Bries G., Brinch L., Brittain D., Bron D., Brune M., Bullorsky E.O., Bunjes D., Burdach S., Burnett A.K., Buzyn A., Caballero D., Cagirgan S., Cahn J.-Y., Canepa C.O., Cao A., Carella A.M., Carrera F.D., Carret A.-S., Cascinu S., Castel V., Caswell M., Cavanna L., Cetto G.L., Chapuis B., Chasty R., Chen Y.-C., Chisesi T., Chopra R., Chybicka A., Clark R.E., Colombat P., Colovic M.D., Constenla-Figueiras M., Contreras M., Contu L., Cordonnier C., Cornelissen J.J., Cornish J., Coser P., Costa N., Coze C., Craddock C., Crown J., Culligan D.J., Danova M., Darbyshire P.J., Davies J.M., de Bock R., de Pablos Gallego J.M., De Prijck B., de Revel T., De Rossi G., De Souza C.A., Deb G., Degos L., Demuynck H., Dervenoulas I., Di Bartolomeo P., Di Renzo N., Diaz M.A., Diehl V., Diez-Martin J.L., Dincer S., Giorgio D., Dmoszynska A., Doelken G., Peter P.D., Dulley F., Easow J., Ebell W., Efremidis A., Ehninger G., Eichler H., Eimermacher H., Enno A., Errazquin L., Aguado J.E., Everaus H., Fagioli F., Fanin R., Fassas A., Fasth A., Faulkner L.B., Fauser A.A., Feldman L., Feremans W., Ferhanoglu B., Fernández M.N., Fernández-Ranada J.M., Ferrant A., Ferrara F., Finke J., Fischer A., Fischer J., Fitzsimons T., Floristan F., Forjaz de Lacerda J.M.F., Fossati-Bellani F., Fosser V., Franklin I., Freund M., Frickhofen N., Gabbas A., Gadner H., Gallamini A., Galvin M.C., López J.G., García-Conde J., Gaska T., Gastl G., Gedikoglu G., Ghavamzadeh A., Gianni A., Gibson B.E., Gil J.L., Gilleece M.H., Gisselbrecht C., Glass B., Gmür J., Göbel U., Goldman J.M., Goldstone A.H., San Miguel J.D.G., González-López M.-A., Grafakos S., Gramatzki M., Grañena A., Gratecos N., Gratwohl A., Greinix H.T., Gugliotta L., Guilhot F., Guimaraes J.E., Gülbas Z., Gulyuz O., Gurman G., Gutierrez M.M., Haas R., Hamladji R.-M., Hamon M.D., Hansen N.E., Harhalakis N., Harousseau J.L., Hartenstein R., Hartmann C.O., Hausmaninger H., Haznedar R., Heit W., Hellmann A., Herrmann R.P., Hertenstein B., Hess U., Hinterberger W., Ho A.D., Hoelzer D., Holowiecki J., Horst H.-A., Hossfeld D.K., Huebsch L., Hunter A.E., Iacopino P., Iannitto E., Indrák K., Iriondo A., Izzi T., Jackson G.L., Jacobs P., Jacobsen N., Janvier M., Jebavy L., Joensuu H., Joerg S., Jones F.G.C., Jouet J.P., Joyner M.V., Juliusson G., Jürgens H., Kalayoglu-Besisik S., Kalman N., Kalmanti M., Kansoy S., Kansu E., Kanz L., Karianakis G., Kernéis Y., Khalifeh O., Khomenko V., Kienast J., Killick S., Kirchner H.H., Klingebiel T., Knauf W., Koenigsmann M., Koistinen P., Koivunen E., Kolb H.-J., Kolbe K., Koller E., Komarnicki M., Koscielniak E., Kovacsovics T., Kowalczyk J.R., Koza V., Kozak T., Kugler J., Kuliczkowski K., Kvaloy S., Labar B., Laciura P., Palacios J.J.L., Lakota J., Lambertenghi D.G., Lange A., Lanza F., Isasti R.L., Lauria F., Le Moine F., Leblond V., Lelli G., Lenhoff S., Leon L.A., Leoncini-Franscini L., Leone G., Leoni P., Levis A., Leyvraz S., Liberati M., Link H., Linkesch W., Liso V., Lisukov I.A., Littlewood T., Ljungman P., Locatelli F., Losonczy H., Lotz J.-P., Ludwig H., Lukac J., Lutz D., Macchia P., Madrigal A., Maiolino A., Majolino I., Eloy-García J.M., Malesevic M., Mandelli F., Marc A., Marcus R., Marianska B., Markuljak I., Marsh J.C.W., Martelli M.F., Marti Tutusaus J.M., Martin S., Martin M., Martinelli G., Martínez-Rubio A.M., Martoni A., Maschan A., Maschmeyer G., Masszi T., Mazza P., McCann S., Meier C.R., Messina C., Mettivier V., Metzner B., Michallet M., Michieli M., Michon J., Milligan D.W., Milone J.H., Giuseppe G.M., Minigo H., Mistrik M., Moicean A.D., Monfardini S., Montserrat E., Moraleda Jimenez J.M., Morales-Lazaro A., Morandi S., Morra E., Mufti G.J., Musso M., Nagler A., Nalli G., Naparstek E., Narni F., Nenadov-Beck M., Neubauer A., Newland A.C., Niederwieser D., Niethammer D., Noens L.A., Nousiainen T., Novik A., Novitzky N., Occhini D., Odriozolas J., Ojanguren J.M., O’meara A., Onat H., Orchard K., Ortega J.J., Osieka R., Ossenkoppele G.J., Othman B., Ovali E., Ozcebe O.I., Ozerkan K., Ozturk A., Papatryfonos A., Parker J.E., Pastore M., Patrone F., Patton N., Pejin D., Peñarrubia M.J., Equiza E.P., Peschel C., Pession A., Pigaditou A., Pignon B., Pihkala U., Pimentel P., Pitini V., Podoltseva E., Pogliani E.M., Anna A.P., Porta F., Potter M., Powles R., Prentice G.H., Pretnar J., Ptushkin V., Quarta G., Reiter A., Remes K., Reykdal S., Santasusana J.M.R., Rifón J., Rio B., Rizzoli V., Robak T., Robinson A.J., Rodeghiero F., Rodríguez Fernández J.M., Rombos Y., Romeril K.R., Rosenmayr A., Rossi J.F., Rosti G., Rotoli B., Rowe J.M., Russell N.H., Ryzhak O., Rzepecki P., Saglio G., Salwender H., Samonigg H., Santoro A., Sanz M.A., Sayer H.G., Scanni A., Schaafsma M.R., Schaefer U.W., Schanz U., Schattenberg A., Schey S.A.M., Schlimok G., Schmoll H.-J., Schots R., Schouten H., Schwarer A.P., Schwerdtfeger R., Scimè R., Segel E., Seger R., Selleslag D., Serban M., Shamaa S., Shaw P.J., Siegert W., Siena S., Sierra J., Simonsson B., Singer C.R.J., Sirchia G., Skotnicki A.B., Slavin S., Snowden J., Sotto J.J., Tanyeli A., Tedeschi L., Tidefelt U., Tissot J.-D., Tobler A., Tomas J.F., Torres J.P., Torres G.A., Touraine J.-L., Trneny M., Uderzo C., Unal E., Unal A., Undar L., Urban C., Van den Berg H., van Marwijk K.M., Vellenga E., Venturini M., Verdonck L.F., Veys P., Vilardell J., Vinante O., Visani G., Vitek A., Vivancos P., Volpe E., Vora A., Vorlicek J., Vowels M., Vujic D., Wachowiak J., Wagner T., Wahlin A., Walewski J., Wandt H., Weissinger F., Wijermans P.W., Wiktor-Jedrzejczak W., Will A.M., Woell E., Wörmann B., Yaniv I., Yesilipek M.A., Yilmaz U., Yong A., Zachée P., Zambelli A., Zander A.R., Zintl F., Zoumbos N.C., Çukurova Üniversitesi, Maltepe Üniversitesi, and Ege Üniversitesi

Subjects
Myeloid, Male, Pathology, Time Factors, Graft vs Host Disease, Biochemistry, Gastroenterology, Blood cell, Bone Marrow, Child, Bone Marrow Transplantation, Leukemia, Remission Induction, Hematology, Middle Aged, Prognosis, Leukemia, Myeloid, Acute, ComputingMilieux_MANAGEMENTOFCOMPUTINGANDINFORMATIONSYSTEMS, medicine.anatomical_structure, Treatment Outcome, Child, Preschool, Female, Stem cell, InformationSystems_MISCELLANEOUS, Homologous, Adult, medicine.medical_specialty, Adolescent, Immunology, Acute, Disease-Free Survival, Internal medicine, medicine, Transplantation, Homologous, Humans, Preschool, Aged, Transplantation, business.industry, ComputerSystemsOrganization_COMPUTER-COMMUNICATIONNETWORKS, Cell Biology, medicine.disease, Peripheral blood, Histocompatibility, Multivariate Analysis, Stem Cell Transplantation, ComputingMethodologies_PATTERNRECOGNITION, Myelocytic leukemia, Bone marrow, business, Settore MED/15 - Malattie del Sangue
Abstract

PubMed ID: 12829583, Several studies have compared bone marrow (BM) and peripheral blood (PB) as stem cell sources in patients receiving allografts, but the cell doses infused have not been considered, especially for BM. Using the ALWP/EBMT registry, we retrospectively studied 881 adult patients with acute myelocytic leukemia (AML), who received a non-T-depleted allogeneic BM (n = 515) or mobilized PB (n = 366) standard transplant, in first remission (CR1), from an HLA-identical sibling, over a 5-year period from January 1994. The BM cell dose ranged from 0.17 to 29 × 10 8 /kg with a median of 2.7 × 10 8 /kg. The PB cell dose ranged from 0.02 to 77 × 10 8 /kg with a median of 9.3 × 10 8 /kg. The median dose for patients receiving BM (2.7 × 10 8 /kg) gave the greatest discrimination. In multivariate analyses, high-dose BM compared to PB was associated with lower transplant-related mortality (RR = 0.61; 95% CI, 0.39-0.98; P = .04), better leukemia-free survival (RR = 0.65; 95% CI, 0.46-0.91; P = .013), and better overall survival (RR = 0.64; 95% CI, 0.44-0. 92; P = .016). The present study in patients with AML receiving allografts in first remission indicates a better outcome with BM as compared to PB, when the dose of BM infused is rich. © 2003 by The American Society of Hematology.

Published
2003

136. Marrow versus peripheral blood for geno-identical allogeneic stem cell transplantation in acute myelocytic leukemia: Influence of dose and stem cell source shows better outcome with rich marrow

Author

Gorin, N.C. Labopin, M. Rocha, V. Arcese, W. Beksac, M. Gluckman, E. Ringden, O. Ruutu, T. Reiffers, J. Bandini, G. Falda, M. Zikos, P. Willemze, R. Frassoni, F. Abecasis, M. Abráhamová, J. Afanassiev, B.V. Aglietta, M. Alabdulaaly, A. Aleinikova, O. Paolo Alessandrino, E. Al Shemmari, S.H. Amadori, D. Amadori, S. Amos, T. Andolina, M. Andreesen, R. Angelucci, E. Anhuf, J. Arnold, R. Arpaci, F. Attal, M. Azevedo, W. Azim, H.A. Baccarani, M. Bacigalupo, A. Barbui, T. Bargetzi, M. Barnard, D.L. Bartsch, H.H. Baruchel, A. Battista, C. Bay, J.-O. Bayik, M. Bazarbachi, A. Beguin, Y. López, J.L.B. Benedek, I. Benedetti, F. Bengala, C. Berrebi, A. Besalduch, J. Biesma, D. Biron, P. Björkholm, M. Blaise, D. Blesing, N.E. Boasson, M. Bobev, D. Boccadoro, M. Bolaman, Z. Boogaerts, M.A. Bordessoule, D. Bosi, A. Aida, B.S. Bourhis, J.H. Bourikas, G. Bowen, D.T. Bregni, M. Bries, G. Brinch, L. Brittain, D. Bron, D. Brune, M. Bullorsky, E.O. Bunjes, D. Burdach, S. Burnett, A.K. Buzyn, A. Caballero, D. Cagirgan, S. Cahn, J.-Y. Canepa, C.O. Cao, A. Carella, A.M. Carrera, F.D. Carret, A.-S. Cascinu, S. Castel, V. Caswell, M. Cavanna, L. Cetto, G.L. Chapuis, B. Chasty, R. Chen, Y.-C. Chisesi, T. Chopra, R. Chybicka, A. Clark, R.E. Colombat, P. Colovic, M.D. Constenla-Figueiras, M. Contreras, M. Contu, L. Cordonnier, C. Cornelissen, J.J. Cornish, J. Coser, P. Costa, N. Coze, C. Craddock, C. Crown, J. Culligan, D.J. Danova, M. Darbyshire, P.J. Davies, J.M. de Bock, R. de Pablos Gallego, J.M. De Prijck, B. de Revel, T. De Rossi, G. De Souza, C.A. Deb, G. Degos, L. Demuynck, H. Dervenoulas, I. Di Bartolomeo, P. Di Renzo, N. Diaz, M.A. Diehl, V. Diez-Martin, J.L. Dincer, S. Giorgio, D. Dmoszynska, A. Doelken, G. Peter, P.D. Dulley, F. Easow, J. Ebell, W. Efremidis, A. Ehninger, G. Eichler, H. Eimermacher, H. Enno, A. Errazquin, L. Aguado, J.E. Everaus, H. Fagioli, F. Fanin, R. Fassas, A. Fasth, A. Faulkner, L.B. Fauser, A.A. Feldman, L. Feremans, W. Ferhanoglu, B. Fernández, M.N. Fernández-Ranada, J.M. Ferrant, A. Ferrara, F. Finke, J. Fischer, A. Fischer, J. Fitzsimons, T. Floristan, F. Forjaz de Lacerda, J.M.F. Fossati-Bellani, F. Fosser, V. Franklin, I. Freund, M. Frickhofen, N. Gabbas, A. Gadner, H. Gallamini, A. Galvin, M.C. López, J.G. García-Conde, J. Gaska, T. Gastl, G. Gedikoglu, G. Ghavamzadeh, A. Gianni, A. Gibson, B.E. Gil, J.L. Gilleece, M.H. Gisselbrecht, C. Glass, B. Gmür, J. Göbel, U. Goldman, J.M. Goldstone, A.H. San Miguel, J.D.G. González-López, M.-A. Grafakos, S. Gramatzki, M. Grañena, A. Gratecos, N. Gratwohl, A. Greinix, H.T. Gugliotta, L. Guilhot, F. Guimaraes, J.E. Gülbas, Z. Gulyuz, O. Gurman, G. Gutierrez, M.M. Haas, R. Hamladji, R.-M. Hamon, M.D. Hansen, N.E. Harhalakis, N. Harousseau, J.L. Hartenstein, R. Hartmann, C.O. Hausmaninger, H. Haznedar, R. Heit, W. Hellmann, A. Herrmann, R.P. Hertenstein, B. Hess, U. Hinterberger, W. Ho, A.D. Hoelzer, D. Holowiecki, J. Horst, H.-A. Hossfeld, D.K. Huebsch, L. Hunter, A.E. Iacopino, P. Iannitto, E. Indrák, K. Iriondo, A. Izzi, T. Jackson, G.L. Jacobs, P. Jacobsen, N. Janvier, M. Jebavy, L. Joensuu, H. Joerg, S. Jones, F.G.C. Jouet, J.P. Joyner, M.V. Juliusson, G. Jürgens, H. Kalayoglu-Besisik, S. Kalman, N. Kalmanti, M. Kansoy, S. Kansu, E. Kanz, L. Karianakis, G. Kernéis, Y. Khalifeh, O. Khomenko, V. Kienast, J. Killick, S. Kirchner, H.H. Klingebiel, T. Knauf, W. Koenigsmann, M. Koistinen, P. Koivunen, E. Kolb, H.-J. Kolbe, K. Koller, E. Komarnicki, M. Koscielniak, E. Kovacsovics, T. Kowalczyk, J.R. Koza, V. Kozak, T. Kugler, J. Kuliczkowski, K. Kvaloy, S. Labar, B. Laciura, P. Palacios, J.J.L. Lakota, J. Lambertenghi, D.G. Lange, A. Lanza, F. Isasti, R.L. Lauria, F. Le Moine, F. Leblond, V. Lelli, G. Lenhoff, S. Leon, L.A. Leoncini-Franscini, L. Leone, G. Leoni, P. Levis, A. Leyvraz, S. Liberati, M. Link, H. Linkesch, W. Liso, V. Lisukov, I.A. Littlewood, T. Ljungman, P. Locatelli, F. Losonczy, H. Lotz, J.-P. Ludwig, H. Lukac, J. Lutz, D. Macchia, P. Madrigal, A. Maiolino, A. Majolino, I. Eloy-García, J.M. Malesevic, M. Mandelli, F. Marc, A. Marcus, R. Marianska, B. Markuljak, I. Marsh, J.C.W. Martelli, M.F. Marti Tutusaus, J.M. Martin, S. Martin, M. Martinelli, G. Martínez-Rubio, A.M. Martoni, A. Maschan, A. Maschmeyer, G. Masszi, T. Mazza, P. McCann, S. Meier, C.R. Messina, C. Mettivier, V. Metzner, B. Michallet, M. Michieli, M. Michon, J. Milligan, D.W. Milone, J.H. Giuseppe, G.M. Minigo, H. Mistrik, M. Moicean, A.D. Monfardini, S. Montserrat, E. Moraleda Jimenez, J.M. Morales-Lazaro, A. Morandi, S. Morra, E. Mufti, G.J. Musso, M. Nagler, A. Nalli, G. Naparstek, E. Narni, F. Nenadov-Beck, M. Neubauer, A. Newland, A.C. Niederwieser, D. Niethammer, D. Noens, L.A. Nousiainen, T. Novik, A. Novitzky, N. Occhini, D. Odriozolas, J. Ojanguren, J.M. O’meara, A. Onat, H. Orchard, K. Ortega, J.J. Osieka, R. Ossenkoppele, G.J. Othman, B. Ovali, E. Ozcebe, O.I. Ozerkan, K. Ozturk, A. Papatryfonos, A. Parker, J.E. Pastore, M. Patrone, F. Patton, N. Pejin, D. Peñarrubia, M.J. Equiza, E.P. Peschel, C. Pession, A. Pigaditou, A. Pignon, B. Pihkala, U. Pimentel, P. Pitini, V. Podoltseva, E. Pogliani, E.M. Anna, A.P. Porta, F. Potter, M. Powles, R. Prentice, G.H. Pretnar, J. Ptushkin, V. Quarta, G. Reiter, A. Remes, K. Reykdal, S. Santasusana, J.M.R. Rifón, J. Rio, B. Rizzoli, V. Robak, T. Robinson, A.J. Rodeghiero, F. Rodríguez Fernández, J.M. Rombos, Y. Romeril, K.R. Rosenmayr, A. Rossi, J.F. Rosti, G. Rotoli, B. Rowe, J.M. Russell, N.H. Ryzhak, O. Rzepecki, P. Saglio, G. Salwender, H. Samonigg, H. Santoro, A. Sanz, M.A. Sayer, H.G. Scanni, A. Schaafsma, M.R. Schaefer, U.W. Schanz, U. Schattenberg, A. Schey, S.A.M. Schlimok, G. Schmoll, H.-J. Schots, R. Schouten, H. Schwarer, A.P. Schwerdtfeger, R. Scimè, R. Segel, E. Seger, R. Selleslag, D. Serban, M. Shamaa, S. Shaw, P.J. Siegert, W. Siena, S. Sierra, J. Simonsson, B. Singer, C.R.J. Sirchia, G. Skotnicki, A.B. Slavin, S. Snowden, J. Sotto, J.J. Tanyeli, A. Tedeschi, L. Tidefelt, U. Tissot, J.-D. Tobler, A. Tomas, J.F. Torres, J.P. Torres, G.A. Touraine, J.-L. Trneny, M. Uderzo, C. Unal, E. Unal, A. Undar, L. Urban, C. Van den Berg, H. van Marwijk, K.M. Vellenga, E. Venturini, M. Verdonck, L.F. Veys, P. Vilardell, J. Vinante, O. Visani, G. Vitek, A. Vivancos, P. Volpe, E. Vora, A. Vorlicek, J. Vowels, M. Vujic, D. Wachowiak, J. Wagner, T. Wahlin, A. Walewski, J. Wandt, H. Weissinger, F. Wijermans, P.W. Wiktor-Jedrzejczak, W. Will, A.M. Woell, E. Wörmann, B. Yaniv, I. Yesilipek, M.A. Yilmaz, U. Yong, A. Zachée, P. Zambelli, A. Zander, A.R. Zintl, F. Zoumbos, N.C. The Acute Leukemia Working Party (ALWP) of the European Cooperative Group for Blood Marrow Transplantation (EBMT)

Abstract

Several studies have compared bone marrow (BM) and peripheral blood (PB) as stem cell sources in patients receiving allografts, but the cell doses infused have not been considered, especially for BM. Using the ALWP/EBMT registry, we retrospectively studied 881 adult patients with acute myelocytic leukemia (AML), who received a non-T-depleted allogeneic BM (n = 515) or mobilized PB (n = 366) standard transplant, in first remission (CR1), from an HLA-identical sibling, over a 5-year period from January 1994. The BM cell dose ranged from 0.17 to 29 × 108/kg with a median of 2.7 × 108/kg. The PB cell dose ranged from 0.02 to 77 × 10 8/kg with a median of 9.3 × 108/kg. The median dose for patients receiving BM (2.7 × 108/kg) gave the greatest discrimination. In multivariate analyses, high-dose BM compared to PB was associated with lower transplant-related mortality (RR = 0.61; 95% CI, 0.39-0.98; P = .04), better leukemia-free survival (RR = 0.65; 95% CI, 0.46-0.91; P = .013), and better overall survival (RR = 0.64; 95% CI, 0.44-0. 92; P = .016). The present study in patients with AML receiving allografts in first remission indicates a better outcome with BM as compared to PB, when the dose of BM infused is rich. © 2003 by The American Society of Hematology.

Published
2003

140. Common genetic variation at 15q25.2 impacts on chronic lymphocytic leukaemia risk

Author

Crowther Swanepoel, D, Di Bernardo, Mc, Jamroziak, K, Karabon, L, Frydecka, I, Deaglio, S, D'Arena, G, Rossi, Dario, Gaidano, G, Olver, B, Lloyd, A, Broderick, P, Laurenti, Luca, Szemraj Rogucka, Z, Robak, T, Catovsky, D, Houlston, Rs, Laurenti, Luca (ORCID:0000-0002-8327-1396), Crowther Swanepoel, D, Di Bernardo, Mc, Jamroziak, K, Karabon, L, Frydecka, I, Deaglio, S, D'Arena, G, Rossi, Dario, Gaidano, G, Olver, B, Lloyd, A, Broderick, P, Laurenti, Luca, Szemraj Rogucka, Z, Robak, T, Catovsky, D, Houlston, Rs, and Laurenti, Luca (ORCID:0000-0002-8327-1396)

Abstract

A genome-wide association study of chronic lymphocytic leukaemia (CLL) suggested that common variants at 15q25.2 (rs783540) and 18q21.1 (rs1036935) influence CLL. To validate these associations and explore their relationship with CLL risk we genotyped case-control datasets from Poland, UK and Italy totalling 1428 cases and 1920 controls. Combined data from these and previously genotyped series (2503 cases and 5789 controls) provided evidence for an association between 15q25.2 and 18q21.1 loci and CLL risk (P(combined) = 1·10 × 10(-7) and 1·30 × 10(-5) respectively). These data provide further evidence for the involvement of common genetic variants in CLL risk and insight into the biological basis of disease development.

Published
2011

141. Combined pegylated liposomal doxorubicin and bortezomib is highly effective in patients with recurrent or refractory multiple myeloma who received prior thalidomide/lenalidomide therapy.

Author

Sonneveld, P., Hajek, R., Nagler, A., Spencer, A., Blade, J., Robak, T., Zhuang, S.H., Harousseau, J.L., Orlowski, R.Z., Raymakers, R.A.P., Sonneveld, P., Hajek, R., Nagler, A., Spencer, A., Blade, J., Robak, T., Zhuang, S.H., Harousseau, J.L., Orlowski, R.Z., and Raymakers, R.A.P.

Abstract

Item does not contain fulltext, BACKGROUND: Recently, the authors reported improved time to disease progression (TTP) with a combination of pegylated liposomal doxorubicin (PLD) and bortezomib compared with bortezomib alone in a phase 3 randomized trial in patients with recurrent/refractory multiple myeloma (MM). In the current analysis, they determined 1) the efficacy of PLD plus bortezomib versus bortezomib alone in patients with MM who had failed on prior thalidomide/lenalidomide (immunomodulatory drug [IMiD]) treatment and 2) the efficacy and safety profile of PLD plus bortezomib in IMiD-exposed and IMiD-naive patients. METHODS: This prespecified analysis included 646 patients who were randomized to receive either PLD with bortezomib (n=324; 194 IMiD-naive patients and 130 IMiD-exposed patients) or bortezomib alone (n=322; 184 IMiD-naive patients and 138 IMiD-exposed patients). The primary efficacy endpoint was TTP, and secondary endpoints included overall survival, response rate, and safety. RESULTS: The median TTP was significantly longer with PLD plus bortezomib compared with bortezomib alone in IMiD-exposed patients (270 days vs 205 days). No statistical difference was noted with respect to TTP between IMiD-naive (295 days) versus IMiD-exposed (270 days) subgroups who received PLD plus bortezomib. A sustained trend favoring combination therapy was observed in analyses of overall survival. In patients who achieved a response, the response duration was comparable for IMiD-naive patients and IMiD-exposed patients in the combination treatment group and lasted a median of 310 days and 319 days, respectively. The incidence of grade 3/4 adverse events was similar with PLD plus bortezomib regardless of prior IMiD exposure. CONCLUSIONS: A significantly prolonged TTP was observed with combined PLD plus bortezomib combination therapy compared with bortezomib alone despite prior IMiD exposure. For the combination treatment arm in the IMiD-naive and IMiD-exposed subgroups, TTP was comparable. Similarly

Published
2008

142. First clinical use of ofatumumab, a novel fully human anti-CD20 monoclonal antibody in relapsed or refractory follicular lymphoma: Results of a phase 1/2 trial

Author

Hagenbeek, A. (Anton), Gadeberg, O. (Ole), Johnson, P. (Jürgen), Pedersen, L.M., Walewski, J. (Jan), Hellmann, A. (Andrzej), Link, B.K. (Brian), Robak, T. (Tadeusz), Wojtukiewicz, M. (Marek), Pfreundschuh, M. (Michael), Kneba, M. (Michael), Engert, A. (Andreas), Sonneveld, P. (Pieter), Flensburg, M. (Mimi), Petersen, J. (Jørgen), Losic, N. (Nedjad), Radford, J. (John), Hagenbeek, A. (Anton), Gadeberg, O. (Ole), Johnson, P. (Jürgen), Pedersen, L.M., Walewski, J. (Jan), Hellmann, A. (Andrzej), Link, B.K. (Brian), Robak, T. (Tadeusz), Wojtukiewicz, M. (Marek), Pfreundschuh, M. (Michael), Kneba, M. (Michael), Engert, A. (Andreas), Sonneveld, P. (Pieter), Flensburg, M. (Mimi), Petersen, J. (Jørgen), Losic, N. (Nedjad), and Radford, J. (John)

Abstract

Ofatumumab is a unique monoclonal antibody that targets a distinct small loop epitope on the CD20 molecule. Preclinical data show that ofatumumab is active against B-cell lymphoma/chronic lymphocytic leukemia cells with low CD20-antigen density and high expression of complement inhibitory molecules. In a phase 1/2 trial evaluating safety and efficacy of ofatumumab in relapsed or refractory follicular non-Hodgkin lymphoma (FL) grade 1 or 2, 4 dose groups of 10 patients received 4 weekly infusions of 300, 500, 700, or 1000 mg. Patients had a median of 2 prior FL therapies and 13% had elevated lactate dehydrogenase. No safety concerns or maximum tolerated dose was identified. A total of 274 adverse events were reported; 190 were judged related to ofatumumab, most occurring on the first infusion day with Common Terminology Criteria grade 1 or 2. Eight related events were grade 3. Treatment caused immediate and profound B-cell depletion, and 65% of patients reverted to negative BCL2 status. Clinical response rates ranged from 20% to 63%. Median time to progression for all patients/ responders was 8.8/ 32.6 months, and median duration of response was 29.9 months at a median/ maximum follow-up of 9.2/38.6 months. Ofatumumab is currently being evaluated in patients with rituximab-refractory FL. This trial was registered at www.clinicaltrials.gov as #NCT00092274.

Published
2008
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143. Combined pegylated liposomal doxorubicin and bortezomib is highly effective in patients with recurrent or refractory multiple myeloma who received prior thalidomide/lenalidomide therapy

Author

Sonneveld, P. (Pieter), Hajek, R. (Roman), Nagler, A. (Arnon), Spencer, A. (Andrew), Bladé, J. (Joan), Robak, T. (Tadeusz), Zhuang, S.H. (Sen), Harousseau, J-L. (Jean-Luc), Orlowski, R.Z. (Robert), Sonneveld, P. (Pieter), Hajek, R. (Roman), Nagler, A. (Arnon), Spencer, A. (Andrew), Bladé, J. (Joan), Robak, T. (Tadeusz), Zhuang, S.H. (Sen), Harousseau, J-L. (Jean-Luc), and Orlowski, R.Z. (Robert)

Abstract

BACKGROUND. Recently, the authors reported improved time to disease progression (TTP) with a combination of pegylated liposomal doxorubicin (PLD) and bortezomib compared with bortezomib alone in a phase 3 randomized trial in patients with recurrent/refractory multiple myeloma (MM). In the current analysis, they determined 1) the efficacy of PLD plus bortezomib versus bortezomib alone in patients with MM who had failed on prior thalidomide/lenalidomide (immunomodulatory drug [IMiD]) treatment and 2) the efficacy and safety profile of PLD plus bortezomib in IMiD-exposed and IMiD-naive patients. METHODS. This prespecified analysis included 646 patients who were randomized to receive either PLD with bortezomib (n = 324; 194 IMiD-naive patients and 130 IMiD-exposed patients) or bortezomib alone (n = 322; 184 IMiD-naive patients and 138 IMiD-exposed patients). The primary efficacy endpoint was TTP, and secondary endpoints included overall survival, response rate, and safety. RESULTS. The median TTP was significantly longer with PLD plus bortezomib compared with bortezomib alone in IMiD-exposed patients (270 days vs 205 days). No statistical difference was noted with respect to TTP between IMiD-naive (295 days) versus IMiD-exposed (270 days) subgroups who received PLD plus bortezomib. A sustained trend favoring combination therapy was observed in analyses of overall surviv

Published
2008
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144. Safety and efficacy of ofatumumab, a fully human monoclonal anti-CD20 antibody, in patients with relapsed or refractory B-cell chronic lymphocytic leukemia: a phase 1-2 study

Author

Coiffier, B., Lepretre, S., Pedersen, L.M., Gadeberg, O., Fredriksen, H., Oers, M.H.J. van, Wooldridge, J., Kloczko, J., Holowiecki, J., Hellmann, A., Walewski, J., Flensburg, M., Petersen, J., Robak, T., Pedersen, Lars Møller, Coiffier, B., Lepretre, S., Pedersen, L.M., Gadeberg, O., Fredriksen, H., Oers, M.H.J. van, Wooldridge, J., Kloczko, J., Holowiecki, J., Hellmann, A., Walewski, J., Flensburg, M., Petersen, J., Robak, T., and Pedersen, Lars Møller

Abstract

Safety and efficacy of the fully human anti-CD20 monoclonal antibody, ofatumumab, was analyzed in a multicenter dose-escalating study including 33 patients with relapsed or refractory chronic lymphocytic leukemia. Three cohorts of 3 (A), 3 (B), and 27 (C) patients received 4, once weekly, infusions of ofatumumab at the following doses: (A) one 100 mg and three 500 mg; (B) one 300 mg and three 1000 mg; (C) one 500 mg and three 2000 mg. Sixty-seven percent of the patients were Binet stage 13, and the median number of previous treatments was 3. The maximum tolerated dose was not reached. The majority of related adverse events occurred at first infusion, and the number of adverse events decreased at each subsequent infusion. Seventeen (51%) of 33 patients experienced infections, 88% of them of grade 1-2. One event of interstitial pneumonia was fatal; all other cases resolved within one month. The response rate of cohort C was 50% (13/26), one patient having a nodular partial remission and 12 patients partial remission. In conclusion, ofatumumab was found to be well tolerated in patients with chronic lymphocytic leukemia (CLL) in doses up to 2000 mg. Preliminary data on safety and objective response are encouraging and support further studies on the role of ofatumumab in CILL patients. This trial was registered at www.clinicaltrials.gov as no. INCT000933114 Udgivelsesdato: 2008/2/1

Published
2008

145. Znaczenie polimorfizmów genetycznych w obrębie nieklasycznej cząsteczki G głównego układu zgodności tkankowej człowieka w przewlekłej białaczce limfocytowej

Author

Klimkiewicz-Wojciechowska, G., primary, Grzybowska-Izydorczyk, O., additional, Borowiec, M., additional, Wyka, K., additional, Chmielewska, M., additional, Cebula-Obrzut, B., additional, Makuch-Łasica, H., additional, Robak, T., additional, Warzocha, K., additional, Młynarski, W., additional, and Lech-Marańda, E., additional

Published
2013
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146. Ocena skuteczności i bezpieczeństwa leczenia bortezomibem chorych na szpiczaka plazmocytowego w Polsce – raport Polskiej Grupy Szpiczakowej (PGSz)

Author

Walter-Croneck, A., primary, Grząśko, N., additional, Soroka-Wojtaszko, M., additional, Torosian, T., additional, Jedrzejczak, W.W., additional, Jurczyszyn, A., additional, Skotnicki, A., additional, Rymko, M., additional, Całbecka, M., additional, Lech-Marańda, E., additional, Mądro, E., additional, Zielińska, P., additional, Grygoruk-Wiśniowska, I., additional, Kyrcz-Krzemień, S., additional, Dawidowska, D., additional, Gawron, L., additional, Hołojda, J., additional, Nowicki, A., additional, Komarnicki, M., additional, Błońska, D., additional, Gadomska, G., additional, Druzd-Sitek, A., additional, Walewski, J., additional, Potoczek, S., additional, Usnarska-Zubkiewicz, L., additional, Kuliczkowski, K., additional, Olszewska-Szopa, M., additional, Rzepecki, P., additional, Iskierka, E., additional, Robak, T., additional, Barchnicka, A., additional, Grosicki, S., additional, Masternak, A., additional, Woszczyk, D., additional, Dmoszyńska, A., additional, Kozińska, J., additional, Boguradzki, P., additional, Charliński, G., additional, Rybicka, M., additional, Dzierżak-Mietła, M., additional, Wiśniewska-Piąty, K., additional, Gontarska, A., additional, Kosmala-Niewiadomska, W., additional, Subocz, E., additional, Hałka, J., additional, Kopacz, A., additional, Blajer, B., additional, and Świderska, A., additional

Published
2013
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147. Ocena skuteczności poszczególnych kursów terapii indukującej i konsolidującej dorosłych chorych na ostrą białaczkę limfoblastyczną z uwzględnieniem odpowiedzi na poziomie minimalnej choroby resztkowej. Analiza pośrednia badania PALG ALL 5-2007

Author

Giebel, S., primary, Jagoda, K., additional, Szmigielska, A., additional, Krawczyk-Kuliś, M., additional, Lech-Marańda, E., additional, Paluszewska, M., additional, Czyż, A., additional, Piszcz, J., additional, Piątkowska-Jakubas, B., additional, Adamczyk-Cioch, M., additional, Hałka, J., additional, Robak, T., additional, Kyrcz-Krzemień, S., additional, Warzocha, K., additional, Jędrzejczak, W.W., additional, Komarnicki, M., additional, Kłoczko, J., additional, Skotnicki, A.B., additional, Pasiarski, M., additional, Hellmann, A., additional, Rzepecki, P., additional, Raźny, M., additional, and Hołowiecki, J., additional

Published
2013
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