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2,530 results on '"Baccarani, M"'

1. European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia

Author

Hochhaus, A., Baccarani, M., Silver, R. T., Schiffer, C., Apperley, J. F., Cervantes, F., Clark, R. E., Cortes, J. E., Deininger, M. W., Guilhot, F., Hjorth-Hansen, H., Hughes, T. P., Janssen, J. J. W. M., Kantarjian, H. M., Kim, D. W., Larson, R. A., Lipton, J. H., Mahon, F. X., Mayer, J., Nicolini, F., Niederwieser, D., Pane, F., Radich, J. P., Rea, D., Richter, J., Rosti, G., Rousselot, P., Saglio, G., Saußele, S., Soverini, S., Steegmann, J. L., Turkina, A., Zaritskey, A., and Hehlmann, R.

Published
2020
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2. A Phase 2 Trial of Ponatinib in Philadelphia Chromosome–Positive Leukemias

Author

Cortes, JE, Kim, D-W, Pinilla-Ibarz, J, le Coutre, P, Paquette, R, Chuah, C, Nicolini, FE, Apperley, JF, Khoury, HJ, Talpaz, M, DiPersio, J, DeAngelo, DJ, Abruzzese, E, Rea, D, Baccarani, M, Müller, MC, Gambacorti-Passerini, C, Wong, S, Lustgarten, S, Rivera, VM, Clackson, T, Turner, CD, Haluska, FG, Guilhot, F, Deininger, MW, Hochhaus, A, Hughes, T, Goldman, JM, Shah, NP, and Kantarjian, H

Subjects
Hematology, Clinical Research, Cancer, Rare Diseases, 6.1 Pharmaceuticals, 6.2 Cellular and gene therapies, Evaluation of treatments and therapeutic interventions, Adolescent, Adult, Aged, Aged, 80 and over, Female, Humans, Imidazoles, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Male, Middle Aged, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Protein Kinase Inhibitors, Pyridazines, Thrombocytopenia, Thrombosis, Young Adult, PACE Investigators, Medical and Health Sciences, General & Internal Medicine
Abstract

BackgroundPonatinib is a potent oral tyrosine kinase inhibitor of unmutated and mutated BCR-ABL, including BCR-ABL with the tyrosine kinase inhibitor-refractory threonine-to-isoleucine mutation at position 315 (T315I). We conducted a phase 2 trial of ponatinib in patients with chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-positive ALL).MethodsWe enrolled 449 heavily pretreated patients who had CML or Ph-positive ALL with resistance to or unacceptable side effects from dasatinib or nilotinib or who had the BCR-ABL T315I mutation. Ponatinib was administered at an initial dose of 45 mg once daily. The median follow-up was 15 months.ResultsAmong 267 patients with chronic-phase CML, 56% had a major cytogenetic response (51% of patients with resistance to or unacceptable side effects from dasatinib or nilotinib and 70% of patients with the T315I mutation), 46% had a complete cytogenetic response (40% and 66% in the two subgroups, respectively), and 34% had a major molecular response (27% and 56% in the two subgroups, respectively). Responses were observed regardless of the baseline BCR-ABL kinase domain mutation status and were durable; the estimated rate of a sustained major cytogenetic response of at least 12 months was 91%. No single BCR-ABL mutation conferring resistance to ponatinib was detected. Among 83 patients with accelerated-phase CML, 55% had a major hematologic response and 39% had a major cytogenetic response. Among 62 patients with blast-phase CML, 31% had a major hematologic response and 23% had a major cytogenetic response. Among 32 patients with Ph-positive ALL, 41% had a major hematologic response and 47% had a major cytogenetic response. Common adverse events were thrombocytopenia (in 37% of patients), rash (in 34%), dry skin (in 32%), and abdominal pain (in 22%). Serious arterial thrombotic events were observed in 9% of patients; these events were considered to be treatment-related in 3%. A total of 12% of patients discontinued treatment because of an adverse event.ConclusionsPonatinib had significant antileukemic activity across categories of disease stage and mutation status. (Funded by Ariad Pharmaceuticals and others; PACE ClinicalTrials.gov number, NCT01207440 .).

Published
2013

3. Differences among young adults, adults and elderly chronic myeloid leukemia patients

Author

Salvi, F., Pini, M., Leoni, P., Rupoli, S., Galieni, P., Bigazzi, C., Cantore, N., Palmieri, F., Albano, F., Russo Rossi, A., Rambaldi, A., Intermesoli, T., Palandri, F., Testoni, N., Luatti, S., Soverini, S., Iacobucci, I., Bochicchio, M.T., Apolinari, M., Fogli, M., Cervello, I., Capucci, A., Malagola, M., Malpignano, A., Girasoli, M., Angelucci, E., Usala, E., Storti, S., De Biasi, E., Tagariello, G., Sartori, R., Di Raimondo, F., Vigneri, P., Impera, S., Molica, S., Lanza, F., Viganò, C., Grasso, M., Rapezzi, D., Cavazzini, F., Bosi, A., Santini, V., Capalbo, S.F., Spinosa, G., Pierri, I., Bergamaschi, M., Carella, A.M., Bacigalupo, A., De Blasio, A., Ciccone, F., Di Renzo, N., Musolino, C., Russo, S., Cortelezzi, A., Morra, E., Pungolino, E.M., Luppi, M., Marasca, R., Pogliani, E.M., Gambacorti-Passerini, C., Luciano, L., Ferrara, F., Annunziata, M., Latte, G., Noli, D., Rege-Cambrin, G., Fava, C., Semenzato, G., Binotto, G., Fabbiano, F., Turri, D., Siragusa, S., Caracciolo, C., Musso, M., Porretto, F., Aversa, F., Crugnola, M., Cazzola, M., Orlandi, E., Falini, B., Falzetti, F., Visani, G., Isidori, A., Fioritoni, G., Di Lorenzo, R., Vallisa, D., Trabacchi, E., Petrini, M., Galimberti, S., Pizzuti, M., Zaccaria, A., Salvucci, M., Ronco, F., Ielo, D., Merli, F., Avanzini, P., Tosi, P., Merli, A., Musto, P., De Stefano, V., Sica, S., Latagliata, R., De Fabritiis, P., Trawiska, M., Majolino, I., Pacilli, L., Ronci, B., Cedrone, M., Petti, M.C., Pisani, F., Tafuri, A., Montefusco, E., Iuliano, F., Dore, F., Pardini, S., Bocchia, M., Defina, M., Liberati, A.M., Luzzi, D., Boccadoro, M., Ferrero, D., Vitolo, U., Gherlinzoni, F., Calistri, E., Fanin, R., Pizzolo, G., Meneghini, V., Rodighiero, F., D'Emilio, A., Castagnetti, F., Gugliotta, G., Baccarani, M., Breccia, M., Specchia, G., Levato, L., Abruzzese, E., Rossi, G., Iurlo, A., Martino, B., Pregno, P., Stagno, F., Cuneo, A., Bonifacio, M., Gobbi, M., Russo, D., Gozzini, A., Tiribelli, M., de Vivo, A., Alimena, G., Cavo, M., Martinelli, G., Pane, F., Saglio, G., and Rosti, G.

Published
2015
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4. Treatment and outcome of 2904 CML patients from the EUTOS population-based registry

Author

Hoffmann, V S, Baccarani, M, Hasford, J, Castagnetti, F, Di Raimondo, F, Casado, L F, Turkina, A, Zackova, D, Ossenkoppele, G, Zaritskey, A, Höglund, M, Simonsson, B, Indrak, K, Sninska, Z, Sacha, T, Clark, R, Bogdanovic, A, Hellmann, A, Griskevicius, L, Schubert-Fritschle, G, Sertic, D, Guilhot, J, Lejniece, S, Zupan, I, Burgstaller, S, Koskenvesa, P, Everaus, H, Costeas, P, Lindoerfer, D, Rosti, G, Saussele, S, Hochhaus, A, and Hehlmann, R

Published
2017
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11. The EUTOS population-based registry: incidence and clinical characteristics of 2904 CML patients in 20 European Countries

Author

Hoffmann, V S, Baccarani, M, Hasford, J, Lindoerfer, D, Burgstaller, S, Sertic, D, Costeas, P, Mayer, J, Indrak, K, Everaus, H, Koskenvesa, P, Guilhot, J, Schubert-Fritschle, G, Castagnetti, F, Di Raimondo, F, Lejniece, S, Griskevicius, L, Thielen, N, Sacha, T, Hellmann, A, Turkina, A G, Zaritskey, A, Bogdanovic, A, Sninska, Z, Zupan, I, Steegmann, J-L, Simonsson, B, Clark, R E, Covelli, A, Guidi, G, and Hehlmann, R

Published
2015
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12. The proportion of different BCR-ABL1 transcript types in chronic myeloid leukemia. An international overview

Author

Baccarani, M, Castagnetti, F, Gugliotta, G, Rosti, G, Soverini, S, Albeer, A, Pfirrmann, M, Bekadja, Ma, Entasoltan, B, Nachi, M, Elghandour, A, El Sorady, M, Abdelfattah, R, El Nahass, Y, Samra, M, Azzazi, M, Elsobki, E, Moussa, M, Fahmy, O, Mattar, M, Shehata, Azmy, Se, (Azmy, E, 9 ), Emad), Bolarinwa, (Bolarinwa, Ra, ( 10 ), Rahman A., Eid, (Eid, S, Samir)( 11, ), Khelif, (Khelif, A, Abderrhaim)( 11, ), Hached, (Hached, F, Farhat)( 11, ), Menif, (Menif, S, Samia)( 12, ), Rahman, (Rahman, H, Hafizur)( 13, ), Huang, (Huang, Xj, Xiaojun)(, 14, 15, ), Jiang, (Jiang, Q, Qian)(, 14, (Ye, Yx, Yuanxin)( 16, ), Zhu, (Zhu, Hl, Huanling)( 16, ), Chen, (Chen, Sn, Suning)( 17, ), Varma, (Varma, N, Neelam)( 18, ), Ganesan, (Ganesan, P, Prasanth)( 19, ), Gundeti, (Gundeti, S, Sadashivudu)( 20, ), Malhotra, (Malhotra, H, Hemant)( 21, ), Radhakrishnan, (Radhakrishnan, Vs, ( 22 ), Vivek S., Kumar, (Kumar, L, Lalit)( 23, ), Sharawat, (Sharawat, Sk, Surender Kumar)( 23, ), Seth, (Seth, T, Tulika)( 24, ), Ausekar, (Ausekar, Bv, ( 25 ), B. V., Balasubramanian, (Balasubramanian, P, Poonkuzhali)( 26, ), Poopak, (Poopak, B, Behzad)(, 27, 28, ), Inokuchi, (Inokuchi, K, Koiti)( 29, ), Kim, (Kim, Dw, Dong-Wook)( 30, ), Kindi, Al, S (Al Kindi, Salam)( 31, ), Mirasol, (Mirasol, A, Angelina)( 32, ), Qari, (Qari, M, Mohammed)( 33, ), Goh, (Goh, Yt, Yeow Tee)( 34, ), Shih, (Shih, Ly, Lee-Yung)(, 35, 36, ), Branford, (Branford, S, Susan)(, 37, 38, ), Lion, (Lion, T, Thomas)( 39, ), Valent, (Valent, P, Peter)( 40, ), Burgstaller, (Burgstaller, S, Sonja)( 41, ), Thaler, (Thaler, J, Joseph)( 41, ), Labar, (Labar, B, Boris)( 42, ), Zadro, (Zadro, R, Renata)( 42, ), Mayer, (Mayer, J, Jiri)(, 43, 44, ), Zackova, (Zackova, D, Daniela)(, 43, Faber, (Faber, E, Edgar)( 45, ), Pallisgaard, (Pallisgaard, N, Niels)( 46, ), Xavier-Mahon, (Xavier-Mahon, F, Francois)( 47, ), Lippert, (Lippert, E, Eric)( 48, ), Cayuela, (Cayuela, Jm, Jean Michel)( 49, ), Rea, (Rea, D, Delphine)( 49, ), Millot, (Millot, F, Frederic)( 50, ), Suttorp, (Suttorp, M, Meinolf)( 51, ), Hochhaus, (Hochhaus, A, Andreas)( 52, ), Niederwieser, (Niederwieser, D, Dietger)( 53, ), Saussele, (Saussele, S, Susanne)( 54, ), Haferlach, (Haferlach, T, Torsten)( 55, ), Jeromine, (Jeromine, S, Sabine)( 55, ), Panayiotidis, (Panayiotidis, P, Panayiotis)(, 56, 57, ), Conneally, (Conneally, E, Eibhlin)( 58, ), Langabeer, (Langabeer, S, Steve)( 58, ), Nagler, (Nagler, A, Arnon)(, 59, 60, ), Rupoli, (Rupoli, S, Serena)( 61, ), Santoro, (Santoro, N, Nicola)( 62, ), Albano, (Albano, F, Francesco)( 63, ), Castagnetti, (Castagnetti, F, Fausto), Ottaviani, (Ottaviani, E, Emanuela)(, 64, 65, ), Rambaldi, (Rambaldi, A, Alessandro)(, 66, 67, ), Stagno, (Stagno, F, Fabio)( 68, ), Molica, (Molica, S, Stefano)( 69, ), Biagiotti, (Biagiotti, C, Caterina)( 70, ), Scappini, (Scappini, B, Barbara)( 70, ), Lemoli, (Lemoli, R, Roberto)( 71, ), Iurlo, (Iurlo, A, Alessandra)(, 72, 73, ), Pungolino, (Pungolino, E, Ester)( 74, ), Menna, (Menna, G, Giuseppe), Pane, (Pane, F, Fabrizio)( 76, ), Gottardi, (Gottardi, E, Enrico)(, 77, 78, ), Rege-Cambrin, (Rege-Cambrin, G, Giovanna)(, 77, Binotto, (Binotto, G, Gianni)( 79, ), Putti, (Putti, Mc, Maria Caterina)( 80, ), Falzetti, (Falzetti, F, Franca)( 81, ), Visani, (Visani, G, Giuseppe)( 82, ), Galimberti, (Galimberti, S, Sara)( 83, ), Musto, (Musto, P, Pellegrino)( 84, ), Abruzzese, (Abruzzese, E, Elisabetta)( 85, ), Breccia, (Breccia, M, Massimo)( 86, ), Giona, (Giona, F, Fiorina)( 86, ), Chiusolo, (Chiusolo, P, Patrizia)( 87, ), Sica, (Sica, S, Simona)( 87, ), Fava, (Fava, C, Carmen)( 88, ), Ferrero, (Ferrero, D, Dario)( 88, ), Tiribelli, (Tiribelli, M, Mario)( 89, ), Bonifacio, (Bonifacio, M, Massimiliano)( 90, ), Griskevicius, (Griskevicius, L, Laimonas)( 91, ), Musteata, (Musteata, V, Vasile)( 92, ), Janssen, (Janssen, J, Jeroen)( 93, ), Prejzner, (Prejzner, W, Witold)( 94, ), Sacha, (Sacha, T, Tomasz)( 95, ), Waclaw, (Waclaw, J, Joanna)( 95, ), Almeida, (Almeida, Am, Antonio Medina)( 96, ), Kulikov, (Kulikov, S, Sergei)( 97, ), Turkina, (Turkina, A, Anna)( 97, ), Bogdanovic, (Bogdanovic, A, Andrija)( 98, ), Zupan, (Zupan, I, Irena)( 99, ), Marce, (Marce, S, Silvia)( 100, ), Cervantes, (Cervantes, F, Francisco)( 101, ), Steegmann, (Steegmann, Jl, Juan Luis)( 102, ), Kotlyarchuk, (Kotlyarchuk, K, Konstyantyn)( 103, ), Milner, (Milner, Bj, ( 104 ), Benedict J., Rose, (Rose, S, Susan)( 105, ), Clench, (Clench, T, Tim)( 106, ), Waits, (Waits, P, Paula)( 107, ), Austin, (Austin, S, Steve)( 108, ), Wickham, (Wickham, C, Caroline)( 109, ), Clark, (Clark, R, Richard)( 110, ), Apperley, (Apperley, J, Jane), Claudiani, (Claudiani, S, Simone)( 111, ), Foroni, (Foroni, L, Letizia)( 111, ), Szydlo, (Szydlo, R, Richard)( 111, ), Burt, (Burt, E, Emma)( 112, ), Bescoby, (Bescoby, R, Ruth)( 113, ), Cork, (Cork, L, Leanne)( 113, ), O'Brien, (O'Brien, S, Stephen)( 113, ), Green, (Green, B, Bethaney)( 114, ), Hawtree, (Hawtree, S, Sarah)( 114, ), Watson, (Watson, M, Mark)( 114, ), Bengio, (Bengio, Rm, Raquel Maria)( 115, ), Larripa, (Larripa, I, Irene)( 115, ), Pavlovsky, (Pavlovsky, C, Carolina)( 116, ), Moiraghi, (Moiraghi, B, Beatriz)( 117, ), Pinna, De, CAR (Requiao de Pinna, Cristiane Almeida)( 118, ), Magalhaes, GHR (Romani Magalhaes, Gustavo Henrique)( 119, ), Pagnano, (Pagnano, K, Katia)( 120, ), Funke, (Funke, V, Vaneuza)( 121, ), Tavares, (Tavares, Rs, Renato Sampaio)( 122, ), Prado, (Prado, A, Adriana)( 123, ), Azevedo, (Azevedo, Aa, Alita Andrade)( 124, ), Fogliatto, (Fogliatto, L, Laura)( 125, ), Bonecker, (Bonecker, S, Simone)( 126, ), Centrone, (Centrone, R, Renato)( 127, ), Moellman, (Moellman, A, Artur)( 128, ), Conchon, (Conchon, M, Monika)( 130, ), Centurion, (Centurion, Me, Maria Elida)( 131, ), (Prado, Ai, Ana-Ines)( 132, ), Lopez, (Lopez, Jl, ( 133 ), J. L., Petruzziello, (Petruzziello, F, Fara)( 75, ), Bendit, (Bendit, I, Israel), Baccarani M., Castagnetti F., Gugliotta G., Rosti G., Soverini S., Albeer A., and Pfirrmann M.

Subjects
Male, 0301 basic medicine, Cancer Research, bcr-abl, Fusion Proteins, bcr-abl, Global Health, 0302 clinical medicine, hemic and lymphatic diseases, 80 and over, Odds Ratio, Prevalence, Age Factor, Chronic, Young adult, Child, MOLECULAR RESPONSE, Leukemic, Aged, 80 and over, Leukemia, Hematology, Gene Expression Regulation, Leukemic, CHRONIC MYELOGENOUS LEUKEMIA, Age Factors, Myeloid leukemia, Middle Aged, Oncology, Child, Preschool, 030220 oncology & carcinogenesis, Female, Life Sciences & Biomedicine, Human, Adult, Transcriptional Activation, medicine.medical_specialty, Adolescent, Immunology, IMATINIB MESYLATE, DENDRITIC CELLS, CML PATIENTS, Young Adult, 03 medical and health sciences, Myelogenous, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Internal medicine, medicine, Humans, 1112 Oncology and Carcinogenesis, BCR/ABL TRANSCRIPT, Preschool, CYTOGENETIC RESPONSE, Aged, Science & Technology, CHRONIC-PHASE, business.industry, Infant, Newborn, Fusion Proteins, ABL FUSION PROTEINS, P190 BCR-ABL, Infant, 1103 Clinical Sciences, Odds ratio, Newborn, medicine.disease, International BCR-ABL Study Group, Settore MED/15 - MALATTIE DEL SANGUE, 030104 developmental biology, Imatinib mesylate, Gene Expression Regulation, BCR-ABL Positive, business, Chronic myelogenous leukemia
Abstract

There are different BCR-ABL1 fusion genes that are translated into proteins that are different from each other, yet all leukemogenic, causing chronic myeloid leukemia (CML) or acute lymphoblastic leukemia. Their frequency has never been systematically investigated. In a series of 45503 newly diagnosed CML patients reported from 45 countries, it was found that the proportion of e13a2 (also known as b2a2) and of e14a2 (also known as b3a2), including the cases co-expressing e14a2 and e13a2, was 37.9% and 62.1%, respectively. The proportion of these two transcripts was correlated with gender, e13a2 being more frequent in males (39.2%) than in females (36.2%), was correlated with age, decreasing from 39.6% in children and adolescents down to 31.6% in patients ≥ 80 years old, and was not constant worldwide. Other, rare transcripts were reported in 666/34561 patients (1.93%). The proportion of rare transcripts was associatedwith gender (2.27% in females and 1.69% in males) and with age (from 1.79% in children and adolescents up to 3.84% in patients ≥ 80 years old). These data show that the differences in proportion are not by chance. This is important, as the transcript type is a variable that is suspected to be of prognostic importance for response to treatment, outcome of treatment, and rate of treatment-free remission.

Published
2019

14. INCB84344-201: Ponatinib and steroids in frontline therapy for unfit patients with Ph1 acute lymphoblastic leukemia

Author

Martinelli, G., Papayannidis, C., Piciocchi, A., Robustelli, V., Soverini, S., Terragna, C., Marconi, G., Lemoli, R. M., Guolo, F., Fornaro, A., Lunghi, M., de Fabritiis, P., Candoni, A., Selleri, C., Simonetti, F., Bocchia, M., Vitale, A., Frison, L., Tedeschi, A., Cuneo, A., Bonifacio, M., Martelli, M. P., D'Ardia, S., Trappolini, S., Tosi, P., Galieni, P., Fabbiano, F., Abbenante, M. C., Granier, M., Zhu, Z., Wang, M., Sartor, C., Paolini, S., Cavo, M., Foa, R., Fazi, P., Vignetti, M., and Baccarani, M.

Published
2022

15. Therapy of Adult Acute Lymphoblastic Leukemia. Preliminary Results of the GIMEMA ALL/0288 Study

Author

Amadori, S., Annino, L., Ferrari, A., Vegna, M. L., Camera, A., Ciolli, S., Deplano, W., Fabiano, F., Ferrara, F., Ladogana, S., Muti, G., Peta, A., Recchia, A., Sica, S., Stasi, R., Tabilio, A., Visani, G., Baccarani, M., Mandelli, F., Büchner, T., editor, Hiddemann, W., editor, Wörmann, B., editor, Schellong, G., editor, and Ritter, J., editor

Published
1994
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16. Frontline nilotinib in patients with chronic myeloid leukemia in chronic phase: results from the European ENEST1st study

Author

Hochhaus, A, Rosti, G, Cross, N CP, Steegmann, J L, le Coutre, P, Ossenkoppele, G, Petrov, L, Masszi, T, Hellmann, A, Griskevicius, L, Wiktor-Jedrzejczak, W, Rea, D, Coriu, D, Brümmendorf, T H, Porkka, K, Saglio, G, Gastl, G, Müller, M C, Schuld, P, Di Matteo, P, Pellegrino, A, Dezzani, L, Mahon, F-X, Baccarani, M, and Giles, F J

Published
2016
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17. Observational study of chronic myeloid leukemia italian patients who discontinued tyrosine kinase inhibitors in clinical practice

Author

Fava, C, Rege-Cambrin, G, Dogliotti, I, Cerrano, M, Berchialla, P, Dragani, M, Rosti, G, Castagnetti, F, Gugliotta, G, Martino, B, Gambacorti-Passerini, C, Abruzzese, E, Elena, C, Pregno, P, Gozzini, A, Capodanno, I, Bergamaschi, M, Crugnola, M, Bocchia, M, Galimberti, S, Rapezzi, D, Iurlo, A, Cattaneo, D, Latagliata, R, Breccia, M, Cedrone, M, Santoro, M, Annunziata, M, Levato, L, Stagno, F, Cavazzini, F, Sgherza, N, Giai, V, Luciano, L, Russo, S, Musto, P, Caocci, G, Sora, F, Iuliano, F, Lunghi, F, Specchia, G, Pane, F, Ferrero, D, Baccarani, M, Saglio, G, Fava C., Rege-Cambrin G., Dogliotti I., Cerrano M., Berchialla P., Dragani M., Rosti G., Castagnetti F., Gugliotta G., Martino B., Gambacorti-Passerini C., Abruzzese E., Elena C., Pregno P., Gozzini A., Capodanno I., Bergamaschi M., Crugnola M., Bocchia M., Galimberti S., Rapezzi D., Iurlo A., Cattaneo D., Latagliata R., Breccia M., Cedrone M., Santoro M., Annunziata M., Levato L., Stagno F., Cavazzini F., Sgherza N., Giai V., Luciano L., Russo S., Musto P., Caocci G., Sora F., Iuliano F., Lunghi F., Specchia G., Pane F., Ferrero D., Baccarani M., Saglio G., Fava, C, Rege-Cambrin, G, Dogliotti, I, Cerrano, M, Berchialla, P, Dragani, M, Rosti, G, Castagnetti, F, Gugliotta, G, Martino, B, Gambacorti-Passerini, C, Abruzzese, E, Elena, C, Pregno, P, Gozzini, A, Capodanno, I, Bergamaschi, M, Crugnola, M, Bocchia, M, Galimberti, S, Rapezzi, D, Iurlo, A, Cattaneo, D, Latagliata, R, Breccia, M, Cedrone, M, Santoro, M, Annunziata, M, Levato, L, Stagno, F, Cavazzini, F, Sgherza, N, Giai, V, Luciano, L, Russo, S, Musto, P, Caocci, G, Sora, F, Iuliano, F, Lunghi, F, Specchia, G, Pane, F, Ferrero, D, Baccarani, M, Saglio, G, Fava C., Rege-Cambrin G., Dogliotti I., Cerrano M., Berchialla P., Dragani M., Rosti G., Castagnetti F., Gugliotta G., Martino B., Gambacorti-Passerini C., Abruzzese E., Elena C., Pregno P., Gozzini A., Capodanno I., Bergamaschi M., Crugnola M., Bocchia M., Galimberti S., Rapezzi D., Iurlo A., Cattaneo D., Latagliata R., Breccia M., Cedrone M., Santoro M., Annunziata M., Levato L., Stagno F., Cavazzini F., Sgherza N., Giai V., Luciano L., Russo S., Musto P., Caocci G., Sora F., Iuliano F., Lunghi F., Specchia G., Pane F., Ferrero D., Baccarani M., and Saglio G.

Abstract

It is judged safe to discontinue treatment with tyrosine kinase inhibitors (TKI) for chronic myeloid leukemia (CML) in experimental trials on treatment-free remission (TFR). We collected a total of 293 Italian patients with chronic phase CML who discontinued TKI in deep molecular response. Seventy-two percent of patients were on treatment with imatinib, and 28% with second generation TKI at the time of discontinuation. Median duration of treatment with the last TKI was 77 months [Interquartile Range (IQR) 54;111], median duration of deep molecular response was 46 months (IQR 31;74). Duration of treatment with TKI and duration of deep molecular response were shorter with second generation TKI than with imatinib (P<0.001). Eighty-eight percent of patients discontinued as per clinical practice, and reasons for stopping treatment were: toxicity (20%), pregnancy (6%), and shared decision between treating physician and patient (62%). After a median follow up of 34 months (range, 12-161) overall estimated TFR was 62% (95%CI: 56;68). At 12 months, TFR was 68% (95%CI: 62;74) for imatinib, 73% (95%CI: 64;83) for second generation TKI. Overall median time to restart treatment was six months (IQR 4;11). No progressions occurred. Although our study has the limitation of a retrospective study, our experience within the Italian population confirms that discontinuation of imatinib and second generation TKI is feasible and safe in clinical practice

Published
2019

23. Differences among young adults, adults and elderly chronic myeloid leukemia patients

Author

Castagnetti, F., Gugliotta, G., Baccarani, M., Breccia, M., Specchia, G., Levato, L., Abruzzese, E., Rossi, G., Iurlo, A., Martino, B., Pregno, P., Stagno, F., Cuneo, A., Bonifacio, M., Gobbi, M., Russo, D., Gozzini, A., Tiribelli, M., de Vivo, A., Alimena, G., Cavo, M., Martinelli, G., Pane, F., Saglio, G., Rosti, G., Salvi, F., Pini, M., Leoni, P., Rupoli, S., Galieni, P., Bigazzi, C., Cantore, N., Palmieri, F., Albano, F., Russo Rossi, A., Rambaldi, A., Intermesoli, T., Palandri, F., Testoni, N., Luatti, S., Soverini, S., Iacobucci, I., Bochicchio, MT., Apolinari, M., Fogli, M., Cervello, I., Capucci, A., Malagola, M., Malpignano, A., Girasoli, M., Angelucci, E., Usala, E., Storti, S., De Biasi, E., Tagariello, G., Sartori, R., Di Raimondo, F., Vigneri, P., Impera, S., Molica, S., Lanza, F., Viganò, C., Grasso, M., Rapezzi, D., Cavazzini, F., Bosi, A., Santini, V., Capalbo, SF., Spinosa, G., Pierri, I., Bergamaschi, M., Carella, AM., Bacigalupo, A., De Blasio, A., Ciccone, F., Di Renzo, N., Musolino, C., Russo, S., Cortelezzi, A., Morra, E., Pungolino, EM., Luppi, M., Marasca, R., Pogliani, EM., Gambacorti-Passerini, C., Luciano, L., Ferrara, F., Annunziata, M., Latte, G., Noli, D., Rege-Cambrin, G., Fava, C., Semenzato, G., Binotto, G., Fabbiano, F., Turri, D., Siragusa, S., Caracciolo, C., Musso, M., Porretto, F., Aversa, F., Crugnola, M., Cazzola, M., Orlandi, E., Falini, B., Falzetti, F., Visani, G., Isidori, A., Fioritoni, G., Di Lorenzo, R., Vallisa, D., Trabacchi, E., Petrini, M., Galimberti, S., Pizzuti, M., Zaccaria, A., Salvucci, M., Ronco, F., Ielo, D., Merli, F., Avanzini, P., Tosi, P., Merli, A., Musto, P., De Stefano, V., Sica, S., Latagliata, R., De Fabritiis, P., Trawiska, M., Majolino, I., Pacilli, L., Ronci, B., Cedrone, M., Petti, MC., Pisani, F., Tafuri, A., Montefusco, E., Iuliano, F., Dore, F., Pardini, S., Bocchia, M., Defina, M., Liberati, AM., Luzzi, D., Boccadoro, M., Ferrero, D., Vitolo, U., Gherlinzoni, F., Calistri, E., Fanin, R., Pizzolo, G., Meneghini, V., Rodighiero, F., and DʼEmilio, A.

Published
2015
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26. Profiling of drug-metabolizing enzymes/transporters in CD33+ acute myeloid leukemia patients treated with Gemtuzumab-Ozogamicin and Fludarabine, Cytarabine and Idarubicin

Author

Iacobucci, I, Lonetti, A, Candoni, A, Sazzini, M, Papayannidis, C, Formica, S, Ottaviani, E, Ferrari, A, Michelutti, A, Simeone, E, Astolfi, A, Abbenante, M C, Parisi, S, Cattina, F, Malagola, M, Russo, D, Damiani, D, Gherlinzoni, F, Gottardi, M, Baccarani, M, Fanin, R, and Martinelli, G

Published
2013
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27. Chronic fatigue is the most important factor limiting health-related quality of life of chronic myeloid leukemia patients treated with imatinib

Author

Efficace, F, Baccarani, M, Breccia, M, Cottone, F, Alimena, G, Deliliers, G L, Baratè, C, Specchia, G, Di Lorenzo, R, Luciano, L, Turri, D, Martino, B, Stagno, F, Dabusti, M, Bergamaschi, M, Leoni, P, Simula, M P, Levato, L, Fava, C, Veneri, D, Sica, S, Rambaldi, A, Rosti, G, Vignetti, M, and Mandelli, F

Published
2013
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30. Ponatinib in Refractory Ph-Positive Leukemias: A Phase 2 Trial

Author

Cortes, J E, Kim, D-W, Pinilla-Ibarz, J, le Coutre, P, Paquette, R, Chuah, C, Nicolini, F E, Apperley, J, Khoury, H J, Talpaz, M, DiPersio, J, DeAngelo, D J, Abruzzese, E, Rea, D, Baccarani, M, Müller, M C, Gambacorti-Passerini, C, Wong, S, Lustgarten, S, Rivera, V M, Clackson, T, Turner, C D, Haluska, F G, Guilhot, F, Deininger, M W, Hochhaus, A, Hughes, T, Goldman, J, Shah, N P, and Kantarjian, H

Published
2013

32. Validation and reference values of the EORTC QLQ-CML24 questionnaire to assess health-related quality of life in patients with chronic myeloid leukemia

Author

Efficace, F., Iurlo, A., Patriarca, A., Stagno, F., Bee, P.C., Ector, G.I.C.G., Capodanno, I., Elena, C., Bonifacio, M., Blijlevens, N.M.A., Caocci, G., Wan, C., Abruzzese, E., Breccia, M., Cottone, F., Okumura, I., Oerlemans, S., Cascavilla, N., Albano, F., Kota, V., Sztankay, M., Miggiano, M.C., Saussele, S., Renzo, N. Di, Sorà, F., Castagnetti, F., Baccarani, M., Vignetti, M., Rosti, G., Efficace, F., Iurlo, A., Patriarca, A., Stagno, F., Bee, P.C., Ector, G.I.C.G., Capodanno, I., Elena, C., Bonifacio, M., Blijlevens, N.M.A., Caocci, G., Wan, C., Abruzzese, E., Breccia, M., Cottone, F., Okumura, I., Oerlemans, S., Cascavilla, N., Albano, F., Kota, V., Sztankay, M., Miggiano, M.C., Saussele, S., Renzo, N. Di, Sorà, F., Castagnetti, F., Baccarani, M., Vignetti, M., and Rosti, G.

Abstract

Item does not contain fulltext, Health-related quality of life (HRQOL) assessment is important to facilitate decisions in the current treatment landscape of chronic myeloid leukemia (CML). Therefore, the availability of a validated HRQOL questionnaire, specifically developed for CML patients treated with tyrosine kinase inhibitors (TKIs), may enhance quality of research in this area. We performed an international study including 782 CML patients to assess the validity of the EORTC QLQ-CML 24 questionnaire, and to generate HRQOL reference values to facilitate interpretation of results in future studies. Internal consistency, assessed with Cronbach's alpha coefficients, ranged from 0.66 to 0.83. In the confirmatory factor analysis, all standardized factor loadings exceeded the threshold of 0.40 (range 0.49-0.97), confirming the hypothesized scale structure. Reference values stratified by age and sex were also generated. Our findings support the use of the EORTC QLQ-CML 24, in conjunction with the EORTC QLQ-C30, as a valuable measure to assess HRQOL in CML patients.

Published
2021

33. Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study.

Author

Gattermann N., Finelli C., Della Porta M., Fenaux P., Ganser A., Guerci-Bresler A., Schmid M., Taylor K., Vassilieff D., Habr D., Domokos G., Roubert B., Rose C., Agaoglu L., Alimena G., Alonso D., Ame S., Angelucci E., Arrizabalaga B., Athanasiou-Metaxa M., Augustson B., Aydinok Y., Baba A., Baccarani M., Beck J., Beris P., Beyne-Rauzy O., Birgens H., Bordessoule D., Borgna-Pignatti C., Bosly A., Bouabdallah K., Bowden D., Bowen D., Bron D., Cappellini M.D., Capra M., Cartron G., Cazzola M., Chalkias C., Chan L.L., Chancharunee S., Chapman C., Charoenkwan P., Chasapopoulou E., Cheze S., Chuansumrit A., Cianciulli P., Dauriac C., Delforge M., Dolken G., Dombret H., Duyster J., Economopoulos T., Ehninger G., Elalfy M., El-Beshlawy A., Enggaard L., Fillet G., Filosa A., Forni G., Galanello R., Gastl G., Giraudier S., Goldfarb A., Grigg A., Gumruk F., Ha S.Y., Haase D., Heinrich B., Hertzberg M., Ho J., Hsu H.-C., Huang S., Hunault-Berger M., Inusa B., Jaulmes D., Jensen J., Kattamis A., Kilinc Y., Kim K.-H., Kinsey S., Kjeldsen L., Koren A., Lai M.E., Lai Y., Lee J.-W., Lee K.-H., Lee S.-H., Legros L., Li C., Li C.-K., Li Q., Lin K.-H., Linkesch W., Lubbert M., Lutz D., Mohamed Thalha A.J., Mufti G., Muus P., Nobile F., Papadopoulos N., Perrotta S., Petrini M., Pfeilstocker M., Piga A., Poole J., Porter J.B., Pungolino E., Quarta G., Ravoet C., Jolimont Lobbes H.H., Remacha A.F., Roy L., Saglio G., Sanz G., Schmugge M., Schots H., Secchi G., Seymour J.F., Shah F., Shah H., Shen Z., Slama B., Sutcharitchan P., Taher A., Tamary H., Tesch H.-J., Thein S.L., Troncy J., Villegas A., Viprakasit V., Wainwright L., Wassmann B., Wettervald M., Will A., Wormann B., Wright J., Yeh S.-P., Yoon S.-S., Zoumbos N.C., Zweegman S., Gattermann N., Finelli C., Della Porta M., Fenaux P., Ganser A., Guerci-Bresler A., Schmid M., Taylor K., Vassilieff D., Habr D., Domokos G., Roubert B., Rose C., Agaoglu L., Alimena G., Alonso D., Ame S., Angelucci E., Arrizabalaga B., Athanasiou-Metaxa M., Augustson B., Aydinok Y., Baba A., Baccarani M., Beck J., Beris P., Beyne-Rauzy O., Birgens H., Bordessoule D., Borgna-Pignatti C., Bosly A., Bouabdallah K., Bowden D., Bowen D., Bron D., Cappellini M.D., Capra M., Cartron G., Cazzola M., Chalkias C., Chan L.L., Chancharunee S., Chapman C., Charoenkwan P., Chasapopoulou E., Cheze S., Chuansumrit A., Cianciulli P., Dauriac C., Delforge M., Dolken G., Dombret H., Duyster J., Economopoulos T., Ehninger G., Elalfy M., El-Beshlawy A., Enggaard L., Fillet G., Filosa A., Forni G., Galanello R., Gastl G., Giraudier S., Goldfarb A., Grigg A., Gumruk F., Ha S.Y., Haase D., Heinrich B., Hertzberg M., Ho J., Hsu H.-C., Huang S., Hunault-Berger M., Inusa B., Jaulmes D., Jensen J., Kattamis A., Kilinc Y., Kim K.-H., Kinsey S., Kjeldsen L., Koren A., Lai M.E., Lai Y., Lee J.-W., Lee K.-H., Lee S.-H., Legros L., Li C., Li C.-K., Li Q., Lin K.-H., Linkesch W., Lubbert M., Lutz D., Mohamed Thalha A.J., Mufti G., Muus P., Nobile F., Papadopoulos N., Perrotta S., Petrini M., Pfeilstocker M., Piga A., Poole J., Porter J.B., Pungolino E., Quarta G., Ravoet C., Jolimont Lobbes H.H., Remacha A.F., Roy L., Saglio G., Sanz G., Schmugge M., Schots H., Secchi G., Seymour J.F., Shah F., Shah H., Shen Z., Slama B., Sutcharitchan P., Taher A., Tamary H., Tesch H.-J., Thein S.L., Troncy J., Villegas A., Viprakasit V., Wainwright L., Wassmann B., Wettervald M., Will A., Wormann B., Wright J., Yeh S.-P., Yoon S.-S., Zoumbos N.C., and Zweegman S.

Abstract

The prospective 1-year EPIC study enrolled 341 patients with myelodysplastic syndromes (MDS); although baseline iron burden was >2500. ng/mL, ~50% were chelation-naive. Overall median serum ferritin decreased significantly at 1 year (p=0.002). Decreases occurred irrespective of whether patients were chelation-naive or previously chelated; changes were dependent on dose adjustments and ongoing iron intake. Sustained reductions in labile plasma iron were observed. Discontinuation rate (48.7%) and adverse event profile were consistent with previously reported deferasirox data in MDS. Alanine aminotransferase levels decreased significantly; change correlated significantly with reduction in serum ferritin (p<0.0001). This large dataset prospectively confirms the efficacy and well characterizes the safety profile of deferasirox in MDS. © 2010 Elsevier Ltd.

Published
2021

34. next-generation sequencing improves BCR-ABL1 mutation detection in Philadelphia chromosome -positive acute lymphoblastic leukemia

Author

Soverini, S, Martelli, M, Bavaro, L, De Benedittis, C, Papayannidis, C, Sartor, C, Sora', Federica, Albano, F, Galimberti, S, Abruzzese, E, Annunziata, M, Russo, S, Stulle, M, Imovilli, A, Bonifacio, M, Maino, E, Stagno, F, Basilico, Cm, Borlenghi, E, Fozza, C, Mignone, F, Minari, R, Stella, S, Baccarani, M, Cavo, M, Martinelli, G, Sora, Federica (ORCID:0000-0002-9607-5298), Soverini, S, Martelli, M, Bavaro, L, De Benedittis, C, Papayannidis, C, Sartor, C, Sora', Federica, Albano, F, Galimberti, S, Abruzzese, E, Annunziata, M, Russo, S, Stulle, M, Imovilli, A, Bonifacio, M, Maino, E, Stagno, F, Basilico, Cm, Borlenghi, E, Fozza, C, Mignone, F, Minari, R, Stella, S, Baccarani, M, Cavo, M, Martinelli, G, and Sora, Federica (ORCID:0000-0002-9607-5298)

Abstract

BCR-ABL1 kinase domain mutation testing in tyrosine kinase inhibitor (TKI)-resistant Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukaemia (ALL) patients is routinely performed by Sanger sequencing (SS). Recently, next-generation sequencing (NGS)-based approaches have been developed that afford greater sensitivity and straightforward discrimination between compound and polyclonal mutations. We performed a study to compare the results of SS and NGS in a consecutive cohort of 171 Ph+ ALL patients. At diagnosis, 0/44 and 3/44 patients were positive for mutations by SS and NGS respectively. Out of 47 patients with haematologic resistance, 45 had mutations according to both methods, but in 25 patients NGS revealed additional mutations undetectable by SS. Out of 80 patients in complete haematologic response but with BCR-ABL1 ≥0·1%, 28 (35%) and 52 (65%) were positive by SS and NGS respectively. Moreover, in 12 patients positive by SS, NGS detected additional mutations. NGS resolved clonal complexity in 34 patients with multiple mutations at the same or different codons and identified 35 compound mutations. Our study demonstrates that, in Ph+ ALL on TKI therapy, NGS enables more accurate assessment of mutation status both in patients who fail therapy and in patients with minimal residual disease above 0·1%.

Published
2021

42. Different isoforms of the B-cell mutator activation-induced cytidine deaminase are aberrantly expressed in BCR–ABL1-positive acute lymphoblastic leukemia patients

Author

Iacobucci, I, Lonetti, A, Messa, F, Ferrari, A, Cilloni, D, Soverini, S, Paoloni, F, Arruga, F, Ottaviani, E, Chiaretti, S, Messina, M, Vignetti, M, Papayannidis, C, Vitale, A, Pane, F, Piccaluga, P P, Paolini, S, Berton, G, Baruzzi, A, Saglio, G, Baccarani, M, Foà, R, and Martinelli, G

Published
2010
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49. Cryotherapy in the prevention of oral mucositis in patients receiving low-dose methotrexate following myeloablative allogeneic stem cell transplantation: a prospective randomized study of the Gruppo Italiano Trapianto di Midollo Osseo nurses group

Author

Gori, E, Arpinati, M, Bonifazi, F, Errico, A, Mega, A, Alberani, F, Sabbi, V, Costazza, G, Leanza, S, Borrelli, C, Berni, M, Feraut, C, Polato, E, Altieri, M C, Pirola, E, Loddo, M C, Banfi, M, Barzetti, L, Calza, S, Brignoli, C, Bandini, G, De Vivo, A, Bosi, A, and Baccarani, M

Published
2007
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