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3. Case Report: Invasive Fungal Infection and Daratumumab: A Case Series and Review of Literature

Author

Farina, Francesca, primary, Ferla, V., additional, Marktel, S., additional, Clerici, D., additional, Mastaglio, S., additional, Perini, T., additional, Oltolini, C., additional, Greco, R., additional, Aletti, F., additional, Assanelli, A., additional, Lupo-Stanghellini, M. T., additional, Bernardi, M., additional, Corti, C., additional, Ciceri, F., additional, and Marcatti, M., additional

Published
2022
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4. P1397: CHRONICALLY REDUCED LEVELS OF THROMBOPOIETIN IMPAIR HEMATOPOIETIC STEM CELL FUNCTION AND MEGAKARYOCYTE BONE MARROW NICHE

Author

Aprile, A., primary, Storto, M., additional, Malara, A., additional, Gulino, A., additional, Raggi, L., additional, Sighinolfi, S., additional, Beretta, S., additional, Merelli, I., additional, Marktel, S., additional, Ponzoni, M., additional, Tripodo, C., additional, Balduini, A., additional, and Ferrari, G., additional

Published
2022
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5. P1519: EXPLORING THE ROLE OF FGF23 IN BETA-THALASSEMIA: A NOVEL THERAPEUTIC TARGET TO AMELIORATE ALTERED INTERACTIONS BETWEEN HSC AND BONE MARROW NICHE

Author

Raggi, L., primary, Aprile, A., additional, Bolamperti, S., additional, Villa, I., additional, Storto, M., additional, Morello, G., additional, Marktel, S., additional, Tripodo, C., additional, Cappellini, M. D., additional, Motta, I., additional, Rubinacci, A., additional, and Ferrari, G., additional

Published
2022
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6. Hematopoietic stem cell function in b-thalassemia is impaired and is rescued by targeting the bone marrow niche

Author

Aprile, A, Gulino, A, Storto, M, Villa, I, Beretta, S, Merelli, I, Rubinacci, A, Ponzoni, M, Marktel, S, Tripodo, C, Lidonnici, M, Ferrari, G, Aprile A., Gulino A., Storto M., Villa I., Beretta S., Merelli I., Rubinacci A., Ponzoni M., Marktel S., Tripodo C., Lidonnici M. R., Ferrari G., Aprile, A, Gulino, A, Storto, M, Villa, I, Beretta, S, Merelli, I, Rubinacci, A, Ponzoni, M, Marktel, S, Tripodo, C, Lidonnici, M, Ferrari, G, Aprile A., Gulino A., Storto M., Villa I., Beretta S., Merelli I., Rubinacci A., Ponzoni M., Marktel S., Tripodo C., Lidonnici M. R., and Ferrari G.

Abstract

Hematopoietic stem cells (HSCs) are regulated by signals from the bone marrow (BM) niche that tune hematopoiesis at steady state and in hematologic disorders. To understand HSC-niche interactions in altered nonmalignant homeostasis, we selected b-thalassemia, a hemoglobin disorder, as a paradigm. In this severe congenital anemia, alterations secondary to the primary hemoglobin defect have a potential impact on HSC-niche cross talk. We report that HSCs in thalassemic mice (th3) have an impaired function, caused by the interaction with an altered BM niche. The HSC self-renewal defect is rescued after cell transplantation into a normal microenvironment, thus proving the active role of the BM stroma. Consistent with the common finding of osteoporosis in patients, we found reduced bone deposition with decreased levels of parathyroid hormone (PTH), which is a key regulator of bone metabolism but also of HSC activity. In vivo activation of PTH signaling through the reestablished Jagged1 and osteopontin levels correlated with the rescue of the functional pool of th3 HSCs by correcting HSC-niche cross talk. Reduced HSC quiescence was confirmed in thalassemic patients, along with altered features of the BM stromal niche. Our findings reveal a defect in HSCs in b-thalassemia induced by an altered BM microenvironment and provide novel and relevant insight for improving transplantation and gene therapy approaches.

Published
2020

7. A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease

Author

Migliavacca, M., Basso Ricci, L., Farinelli, G., Calbi, V., Tucci, F., Barzaghi, F., Ferrua, F., Cicalese, M. P., Darin, S., Barzaghi, L. R., Giglio, F., Peccatori, J., Fumagalli, F., Nicoletti, R., Giannelli, S., Sartirana, C., Bandiera, A., Esposito, M., Milani, R., Mazzi, B., Finocchi, A., Marktel, S., Assanelli, A., Locatelli, Franco, Ciceri, F., Aiuti, A., Bernardo, M. E., Locatelli F. (ORCID:0000-0002-7976-3654), Migliavacca, M., Basso Ricci, L., Farinelli, G., Calbi, V., Tucci, F., Barzaghi, F., Ferrua, F., Cicalese, M. P., Darin, S., Barzaghi, L. R., Giglio, F., Peccatori, J., Fumagalli, F., Nicoletti, R., Giannelli, S., Sartirana, C., Bandiera, A., Esposito, M., Milani, R., Mazzi, B., Finocchi, A., Marktel, S., Assanelli, A., Locatelli, Franco, Ciceri, F., Aiuti, A., Bernardo, M. E., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

X-linked chronic granulomatous disease is a rare disease caused by mutations in the CYBB gene. While more extensive knowledge is available on genetics, pathogenesis, and possible therapeutic options, mitochondrial activity and its implications on patient monitoring are still not well-characterized. We have developed a novel protocol to study mitochondrial activity on whole blood of XCGD patients before and after transplantation, as well as on XCGD carriers. Here we present results of these analyses and of the restoration of mitochondrial activity in hyperinflamed X-linked Chronic Granulomatous Disease after hematopoietic stem cell transplantation. Moreover, we show a strong direct correlation between mitochondrial activity, chimerism, and DHR monitored before and after transplantation and in XCGD carriers. In conclusion, based on these findings, we suggest testing this new ready-to-use marker to better characterize patients before and after treatment and to investigate disease expression in carriers.

Published
2022

9. Incidence, risk factors and clinical outcome of leukemia relapses with loss of the mismatched HLA after partially incompatible hematopoietic stem cell transplantation

Author

Crucitti, L, Crocchiolo, R, Toffalori, C, Mazzi, B, Greco, R, Signori, A, Sizzano, F, Chiesa, L, Zino, E, Lupo Stanghellini, M T, Assanelli, A, Carrabba, M G, Marktel, S, Marcatti, M, Bordignon, C, Corti, C, Bernardi, M, Peccatori, J, Bonini, C, Fleischhauer, K, Ciceri, F, and Vago, L

Published
2015
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10. Sirolimus-based graft-versus-host disease prophylaxis promotes the in vivo expansion of regulatory T cells and permits peripheral blood stem cell transplantation from haploidentical donors

Author

Peccatori, J, Forcina, A, Clerici, D, Crocchiolo, R, Vago, L, Stanghellini, M T L, Noviello, M, Messina, C, Crotta, A, Assanelli, A, Marktel, S, Olek, S, Mastaglio, S, Giglio, F, Crucitti, L, Lorusso, A, Guggiari, E, Lunghi, F, Carrabba, M, Tassara, M, Battaglia, M, Ferraro, A, Carbone, M R, Oliveira, G, Roncarolo, M G, Rossini, S, Bernardi, M, Corti, C, Marcatti, M, Patriarca, F, Zecca, M, Locatelli, F, Bordignon, C, Fleischhauer, K, Bondanza, A, Bonini, C, and Ciceri, F

Published
2015
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12. S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY

Author

Marktel, S, primary, Scaramuzza, S, additional, Giglio, F, additional, Cicalese, M, additional, Lidonnici, M, additional, Rossi, C, additional, Calbi, V, additional, Masera, N, additional, D’Angelo, E, additional, Mirra, N, additional, Origa, R, additional, Tartaglione, I, additional, Perrotta, S, additional, Viarengo, G, additional, Santoleri, L, additional, Milani, R, additional, Gattillo, S, additional, Calabria, A, additional, Montini, E, additional, Graziadei, G, additional, Naldini, L, additional, Cappellini, M, additional, Aiuti, A, additional, Ciceri, F, additional, and Ferrari, G, additional

Published
2022
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22. T CELL DEPLETED HAPLOIDENTICAL STEM CELL TRANSPLANTATION FOLLOWED BY ADD-BACKS OF SUICIDE-GENE MODIFIED DONOR T-CELLS OFFERS LONG TERM SURVIVAL FREE OF IMMUNE SUPPRESSION TREATMENT: A 7 YEARS FOLLOW-UP IN 56 PATIENTS: PH-O084

Author

Lupo-Stanghellini, M. T., Greco, R., Oliveira, G., Peccatori, J., Marcatti, M., Guggiari, E., Lunghi, F., Marktel, S., Giglio, F., Bondanza, A., Malerba, A., Trinca, S., Corti, C., Bernardi, M., Lambiase, A., Bordignon, C., Bonini, C., and Ciceri, F.

Published
2014

29. Genotypes and haplotypes in the 3′ untranslated region of the HLA-G gene and their association with clinical outcome of hematopoietic stem cell transplantation for beta-thalassemia

Author

Sizzano, F., Testi, M., Zito, L., Crocchiolo, R., Troiano, M., Mazzi, B., Turchiano, G., Torchio, M., Pultrone, C., Gregori, S., Chiesa, R., Gaziev, J., Sodani, P., Marktel, S., Amoroso, A., Roncarolo, M. G., Lucarelli, G., Ciceri, F., Andreani, M., and Fleischhauer, K.

Published
2012
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34. Risk of complications of haematopoietic stem cell collection in paediatric sibling donors: an EBMT Paediatric Diseases Working Party prospective observational study: O404

Author

Styczynski, J., Balduzzi, A., Gil, L., Ehlert, K., Fagioli, F., Hamladji, R.-M., Marktel, S., Yesilipek, A., Matulova, M., Dalle, J.-H., Wachowiak, J., Miano, M., Varotto, S., Diaz, M. A., Vermylen, C., Eyrich, M., Badell, I., Dreger, P., Gozdzik, J., Hutt, D., Rascon, J., Labopin, M., Elarouci, N., Dini, G., and Peters, C.

Published
2011

35. Results of a Multicentre, Randomized, Controlled Open-Label Study on the Use of Anti-T-Lymphocyte Globulin (ATLG) and Rituximab for Immunomodulation of Graft-Versus-Host Disease (GvHD) and Graft Failure (GF) in Patients with Non-Malignant Disorders.

Author

Algeri, M, Galimberti, S, Bernardo, M, Rovelli, A, Zecca, M, La Nasa, G, Marktel, S, Merli, P, Bertaina, A, Pagliara, D, Boccieri, E, Del Bufalo, F, Gaspari, S, Ruggeri, A, Capitoli, G, Valsecchi, M, Locatelli, F, Bernardo, ME, Valsecchi, MG, Algeri, M, Galimberti, S, Bernardo, M, Rovelli, A, Zecca, M, La Nasa, G, Marktel, S, Merli, P, Bertaina, A, Pagliara, D, Boccieri, E, Del Bufalo, F, Gaspari, S, Ruggeri, A, Capitoli, G, Valsecchi, M, Locatelli, F, Bernardo, ME, and Valsecchi, MG

Published
2019

36. Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

Author

Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., Simoes, B. P., Ferster, A., Dupont, S., De La Fuente, J., Dalle, J. -H., Zecca, M., Walters, M. C., Krishnamurti, L., Bhatia, M., Leung, K., Yanik, G., Kurtzberg, J., Dhedin, N., Kuentz, M., Michel, G., Apperley, J., Lutz, P., Neven, B., Bertrand, Y., Vannier, J. P., Ayas, M., Cavazzana, M., Matthes-Martin, S., Rocha, V., Elayoubi, H., Kenzey, C., Bader, P., Locatelli, Franco, Ruggeri, A., Eapen, M., Bordon, V., Labarque, V., Pereira, M., Bittencourt, H., Petersen, H., Deconninck, E., Jubert, C., Perrin, J., Cahn, J. Y., Bruno, B., Bordigoni, P., Mechinaud, F., Vernant, J. P., Stephan, J. L., Suttorp, M., Strahm, B., Da Cunha, C. B., Garwer, B., Rothmayer, M., Wendelin, K., Graphakos, S., Tbakhi, A., Naeimi, N., Zuckerman, T., Sharon, P. B., Yaniv, I., Amos, T., Prete, A., Lo Nigro, L., Lanino, E., Faraci, M., Ciceri, F., Marktel, S., De Simone, G., Messina, C., Bartolomeo, P. D. I., Santarone, S., Vallisa, D., Bertaina, A., Arcese, W., Foa, R., Berger, M., Maximova, N., Wallet, S., Bazuaye, G. N., Maschan, A., De Heredia, C. D., Bieler, C. B., Pato, J. R., Heras, I., Trevor, R., Abayomi, K., Thomson, J., Fasth, A., Frodin, U., Ljugman, P., Ansari, M., Gungor, T., Unal, E., Pehlivan, M., Anak, S., Ozturk, G., Unal, A., Lawson, S., Keshani, J., Drake, A., Wynn, R., Williams, J., Jagsia, M., Leung, W., Abraham, A., Sahdey, I., Margolis, D., Eames, G., Horwitz, E., Cowan, M., Kapoor, N., Rowley, S., Megason, G., Rogers, Z., Bolanos-Meade, J., Hudspeth, M., Rosenthal, J., Olson, T., Kassow, K., Selby, G., Haines, H., Chaudhury, S., Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., Simoes, B. P., Ferster, A., Dupont, S., De La Fuente, J., Dalle, J. -H., Zecca, M., Walters, M. C., Krishnamurti, L., Bhatia, M., Leung, K., Yanik, G., Kurtzberg, J., Dhedin, N., Kuentz, M., Michel, G., Apperley, J., Lutz, P., Neven, B., Bertrand, Y., Vannier, J. P., Ayas, M., Cavazzana, M., Matthes-Martin, S., Rocha, V., Elayoubi, H., Kenzey, C., Bader, P., Locatelli, F., Ruggeri, A., Eapen, M., Bordon, V., Labarque, V., Pereira, M., Bittencourt, H., Petersen, H., Deconninck, E., Jubert, C., Perrin, J., Cahn, J. Y., Bruno, B., Bordigoni, P., Mechinaud, F., Vernant, J. P., Stephan, J. L., Suttorp, M., Strahm, B., Da Cunha, C. B., Garwer, B., Rothmayer, M., Wendelin, K., Graphakos, S., Tbakhi, A., Naeimi, N., Zuckerman, T., Sharon, P. B., Yaniv, I., Amos, T., Prete, A., Lo Nigro, L., Lanino, E., Faraci, M., Ciceri, F., Marktel, S., De Simone, G., Messina, C., Bartolomeo, P. D. I., Santarone, S., Vallisa, D., Bertaina, A., Arcese, W., Foa, R., Berger, M., Maximova, N., Wallet, S., Bazuaye, G. N., Maschan, A., De Heredia, C. D., Bieler, C. B., Pato, J. R., Heras, I., Trevor, R., Abayomi, K., Thomson, J., Fasth, A., Frodin, U., Ljugman, P., Ansari, M., Gungor, T., Unal, E., Pehlivan, M., Anak, S., Ozturk, G., Unal, A., Lawson, S., Keshani, J., Drake, A., Wynn, R., Williams, J., Jagsia, M., Leung, W., Abraham, A., Sahdey, I., Margolis, D., Eames, G., Horwitz, E., Cowan, M., Kapoor, N., Rowley, S., Megason, G., Rogers, Z., Bolanos-Meade, J., Hudspeth, M., Rosenthal, J., Olson, T., Kassow, K., Selby, G., Haines, H., Chaudhury, S., France Monacord, Centre Scientifique de Monaco (CSM), CHI Créteil, Centre de Recherche Saint-Antoine (CR Saint-Antoine), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC), Eurocord, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Institut Universitaire d'Hématologie (IUH), Université Paris Diderot - Paris 7 (UPD7)-Université Paris Diderot - Paris 7 (UPD7), Hôpital Universitaire des Enfants - Reine Fabiola, Université Libre de Bruxelles, Equipe NEMESIS - Centre de Recherches de l'Institut du Cerveau et de la Moelle épinière (NEMESIS-CRICM), Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière (CRICM), Centre National de la Recherche Scientifique (CNRS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC)-Centre National de la Recherche Scientifique (CNRS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC), Unité d'Hémato-Immunologie pédiatrique [Hôpital Robert Debré, Paris], Service d'Immuno-hématologie pédiatrique [Hôpital Robert Debré, Paris], Hôpital Robert Debré-Hôpital Robert Debré, Duke University Medical Center, Service de Greffe de Moelle - Unité AJA, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (APHP), Service d'hématologie clinique, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Hôpital de la Timone [CHU - APHM] (TIMONE), Department of Haematology, Hôpital Civil, Hopital Civil, Département d'Immunologie, hématologie et rhumatologie pédiatriques [Hôpital Necker-Enfants malades - APHP], CHU Necker - Enfants Malades [AP-HP], Centre Hospitalier Lyon Sud [CHU - HCL] (CHLS), Hospices Civils de Lyon (HCL), Hemostase, Endothelium, Angiogenese (UMR_S_553), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Diderot - Paris 7 (UPD7), Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices (PANTHER), Université de Rouen Normandie (UNIROUEN), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Imagine - Institut des maladies génétiques (IMAGINE - U1163), Centre National de la Recherche Scientifique (CNRS)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Churchill Hospital [Breast Care Unit], Churchill Hospital Oxford Centre for Haematology, Santa Lucia Foundation, IRCSS, Rome, Medical College of Wisconsin, National Institute for Health Research, Centre de Recherche Saint-Antoine (UMRS893), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut Universitaire d'Hématologie (IUH), Hôpital Universitaire des Enfants Reine Fabiola [Bruxelles, Belgique] (HUDERF), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, and UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique

Subjects
Male, HYDROXYUREA, Transplantation Conditioning, [SDV]Life Sciences [q-bio], medicine.medical_treatment, CHILDREN, Hematopoietic stem cell transplantation, Biochemistry, THALASSEMIA, 0302 clinical medicine, HLA Antigens, Surveys and Questionnaires, 1114 Paediatrics And Reproductive Medicine, Child, ComputingMilieux_MISCELLANEOUS, CÉLULAS-TRONCO, Hazard ratio, Graft Survival, BONE-MARROW TRANSPLANT, Hematopoietic Stem Cell Transplantation, Hematology, Sickle cell anemia, 3. Good health, Survival Rate, medicine.anatomical_structure, Treatment Outcome, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, 030220 oncology & carcinogenesis, Child, Preschool, Histocompatibility, [SDV.IMM]Life Sciences [q-bio]/Immunology, Female, Life Sciences & Biomedicine, medicine.medical_specialty, Adolescent, Anemia, Immunology, Anemia, Sickle Cell, 1102 Cardiovascular Medicine And Haematology, Disease-Free Survival, 03 medical and health sciences, Internal medicine, MANAGEMENT, medicine, Humans, ANEMIA, Survival rate, Transplantation, Science & Technology, business.industry, Siblings, Sickle cell disease, Infant, 1103 Clinical Sciences, ADULTS, Cell Biology, medicine.disease, LIFE, EXPERT PANEL, Bone marrow, FOLLOW-UP, business, 030215 immunology
Abstract

Despite advances in supportive therapy to prevent complications of sickle cell disease (SCD), access to care is not universal. Hematopoietic cell transplantation is, to date, the only curative therapy for SCD, but its application is limited by availability of a suitable HLA-matched donor and lack of awareness of the benefits of transplant. Included in this study are 1000 recipients of HLA-identical sibling transplants performed between 1986 and 2013 and reported to the European Society for Blood and Marrow Transplantation, Eurocord, and the Center for International Blood and Marrow Transplant Research. The primary endpoint was event-free survival, defined as being alive without graft failure; risk factors were studied using a Cox regression models. The median age at transplantation was 9 years, and the median follow-up was longer than 5 years. Most patients received a myeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bone marrow was the predominant stem cell source (n = 839; 84%); peripheral blood and cord blood progenitors were used in 73 (7%) and 88 (9%) patients, respectively. The 5-year event-free survival and overall survival were 91.4% (95% confidence interval, 89.6%-93.3%) and 92.9% (95% confidence interval, 91.1%-94.6%), respectively. Event-free survival was lower with increasing age at transplantation (hazard ratio [HR], 1.09; P < .001) and higher for transplantations performed after 2006 (HR, 0.95; P = .013). Twenty-three patients experienced graft failure, and 70 patients (7%) died, with the most common cause of death being infection. The excellent outcome of a cohort transplanted over the course of 3 decades confirms the role of HLA-identical sibling transplantation for children and adults with SCD.

Published
2017

37. Safety of retroviral gene marking with a truncated NGF receptor

Author

Bonini, C., Grez, M., Traversari, C., Ciceri, F., Marktel, S., Ferrari, G., Dinauer, M., Sadat, M., Aiuti, A., Deola, S., Radrizzani, M., Hagenbeek, A., Apperley, J., Ebeling, S., Martens, A., Kolb, H.J., Weber, M., Lotti, F., Grande, A., Weissinger, E., Bueren, J.A., Lamana, M., Falkenburg, J.H.F., Heemskerk, M.H.M., Austin, T., Kornblau, S., Marini, F., Benati, C., Magnani, Z., Cazzaniga, S., Toma, S., Gallo-Stampino, C., Introna, M., Slavin, S., Greenberg, P.D., Bregni, M., Mavilio, F., and Bordignon, C.

Abstract

Author(s): C. Bonini (corresponding author) [1]; M. Grez [2]; C. Traversari [3]; F. Ciceri [1]; S. Marktel [1, 4]; G. Ferrari [5]; M. Dinauer [6]; M. Sadat [6]; A. Aiuti [...]

Published
2003
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39. Longitudinal microbiome profile associates with the risk of major transplant-related complications in allogeneic HSCT recipients

Author

Greco R, Mancini N, Pasciuta R, Barbanti MC, Morelli M, Vago L, Giglio F, Stanghellini MTL, Forcina A, Infurnari L, Marktel S, Assanelli A, Carrabba M, Bernardi M, Corti C, Peccatori J, Clementi M, Ciceri F, Greco, R, Mancini, N, Pasciuta, R, Barbanti, Mc, Morelli, M, Vago, L, Giglio, F, Stanghellini, Mtl, Forcina, A, Infurnari, L, Marktel, S, Assanelli, A, Carrabba, M, Bernardi, M, Corti, C, Peccatori, J, Clementi, M, and Ciceri, F

Published
2016

40. Treosulfan based reduced toxicity conditioning followed by allogeneic stem cell transplantation in patients with myelofibrosis

Author

Claudiani S., Marktel S., Piemontese S., Assanelli A., Lupo-Stanghellini M. T., Carrabba M., Guggiari E., Giglio F., De Freitas T., Marcatti M., Bernardi M., Corti C., Peccatori J., Lunghi F., Ciceri F., Claudiani, S., Marktel, S., Piemontese, S., Assanelli, A., Lupo-Stanghellini, M. T., Carrabba, M., Guggiari, E., Giglio, F., De Freitas, T., Marcatti, M., Bernardi, M., Corti, C., Peccatori, J., Lunghi, F., and Ciceri, F.

Subjects
Male, Transplantation Conditioning, bone marrow transplantation, Middle Aged, Allografts, Disease-Free Survival, Survival Rate, Primary Myelofibrosis, hematopoietic stem cell transplantation, reduced-toxicity conditioning, Humans, Female, myeloproliferative disease, Unrelated Donors, treosulfan, Busulfan, Vidarabine, Aged, Stem Cell Transplantation
Abstract

Allogeneic transplantation is the only potentially curative strategy for myelofibrosis, even in the era of new drugs that so far only mitigate symptoms. The choice to proceed to allogeneic transplantation is based on several variables including age, disease phase, degree of splenomegaly, donor availability, comorbidities and iron overload. These factors, along with conditioning regimen and time to transplantation, may influence the outcome of ASCT. We report 14 patients affected by myelofibrosis with a median age of 57 years (range, 41–76) receiving a treosulfan-fludarabine based reduced toxicity conditioning. Patients (pts) received a stem cell transplantation from an HLA identical (n = 10) or matched unrelated donor (n = 4). All pts had a complete myeloablation followed by engraftment and in 12 out of 13 evaluated pts donor chimerism was 100% at 1 month. In most cases a reduction of splenomegaly and a reduction (or resolution) of bone marrow fibrosis was observed. After a median follow-up of 39 months (range, 3–106), the 3-year probability of overall survival and disease free survival was 54 +/− 14% and 46 +/− 14%, respectively. The cumulative incidence of non-relapse mortality at 2 years was 39 +/− 15%. Causes of non-relapse mortality were: infection (n = 2), GvHD (n = 2) and haemorrhage (n = 1). We can conclude that a treosulfan and fludarabine based conditioning has a potent myeloablative and anti-disease activity although non-relapse mortality remains high in this challenging clinical setting. Copyright © 2014 John Wiley & Sons, Ltd.

Published
2016

41. Nanosphere's Verigene (R) System to detect Multidrug-Resistant Gram-negative Bacterial Outbreak: a prospective study in 60 patients

Author

Barbanti MC, Mancini N, Infurnari L, Pasciuta R, Forcina A, Vago L, Giglio F, Messina C, Morelli M, Perini T, Girlanda S, Carrabba M, Stanghellini MTL, Marktel S, Assanelli A, Bernardi M, Corti C, Peccatori J, Clementi M, Ciceri F, Greco R, Barbanti, Mc, Mancini, N, Infurnari, L, Pasciuta, R, Forcina, A, Vago, L, Giglio, F, Messina, C, Morelli, M, Perini, T, Girlanda, S, Carrabba, M, Stanghellini, Mtl, Marktel, S, Assanelli, A, Bernardi, M, Corti, C, Peccatori, J, Clementi, M, Ciceri, F, and Greco, R

Published
2016

42. T-Cell Replete Haploidentical PBSC Transplantation Using Intensified Myeloablative Conditioning, Post-Transplant Cyclophosphamide and Sirolimus

Author

Greco R, Piemontese S, Cieri N, Giglio F, Morelli M, Messina C, Marcatti M, Marktel S, Carrabba M, Bernardi M, Corti C, Assanelli A, Stanghellini MTL, Bonini C, Vago L, Ciceri F, Peccatori J, Greco, R, Piemontese, S, Cieri, N, Giglio, F, Morelli, M, Messina, C, Marcatti, M, Marktel, S, Carrabba, M, Bernardi, M, Corti, C, Assanelli, A, Stanghellini, Mtl, Bonini, C, Vago, L, Ciceri, F, and Peccatori, J

Published
2016

43. Secondary solid tumors after allogeneic stem cell transplantation: active surveillance in long term follow-up

Author

Lupo-Stanghellini MT, Assanelli A, Greco R, Giglio F, Mastaglio S, Morelli M, Pavesi F, Sala E, Brambilla M, Piemontese S, Vago L, Messina C, Dalto SC, Francesca L, Perini T, Girlanda S, Malerba A, Marcatti M, Guggiari E, Marktel S, Carrabba M, Lunghi F, Bernardi M, Bonini C, Corti C, Peccatori J, Ciceri F, Lupo-Stanghellini, Mt, Assanelli, A, Greco, R, Giglio, F, Mastaglio, S, Morelli, M, Pavesi, F, Sala, E, Brambilla, M, Piemontese, S, Vago, L, Messina, C, Dalto, Sc, Francesca, L, Perini, T, Girlanda, S, Malerba, A, Marcatti, M, Guggiari, E, Marktel, S, Carrabba, M, Lunghi, F, Bernardi, M, Bonini, C, Corti, C, Peccatori, J, and Ciceri, F

Published
2016

44. HHV6 Specific T-cells are Predictor of Clinically Relevant HHV6 Infection after Allogeneic HSCT

Author

Greco R, Noviello M, Crucitti L, Racca S, Valtolina V, Pozzato M, Morelli M, Giglio F, Mannina D, Barbanti MC, Forcina A, Rolla S, Assanelli A, Carrabba M, Marktel S, Bernardi M, Corti C, Stanghellini MTL, Vago L, Peccatori J, Clementi M, Bonini C, Ciceri F, Greco, R, Noviello, M, Crucitti, L, Racca, S, Valtolina, V, Pozzato, M, Morelli, M, Giglio, F, Mannina, D, Barbanti, Mc, Forcina, A, Rolla, S, Assanelli, A, Carrabba, M, Marktel, S, Bernardi, M, Corti, C, Stanghellini, Mtl, Vago, L, Peccatori, J, Clementi, M, Bonini, C, and Ciceri, F

Published
2016

45. BIOMARKERS PREDICTING ACUTE GRAFT-VERSUS-HOST DISEASE IN ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANT RECIPIENTS: A PROSPECTIVE STUDY IN 120 PATIENTS

Author

Vaccari LC, Barbanti MC, Giglio F, Morelli M, Mannina D, Perini T, Lazzari L, Mastaglio S, Piemontese S, Lorentino F, Forcina A, Assanelli A, Marktel S, Carrabba M, Vago L, Corti C, Bernardi M, Stanghellini MTL, Peccatori J, Ciceri F, Greco R, Vaccari, Lc, Barbanti, Mc, Giglio, F, Morelli, M, Mannina, D, Perini, T, Lazzari, L, Mastaglio, S, Piemontese, S, Lorentino, F, Forcina, A, Assanelli, A, Marktel, S, Carrabba, M, Vago, L, Corti, C, Bernardi, M, Stanghellini, Mtl, Peccatori, J, Ciceri, F, and Greco, R

Published
2016

46. Predicting failure of hematopoietic stem cell mobilization before it starts: The predicted poor mobilizer (pPM) score

Author

Olivieri, J., Attolico, I., Nuccorini, R., Pascale, S. P., Chiarucci, M., Poiani, M., Corradini, P., Farina, L., Gaidano, G., Nassi, L., Sica, S., Piccirillo, N., Pioltelli, P. E., Martino, M., Moscato, T., Pini, M., Zallio, F., Ciceri, F., Marktel, S., Mengarelli, A., Musto, P., Capria, S., Merli, F., Codeluppi, K., Mele, G., Lanza, F., Specchia, G., Pastore, D., Milone, G., Saraceni, F., Di Nardo, E., Perseghin, P., Olivieri, A., Sica S. (ORCID:0000-0003-2426-3465), Piccirillo N. (ORCID:0000-0002-1688-1987), Olivieri, J., Attolico, I., Nuccorini, R., Pascale, S. P., Chiarucci, M., Poiani, M., Corradini, P., Farina, L., Gaidano, G., Nassi, L., Sica, S., Piccirillo, N., Pioltelli, P. E., Martino, M., Moscato, T., Pini, M., Zallio, F., Ciceri, F., Marktel, S., Mengarelli, A., Musto, P., Capria, S., Merli, F., Codeluppi, K., Mele, G., Lanza, F., Specchia, G., Pastore, D., Milone, G., Saraceni, F., Di Nardo, E., Perseghin, P., Olivieri, A., Sica S. (ORCID:0000-0003-2426-3465), and Piccirillo N. (ORCID:0000-0002-1688-1987)

Abstract

Predicting mobilization failure before it starts may enable patient-tailored strategies. Although consensus criteria for predicted PM (pPM) are available, their predictive performance has never been measured on real data. We retrospectively collected and analyzed 1318 mobilization procedures performed for MM and lymphoma patients in the plerixafor era. In our sample, 180/1318 (13.7%) were PM. The score resulting from published pPM criteria had sufficient performance for predicting PM, as measured by AUC (0.67, 95%CI: 0.63-0.72). We developed a new prediction model from multivariate analysis whose score (pPM-score) resulted in better AUC (0.80, 95%CI: 0.76-0.84, p < 0001). pPM-score included as risk factors: increasing age, diagnosis of NHL, positive bone marrow biopsy or cytopenias before mobilization, previous mobilization failure, priming strategy with G-CSF alone, or without upfront plerixafor. A simplified version of pPM-score was categorized using a cut-off to maximize positive likelihood ratio (15.7, 95%CI: 9.9-24.8); specificity was 98% (95%CI: 97-98.7%), sensitivity 31.7% (95%CI: 24.9-39%); positive predictive value in our sample was 71.3% (95%CI: 60-80.8%). Simplified pPM-score can "rule in" patients at very high risk for PM before starting mobilization, allowing changes in clinical management, such as choice of alternative priming strategies, to avoid highly likely mobilization failure.

Published
2018

47. Management of veno-occlusive disease: the multidisciplinary approach to care

Author

Wallhult, E, Kenyon, M, Liptrott, S, Mank, A, Ni Chonghaile, M, Babic, A, Bijkerk, J, Bompoint, C, Corbacioglu, S, de Weijer, R, Fink, C, Marktel, S, Soni, V, Sprenger, S, Arjona, ET, and Mohty, M

Subjects
supportive care, bone marrow transplantation, veno-occlusive disease, multidisciplinary care
Abstract

Although it is considered a relatively rare disorder, veno-occlusive disease (VOD) is one of the main causes of overall, non-relapse mortality associated with haematopoietic stem cell transplantation (HSCT). This article, based on the consensus opinion of haemato-oncology nurses, haemato-oncologists and pharmacists from both adult and paediatric services at the VOD International Multi-Disciplinary Advisory Board at the European Society for Blood and Marrow Transplantation (EBMT) meeting, Istanbul, 2015, aims to explore the multidisciplinary approach to care for the management of VOD, with an emphasis on current challenges in this area. The careful monitoring of HSCT patients allows early detection of the symptoms associated with VOD and timely treatment, ultimately improving patient outcomes. As part of a multidisciplinary team, nurses have an essential role to play, from pretransplant assessment to medical management and overall care of the patient. Physicians and pharmacists have a responsibility to facilitate education and training so that nurses can work effectively within that team.

Published
2017

48. Allogeneic haematopoietic stem cell transplantation in patients older than 65 years with high risk acute myeloid leukemia: results in 46 consecutive patients

Author

Sala E, Carrabba MG, Messina C, Pavesi F, Xue E, Gentner B, Vago L, Morelli M, Assanelli A, Marktel S, Lupo-Stanghellini MT, Corti C, Peccatori J, Ciceri F, Bernardi M, Sala, E, Carrabba, Mg, Messina, C, Pavesi, F, Xue, E, Gentner, B, Vago, L, Morelli, M, Assanelli, A, Marktel, S, Lupo-Stanghellini, Mt, Corti, C, Peccatori, J, Ciceri, F, and Bernardi, M

Published
2015

49. T-cell immune reconstitution predicts the outcome of Human Herpes Virus 6 Infection in 54 Patients after Allogeneic Hematopoietic Stem Cell Transplantation

Author

Greco R, Crucitti L, Noviello M, Racca S, Barbanti MC, Valtolina V, Dvir R, Vago L, Stanghellini MTL, Giglio F, Morelli M, Rolla S, Lorentino F, Orsini A, Forcina A, Assanelli A, Carrabba M, Marktel S, Bernardi M, Corti C, Peccatori J, Bonini C, Clementi M, Ciceri F, Greco, R, Crucitti, L, Noviello, M, Racca, S, Barbanti, Mc, Valtolina, V, Dvir, R, Vago, L, Stanghellini, Mtl, Giglio, F, Morelli, M, Rolla, S, Lorentino, F, Orsini, A, Forcina, A, Assanelli, A, Carrabba, M, Marktel, S, Bernardi, M, Corti, C, Peccatori, J, Bonini, C, Clementi, M, and Ciceri, F

Published
2015

50. Targeting WT1 reactivation in patients in complete remission after allogeneic haematopoietic stem cell transplantation for AML and MDS: results in 111 patients

Author

Sala E, Carrabba MG, Messina C, Vago L, Tresoldi C, Pavesi F, Xue E, Gentner B, Assanelli A, Marktel S, Lupo-Stanghellini MT, Corti C, Peccatori J, Ciceri F, Bernardi M, Sala, E, Carrabba, Mg, Messina, C, Vago, L, Tresoldi, C, Pavesi, F, Xue, E, Gentner, B, Assanelli, A, Marktel, S, Lupo-Stanghellini, Mt, Corti, C, Peccatori, J, Ciceri, F, and Bernardi, M

Published
2015

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