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Long‐term results of survival in patients with thalassemia major treated with bone marrow transplantation
Author(s) -
Di Bartolomeo Paolo,
Santarone Stella,
Di Bartolomeo Erminia,
Olioso Paola,
Bavaro Pasqua,
Papalinetti Gabriele,
Di Carlo Paolo,
Papola Franco,
Nicolucci Antonio,
Di Nicola Marta,
Iacone Antonio
Publication year - 2008
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/ajh.21175
Subject(s) - medicine , thalassemia , busulfan , cohort , surgery , transplantation , hemoglobinopathy , graft versus host disease , bone marrow , pediatrics , disease , hematopoietic stem cell transplantation
Allogeneic bone marrow transplantation (BMT) is the only available curative approach for thalassemia major, although long‐term morbidity and mortality are not established. The aim of this study was to assess the long‐term clinical and hematological results in children and adults with thalassemia major treated with BMT. We analyzed the outcome of 115 patients (median age 9 years, range 11 months to 28 years) with thalassemia major undergoing BMT from a related donor between 1983 and 2006. All patients received the same protocol, consisting of busulfan and cyclophoshamide as conditioning therapy and cyclosporin (CSA) alone or CSA and methotrexate for graft‐versus‐host disease (GvHD) prophylaxis. The cumulative probability of graft rejection was 6.7%. The transplant‐related mortality at 1 year was 8.7%. The 20‐year Kaplan‐Meier estimate of overall survival and disease‐free survival was 89.2% and 85.7%, respectively. Ninety‐nine patients out of 103 survivors were in excellent clinical and hematological conditions at last visit following a median follow‐up of 15 years (range, 1–24 years) with the exception of two patients who had invalidating chronic GvHD. This study conducted with a large cohort of patients and covering a long period of observation time, showed BMT to be curative for the majority of patients with thalassemia major. The impact of long‐term transplant‐related sequelae was very limited. Am. J. Hematol. 2008. © 2008 Wiley‐Liss, Inc.