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Bone Marrow Transplantation in Adults with Thalassemia: Treatment and Long‐Term Follow‐Up
Author(s) -
GAZIEV JAVID,
SODANI PIETRO,
POLCHI PAOLA,
ANDREANI MARCO,
LUCARELLI GUIDO
Publication year - 2005
Publication title -
annals of the new york academy of sciences
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.712
H-Index - 248
eISSN - 1749-6632
pISSN - 0077-8923
DOI - 10.1196/annals.1345.024
Subject(s) - medicine , thalassemia , transplantation , regimen , surgery , pediatrics
A bstract : Current regular blood transfusion programs and chelation treatment have considerably improved survival of patients with thalassemia, which resulted in a larger proportion of adult patients. However, disease‐ and treatment‐related complications in these patients progress over time, causing severe morbidity and shortened life expectancy. Stem cell transplantation still remains the only cure currently available for patients with thalassemia. This study updates transplant outcomes in 107 adult patients with median age of 22 years (range, 17‐35 years) who received bone marrow transplantation (BMT) from human leukocyte antigen (HLA)‐identical related donors between 1988 and 1996 (group A) and describes the results of BMT in 15 adult patients with median age of 21 years (range, 17‐31 years) who were treated with a new treatment protocol (Protocol 26) between 1997 and 2003 (group B). The probability of survival, event‐free survival, nonrejection mortality, and rejection for group A patients were 66%, 62%, 37%, and 4%, respectively, with a median follow‐up of 12 years (range, 8.3‐16.2 years). Group B patients treated with the new protocol had some improvement in thalassemia‐free survival (67%) and lower transplant‐related mortality (27%) than that of previous protocols. However, transplant‐related mortality in these high‐risk patients remains elevated. Current myeloablative BMT in adult patients is characterized by higher transplant‐related toxicity due to an advanced phase of disease. Although this new approach to transplant adult patients with a reduced‐dose intensity‐conditioning regimen has improved thalassemia‐free survival, transplant‐related mortality in these high‐risk patients remains elevated.