Bone Marrow Transplantation in Adults with Thalassemia: Treatment and Long-Term Follow-Up

: 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 m...

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Published in:Annals of the New York Academy of Sciences 2005-11, Vol.1054 (1), p.196-205
Main Authors: GAZIEV, JAVID, SODANI, PIETRO, POLCHI, PAOLA, ANDREANI, MARCO, LUCARELLI, GUIDO
Format: Article
Language:English
Subjects:
Adolescent
Adult
adult thalassemia
Azathioprine - administration & dosage
BMT
Bone Marrow Transplantation - adverse effects
Bone Marrow Transplantation - mortality
Bone Marrow Transplantation - statistics & numerical data
Busulfan - administration & dosage
Chelation Therapy
Clinical Protocols
Combined Modality Therapy
Comorbidity
Deferoxamine - therapeutic use
Disease-Free Survival
Erythrocyte Transfusion
Erythropoietin - administration & dosage
Female
Follow-Up Studies
Graft vs Host Disease - epidemiology
Graft vs Host Disease - etiology
Graft vs Host Disease - prevention & control
Granulocyte Colony-Stimulating Factor - administration & dosage
Hematopoietic Cell Growth Factors - administration & dosage
Hemosiderosis - epidemiology
Hemosiderosis - etiology
Hemosiderosis - therapy
Humans
Hydroxyurea - administration & dosage
Immunosuppressive Agents - administration & dosage
Iron Chelating Agents - therapeutic use
Life Tables
Liver Cirrhosis - complications
long-term follow-up
Male
Phlebotomy
Postoperative Complications - mortality
Survival Analysis
Thalassemia - complications
Thalassemia - drug therapy
Thalassemia - mortality
Thalassemia - surgery
Thalassemia - therapy
Transfusion Reaction
Transplantation Conditioning - methods
Transplantation Conditioning - mortality
Transplantation Conditioning - statistics & numerical data
Transplantation, Homologous - adverse effects
Transplantation, Homologous - mortality
Transplantation, Homologous - statistics & numerical data
Treatment Outcome
Vidarabine - administration & dosage
Vidarabine - analogs & derivatives
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Summary:: 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.
ISSN:0077-8923
1749-6632
DOI:10.1196/annals.1345.024