Bone Marrow Transplantation for Thalassemia

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Abstract

Purpose

We reviewed the results of transplanting allogeneic marrow from HLA-identical donors in patients with β-thalassemia. Among the 484 consecutive patients who have received transplants since 1981, survival and disease-free survival rates leveled off at ∼1 year after transplantation, at 82 and 75%, respectively.

Patients and Methods

Clinical characteristics of patients before transplant have been studied to determine their impact on survival, disease-free survival, and graft rejection. By multivariate analysis, portal fibrosis, hepatomegaly, and a history of inadequate chelation therapy were identified as risk factors. The patients were then divided into three classes of risk.

Results

The rate of prolonged disease-free survival was 98% and 87% for class 1 and class 2 patients. This rate of disease-free survival is 70% with the use of our last conditioning protocol for class 3 patients. Older patients (17–32 years) have a 79% probability of prolonged diseasefree survival.

Conclusions

We conclude that for patients with thalassemia major, transplantation of bone marrow from a human leukocyte antigen-identical donor offers a high probability of disease-free survival, particularly for those patients in early stages of their disease.

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