Bone marrow transplantation for beta-thalassemia.

Bone marrow transplantation in thalassemia represents the only form of radical cure of this disease. Patients younger than 17 years are divided into three classes of risk according to the presence or absence of portal fibrosis, hepatomegaly, and history of adequate chelation treatment. Patients older than 16 years are categorized as adult thalassemics. Survival and event-free survival are 93% and 91% for Class 1, 87% and 83% for Class 2, and 79% and 58% for Class 3, respectively. Adult patient survival is 66% and event-free survival is 62%.