Bone marrow and cord blood stem cell transplantation for thalassemia in Thailand

Abstract

Thalassemias and hemoglobinopathies are prevalent in Thailand. Bone marrow transplantation is the only curative therapy for thalassemia. We report 28 transplants in 26 patients with thalassemia. Twenty-one patients underwent bone marrow transplantation after conditioning with busulfan 14 mg/kg and cyclophosphamide 200 mg/kg. The outcome was favorable in those with hepatomegaly and splenomegaly ≤ 2 cm below costal margin. Graft rejection is a problem in the patients who have severe manifestations as determined by enlargement of liver and spleen > 2 cm below costal margin. A higher dose of busulfan 600 mg/m2 was used in seven patients who had severe manifestations with success. Cord blood is an alternative source of stem cells for transplant. Cord blood was collected from HLA-identical unaffected neonates and used for transplant into affected siblings. Five out of 6 pateints who underwent cord blood transplantation are alive and well.