Collection of cord blood stem cells for transplantation in thalassemic patients.

Thalassemia is widely distributed throughout the world and is one of the major public health problems. The use of bone marrow transplantation, the only curative therapy for thalassemia, is limited because less than 30% of the patients have unaffected and HLA-identical siblings as donors. Cord blood stem cells, an alternative source of stem cells for transplantation, have been successfully transplanted into patients with several diseases after myeloablative therapy. Twenty cord blood samples from unaffected neonates whose siblings had severe thalassemia were collected. The median volume was 80 ml. The median number of cells and colony forming units-granulocyte-macrophage in cord blood was 9.2 x 10(8) and 3.4 x 10(5), respectively. Four of 20 cord blood samples had HLA-matched to the affected siblings. One patient underwent cord blood transplantation with success; one patient is waiting for transplantation.