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.

Study of hematopoietic progenitor cells in thalassemia after bone marrow transplantation.

The hematopoietic committed progenitor cells (BFU-E and CFU-GM) in blood and bone marrow were studied in thalassemic patients before and after bone marrow transplantation. Eighteen transplants were performed in 17 patients with thalassemia. Five were homozygous beta-thalassemia and 12 were beta-thalassemia/hemoglobin E disease.

Abnormalities of hematopoietic progenitor cells in patients with aplastic anemia after hematologic recovery.

Hematopoietic progenitor cells were studied in 11 patients with aplastic anemia who had hematologic recovery after androgen therapy. The mean numbers of colonies derived from erythroid and granulocyte-macrophage progenitor cells (BFU-E and CFU-GM) were markedly decreased compared to normal controls. Cell-mediated suppression of colony growth as detected by coculture studies was observed in 5 patients; 4 patients for CFU-GM and one for both CFU-GM and BFU-E.

Studies on the pathogenesis of aplastic anemia in Thailand: evidence of immune-mediated mechanism.

The pathogenesis of aplastic anemia in Thailand was studied by using in vitro progenitor cells culture. In 37 patients who had active disease, the numbers of colonies derived from erythroid and granulocyte-macrophage progenitor cells (BFU-E and CFU-GM) were markedly decreased both in the blood and bone marrow as compared to normal controls. Co-culture of patients' cells with normal blood cells was performed in order to verify an immunologically mediated mechanism.

Methylprednisolone therapy in aplastic anaemia: correlation of in vitro tests and lymphocyte subsets with clinical response.

20 patients with aplastic anaemia were treated with methylprednisolone 1 g/d for 3 d followed by prednisolone 60 mg on alternate days. At 3 months after therapy, 7 of 20 patients (35%) showed recovery, 2 had died and 11 were non-responders. Only newly diagnosed patients were responders.