We report our experience of bone marrow transplantation for thalassemi
a in Thailand. From July 1988 to September 1992, 10 thalassemic patien
ts underwent allogeneic bone marrow transplantation. Two of them were
homozygous beta-thalassemia and 8 were beta-thalassemia/Hb E disease.
Seven were male and 3 female. The age ranged from 1.8-14 years. The co
nditioning regimen comprised busulfan 14 mg/kg and cyclophosphamide 20
0 mg/kg. For GVHD prophylaxis, either cyclosporine alone or in combina
tion with short methotrexate was given. Five patients were alive and w
ell 104-1534 days after transplantation. Three patients with severe ma
nifestations at the time of transplant had partial engraftment, and lo
st their graft within 3 months. They survived with thalassemia 1041-13
57 days after transplantation. One patient who received one antigen mi
smatched marrow from her brother had no engraftment and was alive with
thalassemia 1429 days posttrans plant. One patient died early on day
9 from CNS complication. No GVHD was observed in this series. These re
sults indicate that bone marrow transplantation can cure thalassemia b
ut there is still high autologous recovery rate in those with severe m
anifestations. In Southeast Asia, both thalassemias and hemoglobinopat
hies are prevalent(1,2). Those abnormal genes in various combinations
lead to more than 60 different thalassemic syndromes. Of these, homozy
gous beta-thalassemia and double heterozygosity between beta-thalassem
ia and hemoglobin(Hb)E are the most severe forms in which patients can
be born alive. It has been estimated that 600 new cases of homozygous
beta-thalassemia and 3000 new cases of beta-thalassemia /Hb E disease
are born annually, beta-thalassemia/Hb E disease is more common than
homozygous beta-thalassemia in Southeast Asia because of much higher f
requencies of Hb E(1,2). At present bone marrow transplantation is the
only mode of curative therapy.for thalassemia(3-7). Up to now more th
an 500 patients have been successfully transplanted(8). We report here
in our experience of bone marrow transplantation for the treatment of
thalassemia in Thailand.