Premium
Bone‐marrow transplantation for thalassaemia
Author(s) -
Vowels Marcus R.,
Berdoukas Vasili,
LamPoTang P. Reg L.,
Ford David
Publication year - 1986
Publication title -
medical journal of australia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 131
eISSN - 1326-5377
pISSN - 0025-729X
DOI - 10.5694/j.1326-5377.1986.tb115927.x
Subject(s) - medicine , transplantation , surgery , chelation therapy , bone marrow , cyclophosphamide , transfusion therapy , thalassemia , blood transfusion , chemotherapy
An 18‐month‐old boy with betathalassaemia major underwent bone‐marrow transplantation with marrow from“ his 30‐month‐old brother. The brother was HLA‐identical, mixed‐lymphocyte culture non‐reactive and had thalassaemia minor. The patient was ”conditioned“ with busulphan and cyclophosphamide before transplantation and received methotrexate to prevent graft‐versus‐host disease. Immediately after the transplant, complications arose, which included mild graft‐versus‐host disease, gastrointestinal bleeding and fever. The boy is alive 18 months after transplantation, is leading a normal life, is receiving no therapy and has a normally functioning donor marrow with thalassaemia minor. Bone‐marrow transplantation may be considered as alternative therapy in patients with betathalassaemia who are young, and who have no organ dysfunction or iron overload. Chronic transfusion and chelation therapy and its problems must be weighed against the 13% risk of mortality and the 73% chance of a normal life that are associated with transplantation. Older patients, who have received multiple blood transfusions, have iron overload or have organ dysfunction, have a low survival rate after transplantation and this therapy is inappropriate for such patients.