Bone marrow transplantation in two patients with aplastic anaemia

Abstract

Bone marrow transplantation is now established as a significant advance in the management of patients with severe aplastic anaemia. Two such patients, a male of 19 yr and a female aged 14, received bone marrow transplants from female sibling donors at Royal Perth Hospital in January 1979. In each case, donor and recipient were HLA identical and mutually non-stimulatory in mixed lymphocyte culture. The patients were isolated in laminar flow (Clemco) units for 2 wk prior to transplantation and for 4 wk subsequently. They received cyclophosphamide 50 mg/kg daily from day –5 to day –2 as preconditioning, and methotrexate 15 mg weekly post-transplant as prophylaxis against graft versus host disease (GVHD). The girl's transplant was ABO incompatible, the donor being group AB and the recipient group O. A Haemonetics-30 cell separator was used to reduce the anti-A and anti-B titres of the recipient prior to the marrow infusion. Plasmaphereses, each of 4 1, were performed on days – 2, – 1, and 0. No haemolysis was encountered during marrow transfusion. She received 3.0×10⁸ marrow cells/kg. Engraftment was observed on day 15 and the peripheral count rose rapidly to normal levels. Her blood group gradually converted to AB. and the anti-A and anti-B titres disappeared. The pre-engraftment phase was complicated by severe oral herpes simplex ulceration and adenovirus cystitis. On day 39 GVHD became evident. Prednisolone in a dose of 2 mg/kg/d improved the skin rash, but diarrhoea and vomiting persisted and hepatic function deteriorated. Antithymocyte globulin in a dose of 10 mg/kg on alternate days was commenced on day 57, and continued for 3 wk without effect. She eventually became thrombocytopenic with severe megakaryocyte hypoplasia and died from gastrointestinal haemorrhage on day 114. The boy received 2.15×10⁸ marrow cells/kg. Engraftment was first evident on day 18. The peripheral blood count improved slowly, not reaching normal levels until day 110. Apart from transient GVHD on day 70, a mild chest infection around day 100, and a photosensitivity rash attributed to Septrin, he has remained well throughout.