Abstract

Successful BMT was performed in a 9 yr. old black female with severe idiopathic aplastic anemia from her HLA-MLC non-identical father. Initial Hgb.=3.8 gm.%, retic. count=0.1%, WBC=700 cmm., platelets=10,000 cmm.; bone marrow biopsy was markedly hypocellular with 85% non-hematopoietic elements. Tissue typing showed the patient to be HLA A-1 B-8, A-26 B-7; the father was HLA A-1 B-8, A-3 B-7. In mixed leukocyte culture (MLC), there was low but significant bidirectional (10%; 16%) stimulation. Cell mediated lympholysis demonstrated no significant killing. Pre-transplant preparation included Procarbazine 15 mg/kg/day × 3 days, anti-thymocyte globulin (ATG) 15 mg/kg/day x 3 days, and 750 rads of total body irradiation by linear accelerator. Cells from the donor (6.9 × 108/kg) were infused intravenously. Graft versus host (GVH) prophylaxis following BMT consisted of IV Methotrexate weekly × 100 days and ATG and corticosteroids on days 7-21. By day 28 post BMT, cells of donor HLA type were present and marrow chromosomes were 100% XY. On days 29-31 mild GVH developed with fever, rash and slight liver enzyme elevations. Skin biopsy was consistent with GVH. Skin GVH resolved with Prednisone treatment. At 7 months post BMT peripheral counts are normal, however liver enzyme elevations persist. Marrow engraftment with minimal GVH in this patient should encourage further trials of BMT in patients without HLA-MLC identical siblings.

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