Complete Recovery of Hemopoiesis Following Bone Marrow Transplantation in a Patient with Unresponsive Congenital Pure Red Cell Anemia

Abstract

Bone marrow transplantation (BMT) was carried out on a 1-year-old boy with congenital pure red cell anemia who did not respond to corticosteroids, cyclophosphamide, and antilymphocyte globulin. Peripheral blood examination at diagnosis showed WBC 6, 900/mm³, RBC 236×10⁴./mm³, Hb 8.3 g/dl, Plat. 62×10⁴/mm³, Reticulo. 0 ‰, and blood type was group O Rh (D) +. He had an HLA identical 4-year-old brother whose blood type was group A Rh (D) +. After he received cyclophosphamide (50 mg/kg for 4 days) and busulfan (3 mg/kg for 4 days) followed by 350 rads of total body irradiation, nucleated marrow cells (2.8×10⁸/kg) were transfused on January 26th, 1987. Short-term MTX and CSA was given to prevent acute GVHD. No evidence of severe complication has developed after transplantation. The recovery of WBC over 1, 000/mm³, neutrophil over 500/mm³, and platelet over 5×10⁴/mm³ was day 19, 28, and 26, respectively. Bone marrow examination on day 58 showed the marked evidence of erythropoiesis and engraftment was confirmed by the blood type on day 120. He has been completely healthy and hematologically normal for more than 2 years. This is the first successful case of allogeneic transplant for congenital pure red cell anemia in Japan.