Bone marrow transplantation for primary immunodeficiencies using matched unrelated donor and parental graft

Abstract

Primary immunodeficiencies (PID) such as severe combined immunodeficiency (SCID) and Wiskott-Aldrich syndrome (WAS) are lethal unless bone marrow transplantation (BMT) is available. However, only about 25% of pediatric patients in Hong Kong had matched sibling donors. Therefore, between April 1992 and August 1994, we performed one BMT using matched unrelated donor (MUD) in a boy with WAS, three BMT using mismatched (2-3 Ag mismatched) parents in two boys with WAS and one girl with SCID, and one BMT using a phenotypically matched parent in a boy with SCID. Their ages at BMT ranged from 0.5 to 4. 7 years. Their conditioning regimen included busulfan, cyclophosphamide, and Campath 1-G (2 WAS, 1 SCID), CBV (1 WAS with EBV-associated lymphoma who had MUD BMT), and nil for the SCID patient who received BMT from a phenotypically matched parent. Prophylaxis for GVHD included cyclosporin A and methotrexate for all five patients. Engraftment occurred between Days 13 and 25. Acute GVHD (grade 1-2) occurred in all five patients and one patient (WAS) had chronic GVHD. Mixed chimerism was demonstrated in four patients and the follow-up time ranged from 6 months to 2 years, 10 months. All five patients are alive and at home at the time of reporting. We conclude that using MUD and mismatched parents as alternative donors for children with SCID and WAS is feasible in Hong Kong. (Acknowledgment: We thank Dr. Gareth Morgan (London) for advice and the supply of Campath 1-G.)