Neonatal bone marrow transplantation for severe combined immunodeficiency

Aims-To evaluate outcome following neonatal bone marrow transplantation (BM T) for severe combined immunodeficiency (SCID) when there is a family histo ry of a previously affected sibling, and to compare results with those publ ished for in utero BMT. Methods-A retrospective review of cases referred and transplanted between 1 987 and 1999, focusing on infectious and graft versus host disease (GvHD) c omplications after BMT, and T and B lymphocyte function. Thirteen patients received 18 stem cell transplants: four whole marrow, one cord blood, 10 pa rental T cell depleted (TCD) haplo-identical, and three TCD unrelated donor BMT. Nine were conditioned with busulphan and cyclophosphamide. Results-All are alive and well (six months to 11.5 years after BMT). Six ha d grade I-II acute GvHD and two chronic GvHD (now resolved). Three had a to p up BMT for poor T cell function, one had a third BMT for graft failure an d chronic GvHD, and one had a third BMT for graft failure. Twelve have good in vitro proliferation to T cell mitogens, and all have normal serum IgA l evels. Three receive intravenous immunoglobulin; for one of these, it is le ss than one year since BMT. Nine are above the 2nd centile, and 10 of 12 ol d enough to be assessed have normal neurodevelopment. Conclusion-These results are better than those published for in utero BMT f or SCID. Early postnatal BMT should be the preferred option in neonatal SCI D.