melphalan and Pulmonary-Aspergillosis

We report on 12 patients with chronic granulomatous disease transplanted with hematopoietic stem cells from matched unrelated (n = 9) or matched sibling donors (n = 3). The most common infectious complication was pulmonary aspergillosis, which nine patients had previously developed. Only 5 of 12 individuals had normal lung function prior to transplantation. At a mean follow-up of 53 months 9 of the 12 patients are alive including 7 of 9 following matched unrelated donor (MUD) transplantation. One patient died from ARDS, another from systemic BK virus infection, the third from complications of chronic graft-versus-host disease. Seven of nine surviving patients have normal lung function now. HSCT from a MUD is an option worth considering when no matched family donor is available. Restricted lung function prior to HSCT does not appear to be a limiting factor for such treatment.

Topics: Adolescent; Busulfan; Child; Child, Preschool; Chimerism; Cyclophosphamide; Follow-Up Studies; Granulomatous Disease, Chronic; Hematopoietic Stem Cell Transplantation; Histocompatibility; Humans; Kaplan-Meier Estimate; Male; Melphalan; Postoperative Complications; Pulmonary Aspergillosis; Retrospective Studies; Treatment Outcome; Vidarabine; Young Adult