Hematopoietic stem cell transplantation from matched unrelated donors in chronic granulomatous disease.
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.
Schuetz
C
C
Department of Paediatrics, University Hospital Ulm, Paediatrics Eythstrasse 24, 89075, Ulm, Germany. catharinaschuetz@gmx.de
Hoenig
M
M
Gatz
S
S
Speth
F
F
Benninghoff
U
U
Schulz
A
A
Debatin
K M
KM
Friedrich
W
W
D000293:Adolescent; D002066:Busulfan / Q000008:administration & dosage / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D046528:Chimerism; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000627:therapeutic use; D005500:Follow-Up Studies; D006105:Granulomatous Disease, Chronic / Q000276:immunology / Q000401:mortality / Q000601:surgery*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006648:Histocompatibility / Q000276:immunology; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use; D011183:Postoperative Complications / Q000276:immunology / Q000401:mortality; D055732:Pulmonary Aspergillosis / Q000209:etiology / Q000401:mortality; D012189:Retrospective Studies; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives / Q000627:therapeutic use; D055815:Young Adult
D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine; D008558:Melphalan