Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency. Pediatrics 2009 Mar;123(3):836-40
Date
03/04/2009Pubmed ID
19255011Pubmed Central ID
PMC3380632DOI
10.1542/peds.2008-1191Scopus ID
2-s2.0-63149138368 (requires institutional sign-in at Scopus site) 99 CitationsAbstract
OBJECTIVES: Leukocyte adhesion deficiency is a rare primary immune disorder caused by defects of the CD18 beta-integrin molecule on immune cells. The condition usually presents in early infancy and is characterized by deep tissue infections, leukocytosis with impaired formation of pus, and delayed wound healing. Allogeneic hematopoietic stem-cell transplantation offers the possibility of curative therapy, and with patient numbers at any individual center being limited, we surveyed the transplant experience at 14 centers worldwide.
METHODS: The course of 36 children with a confirmed diagnosis of leukocyte adhesion deficiency who underwent hematopoietic stem-cell transplantation between 1993 and 2007 was retrospectively analyzed. Data were collected by the registries of the European Society for Immunodeficiencies/European Group for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research.
RESULTS: At a median follow-up of 62 months (extending to 14 years), the overall survival rate was 75%. Myeloablative conditioning regimens were used in 28 patients, and reduced-intensity conditioning in 8 patients, with no deaths in this subgroup. Survival rates after matched family donor and unrelated donor transplants were similar, with 11 of 14 matched family donor and 12 of 14 unrelated donor recipients alive; mortality was greatest after haploidentical transplants, after which 4 of 8 children did not survive. Twenty-seven transplant recipients were alive, with full donor engraftment in 17 cases, mixed multilineage chimerism in 7 patients, and mononuclear cell-restricted chimerism in an additional 3 cases.
CONCLUSIONS: Hematopoietic stem-cell transplantation offers long-term benefit in leukocyte adhesion deficiency and should be considered as an early therapeutic option if a suitable HLA-matched stem-cell donation is available. Reduced-intensity conditioning was particularly safe, and mixed-donor chimerism seems sufficient to prevent significant symptoms, although careful long-term monitoring will be required for these patients.
Author List
Qasim W, Cavazzana-Calvo M, Davies EG, Davis J, Duval M, Eames G, Farinha N, Filopovich A, Fischer A, Friedrich W, Gennery A, Heilmann C, Landais P, Horwitz M, Porta F, Sedlacek P, Seger R, Slatter M, Teague L, Eapen M, Veys PAuthor
Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AdolescentBone Marrow Purging
Child
Child, Preschool
Chimerism
Female
Follow-Up Studies
Graft Survival
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Histocompatibility Testing
Humans
Infant
Leukocyte-Adhesion Deficiency Syndrome
Male
Opportunistic Infections
Retrospective Studies
Risk Factors
Survival Rate
Virus Activation
Virus Diseases
