title: Results of allogeneic bone marrow transplantation in patients with leukocyte adhesion deficiency.
abstract: We have retrospectively analyzed the outcome of bone marrow transplantation (BMT) in 14 patients with leukocyte adhesion deficiency (LAD) performed in two centers between 1981 and 1993. Five patients received BMT from HLA-identical donors. Nine received T-depleted marrow from two HLA antigen- or haplotype-incompatible parents. Conditioning regimen consisted of chemotherapy exclusively in 13 patients and associated with total body irradiation (TBI) in one patient. In five cases, failure of engraftment occurred as a result of either insufficient myeloablation (n = 3) or, possibly, graft rejection in two cases of moderate phenotype of LAD. The second conditioning regimen consisted of TBI and chemotherapy with the use of anti-lymphocyte function-associated antigen 1 (LFA-1) and anti-CD2 monoclonal antibodies for patients with the moderate phenotype of LAD. These patients were successfully retransplanted. Eight patients developed acute graft-versus-host disease (GVHD). Chronic GVHD occurred in five cases. GVHD led to the death of three patients. Ten patients are alive and well 12 months to 12 years after BMT. Chimerism is full in six of these patients and mixed but stable in four with variable proportion of donor leukocytes. One patient with less than 15% donor leukocytes has mild gingivitis, while the others are well. Sequelae from BMT are limited in two cases to growth retardation caused by TBI. Success of BMT in cases of LAD including seven of nine recipients of HLA nonidentical marrow indicates that this procedure can be proposed as a curative approach to LAD regardless of an available HLA-identical donor. Great care should be taken in GVHD prophylaxis and treatment.
authors.affiliation: Unité d'Immuno-Hématologie Pédiatrique, Hôpital Necker, Paris, France.
authors.forename: C
authors.initials: C
authors.lastname: Thomas
authors.forename: F
authors.initials: F
authors.lastname: Le Deist
authors.forename: M
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: M
authors.initials: M
authors.lastname: Benkerrou
authors.forename: E
authors.initials: E
authors.lastname: Haddad
authors.forename: S
authors.initials: S
authors.lastname: Blanche
authors.forename: W
authors.initials: W
authors.lastname: Hartmann
authors.forename: W
authors.initials: W
authors.lastname: Friedrich
authors.forename: A
authors.initials: A
authors.lastname: Fischer
mesh_terms: D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D018821:CD18 Antigens / Q000378:metabolism; D002448:Cell Adhesion; D002634:Chemotaxis, Leukocyte; D002648:Child; D002675:Child, Preschool; D002678:Chimera; D003602:Cytotoxicity, Immunologic; D005260:Female; D006086:Graft vs Host Disease / Q000150:complications; D006098:Granulocytes / Q000502:physiology; D006801:Humans; D016023:Integrins / Q000172:deficiency*; D007694:Killer Cells, Natural / Q000276:immunology; D018370:Leukocyte-Adhesion Deficiency Syndrome / Q000628:therapy*; D008297:Male
chemical_list: D018821:CD18 Antigens; D016023:Integrins