title: Cure of X-linked lymphoproliferative disease (XLP) with allogeneic hematopoietic stem cell transplantation (HSCT): report from the XLP registry.
abstract: Seven male patients in the David T Purtilo International X-linked Lymphoproliferative Disease (XLP) Registry have undergone allogeneic hematopoietic stem cell transplantation (HSCT). All patients received HSCT from HLA-identical donors: sibling BM, five; unrelated BM, one; and sibling umbilical cord blood, one. Ages at time of HSCT ranged from 5 to 30 years. Pre-HSCT clinical course varied, but four boys had a significant history of chronic and/or serious infections. Conditioning regimens varied: TBI containing regimens, four, chemotherapy only, three. All patients engrafted. Six developed grade I-II acute GVHD but no chronic GVHD. Four are alive and well with normal immune function greater than 3 years following HSCT. Three died within 100 days: disseminated adenovirus, one; polymicrobial sepsis, one; and multiple organ system failure and bleeding diathesis, one. No EBV-associated post-transplant complications were observed, even though all donors except the umbilical cord blood were EBV-seropositive. Unsuccessful HSCT was associated with age at HSCT (> 15 years), TBI-containing regimen and significant history for pre-HSCT infections. These results provide evidence that HSCT performed during childhood with HLA-identical sibling donors, regardless of EBV serostatus, offers the only curative therapy for XLP.
authors: Department of Pathology@Microbiology, University of Nebraska Medical Center, Omaha 68198-5660, USA.
authors: T G
authors: TG
authors: Gross
authors: A H
authors: AH
authors: Filipovich
authors: M E
authors: ME
authors: Conley
authors: E
authors: E
authors: Pracher
authors: K
authors: K
authors: Schmiegelow
authors: J D
authors: JD
authors: Verdirame
authors: M
authors: M
authors: Vowels
authors: L L
authors: LL
authors: Williams
authors: T A
authors: TA
authors: Seemayer
mesh_terms: D000293:Adolescent; D000328:Adult; D000914:Antibodies, Viral / Q000097:blood; D002648:Child; D002675:Child, Preschool; D005260:Female; D005312:Fetal Blood / Q000166:cytology; D008040:Genetic Linkage; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006680:HLA Antigens; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D004854:Herpesvirus 4, Human / Q000276:immunology; D006801:Humans; D019520:Living Donors; D008232:Lymphoproliferative Disorders / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D008297:Male; D012042:Registries; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D014960:X Chromosome / Q000235:genetics*
chemical_list: D000914:Antibodies, Viral; D006680:HLA Antigens