CURE OF X-LINKED LYMPHOPROLIFERATIVE DISEASE (XLP) WITH ALLOGENEIC HEMATOPOIETIC STEM-CELL TRANSPLANTATION (HSCT) - REPORT FROM THE XLP REGISTRY

Seven male patients in the David T Purtilo International X-Linked Lymp hoproliferative Disease (XLP) Registry have undergone allogeneic hemat opoietic stem cell transplantation (HSCT), All patients received HSCT from HLA-identical donors: sibling BM, five; unrelated BM, one; and si bling 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 signif icant history of chronic and/or serious infections, Conditioning regim ens varied: TBI containing regimens, four, chemotherapy only, three, A ll patients engrafted, Six developed grade I-II acute GVHD but no chro nic 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-transplan t complications were observed, even though all donors except the umbil ical cord blood were EBV-seropositive. Unsuccessful HSCT was associate d 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, reg ardless of EBV serostatus, offers the only curative therapy for XLP.