Haploidentical haematopoietic stem cell transplantation for malignant infantile osteopetrosis and intermediate osteopetrosis: a retrospective analysis of a single centre

Abstract Objective To evaluate the clinical efficacy of haploidentical haematopoietic stem cell transplantation (haplo-HSCT) for treatment malignant infantile osteopetrosis (MIOP) and intermediate osteopetrosis. Methods Children with MIOP IOP who underwent haplo-HSCT in Beijing Children’s Hospital, Capital Medical University, from January 2010 to May 2018 were retrospectively analysed. Data relating manifestations, engraftment, prognosis children extracted medical records. Results Twenty-seven patients, including 18 males 9 females, an onset age 12 (0.04–72) months enrolled this study. The median time diagnosis was 4 (1–23) months. All patients received a myeloablative conditioning regimen (including fludarabine, busulfan, cyclophosphamide). Graft versus host disease (GVHD) prophylaxis based on anti-human T lymphocyte porcine immunoglobulin/anti-human thymus globulin, methotrexate, mycophenolate mofetil. observation 55.2 (0.3–126.2) By end follow-up, twenty survived seven died. 5 year overall survival rate 73.9%. Stage I-II acute GVHD observed 20 stage III 1 patient no had IV disease. Chronic 11 (40.7%) controlled by anti-GVHD therapy. Conclusions Haplo-HSCT effective IOP, high significantly improved symptoms. For vision impairment before HSCT, improvement slow after transplantation. incidence but mild effectively appropriate treatment. These data indicated that feasible IOP.