Clinical study on haploidentical hematopoietic stem cell transplantation for children with bone marrow failure syndromes / 中华实用儿科临床杂志

ABSTRACT

Objective:To explore the efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSC) combined with tpCB in the treatment of children with bone marrow failure syndromes (BMFs).Methods:The clinical chara-cteristics of 78 BMFs pediatric patients, including inherited BMFs (4 cases) and acquired BMFs (74 cases) under-went haplo-HSC combined with the third-party cord blood (tpCB) in Chinese People′s Liberation Army General Hospital-Sixth Medical Center between July 2012 and July 2019 and were retrospectively analyzed, with 41 males and 37 females.Among them, 73 cases experienced first transplantation and 5 cases accepted second transplantation, with the median age of 5.6 years.The conditioning regimen was based on Busulfan, with 74 acquired BMFs cases using non-myeloablative and the remaining 4 cases using myeloablative.The prophylaxis of acute graft versus host disease (aGVHD) includes Cyclosporine, Mycophenolate mofetil (MMF) and Methotrexate.All patients received bone marrow from haploid donor and tpCB on day 1 and peripheral stem cell from haploid donor on day 2.The median dose of the total donor nucleated cells was 12.19×10 8/kg of recipient weight and CD 34+ cell dose was 6.13×10 6/kg of recipient weight. Results:The median time of granulocytes over 0.5×10 9/L and platelets over 20×10 9/L were + 13 d and + 17 d, respectively.All patients displayed complete donor-type chimerism at + 30 d. No primary graft failure occurred in any patient and second graft failure occurred in two cases.The incidence rate of grade ⅡtoⅣ and grade Ⅲ to Ⅳ aGVHD were 39.0% and 13.9%, respectively.The incidence of chronic GVHD with limited type and extensive type were 7.8% (95% CI 7.1%-8.5%) and 2.6% (95% CI 2.1%-3.1%), respectively.the median follow-up was 1 550 days, and 76 patients survived with free disease.The rate of transplant related mortality was 2.8%, and both of the estimated 5-year overall survival and failure-free survival rate were 97.2%(95% CI 96.8%-97.6%). Conclusions:Haplo-HSC and umbilical cord blood can quickly provide hematopoietic stem cells.The results of haplo-HSC combined with the tpCB in pediatric patients with life-threatening BMFs are promising.