PMID- 32654467 OWN - NLM STAT- MEDLINE DCOM- 20200901 LR - 20200901 IS - 0253-2727 (Print) IS - 2707-9740 (Electronic) IS - 0253-2727 (Linking) VI - 41 IP - 6 DP - 2020 Jun 14 TI - [Clinical study of haploidentical hematopoietic stem cell transplantation on 15 cases of adult-onset primary hemophagocytic lymphohistiocytosis]. PG - 511-516 LID - 10.3760/cma.j.issn.0253-2727.2020.06.014 [doi] AB - Objective: This study was designed to evaluate the efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for adult-onset primary hemophagocytic lymphohistiocytosis (HLH) . Method: A retrospective study was carried out to analyze the clinical data of 15 adult patients with primary HLH who received haplo-HSCT from January 2013 to October 2019 in Beijing Friendship Hospital, Capital Medical University, Beijing, China. Results: Among the 15 patients included in the study, ten were males and five were females, with a median age of 21 years old (18-52) . Eight of the patients had familial hemophagocytic lymphohistiocytosis type 2 (FHL-2) , four had FHL-3, one had Griscelli syndrome type 2 (GS-2) , one had X-linked lymphoproliferative disease type 1 (XLP-1) , and the other had XLP-2. The median time from HLH diagnosis to transplantation was 7 months (2-46 months) . Seven patients were treated with Bu/Cy condition regimen prior to transplantation. Meanwhile, the other eight cases were treated with TBI/Cy. The median concentration of mononuclear cell (MNC) infusion was 12.6 (9.2-20.3) x10(8)/kg and CD34(+) cells was 4.91 (2.51-8.37) x10(6)/kg. The median time of leukocyte engraftment was on day 13 following transplantation (10-23 days) , and the platelet engraftment was on day 12 (9-36) . Graft failure (GF) finally occurred in two patients (one primary GF and one secondary GF) . The cumulative incidence of acute graft-versus-host-disease (GVHD) grades 2 to 4 was 71.4% (10/14) and chronic GVHD was 30.8% (4/13) , respectively. The five-year overall survival (OS) for all 15 cases of primary HLH was 65.5% (95% CI, 34.9%-73.3%) and the transplant-related mortality (TRM) was 26.7% (4/15) . The five-year OS was 87.5% (95% CI, 38.7%-66.3%) in eight patients who received haplo-HSCT subsequent to initial therapy and 42.9% (95% CI, 8.5%-65.2%) in patients seven patients who needed salvage therapy prior to haplo-HSCT (chi(2)=2.387, P=0.122) . The five-year OS was 85.7% (95% CI, 50.4%-89.8%) in eight patients who achieved complete response before haplo-HSCT and 42.9% (95% CI, 6.4%-53.0%) in seven patients with partial response (chi(2)=3.185, P=0.074) . Conclusion: The results indicated that haplo-HSCT is a promising method for the treatment of primary HLH in adults. FAU - Wang, Y N AU - Wang YN AD - Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China. FAU - Wang, J S AU - Wang JS AD - Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China. FAU - Wei, N AU - Wei N AD - Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China. FAU - Wu, L AU - Wu L AD - Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China. FAU - Jin, Z L AU - Jin ZL AD - Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China. FAU - Wang, Z AU - Wang Z AD - Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China. LA - chi GR - 81871633/National Natural Science Foundation of China/ GR - 7181003/Beijing Natural Science Foundation/ GR - XMLX201823/Beijing Municipal Administration of Hospital Clinical Medicine Development of Special Funding Support/ GR - DFL20180101/Beijing Municipal Administration of Hospitals' Ascent Plan/ PT - Journal Article PL - China TA - Zhonghua Xue Ye Xue Za Zhi JT - Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi JID - 8212398 SB - IM MH - Adolescent MH - Adult MH - China MH - Female MH - *Graft vs Host Disease MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - *Lymphohistiocytosis, Hemophagocytic MH - Male MH - Retrospective Studies MH - Transplantation Conditioning MH - Treatment Outcome MH - Young Adult PMC - PMC7378280 OTO - NOTNLM OT - Adult onset OT - Haploidentical OT - Hematopoietic stem cell transplantation OT - Primary hemophagocytic lymphohistiocytosis EDAT- 2020/07/14 06:00 MHDA- 2020/09/02 06:00 PMCR- 2020/06/01 CRDT- 2020/07/14 06:00 PHST- 2020/07/14 06:00 [entrez] PHST- 2020/07/14 06:00 [pubmed] PHST- 2020/09/02 06:00 [medline] PHST- 2020/06/01 00:00 [pmc-release] AID - cjh-41-06-511 [pii] AID - 10.3760/cma.j.issn.0253-2727.2020.06.014 [doi] PST - ppublish SO - Zhonghua Xue Ye Xue Za Zhi. 2020 Jun 14;41(6):511-516. doi: 10.3760/cma.j.issn.0253-2727.2020.06.014.