Application of umbilical cord blood transplantation in the treatment of paroxysmal nocturnalhemoglobinuria
10.3760/cma.j.cn421203-20200402-00105
- VernacularTitle:非血缘脐血移植治疗阵发性睡眠性血红蛋白尿症疗效分析
- Author:
Kaidi SONG
1
;
Xiaoyu ZHU
;
Baolin TANG
;
Xiang WAN
;
Wen YAO
;
Guangyu SUN
;
Huilan LIU
;
Zimin SUN
Author Information
1. 中国科学技术大学附属第一医院（安徽省立医院）血液科，合肥 230001
- Keywords:
Hematopoietic stem cell transplantation;
Paroxysmal nocturnal hemoglobinuria;
Cord blood
- From:
Chinese Journal of Organ Transplantation
2021;42(7):422-425
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To explore the feasibility and efficacy of umbilical cord blood transplantation (UCBT) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH).Methods:From May 2014 to December 2019, clinical data were retrospectively reviewed for 7 PNH patients undergoing UCBT. The grades were severe ( n=6) and extremely severe ( n=1). The causes were primary PNH ( n=4) and PNH-aplastic anemia (AA) syndrome ( n=3). There were 5 males and 2 females with a median age of 29 (20-47) years, a median weight of 60(50-71) kg and a median time from diagnosis to transplantation of 62.5(7.7-171) months. All of them were accompanied by transfusion dependence. Myeloablative ( n=6) and reduced-intensity ( n=1) pretreatment was offered. The regimen of preventing GVHD was cyclosporine A plus short-term mycophenolate mofetil without ATG. The median number of input nucleated cells was 2.4(1.71-4.28)×10 7/kg and the median number of CD34+ cells 1.58(0.88-3.02)×10 5/kg. Results:Neutrophil and erythroid engraftment was obtained with a median neutrophil engraftment time of 17(15-21) days and a median erythroid engraftment time of 27. Engraftment time of 37(25-101) days for platelets >20×10 9/L and 62(27-157) days for platelets >50×10 9/L. The incidence of 100-day acute GVHD was 28.6%(95%CI 0-55.3%). The severity of GVHD was grade Ⅱ° acute ( n=2) and mild ( n=1). The median follow-up period was 13.5(3-71.4) months. Six patients survived while another with PNH-AA syndrome with iron overload died of gastrointestinal hemorrhage. The 2-year overall survival rate was 83.3%(95%CI 27.3-97.5%). Conclusions:With excellent engraftment and survival in the treatment of PNH, UCBT is indicated for patients without HLA full-match donor. PNH-AA syndrome with iron overload may be one of the important prognostic factors.
