Allogeneic haematopoietic stem cell transplantation with decitabine-containing preconditioning regimen in TP53-mutant myelodysplastic syndromes: A case study

Abstract

Myelodysplastic syndrome (MDS) with TP53 mutations has a poor prognosis after transplantation, and novel therapeutic means are urgently needed. Decitabine (Dec) monotherapy has demonstrated improved overall response rates in MDS and acute myeloid leukaemia, although these responses were not durable. This study aimed to preliminary evaluate the efficacy of a Dec-containing allogeneic haematopoietic stem cell transplantation (allo-HSCT) preconditioning regimen in TP53-mutant MDS. Nine patients with TP53-mutant myelodysplastic syndromes received the decitabine-containing preconditioning regimen and subsequent myeloablative allo-HCT between April 2013 and September 2021 in different centres. At a median follow-up of 42 months (range, 5 to 61 months), the overall survival (OS) was 89% (8/9), progression-free survival (PFS) was 89% (8/9), and relapse incidence was 11.1%. The incidence of severe acute (grade III-IV) graft-versus-host disease (GVHD) was 22.2% (2/9) and that of chronic moderate-to-severe GVHD was 11.1% (1/9). The 1-year GVHD-free/relapse-free survival (GRFS) was 56% (5/9). In conclusion, we found real-world clinical data that supports the use of a Dec-containing preconditioning regimen before allo-HSCT for possible improved outcomes in TP53-mutant MDS patients; there is therefore an urgent call for an in-depth exploration of the involved mechanism to confirm these preliminary findings.

Document Details

Document Type
Pub Defense Publication
Publication Date
Jul 18, 2022
Source ID
10.3389/fonc.2022.928324

Entities

People

  • Bin Zhang
  • Botao Li
  • Jiangwei Hu
  • Jianlin Chen
  • Jing Ren
  • Jing Xie
  • Jingwen Niu
  • Lei Wang
  • Liangding Hu
  • Liping Dou
  • Liren Qian
  • Na Liu
  • Sanchun Lan
  • Yao Sun
  • Yehui Tan
  • Yu Zhang
  • Yuhang Li
  • Yuxin Wang
  • Zhuoqing Qiao

Organizations

  • Army Research Office
  • National Natural Science Foundation of China

Tags

Fields of Study

  • Biology
  • Medicine

Readers

  • Immunology and Pathology
  • Molecular and genetic basis of cancer.
  • Trauma or Military Medicine

Technology Areas

  • Biotechnology