Haplo-identical transplantation for acquired severe aplastic anaemia in a multicentre prospective study

Br J Haematol. 2016 Oct;175(2):265-274. doi: 10.1111/bjh.14225. Epub 2016 Jun 28.

Abstract

We conducted a prospective, multicentre study to confirm the feasibility of haplo-identical transplantation in treatment of severe aplastic anaemia (SAA) as salvage therapy, by analysing the outcomes of 101 patients who received haplo-identical transplantation between June 2012 and October 2015. All cases surviving for more than 28 d achieved donor myeloid engraftment. The median time for myeloid engraftment was 12 (range, 9-25) days and 15 (range, 7-101) days for platelets, with a cumulative platelet engraftment incidence of 94·1 ± 0·1%. With a median follow-up of 18·3 (3·0-43·6) months, recipients from haplo-identical transplantation had more cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD, 33·7% vs. 4·2%, P < 0·001), more chronic GVHD (22·4% vs. 6·6%, P = 0·014) at 1 year, but similar grade III-IV aGVHD (7·9% vs. 2·1%, P = 0·157), 3-year estimated overall survival (OS, 89·0% vs. 91·0%, P = 0·555) and failure-free survival (FFS, 86·8% vs. 80·3%, P = 0·659) when compared with 48 patients who received contemporaneous transplantation from matched related donors. Multivariate analysis showed no significant difference in engraftment and survival between the two cohorts. Both OS and FFS for the entire population correlated significantly with grades III-IV aGVHD. In conclusion, haplo-identical transplantation is a feasible choice for SAA with favourable outcomes.

Keywords: haematopoietic stem cell transplantation; haploidentical; severe aplastic anaemia.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / diagnosis*
  • Anemia, Aplastic / etiology
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / therapy*
  • Biomarkers
  • Child
  • Child, Preschool
  • Female
  • Graft Survival
  • Graft vs Host Disease / epidemiology
  • Graft vs Host Disease / etiology
  • HLA Antigens / genetics
  • Haplotypes*
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / methods
  • Humans
  • Incidence
  • Male
  • Middle Aged
  • Prospective Studies
  • Retreatment
  • Severity of Illness Index
  • Survival Analysis
  • Tissue Donors*
  • Transplantation Conditioning
  • Transplantation, Homologous
  • Treatment Outcome
  • Young Adult

Substances

  • Biomarkers
  • HLA Antigens