Combination of haploidentical haematopoietic stem cell transplantation with an unrelated cord-blood unit in patients with severe aplastic anemia: a report of 146 cases

Bone Marrow Transplant. 2020 Oct;55(10):2017-2025. doi: 10.1038/s41409-020-0874-9. Epub 2020 Mar 26.

Abstract

We analyzed the outcomes of 146 severe aplastic anemia (SAA) patients who received a combination of haploidentical haematopoietic stem cell transplantation (haplo-HSCT) and an unrelated cord-blood (UCB) unit between September 2011 and December 2017. One hundred and seventeen patients underwent transplantation as first-line therapy. Seven patients experienced early mortality, and among the evaluable 139 patients, one patient experienced primary graft failure (GF), while the other 138 patients achieved successful haploidentical donor engraftment; additionally, three patients experienced secondary GF. Six patients demonstrated delayed platelet recovery, and three patients demonstrated platelet GF. The median time for myeloid and platelet engraftment was 11 (range: 9-28) days and 15 (range: 9-330) days, respectively. With a median follow-up of 40 (range: 18-93) months, the cumulative incidences were 31.43% and 10.00% for grades II-IV and grades III-IV acute graft-versus-host disease (GVHD), respectively. The cumulative incidences of chronic GVHD (cGVHD) and moderate-severe cGVHD were 36.23% and 11.71%, respectively. There was no patient relapse. The probabilities of 4-year overall survival and GVHD-free, failure-free survival were 81.4 ± 3.3% and 69.2 ± 3.9%, respectively. These encouraging preliminary results indicated that haplo-HSCT combined with the infusion of UCB is a feasible choice for SAA patients without matched donors.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia, Aplastic* / therapy
  • Fetal Blood*
  • Graft vs Host Disease*
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Tissue Donors
  • Transplantation Conditioning