Funds from Bloodwise, Cancer Research UK and the AAT, enable Professor Judith Marsh and colleagues to make a compelling argument. Published in Biology of Blood and Marrow Transplantation, May 2019

The proposal to consider upfront matched unrelated donor (MUD) stem cell transplant in adults who would have been considered for a matched sibling donor (MSD) SCT but who lack such a donor, as an alternative to immuno-suppressive treatment if an MUD is readily available is compelling and now warrants similar acceptance as for children with Severe Aplastic Anaemia.

Lay summary:

  • Recent decades have seen significant successes in HLA-matched unrelated donor stem cell transplantation for severe aplastic anaemia – mainly due to improved HLA matching, better conditioning regimens with fludarabine, avoidance of high-dose total body irradiation, introduction of ciclosporin as post-grafting immunosuppression to reduce Graft vs Host Disease and aid engraftment, and other breakthroughs
  • Previously this was reserved as an option following at least 2 courses of IST in SAA in adults, more recently – after 1 failed course of IST
  • Recent national and international studies and guidelines now recommend upfront MUD SCT for children for whom a matched unrelated donor is readily available because outcomes are similar to those of a matched sibling donor SCT
  • Professors Judith Marsh, Antonio M. Risitano and Ghulam Mufti examine the evidence supporting consideration of early matched unrelated donor SCT, who have such a donor readily available, especially using an irradiation-free conditioning regimen, with a low risk of GVHD, as another treatment option.
  • This option may be offered to patients as an informed choice, with the aim of curing their AA rather than achieving freedom from disease – a relapse-free survival, or overall survival.

You can read the full article here.