Emerging treatments

Umbilical cord blood as stem cell source

Umbilical cord blood is increasingly being used as a source of stem cells, as this allows transplantation in patients without a human leukocyte antigen (HLA- antigen) matched donor. Results show that this approach is feasible in aplastic anaemia, but more experience is needed to determine the long-term outcome.[90][91][92][93][94]​​​ Furthermore, it is an expensive procedure as high stem cell dose (usually two cord units) is needed to reduce the risk of graft rejection.

Haploidentical stem cell transplantation (SCT)

Haploidentical SCT may be considered in patients whose disease has not responded to immunosuppressive treatment, or for whom a matched sibling or unrelated donor is unavailable and is preferable to cord blood SCT in these patients.​[1]​ Haploidentical donors are readily available and most patients will have at least one potential haploidentical stem cell donor in their family.[1][95]​​ Evidence from meta-analysis suggests promising results in terms of engraftment rates and risk of complications.[96]​ Overall survival rates in studies of haploidentical SCT (with post-transplantion cyclophosphamide to prevent graft versus host disease) have been reported as 81% to 94% at 1 year, 78% at 2 years and 63% at 3 years.[97][98][99][100][101]

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