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 an HLA-matched donor. Results show that this approach is feasible in AA, but more experience is needed to determine the long-term outcome of this approach.[66][67][68][69] 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

Successful stem cell transplant using haploidentical donors with post-transplant cyclophosphamide has been reported in 2 patients with paroxysmal nocturnal haemoglobinuria.[70] This approach has been further explored with early promising results, especially in patients who lack donor-specific HLA antibodies.[71][72]

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