Clinical Scenario

This protocol covers patients with acquired severe or very severe aplastic anaemia (SAA/VSAA) who either have no available HLA-matched sibling donor, or who have such a donor but are aged over 40 years — a population in whom the standard sibling transplant pathway is not the primary approach.

Patient Population

Two distinct groups are addressed: SAA/VSAA patients in the absence of a matched sibling donor, and SAA/VSAA patients with a matched sibling donor who are over 40 years of age. Patients aged between 40 and 50 years with a matched sibling donor require individual assessment taking into account performance status and comorbidities, as well as centre expertise, before the appropriate pathway is confirmed.

Treatment Approach (Partial Overview)

The structured regimen for this scenario centres on an alternative donor transplant strategy with a specific conditioning approach. The preferred donor type, full conditioning details, sequencing, and evidence grading are set out in the complete protocol below.

References

DOI: 10.1111/bjh.19236

SAA/VSAA in the absence of a MSD.
SAA/VSAA with a MSD and >40 years of age (patients aged between 40 and 50 years with MSD could be considered for HSCT depending on performance status and comorbidities, in addition to centre expertise)
Alternative donor HSCT using either cord blood or a haploidentical family donor, a 9/10 MUD may be considered, among other treatment options, after failure to respond to IST and in the absence of a MSD or a suitably matched MUD.
Alternative donor HSCT is an option for patients who lack a suitably matched donor; a haploidentical family donor is preferable to cord blood HSCT, using a reduced-intensity regimen with post-transplant high-dose cyclophosphamide (2B).

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Severe or Very Severe Aplastic Anaemia: Treatment When No HLA-Matched Sibling Donor Is Available, or Patient Is Aged Over 40

References