Treatment of Severe Aplastic Anemia in Patients Under 40 with an Available HLA-Matched Sibling Donor
This page addresses first-line treatment for patients with acquired severe or very severe aplastic anaemia who are aged under 40 and have a confirmed HLA-matched sibling donor — a specific combination that defines a distinct clinical pathway.
Clinical Scenario
Acquired severe or very severe aplastic anaemia, defined by marrow cellularity below 25%, with at least two of the following: neutrophils <0.5 × 109/L, platelets <20 × 109/L, or reticulocytes <60 × 109/L. Patient age under 40 years, with a confirmed HLA-matched sibling donor available.
References
DOI: 10.1111/bjh.19236
- Severe AA (SAA): Marrow cellularity <25% (or 25%–50% with <30% residual haematopoietic cells), plus at least two of: (a) neutrophil count <0.5 × 109/L; (b) platelet count <20 × 109/L; (c) reticulocyte count <60 × 109/L (using an automated reticulocyte count)
- Up-front HSCT from a MSD is indicated for SAA in young and adult patients aged <40 years who have a MSD.
- Standard treatment for newly diagnosed acquired SAA patients is ATG-based IST with eltrombopag or allogeneic HSCT from a matched sibling donor (MSD)