Aplastic anemia
ICD-10 D61.9 · ICD-11 3A70

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.

Treatment Approach — Partial Overview

Up-front allogeneic haemopoietic stem cell transplant from the matched sibling donor is the recommended intervention in this setting. The specific conditioning and post-transplant immunosuppression strategy are determined by the patient's age within this under-40 group.

Complete conditioning details, agent selection, sequencing, and post-graft management are in the full structured protocol.

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)
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