This protocol addresses patients with non-severe aplastic anemia (NSAA) — those who do not fulfil the diagnostic criteria for severe or very severe AA — who nonetheless present with clear indications for treatment: transfusion dependency, clinically significant bleeding, or recurrent infections.
Although classified as non-severe, patients in this sub-group have disease that substantially affects their clinical status or quality of life, warranting active management rather than observation alone.
For NSAA meeting treatment criteria, the approach centres on immunosuppressive combination therapy as the primary intervention. The complete protocol specifies the regimen components, evidence basis, and available alternatives — including what is and is not approved for this indication.
The target is a meaningful haematological response, defined as achieving transfusion independence or measurable cell-line recovery:
DOI: 10.1111/bjh.19236
Non-severe AA (NSAA): AA not fulfilling the criteria for SAA or VSAA
Indications for treatment are transfusion dependency, progression to SAA and where patient lifestyle dictates.
Based on the only prospective randomised trial in NSAA, the combination of ATG and CSA results in similar OS but better failure-free survival and a faster and better response (74% vs. 46%, respectively), compared to CSA alone.
Danazol is another option, but there are limited data on efficacy and supply issues.
The use of eltrombopag for NSAA is not approved.
Partial | Transfusion independence (if previously dependent) or doubling or normalisation of at least one cell line or increase of baseline • Haemoglobin concentration of >30 g/L (if initially <60) • Neutrophils of >0.5 × 10⁹/L (if initially <0.5) • Platelets of >20 × 10⁹/L (if initially <20)
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