This protocol applies to patients with acquired severe or very severe aplastic anaemia (SAA/VSAA) who have not achieved an adequate haematological response after a first course of immunosuppressive therapy, and who either have no HLA-matched sibling donor available or are over 40 years of age with a matched sibling.
First-line treatment with horse anti-thymocyte globulin, ciclosporin, and eltrombopag did not achieve the required response by 6 months: transfusion independence, or a complete response defined by haemoglobin normal for age and gender, neutrophils above 1.5 × 10&sup9;/L, and platelets above 150 × 10&sup9;/L.
Acquired SAA or VSAA in patients with no available HLA-matched sibling donor, or — when a matched sibling is present — in patients aged over 40 years. Patients between 40 and 50 years with a matched sibling may require individual evaluation based on performance status, comorbidities, and centre expertise.
Salvage therapy in this setting may involve transplant-based or non-transplant pathways, chosen based on donor availability and individual patient eligibility — full selection criteria, sequencing, and treatment details are in the complete protocol.
DOI: 10.1111/bjh.19236