Safety and efficacy of immunosuppressive therapy for elderly patients with severe aplastic anaemia

Summary Uncertainty remains regarding the safety and tolerability of immunosuppressive therapy (IST) with anti‐thymocyte globulin (ATG) and cyclosporine (CSA) in older patients. We retrospectively analysed two prospective clinical trials of IST in treatment‐naïve severe aplastic anaemia (SAA) to assess safety in older compared to younger patients. Patients ≥18 years of age who had received IST with ATG and CSA +/− eltrombopag (EPAG) were included. Pre‐treatment baseline characteristics and co‐morbidities were assessed as predictors of therapy‐related complications in younger (<60 years) versus older (≥60 years) patients. Out of 245 eligible patients, 54 were older and 191 were younger. Older patients had a similar frequency of SAEs, ICU admissions and hospital length of stay compared to younger patients. Older patients had a higher frequency of cardiac events related to IST, but none resulted in death. Older patients had worse long‐term overall survival, and more relapse and clonal evolution post‐IST. However, older patients who responded to IST had a similar survival at a median follow‐up to younger patients. Disease‐related factors and limited therapeutic options in refractory disease likely contribute to poorer outcomes in older patients, not complications of upfront IST. Therefore, IST should be considered first‐line therapy for most older SAA patients.