Award-winning study ‘practice changing’ for severe aplastic anaemia

Adding the thrombopoietin receptor agonist eltrombopag (Revolade) to standard immunosuppressive treatment increases response rates in patients with Severe Aplastic Anaemia (SAA), preliminary results from the phase III RACE trial show.

The trial (see abstract here), which was awarded the prestigious van Bekkum award at this year’s European Society for Blood and Marrow Transplantation virtual 46th meeting, involved 197 patients aged 15 and over with SAA who had not received prior immunosuppressive treatment.

Patients were randomised to receive either standard immunosuppression (horse anti-thymocyte globulin, ATG) 40 mg/kg x 4d and cyclosporine A (CsA) 5 mg/kg/d, or standard immunosuppression plus eltrombopag at the dose of 150 mg/d from day 14 until six months (or three months, in case of early complete response).

The primary endpoint of the trial was complete response at three months, which was defined as haemoglobin 100 g/L, neutrophils 1.0 g/L and platelets 100g/L, according to standard international criteria.

Results showed that three months after treatment patients who received the combination of hATG, CsA plus eltrombopag had a significantly higher complete response rate that was sustained at six months (21.9%) compared to patients treated with hATG and CsA alone (9.9%).

Overall response (OR=CR+PR) rates were 31.7% and 59.4%, respectively.

Eltrombopag was generally well-tolerated, with a comparable occurrence of adverse events in the two treatment arms, said the research team who described the results as ‘practice changing’.

Principal investigator of the study, Prof. Antonio Risitano from the Federico II University, Naples, and Head of Hematology and the BMT Unit, Ospedale Moscati, Avellino, Italy said: “Long-term follow up data will eventually document the impact of this treatment strategy on long-term overall survival and disease-free survival. ”

Based on available data and on the well-known association between haematological response and long-term outcome, the triple therapy with eltrombopag, hATG and CsA will likely become the reference treatment for naïve patients with SAA not eligible for transplantation, he added.

The RACE trial is sponsored by the EBMT with the financial support of Novartis and Pfizer.

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