07 September 2020

Adding a medication to standard immunosuppressive treatment is safe and increases response rates in patients with severe aplastic anaemia, according to the findings of a major European study.

First results from the phase III RACE trial show that eltrombopag is beneficial to those being treated for severe aplastic anaemia (SAA).

The most commonly used immunosuppressive regimen for SAA includes horse ATG (hATG) and Cyclosporine A (CsA). However, about 35% of patients do not respond to the regimen or they suffer a relapse.

Eltrombopag is a thrombopoietin receptor agonist that was originally developed to stimulate thrombopoiesis but was in fact found to restore trilineage haematopoiesis. A previous single-arm study showed that adding eltrombopag to standard immunosuppressive treatment appeared to improve the response rate compared versus using hATG plus CsA alone.

Now, the first results of the randomised controlled RACE trial confirm that adding eltrombopag to standard immunosuppression leads to significantly higher response rates.

The findings were announced at the European Society for Blood and Marrow Transplantation (EBMT) virtual 46th annual meeting. The study is being jointly led by Prof Régis Peffault de Latour, head of the French Reference Centre for Aplastic Anaemia and PNH, Saint-Louis Hospital, and University of Paris, France, and Prof Antonio Risitano from Federico II University, Naples, and Head of Hematology and the BMT Unit, Ospedale Moscati, Avellino, Italy.

Their research was granted the Van Bekkum Award, the most prestigious EBMT award for the best abstract submitted to the physician's programme.

The international, open-label, phase III, randomised trial evaluated 197 patients with SAA. All patients were aged 15 years or older and had not received prior immunosuppressive treatment for their SAA.

They were randomised to receive either standard immunosuppression hATG 40 mg/kg x4d and CsA 5 mg/kg/d, or standard immunosuppression with eltrombopag, at 150 mg/d from day 14 until six months, or three months in case of early complete response.

The researchers found that three months after treatment started, patients who received hATG, CsA and eltrombopag had a significantly higher complete response rate compared to patients treated with hATG and CsA alone.

These higher response rates were sustained at six months and eltrombopag was generally well-tolerated, the virtual meeting was told.

Prof Risitano from Federico II University, Naples, and the BMT Unit, Ospedale Moscati, Avellino, Italy, said: “The RACE trial data shows that eltrombopag increased response rates for naïve SAA patients who are not eligible for haematopoietic stem cell transplantation.

“The RACE study team is continuing to follow up the trial participants up to two years and furthermore aims to set up a long-term follow-up study to monitor the effectiveness and safety of eltrombopag up to ten years.”


Source: European Society for Blood and Marrow Transplantation

https://www.ebmt.org/sites/default/files/2020-08/Press-release_EBMT-RACE-trial_Final.pdf [PDF]


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