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Aplastic anemia is a rare disease that we treat from time to time in many centers over the country.
Since the publication by Philip Scheinberg in 2011 in the New England Journal of Medicine, we know that horse ATG, aka ATGAM, in combination with cyclosporine lead to better response rate compared to rabbit ATG.
Unfortunately, since horse ATG is no longer available in Belgium, we are not able to offer the best therapy to our patients. However, through the solidarity fund, we have been able to provide patients with ATGAM until the price has been revised a few years ago. Indeed, the price of ATGAM has been raised 4 to 5 times and even with the support of the solidarity fund, ATGAM is no longer affordable for most patients.
On the other hand, the RACE study published by Regis Peffault de la Tour in 2022 showed that the addition of eltrombopag to ATGAM with cyclosporine improved significantly the rate and the quality of responses, which makes this regimen the new first-line standard regimen. As we do have easily access to eltrombopag nowadays, many of us have start to treat aplastic anemia with the combination of Thymoglobuline, cyclosporine and eltrombopag with the idea that added value of eltrombopag may overcome the less effectiveness of Thymoglobine. This data is lacking in the literature, and I doubt that any trial will compare this 2 treatments in the next future.
CALL FOR DATA
Therefore, we would like to collect data on all aplastic anemia patients treated in Belgium with the association of eltrombopag, Thymoglobuline and cyclosporine. We will try to combine our data with those from Brazil where they also adopted this strategy. At the end, we will try to match our final population with patients from the RACE study with the help of Regis Peffault de la Tour. We might have a very nice dataset and a very important message to deliver.
How to participate and have patients to report? Please send an email to Prof. Xavier Poiré (UCL Saint-Luc): Xavier.Poire@uclouvain.be and he will get back to you with an excel file to fill in the data.
We thank you in advance for your cooperation.
Best regards,
The BHS Board,
on behalf of Prof. Xavier Poiré (Hematology department, UCL Saint-Luc)
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