Patient recruitment and treatment with Sirolimus
blisters, blood transfusions, Thalassemia
RECRUITMENT, THALASSEMIA
Recruitment of the Sirthalaclin pilot clinical trial and data collection (EudraCT n° 2018–001942‐33, NCT 03877809) was at the Thalassemia Centre of Azienda Ospedaliera‐Universitaria S.Anna, as extensively reported in Zuccato et al.Characteristics of the nine transfusion‐dependent patients included in the studyThe investigational drug in the form of coated tablets (0.5 mg Sirolimus) has been provided to the patients in adequately labelled blisters. The starting Sirolimus dosage was 1 mg/day. All other standard treatments, including blood transfusions and iron chelation therapy, have been continued, in agreement with the International Thalassemia Federation (T.I.F.) Guidelines.