Adult patients with acquired moderate Aplastic Anaemia (mAA) who have not received a specific treatment for their mAA may be included in the EMAA trial of Ulm University (Eltrombopag in Moderate Aplastic Anaemia). They receive either ciclosporin and eltrombopag or ciclosporin and a placebo. The aim of the trial is to evaluate the efficacy and safety of the combination therapy of ciclosporin and eltrombopag. Patients with or without prior transfusions can be included.
A moderate Aplastic Anaemia is defined as the bone marrow containing not enough cells and any two of the following criteria are fulfilled:
if not transfuion dependent: (at least one criterion)
neutrophils (ANC) less than 1.0 G/l
haemoglobin (HGB, Hb) < 8.5 g/dl or ca. 5,28 mmol/L
thrombozytes (platelets, PLT) < 30 G/l
if transfusion dependent: (at least one criterion)
more than 4 PRBCs in 12 weeks (PRBC = Packed Red Blood Cells)
platelet transfusion for therapeutic reasons in case of bleeding/signs of bleeding
prophylactic platelet transfusion when platelets are less than 10 G/l
Aachen, Basel, Berlin, Chemnitz, Essen, Hamburg, Hanover, Ulm
The independent Data Safety Monitoring Board recently evaluated the risk-benefit ratio and recommends the continuation of the trial.