Last updated on February 2018

Efficacy and Safety of Eltrombopag + CSA in Patients With Moderate Aplastic Anemia (EMAA)


Brief description of study

The aim of this study is to improve treatment of Moderate Aplastic Anemia (MAA) by evaluating the safety and efficiency of Eltrombopag as a new treatment option in patients with therapy requiring MAA.

Detailed Study Description

After enrollment (see detailed inclusion and exclusion criteria below) the patients are randomized either to the Placebo or Eltrombopag arm. The randomization is double blinded. Randomization will take in account patient's age and disease severity by stratifying into 4 block combinations to ensure homogeneity between treatment arms. All patients receive background therapy with CSA, regardless of randomisation group, to treat MAA according to current standard of care.

Eltrombopag (or Placebo) is given at a daily starting dose of 150 mg orally as 75 mg tablets once daily (2 tablets Eltrombopag or placebo per day), (Olnes et al NEJM 2012).

In Asian patients Eltrombopag (or Placebo) is given at a daily starting dose of 75 mg orally (1 tablet Eltrombopag or placebo per day). In Asian-Caucasian patients no dose reduction of the starting dose is carried out, but cautious observation of the liver function due to the possibility of altered Eltrombopag metabolism is recommended.

Dose reduction:

In patients without history of thromboembolism or known risk factors for thrombembolism dose reduction (the possibility of an alternating dose schedule is given) is recommended if the platelet count is increasing > 150 G/L.

  • Dosage should be decreased to achieve a platelet count between 100 and 150 G/L after reaching a sufficient erythrocyte and granulocyte response (see 10.1).
  • If the platelet count decreases below 100 G/L the Eltrombopag dose should be escalated again.
  • Eltrombopag should be discontinued if the platelet count exceeds 450 G/L and could be restarted with a lower dose after decrease of the platelet count below 150 G/L.

In patients with history of thromboembolism or known risk factors for thromboembolism (e. g. Factor V Leiden, ATIII deficiency, antiphospholipid syndrome, PNH with GPI-deficient granulocyte population > 50 %, prolonged periods of immobilization, contraceptives and hormone replacement therapy or surgery) dose reduction is recommended if the platelet count is increasing > 100 G/L

  • The target platelet count will be 70-120 G/L after reaching a sufficient erythrocyte and granulocyte response (see 10.1).
  • If the platelet count decreases below 70 G/L the Eltrombopag dose should be escalated again.
  • Eltrombopag should be discontinued in patients with history of and risk factors for thromboembolism if the platelet count exceeds 150 G/L.

Duration of follow up: Last Follow up 24 months after end of study treatment. Patients will receive Eltrombopag or placebo within the study for a minimum of 6 months. Exceptions are patients with disease progression in Severe or Very Severe AA or patients with inacceptable adverse events within the first 6 months.

Eltrombopag will be administered for a maximum period of 12 months within the protocol. Recent data show that the response of hematopoiesis in refractory severe aplastic anemia can be sustained on discontinuation of Eltrombopag25.

As long-term effects of investigational treatments are an objective of the study, the follow-up of patients will cover 24 months after the end of the study treatment according to the protocol

Clinical Study Identifier: NCT02773225

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Britta Höchsmann, MD

University Hospital Ulm
Ulm, Germany
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