Background and importance To investigate the use of eltrombopag in our centre and its suitability in routine clinical practice.
Aim and objectives To evaluate the use of eltrombopag in routine clinical practice and its compliance with the Summary of Product Characteristics (SmPC).
To measure the efficacy according to platelet count response and the need for other concomitant treatments.
Material and methods Observational, retrospective and descriptive study of adult patients who received treatment with eltrombopag from June 2020 to October 2021.
The following variables were collected from the electronic medical record: demographic data, indication for use, initial dose, platelet count and concomitant treatments related to hemostasis.
Efficacy was established according to platelet counts at 3 and 3 months after initiation of treatment, aiming for values between 50 000 and 150 000 platelets/µL.
Results Thirty-five patients (54% women), median age 68 (26–95) years, were included in the study. It was used on-label in 77% (27) of patients: primary immune thrombocytopenia (n=26) and severe aplastic anaemia (n=1). The uses off-label were: recovery of normal platelet counts in patients with oncohaematological diseases 11% (4), patients with hereditary bleeding disorders 6% (2) and due to secondary thrombopenias 6% (2).
60% (21) patients started with doses of 50 mg per day as indicated by the SmPC, 29% (10) started with a 25 mg daily regimen, 9% (3) with a dosage of less than 25 mg per day and 1 patient with 75 mg per day.
The mean platelet count at the start of treatment was 41 710 platelets/µL. Three months afterwards it was 113 910 platelets/µL; 10 patients had values above 150 000 platelets/µL, 6 had values below 50 000 platelets/µL and 6 discontinued treatment. After 6 months the mean was 97 090 platelets/µL; 7 had values higher than 150 000 platelets/µL, 6 had values lower than 50000 platelets/µL; 3 patients who had discontinued eltrombopag restarted it and two more discontinued it.
In 54% (19) of the patients, eltrombopag was started after cycles of corticosteroids, which were progressively withdrawn. 20% (11) required other adjuvant treatment after starting eltrombopag: prednisone (6), immunoglobulins (2), cyclosporine (2) or rituximab (1).
Conclusion and relevance Eltrombopag was used on-label in most patients and a high percentage started with the recommended dose according to the SmPC.
The evolution of the platelet count shows the efficacy of eltrombopag, with a minority of patients having platelet counts below 50 000 platelets/µL and only 11 patients requiring adjuvant treatment.
Conflict of interest No conflict of interest
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