This is a single-center, open label pilot trial where patients with primary ITP who require second line treatment will be offered avatrombopag at a reduced starting dose, adjusted thereafter according to the response and continued for up to 24 weeks. The study aims to acquire experience on use of avatrombopag and explore the efficacy and safety of lower starting dose of avatrombopag
Male or female aged ≥18 years.
Diagnosis of primary ITP and having a platelet count of < 30 x109/L measured within two weeks prior to inclusion with failure to achieve response or relapse after at least one cycle of dexamethasone (20-40 mg daily for 4 days) or prednisone /prednisolone (1 mg/kg for at least two weeks). Shorter courses or lower doses are allowed if discontinued or modified due to side effects.
Clinical need for second (subsequent) line treatment with a platelet elevating therapy assessed by the physician in charge.
Signed and dated written informed consent.
Previous treatment with TPO-RA.
Pregnancy or lactation.
Patients with active serious bleeding or at high risk of bleeding as judged by physician in charge.
Females of child-bearing potential refusing to follow effective contraceptive methods (as described in SmPC) during treatment with Avatrombopag.
Single group
Avatrombopag 20 mg Oral Tablet
DrugDescription: Patients will start on avatrombopag 20 mg every other day for a week then the dose will be readjusted according to the platelet count.
Total Response Time
Median cumulative number of weeks with platelet count \>50 x 109/L from time of Avatrombopag initiation to end of week 20. A period of 2 week will be deducted after IVIG and 4 weeks after dexamethasone.
TTR
Time from initiation of dose to first platelet count \> 50 x 109/L without any rescue therapy during the 2 weeks prior to the blood sampling.
Response by day 8
Number of patients achieving platelet count \> 50 x 109/L by day 8 without any rescue therapy during the 2 weeks prior to the blood sampling.
Rates of platelet overshoot.
Number of episode with platelet count \> 200 and \>400 x 109/L during the first 6 weeks after the initiation of avatrombopag.
Durable response rate.
Number of patients achieving 4 consecutive platelet counts \> 50 x 109/L between weeks 12 and 20 including the last count, without the use of rescue therapy, corticosteroids or any platelet elevating agent after week 6.
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