Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial
Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial
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AbstractIntroduction The therapeutic options for thrombocytopenia in non-severe aplastic anaemia (NSAA) are limited.Avatrombopag (AVA) is prescribed for thrombocytopenic diseases but not for NSAA.Methods Herein, we conducted a phase 2, non-randomized, single-arm trial to explore the efficacy and safety of AVA in refractory/relapsed/intolerant NSAA.
AVA dose was initiated at 20 mg/d and titrated to a maximum of 60 mg/d.The primary endpoint was the haematological response at click here 3 months.Results Twenty-five patients were analyzed.
The overall response rate (ORR) at 3 months was 56% (14/25), with 12% (3/25) achieving a complete response (CR).At a median follow-up of 7 (3–10) months, the OR and CR rates were 52% and 20%, respectively.Responders had a shorter duration of diagnosis of AVA administration than non-responders (10 (6–80) vs 37 (6–480) months, p = 0.
027) and belonged to the relapsed/intolerant NSAA type (71% vs 27%, p = 0.047); 44% (8/18) patients previously treated with eltrombopag before enrollment responded at 3 months, with an average prior eltrombopag dose of cocktail tree for sale median 72.5 (50–100) mg/d and an average AVA dose for a response of median 43.
5 (20–60) mg/d.3-month ORR had no significant correlation with eltrombopag exposure (p = 0.09), prior eltrombopag length (R2=0.
11), or cumulative eltrombopag dose (R2=0.30).Only one patient relapsed after stopping AVA for 1 month.
No serious AVA-related side effects or clone evolution were detected.Conclusion AVA is effective and well-tolerated in NSAA patients who are refractory, relapsed, or intolerant to CsA/tacrolimus ± eltrombopag.Earlier treatment and relapsed/intolerant AA may show a better short-term response rate.
More studies are needed to define the optimal dose and the long-term efficacy (NCT04728789).