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Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China.

BACKGROUND: Eltrombopag (ELT), a thrombopoietin receptor agonist (TPO-RA), has been approved for relapsed/refractory aplastic anemia (AA). However, data on avatrombopag (AVA), another TPO-RA, are limited, and the comparisons between the two TPO-RAs are lacking.

OBJECTIVES: We aimed to compare the efficacy and safety between ELT and AVA in relapsed/refractory AA patients.

DESIGN: In this retrospective study, patients with relapsed/refractory AA who had been treated with ELT ( N  = 45) or AVA ( N  = 30) alone and had compatible baseline hematological parameters were compared.

METHODS: Data from patients diagnosed with acquired AA were retrospectively collected. All patients were refractory/relapsed to standard immunosuppressive therapy (IST) for at least 6 months before ELT or AVA. Patients had to be treated with ELT or AVA alone for at least 6 months before evaluation if they did not respond. Baseline characteristics, overall response (OR), complete response (CR), relapse, adverse events, and factors that may affect efficacy were analyzed.

RESULTS: Of the 75 patients enrolled, 45 received ELT and 30 received AVA. Patients with AVA had a higher percentage of abnormal liver or renal function than those with ELT ( p =  0.036). No significant difference was found in the OR/CR rate in the first/second/third/sixth month between the two cohorts ( p >  0.05). Patients treated with AVA had a shorter median time to response than those treated with ELT ( p =  0.012) and had a higher platelet level in the second month ( p =  0.041). AVA had fewer adverse events than ELT ( p =  0.046). Under compatible follow-up time ( p =  0.463), no difference was found between the ELT and AVA cohorts in relapse ( p =  1.000) or clone evolution ( p =  0.637). No predictive factors for OR and CR in the sixth month were found for either ELT or AVA.

CONCLUSION: With worse liver or renal function, AVA had a similar OR/CR rate but a shorter median time to response and fewer adverse events for patients with relapsed/refractory AA.

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