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Efficacy and safety of romiplostim in refractory aplastic anaemia: a Phase II/III, multicentre, open-label study.

Abstract：

:A previous dose-finding study has suggested that romiplostim is effective in patients with refractory aplastic anaemia (AA) and 10 µg/kg once weekly was recommended as a starting dose. In this Phase II/III, multicentre, open-label study, romiplostim was administered subcutaneously at a fixed dose of 10 µg/kg once weekly for 4 weeks (weeks 1-4) followed by weekly doses (5, 10, 15 and 20 µg/kg) titrated by platelet response for up to 52 weeks (weeks 5-52). A total of 31 patients with AA who were refractory to immunosuppressive therapy (IST) and thrombocytopenia (platelet count of ≤30 × 109 /l) were enrolled. The primary efficacy endpoint of the proportion of patients achieving any haematological (platelet, neutrophil and erythrocyte) response at week 27 was 84% [95% confidence interval (CI) 66-95%]. Trilineage response was 39% (95% CI 22-58%) at week 53. The most common treatment-related adverse events (AEs) were headache and muscle spasms (each 13%). All AEs were mild or moderate except for three patients with Grade 3 hepatic AEs; no AEs necessitated romiplostim discontinuation. Two patients developed cytogenetic abnormalities, of whom one returned to normal karyotype at last follow-up. High-dose romiplostim is effective and well tolerated in the treatment of patients with AA refractory to IST.

journal_name

Br J Haematol

journal_title

British journal of haematology

authors

Jang JH,Tomiyama Y,Miyazaki K,Nagafuji K,Usuki K,Uoshima N,Fujisaki T,Kosugi H,Matsumura I,Sasaki K,Kizaki M,Sawa M,Hidaka M,Kobayashi N,Ichikawa S,Yonemura Y,Enokitani K,Matsuda A,Ozawa K,Mitani K,Lee JW,Nakao

doi

10.1111/bjh.17190

subject

Has Abstract

pub_date

2021-01-01 00:00:00

pages

190-199

issue

1

eissn

0007-1048

issn

1365-2141

journal_volume

192

pub_type

杂志文章

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