{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,17]],"date-time":"2026-10-17T16:05:48Z","timestamp":1792253148021,"version":"4.4.0"},"reference-count":17,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2020,11,5]],"date-time":"2020-11-05T00:00:00Z","timestamp":1604534400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2021,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    A previous dose\u2010finding study has suggested that romiplostim is effective in patients with refractory aplastic anaemia (AA) and 10\u00a0\u00b5g\/kg once weekly was recommended as a starting dose. In this Phase II\/III, multicentre, open\u2010label study, romiplostim was administered subcutaneously at a fixed dose of 10\u00a0\u00b5g\/kg once weekly for 4\u00a0weeks (weeks 1\u20134) followed by weekly doses (5, 10, 15 and 20\u00a0\u00b5g\/kg) titrated by platelet response for up to 52\u00a0weeks (weeks 5\u201352). A total of 31 patients with AA who were refractory to immunosuppressive therapy (IST) and thrombocytopenia (platelet count of \u226430\u00a0\u00d7\u00a010\n                    <jats:sup>9<\/jats:sup>\n                    \/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\u201395%]. Trilineage response was 39% (95% CI 22\u201358%) at week 53. The most common treatment\u2010related 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\u2010up. High\u2010dose romiplostim is effective and well tolerated in the treatment of patients with AA refractory to IST.\n                  <\/jats:p>","DOI":"10.1111\/bjh.17190","type":"journal-article","created":{"date-parts":[[2020,11,5]],"date-time":"2020-11-05T15:57:19Z","timestamp":1604591839000},"page":"190-199","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":49,"title":["Efficacy and safety of romiplostim in refractory aplastic anaemia: a Phase II\/III, multicentre, open\u2010label study"],"prefix":"10.1111","volume":"192","author":[{"given":"Jun Ho","family":"Jang","sequence":"first","affiliation":[{"name":"Department of Hematology Sungkyunkwan University Samsung Medical Center Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshiaki","family":"Tomiyama","sequence":"additional","affiliation":[{"name":"Department of Blood Transfusion Osaka University Hospital Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Koji","family":"Miyazaki","sequence":"additional","affiliation":[{"name":"Department of Transfusion and Cell Transplantation Kitasato University School of Medicine Sagamihara Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4795-121X","authenticated-orcid":false,"given":"Koji","family":"Nagafuji","sequence":"additional","affiliation":[{"name":"Department of Hematology Kurume University Hospital Kurume Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kensuke","family":"Usuki","sequence":"additional","affiliation":[{"name":"Department of Hematology NTT Medical Center Tokyo Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nobuhiko","family":"Uoshima","sequence":"additional","affiliation":[{"name":"Department of Hematology Japanese Red Cross Kyoto Daini Hospital Kyoto Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tomoaki","family":"Fujisaki","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine Matsuyama Red Cross Hospital Matsuyama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hiroshi","family":"Kosugi","sequence":"additional","affiliation":[{"name":"Department of Hematology Ogaki Municipal Hospital Ogaki Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Itaru","family":"Matsumura","sequence":"additional","affiliation":[{"name":"Department of Hematology and Rheumatology Kindai University Faculty of Medicine Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ko","family":"Sasaki","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Dokkyo Medical University Tochigi Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masahiro","family":"Kizaki","sequence":"additional","affiliation":[{"name":"Department of Hematology International Medical Center Saitama Medical University Saitama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masashi","family":"Sawa","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Anjo Kosei Hospital Anjo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michihiro","family":"Hidaka","sequence":"additional","affiliation":[{"name":"Department of Hematology National Hospital Organization Kumamoto Medical Center Kumamoto Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naoki","family":"Kobayashi","sequence":"additional","affiliation":[{"name":"Department of Hematology Sapporo Hokuyu Hospital Sapporo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3163-7197","authenticated-orcid":false,"given":"Satoshi","family":"Ichikawa","sequence":"additional","affiliation":[{"name":"Department of Hematology and Rheumatology Tohoku University Hospital Sendai Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuji","family":"Yonemura","sequence":"additional","affiliation":[{"name":"Department of Transfusion Medicine and Cell Therapy Kumamoto University Hospital Kumamoto Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8360-0102","authenticated-orcid":false,"given":"Kouki","family":"Enokitani","sequence":"additional","affiliation":[{"name":"Kyowa Kirin Co., Ltd. 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