{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:45:39Z","timestamp":1787013939114,"version":"build-2736575974"},"reference-count":31,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,7,10]],"date-time":"2025-07-10T00:00:00Z","timestamp":1752105600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004095","name":"Kyowa Kirin","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004095","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2025,8]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>Romiplostim has been shown to restore multi\u2010lineage haematopoiesis and is effective in patients with aplastic anaemia (AA) refractory to immunosuppressive therapy (IST). This open\u2010label, phase 2\/3 study (NCT04095936) recruited adult AA patients in Japan and Korea who had not received prior IST and evaluated the efficacy and safety of romiplostim plus ciclosporin A (CsA). Romiplostim was initiated at 10\u2009\u03bcg\/kg once weekly through Week 4 and adjusted between 0 and 20\u2009\u03bcg\/kg from Week 5 onwards. CsA was administered at 5\u20136\u2009mg\/kg\/day in two divided doses through Week 26. A total of 24 patients (median [range] age, 52 [19\u201380] years) were enrolled, and 22 (91.7%) completed the study. Four patients (16.7%) had very severe AA (VSAA), 13 (54.2%) had severe AA (SAA) and seven (29.2%) had transfusion\u2010dependent non\u2010severe AA (NSAA). A haematological response at Week 27 was observed in 10\/24 patients (overall response, 41.7%; 95% confidence interval, 22.1%\u201363.4%). At Week 27, the subgroup overall response rates were 0.0% in VSAA, 46.2% in SAA and 57.1% in NSAA. Nearly 92% of patients experienced at least one treatment\u2010emergent adverse event (TEAE), but no drug\u2010related Grade \u22653 TEAEs were reported. One patient developed myelodysplastic syndromes. Treatment with romiplostim plus CsA was effective and well tolerated in patients with AA who had not previously received IST.<\/jats:p>","DOI":"10.1111\/bjh.20206","type":"journal-article","created":{"date-parts":[[2025,7,11]],"date-time":"2025-07-11T01:15:05Z","timestamp":1752196505000},"page":"582-590","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Romiplostim with ciclosporin A in patients with aplastic anaemia na\u00efve to immunosuppressive therapy: A phase 2\/3 study"],"prefix":"10.1111","volume":"207","author":[{"given":"Jong Wook","family":"Lee","sequence":"first","affiliation":[{"name":"Division of Hematology\u2010Oncology Hanyang University Seoul Hospital  Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7423-4676","authenticated-orcid":false,"given":"Jun Ho","family":"Jang","sequence":"additional","affiliation":[{"name":"Department of Hematology\u2010Oncology Samsung Medical Center, Sungkyunkwan University School of Medicine  Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7803-7338","authenticated-orcid":false,"given":"Shigeru","family":"Chiba","sequence":"additional","affiliation":[{"name":"Department of Hematology University of Tsukuba  Tsukuba Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sung\u2010Soo","family":"Yoon","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine Seoul National University Hospital  Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaku","family":"Oshikawa","sequence":"additional","affiliation":[{"name":"Department of Hematology Japan Red Cross Musashino Hospital Tokyo  Tokyo 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":"Yeung\u2010Chul","family":"Mun","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine Ewha Womans' University College of Medicine  Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Toshiro","family":"Kawakita","sequence":"additional","affiliation":[{"name":"Department of Hematology National Hospital Organization Kumamoto Medical Center  Kumamoto Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kazunori","family":"Imada","sequence":"additional","affiliation":[{"name":"Department of Hematology Japanese Red Cross Osaka Hospital  Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"June\u2010Won","family":"Cheong","sequence":"additional","affiliation":[{"name":"Division of Hematology, Department of Internal Medicine, Severance Hospital Yonsei University College of Medicine  Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masayoshi","family":"Noshiro","sequence":"additional","affiliation":[{"name":"Kyowa Kirin Co., Ltd.  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