Clinical Trial

. 2024 Mar 26;8(6):1415-1419.

doi: 10.1182/bloodadvances.2023010959.

Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study

Kinuko Mitani¹, Jong Wook Lee², Jun Ho Jang³, Yoshiaki Tomiyama⁴, Koji Miyazaki⁵, Koji Nagafuji⁶, Kensuke Usuki⁷, Nobuhiko Uoshima⁸, Tomoaki Fujisaki⁹, Hiroshi Kosugi¹⁰, Itaru Matsumura¹¹, Ko Sasaki¹, Masahiro Kizaki¹², Masashi Sawa¹³, Michihiro Hidaka¹⁴, Naoki Kobayashi¹⁵, Satoshi Ichikawa¹⁶, Yuji Yonemura¹⁷, Kenta Murotani¹⁸, Mami Shimizu¹⁹, Akira Matsuda²⁰, Keiya Ozawa²¹, Shinji Nakao²²

Affiliations

¹ Department of Hematology and Oncology, Dokkyo Medical University, Tochigi, Japan.
² Division of Hematology, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea.
³ Department of Hematology, Sungkyunkwan University Samsung Medical Center, Seoul, Republic of Korea.
⁴ Department of Blood Transfusion, Osaka University Hospital, Osaka, Japan.
⁵ Department of Transfusion and Cell Transplantation, Kitasato University School of Medicine, Sagamihara, Japan.
⁶ Department of Hematology, Kurume University Hospital, Kurume, Japan.
⁷ Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan.
⁸ Department of Hematology, Japanese Red Cross Kyoto Daini Hospital, Kyoto, Japan.
⁹ Department of Internal Medicine, Matsuyama Red Cross Hospital, Matsuyama, Japan.
¹⁰ Department of Hematology, Ogaki Municipal Hospital, Ogaki, Japan.
¹¹ Department of Hematology and Rheumatology, Kindai University Faculty of Medicine, Osaka, Japan.
¹² Department of Hematology, Saitama Medical Center, Saitama Medical University, Saitama, Japan.
¹³ Department of Hematology and Oncology, Anjo Kosei Hospital, Anjo, Japan.
¹⁴ Department of Hematology, National Hospital Organization Kumamoto Medical Center, Kumamoto, Japan.
¹⁵ Department of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan.
¹⁶ Department of Hematology, Tohoku University Hospital, Sendai, Japan.
¹⁷ Department of Transfusion Medicine and Cell Therapy, Kumamoto University Hospital, Kumamoto, Japan.
¹⁸ Biostatistics Center, Kurume University, Kurume, Japan.
¹⁹ Department of Medical Affairs, Kyowa Kirin Co, Ltd, Tokyo, Japan.
²⁰ Department of Hemato-Oncology, Saitama Medical University International Medical Center, Saitama Medical University, Saitama, Japan.
²¹ Division of Hematology, Jichi Medical University, Tochigi, Japan.
²² Division of Hematology/Respiratory Medicine, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University, Kanazawa, Japan.

PMID: 38134300
PMCID: PMC10950812
DOI: 10.1182/bloodadvances.2023010959

Clinical Trial

Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study

Kinuko Mitani et al. Blood Adv. 2024.

. 2024 Mar 26;8(6):1415-1419.

doi: 10.1182/bloodadvances.2023010959.

Authors

Affiliations

¹ Department of Hematology and Oncology, Dokkyo Medical University, Tochigi, Japan.
² Division of Hematology, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea.
³ Department of Hematology, Sungkyunkwan University Samsung Medical Center, Seoul, Republic of Korea.
⁴ Department of Blood Transfusion, Osaka University Hospital, Osaka, Japan.
⁵ Department of Transfusion and Cell Transplantation, Kitasato University School of Medicine, Sagamihara, Japan.
⁶ Department of Hematology, Kurume University Hospital, Kurume, Japan.
⁷ Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan.
⁸ Department of Hematology, Japanese Red Cross Kyoto Daini Hospital, Kyoto, Japan.
⁹ Department of Internal Medicine, Matsuyama Red Cross Hospital, Matsuyama, Japan.
¹⁰ Department of Hematology, Ogaki Municipal Hospital, Ogaki, Japan.
¹¹ Department of Hematology and Rheumatology, Kindai University Faculty of Medicine, Osaka, Japan.
¹² Department of Hematology, Saitama Medical Center, Saitama Medical University, Saitama, Japan.
¹³ Department of Hematology and Oncology, Anjo Kosei Hospital, Anjo, Japan.
¹⁴ Department of Hematology, National Hospital Organization Kumamoto Medical Center, Kumamoto, Japan.
¹⁵ Department of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan.
¹⁶ Department of Hematology, Tohoku University Hospital, Sendai, Japan.
¹⁷ Department of Transfusion Medicine and Cell Therapy, Kumamoto University Hospital, Kumamoto, Japan.
¹⁸ Biostatistics Center, Kurume University, Kurume, Japan.
¹⁹ Department of Medical Affairs, Kyowa Kirin Co, Ltd, Tokyo, Japan.
²⁰ Department of Hemato-Oncology, Saitama Medical University International Medical Center, Saitama Medical University, Saitama, Japan.
²¹ Division of Hematology, Jichi Medical University, Tochigi, Japan.
²² Division of Hematology/Respiratory Medicine, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University, Kanazawa, Japan.

PMID: 38134300
PMCID: PMC10950812
DOI: 10.1182/bloodadvances.2023010959

Erratum in

Mitani K, Lee JW, Jang JH, et al. Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study. Blood Adv. 2024;8(6):1415-1419.
[No authors listed] [No authors listed] Blood Adv. 2024 Dec 10;8(23):6097. doi: 10.1182/bloodadvances.2024014868. Blood Adv. 2024. PMID: 39625711 Free PMC article. No abstract available.

No abstract available

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Conflict of interest statement

Conflict-of-interest disclosure: K. Mitani received speakers bureau fees from Celgene K.K., Otsuka Pharmaceutical Co, Ltd, Bristol Myers Squibb K.K., Novartis Pharma K.K., and Kyowa Kirin Co, Ltd; and received research funding from Chugai Pharmaceutical Co, Ltd, Kyowa Kirin Co, Ltd, and Takeda Pharmaceutical Co, Ltd. J.W.L. received speakers’ bureau fees and grants from Alexion and AstraZeneca Rare Disease; and consultancy fees from Allovir, Arrowhead, Kira, and Samsung. Y.T. received speakers’ bureau fees from Kyowa Kirin Co, Ltd, Novartis Pharma K.K., and Sysmex Corporation; received honoraria from Chugai Pharmaceutical Co, Ltd, UCB Japan Co Ltd, Kyowa Kirin Co, Ltd, Novartis Pharma, K.K., and Sysmex Corporation; consultancy fees from SobiTM Japan, Medical & Biological Laboratories Co, Ltd, Kissei Pharmaceutical Co, Ltd, Novartis Pharma K.K., and Sysmex Corporation; and had membership on board of directors or advisory committees for Novartis Pharma K.K. and Sysmex Corporation. K. Miyazaki received speakers’ bureau fees from Kyowa Kirin Co, Ltd, Chugai Pharmaceutical Co, Ltd, Pfizer Japan Inc, Takeda Pharmaceutical Co, Ltd, MSD, K.K., Novartis Pharma, K.K., Novo Nordisk Pharma Ltd, KM Biologics Co, Ltd, Nihon Pharmaceutical Co, Ltd, and CSL Behring K.K., and research funding from Kyowa Kirin Co, Ltd, Chugai Pharmaceutical Co, Ltd, Pfizer Japan Inc, Daiichi Sankyo Co, Ltd, Astellas Pharma Inc, and Otsuka Pharmaceutical Co, Ltd. K.N. received speakers’ bureau fees from Kyowa Kirin Co, Ltd. K.U. received speakers bureau fees from Alexion Pharmaceuticals, Inc, Nippon Shinyaku Co, Ltd, Otsuka Pharmaceutical Co, Ltd, Chugai Pharmaceutical Co, Ltd, Sanofi K.K., Takeda Pharmaceutical Co, Ltd, Kyowa Kirin Co, Ltd, Astellas Pharma Inc, Novartis Pharma K.K., AbbVie G.K., Bristol Myers Squibb K.K., Ono Pharmaceutical Co, Ltd, Daiichi Sankyo Co, Ltd, MSD K.K., Pfizer Japan Inc, Celgene K.K., Eisai Co, Ltd; PharmaEssentia Japan K.K., and Yakult Honsha Co, Ltd; research funding from Alexion Pharmaceuticals, Inc, SymBio Pharmaceuticals Ltd, Nippon Shinyaku Co, Ltd, Otsuka Pharmaceutical Co, Ltd, Chugai Pharmaceutical Co, Ltd, Takeda Pharmaceutical Co, Ltd, Kyowa Kirin Co, Ltd, Astellas Pharma Inc, Amgen K.K., Apellis Pharmaceuticals, Inc, Novartis Pharma K.K., AbbVie G.K., Janssen Pharmaceutical, K.K., Bristol Myers Squibb K.K., Ono Pharmaceutical Co, Ltd, Daiichi Sankyo Co, Ltd, MSD K.K., Gilead Sciences, Inc, Pfizer Japan Inc, Incyte Biosciences Japan G.K., Celgene K.K., Sumitomo Pharma Co, Ltd, Mundipharma K.K., Eisai Co, Ltd, Symbio Technologies LLC, and Yakult Honsha Co, Ltd; and consultancy fees from Alexion Pharmaceuticals Inc, SymBio Pharmaceuticals Ltd, Nippon Shinyaku Co, Ltd, Otsuka Pharmaceutical Co, Ltd, Chugai Pharmaceutical Co, Ltd, Sanofi K.K., Takeda Pharmaceutical Co, Ltd, Kyowa Kirin Co, Ltd, Astellas Pharma Inc, and Alnylam Japan K.K. N.U. received honoraria from Bristol Myers Squibb K.K., Janssen Pharmaceutical K.K., Chugai Pharmaceutical Co, Ltd, Nippon Shinyaku Co, Ltd, and Otsuka Pharmaceutical Co, Ltd. H.K. received honoraria from Bristol Myers Squibb K.K., Celgene K.K., Chugai Pharmaceutical, Janssen Pharmaceutical K.K., Japan Blood Products Organization, Kyowa-Kirin K.K., MSD K.K., Novartis Pharma K.K., Ono Pharmaceutical, Sanofi K.K., and Takeda Pharmaceutical Co, Ltd. I.M. received speakers’ bureau fees from Takeda Pharmaceutical Co Ltd, Daiichi Sankyo Co Ltd, Pfizer Japan Inc, SymBio Pharmaceuticals Ltd, Astellas Pharma Inc, Ono Pharmaceutical Co Ltd, Novartis Pharma K.K., AbbVie G.K., Janssen Pharmaceutical K.K., Bristol Myers Squibb K.K., Sanofi K.K., AstraZeneca K.K., and Otsuka Pharmaceutical Co, Ltd; research funding from Chugai Pharmaceutical Co, Ltd, Kyowa Kirin Co, Ltd, Sumitomo Pharma Co, Ltd, Takeda Pharmaceutical Co, Ltd, Alexion Pharmaceuticals Inc, Pfizer Japan Inc, Astellas Pharma Inc, Ono Pharmaceutical Co, Ltd, Sanofi K.K., Mitsubishi Tanabe Pharma Corp, Novartis Pharma K.K., Nippon Shinyaku Co, Ltd, Eisai Co, Ltd, Asahi Kasei Pharma Corp, AbbVie G.K., Janssen Pharmaceutical K.K, Taiho Pharmaceutical Co, Ltd, Shionogi & Co, Ltd, and Otsuka Pharmaceutical Co, Ltd; and consultancy fees from Otsuka Pharmaceutical Co, Ltd. M.K. received speakers’ bureau fees from Ono Pharmaceutical Co, Ltd, Takeda Pharmaceutical Co, Ltd, AbbVie G.K., Janssen Pharmaceutical K.K., Bristol Myers Squibb K.K., and Novartis Pharma K.K.; and research funding from Kyowa Kirin Co, Ltd, Daiichi Sankyo Co, Ltd, Ono Pharmaceutical Co, Ltd, Takeda Pharmaceutical Co, Ltd, Chugai Pharmaceutical Co, Ltd, and AbbVie G.K. M.S. received honoraria from GSK plc., Daiichi Sankyo Co, Ltd, AstraZeneca K.K., Janssen Pharmaceutical K.K., SymBio Pharmaceuticals Ltd, CSL Behring K.K., AbbVie G.K., Mundipharma K.K., Shire plc, Mochida Pharmaceutical Co, Ltd, Celgene K.K., Sanofi K.K., Takeda Pharmaceutical Co, Ltd, Sumitomo Pharma Co, Ltd, Otsuka Pharmaceutical Co, Ltd, Eisai Co, Ltd, Novartis Pharma K.K., Asahi Kasei Pharma Corp, Bristol Myers Squibb K.K., MSD K.K., Ono Pharmaceutical Co, Ltd, Nippon Shinyaku Co, Ltd, Astellas Pharma Inc, Pfizer Japan Inc, Chugai Pharmaceutical Co, Ltd, and Kyowa Kirin Co, Ltd. M.H. received honoraria from Chugai Pharmaceutical Co, Ltd and JCR Pharmaceuticals Co, Ltd. S.I. received honoraria from Meijiseika; and research funding from AstraZeneca K.K. and Beigene. Y.Y. received honoraria from Alexion Pharmaceuticals, Inc and Novartis Pharma K.K.; and research funding from Chugai Pharmaceutical Co, Ltd. K.Murotani received honoraria from Kyowa Kirin Co, Ltd, Chugai Pharmaceutical Co, Ltd, AstraZeneca K.K., Taiho Pharmaceutical Co, Ltd, MSD K.K., Nippon Boehringer Ingelheim Co, Ltd, Takeda Pharmaceutical Co, Ltd, and Yakult Pharmaceutical Industry Co, Ltd. M.S. is currently employed at Kyowa Kirin Co, Ltd. A.M. received honoraria from Alexion Pharmaceuticals, Inc, Nippon Shinyaku Co, Ltd, Sumitomo Dainippon Pharma Co, Ltd, and Novartis Pharma; and consultancy fees from Kyowa Kirin Co, Ltd. K.O. received honoraria from Human Life CORD Japan Inc, Otsuka Pharmaceutical Co, Ltd, Mitsubishi Tanabe Pharma Corp, AbbVie G.K., and Novartis Pharma K.K.; research funding from Japan Agency for Medical Research and Development; consultancy fees from Takara Bio Inc and Human Life CORD Japan Inc; is a current equity holder in Terumo Corporation and has a membership on their board of directors or advisory committees. S.N. received honoraria from Novartis Pharma K.K. and Kyowa Kirin Co, Ltd; consultancy fees from SymBio Pharmaceuticals Ltd; and research funding from Alexion Pharmaceuticals, Inc. The remaining authors declare no competing financial interests.

Figures

**Figure 1.**
**Long-term efficacy of romiplostim.** (A) Change in hematologic parameters during the entire period; (i) platelet count, (ii) hemoglobin concentration, (iii) neutrophil count, and (iv) reticulocyte count. (B) Hematologic responses to romiplostim at (i) 52 weeks, (ii) 104 weeks, and (iii) 156 weeks. The numbers of cases with missing data were 5 at 104 weeks and 21 at 156 weeks. Of the patients for whom data could be collected, no response was 1 at 104 weeks and 0 at 156 weeks.

**Figure 2.**
**Proportion of transfusion-dependent patients based on the type of transfusion during the entire period.** (A) Platelet and (B) red blood cell.

See this image and copyright information in PMC

References

1. Frampton JE, Lyseng-Williamson KA. Romiplostim. Drugs. 2009;69(3):307–317. - PubMed
1. Lee JW, Lee SE, Jung CW, et al. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019;6(11):e562–e572. - PubMed
1. Jang JH, Tomiyama Y, Miyazaki K, et al. Efficacy and safety of romiplostim in refractory aplastic anaemia: a phase II/III, multicentre, open-label study. Br J Haematol. 2021;192(1):190–199. - PMC - PubMed
1. Winkler T, Fan X, Cooper J, et al. Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag. Blood. 2019 13;133(24):2575–2585. - PMC - PubMed

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Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study

Affiliations

Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study

Authors

Affiliations

Erratum in

Conflict of interest statement

Figures

References

Publication types

MeSH terms

Substances

LinkOut - more resources

Full Text Sources

Medical