Eltrombopag in Combination with Rabbit Anti-thymocyte Globulin/Cyclosporine A in Immunosuppressive Therapy-naïve Patients with Aplastic Anemia in Japan

• Imada Kazunori

  Department of Hematology, Japanese Red Cross Osaka Hospital, Japan

• Obara Naoshi

  Department of Hematology, University of Tsukuba, Japan

• Iida Hiroatsu

  Department of Hematology, National Hospital Organization Nagoya Medical Center, Japan

• Imajo Kenji

  Department of Hematology, Okayama City Hospital, Japan

• Maeda Tetsuo

  Department of Hematology, Suita City Hospital, Japan

• Usuki Kensuke

  Department of Hematology, NTT Medical Center Tokyo, Japan

• Fanghong Zhang

  Novartis Pharma KK, Japan

• Hombo Yosuke

  Novartis Pharma KK, Japan

• Tajima Takeshi

  Novartis Pharma KK, Japan

• Kumagai Akiko

  Novartis Pharma KK, Japan

• Matsuda Akira

  Department of Hemato-Oncology, International Medical Center, Saitama Medical University, Japan

• Nakao Shinji

  Department of Hematology, Kanazawa University Institute of Medical Pharmaceutical and Health Sciences, Japan

Abstract

Objective In Japan, immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG), and cyclosporine A (CsA) is the standard of care in patients with aplastic anemia (AA) who are not indicated for stem-cell transplantation, although some patients may experience relapse. This study assessed the efficacy and safety of eltrombopag in combination with rabbit-ATG/CsA in IST-naïve patients with non-severe or severe AA in Japan. Methods In this non-randomized, open-label, single-arm, phase II study, rabbit-ATG/CsA and eltrombopag were initiated on Days 1 and 15 (±3 days), respectively, and continued for ≥26 weeks; rabbit-ATG was given for 5 days (Days 1 to 5). The primary endpoint was the overall response rate (ORR) at Week 26. Patients Patients with AA who were IST-naïve and ≤70 years old or between 71 and 75 years old based on the recommendation of the investigator were enrolled in Japan. Results Of the 11 enrolled patients, 10 started treatment with eltrombopag. The ORRs at Weeks 26 and 52 were 70.0% and 60.0%, respectively. The ORR at Week 26 was 100% (all 3 patients) in patients with non-severe AA and 57.1% (4/7) in patients with severe AA. Among transfusion-dependent patients, 66.7% (4/6) and 62.5% (5/8) became red blood cell- and platelet-transfusion independent, respectively. The most common adverse events were nausea and headache. No deaths or hematologic malignancies were reported. A cytogenetic abnormality was reported in one patient. Conclusion This study confirmed the clinical benefit of eltrombopag plus rabbit-ATG/CsA in IST-naïve patients with non-severe or severe AA in Japan.

Journal

• Internal Medicine

  Internal Medicine 60 (8), 1159-1168, 2021-04-15

  The Japanese Society of Internal Medicine

Keywords

• eltrombopag
• aplastic anemia
• rabbit-ATG/CsA
• Japan

Details

• CRID: 1390569171137476480
• NII Article ID: 130008025054
• NII Book ID: AA10827774
• DOI: 10.2169/internalmedicine.6063-20
• ISSN: 13497235
• ISSN: 09182918
• HANDLE: 2241/0002003836
• PubMed: 33229810
• Web Site: https://tsukuba.repo.nii.ac.jp/records/2003836
• Web Site: https://www.jstage.jst.go.jp/article/internalmedicine/60/8/60_6063-20/_pdf
• Web Site: https://search.jamas.or.jp/link/ui/2022006922
• Text Lang: en
• Data Source:
  • JaLC
  • IRDB
  • Crossref
  • PubMed
  • CiNii Articles
  • KAKEN
• Abstract License Flag: Disallowed