International Journal of Hematology

DOI: 10.1007/s12185-015-1887-9 Pages: 654-661

Efficacy and safety of rituximab in Japanese patients with relapsed chronic immune thrombocytopenia refractory to conventional therapy

1. Saitama Medical University, Department of General Internal Medicine

2. Hiroshima University, Department of Hematology and Oncology

3. National Hospital Organization Tokyo Medical Center, Hematology, Internal Medicine

4. Kansai Medical University Hirakata Hospital, First Department of Internal Medicine

5. The Jikei University School of Medicine, Kashiwa Hospital, Department of Oncology and Hematology

6. Osaka University Hospital, Department of Blood Transfusion

7. Kitasato University Hospital, Department of Hematology

8. Tokai University Hospital, Department of Hematology and Oncology

9. Mie University Hospital, Clinical Research Support Center

10. Jichi Medical University, Division of Hematology

11. Keio University School of Medicine, Center for Clinical Research

12. Osaka University Graduate School of Medicine, Department of Hematology and Oncology

13. Yasuda Women’s University

14. Waseda University, Faculty of Science and Engineering

15. Keio University School of Medicine, Division of Hematology, Department of Internal Medicine

Correspondence to:
Yoshitaka Miyakawa
Tel: +81-49-276-1111
Email: miyakawa@saitama-med.ac.jp

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Abstract

Primary immune thrombocytopenia (ITP) is an autoimmune disease mediated by the production of auto-antibody against platelets. Rituximab, an anti-CD20 antibody, is reported to be useful for treatment of ITP. In Japan, however, robust evidence on this treatment has not been accumulated. Hence, we conducted this open-label phase III clinical trial to confirm the efficacy and safety of rituximab, administered at 375 mg/m2 once per week at weekly intervals for 4 consecutive weeks in Japanese patients with chronic ITP, who had relapsed and were refractory to conventional therapy. The primary endpoint was defined as the percentage of patients with a platelet count above 50 × 109/L at week 24 after the first dose of rituximab, which was 30.8 % of 26 patients (95 % confidence interval 14.3–51.8 %). Although the lower confidence limit of primary endpoint failed to meet the pre-specified threshold of 20 %, the clinical efficacy of rituximab is substantial in consideration of the 2 % response rate in the placebo arm in other clinical studies in patients with chronic ITP. We conclude that rituximab is clinically useful and safe in the treatment of Japanese patients with chronic ITP, achieving the goal of maintaining platelet count and reducing risk of bleeding while minimizing treatment-related toxicity.

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