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

Yoshitaka Miyakawa, Shinya Katsutani, Takahiro Yano, Shosaku Nomura, Kaichi Nishiwaki, Yoshiaki Tomiyama, Masaaki Higashihara, Yukari Shirasugi, Masakatsu Nishikawa, Katsutoshi Ozaki, Takayuki Abe, Kayoko Kikuchi, Yuzuru Kanakura, Kingo Fujimura, Yasuo Ikeda, Shinichiro Okamoto

Research output: Contribution to journalArticle

5 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)654-661
Number of pages8
JournalInternational Journal of Hematology
Volume102
Issue number6
DOIs
Publication statusPublished - 2015 Dec 1

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Idiopathic Thrombocytopenic Purpura
Safety
Platelet Count
Therapeutics
Phase III Clinical Trials
Autoimmune Diseases
Antibody Formation
Anti-Idiotypic Antibodies
Japan
Blood Platelets
Placebos
Rituximab
Confidence Intervals
Hemorrhage

Keywords

  • Immune thrombocytopenia
  • Platelets
  • Rituximab

ASJC Scopus subject areas

  • Hematology

Cite this

Efficacy and safety of rituximab in Japanese patients with relapsed chronic immune thrombocytopenia refractory to conventional therapy. / Miyakawa, Yoshitaka; Katsutani, Shinya; Yano, Takahiro; Nomura, Shosaku; Nishiwaki, Kaichi; Tomiyama, Yoshiaki; Higashihara, Masaaki; Shirasugi, Yukari; Nishikawa, Masakatsu; Ozaki, Katsutoshi; Abe, Takayuki; Kikuchi, Kayoko; Kanakura, Yuzuru; Fujimura, Kingo; Ikeda, Yasuo; Okamoto, Shinichiro.

In: International Journal of Hematology, Vol. 102, No. 6, 01.12.2015, p. 654-661.

Research output: Contribution to journalArticle

Miyakawa, Y, Katsutani, S, Yano, T, Nomura, S, Nishiwaki, K, Tomiyama, Y, Higashihara, M, Shirasugi, Y, Nishikawa, M, Ozaki, K, Abe, T, Kikuchi, K, Kanakura, Y, Fujimura, K, Ikeda, Y & Okamoto, S 2015, 'Efficacy and safety of rituximab in Japanese patients with relapsed chronic immune thrombocytopenia refractory to conventional therapy', International Journal of Hematology, vol. 102, no. 6, pp. 654-661. https://doi.org/10.1007/s12185-015-1887-9
Miyakawa, Yoshitaka ; Katsutani, Shinya ; Yano, Takahiro ; Nomura, Shosaku ; Nishiwaki, Kaichi ; Tomiyama, Yoshiaki ; Higashihara, Masaaki ; Shirasugi, Yukari ; Nishikawa, Masakatsu ; Ozaki, Katsutoshi ; Abe, Takayuki ; Kikuchi, Kayoko ; Kanakura, Yuzuru ; Fujimura, Kingo ; Ikeda, Yasuo ; Okamoto, Shinichiro. / Efficacy and safety of rituximab in Japanese patients with relapsed chronic immune thrombocytopenia refractory to conventional therapy. In: International Journal of Hematology. 2015 ; Vol. 102, No. 6. pp. 654-661.
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