title: Efficacy and safety of rituximab in Japanese patients  with relapsed chronic immune thrombocytopenia refractory  to conventional therapy.
abstract: Primary immune thrombocytopenia (ITP) is an autoimmune disease mediated by the production of autoantibody 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.
authors.forename: Yoshitaka
authors.initials: Y
authors.lastname: Miyakawa
authors.forename: Shinya
authors.initials: S
authors.lastname: Katsutani
authors.forename: Takahiro
authors.initials: T
authors.lastname: Yano
authors.forename: Shosaku
authors.initials: S
authors.lastname: Nomura
authors.forename: Kaichi
authors.initials: K
authors.lastname: Nishiwaki
authors.forename: Yoshiaki
authors.initials: Y
authors.lastname: Tomiyama
authors.forename: Masaaki
authors.initials: M
authors.lastname: Higashihara
authors.forename: Yukari
authors.initials: Y
authors.lastname: Shirasugi
authors.forename: Masakatsu
authors.initials: M
authors.lastname: Nishikawa
authors.forename: Katsutoshi
authors.initials: K
authors.lastname: Ozaki
authors.forename: Takayuki
authors.initials: T
authors.lastname: Abe
authors.forename: Kayoko
authors.initials: K
authors.lastname: Kikuchi
authors.forename: Yuzuru
authors.initials: Y
authors.lastname: Kanakura
authors.forename: Kingo
authors.initials: K
authors.lastname: Fujimura
authors.forename: Yasuo
authors.initials: Y
authors.lastname: Ikeda
authors.forename: Shinichiro
authors.initials: S
authors.lastname: Okamoto
mesh_terms: D000328:Adult; D044466:Asian People; D002908:Chronic Disease; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage* / Q000009:adverse effects; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000097:blood / Q000188:drug therapy*; D012008:Recurrence; D000069283:Rituximab / Q000008:administration & dosage* / Q000009:adverse effects; D016896:Treatment Outcome
chemical_list: D007155:Immunologic Factors; D000069283:Rituximab