Long-term follow-up of children with refractory immune thrombocytopenia treated with rituximab

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• 作者：Kousaku Matsubara (1)
  Yoshiyuki Takahashi (2)
  Akira Hayakawa (3)
  Fumiko Tanaka (4)
  Hisaya Nakadate (5)
  Michio Sakai (6)
  Naoko Maeda (7)
  Toshiaki Oka (8)
  Eiichi Ishii (9)
  Fumio Bessho (10)
  Tsuyoshi Morimoto (11)
  Hiroaki Goto (12)
  Yoshiko Hashii (13)
  Naoki Hatakeyama (14)
  Akira Shirahata (6)
  Masue Imaizumi (15)
  
• 关键词：Rituximab ; Children ; Refractory ; Immune thrombocytopenia ; Long ; term follow ; up
• 刊名：International Journal of Hematology
• 出版年：2014
• 出版时间：April 2014
• 年：2014
• 卷：99
• 期：4
• 页码：429-436
• 全文大小：252 KB
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• 作者单位：Kousaku Matsubara (1)
  Yoshiyuki Takahashi (2)
  Akira Hayakawa (3)
  Fumiko Tanaka (4)
  Hisaya Nakadate (5)
  Michio Sakai (6)
  Naoko Maeda (7)
  Toshiaki Oka (8)
  Eiichi Ishii (9)
  Fumio Bessho (10)
  Tsuyoshi Morimoto (11)
  Hiroaki Goto (12)
  Yoshiko Hashii (13)
  Naoki Hatakeyama (14)
  Akira Shirahata (6)
  Masue Imaizumi (15)
  
  1. Department of Pediatrics, Nishi-Kobe Medical Center, 5-7-1 Kojidai, Nishi-ku, Kobe, 651-2273, Japan
  2. Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan
  3. Department of Pediatrics, Kobe University Graduate School of Medicine, Kobe, Japan
  4. Department of Pediatrics, Saiseikai Yokohamashi Nanbu Hospital, Yokohama, Kanagawa, Japan
  5. Department of Hematology, National Center for Child Health and Development, Tokyo, Japan
  6. Department of Pediatrics, University of Occupational and Environmental Health, Kitakyushu/Fukuoka, Japan
  7. Department of Pediatrics, National Hospital Organization Nagoya Medical Center, Nagoya, Japan
  8. Department of Pediatrics, Sapporo Tokushukai Hospital, Sapporo, Japan
  9. Department of Pediatrics, Ehime University Graduate School of Medicine, Matsuyama, Ehime, Japan
  10. Department of Pediatrics, Kyorin University, School of Medicine, Tokyo, Japan
  11. Department of Pediatrics, Tokai University School of Medicine, Isehara, Kanagawa, Japan
  12. Department of Pediatrics, Yokohama City University, School of Medicine, Yokohama, Japan
  13. Department of Developmental Medicine, Osaka University Graduate School of Medicine, Suita, Japan
  14. Department of Pediatrics, Sapporo Medical University School of Medicine, Sapporo, Japan
  15. Department of Hematology and Oncology, Miyagi Children’s Hospital, Sendai, Japan
  
• ISSN：1865-3774

文摘

Data on long-term outcomes of children with refractory immune thrombocytopenia (ITP) treated with rituximab are limited. We retrospectively analyzed the long-term effect of rituximab on 22 pediatric ITP patients (11 boys and 11 girls). Compete response (CR) (platelet count ?00?×?109/L) and partial response (PR) (platelet count 30-9?×?109/L) were achieved in nine (41?%) and two (9?%) patients, respectively. Of the 11 responders, eight subsequently relapsed 2-6?months after initial rituximab treatment. The 5-year relapse-free rate was 14?% (3/22, 95?% confidence interval: 0-7?%) with a median follow-up period of 6.4?years. Five initial responders with subsequent relapse and one non-responder received multiple rituximab treatments of nine courses; all patients responded to the second rituximab therapy without any significant toxicity. All eight patients who relapsed after an initial response and six of 11 non-responders achieved CR or PR with subsequent treatment, including repeated courses of rituximab, splenectomy, steroids, and other immunomodulating agents. Our findings indicated that the sustained effect of rituximab on children with refractory ITP is low, but that the long-term outcome of ITP itself is not poor. Furthermore, repeated rituximab administration may be a promising therapy for those who relapse after an initial response.