B-cell function after unrelated umbilical cord blood transplantation using a minimal-intensity conditioning regimen in patients with X-SCID

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• 作者：Satoru Kumaki (1)
  Yoji Sasahara (1)
  Yoshiro Kamachi (2)
  Hideki Muramatsu (2)
  Tomohiro Morio (3)
  Kumiko Goi (4)
  Kanji Sugita (4)
  Tomonari Urabe (5)
  Hidetoshi Takada (6)
  Seiji Kojima (2)
  Shigeru Tsuchiya (1)
  Toshirou Hara (6)
  
• 关键词：X ; SCID ; Reduced ; intensity conditioning ; Umbilical cord blood transplantation ; Fludarabine/melphalan
• 刊名：International Journal of Hematology
• 出版年：2013
• 出版时间：September 2013
• 年：2013
• 卷：98
• 期：3
• 页码：355-360
• 全文大小：198KB
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• 作者单位：Satoru Kumaki (1)
  Yoji Sasahara (1)
  Yoshiro Kamachi (2)
  Hideki Muramatsu (2)
  Tomohiro Morio (3)
  Kumiko Goi (4)
  Kanji Sugita (4)
  Tomonari Urabe (5)
  Hidetoshi Takada (6)
  Seiji Kojima (2)
  Shigeru Tsuchiya (1)
  Toshirou Hara (6)
  
  1. Department of Pediatrics, Tohoku University Graduate School of Medicine, 1-1 Seiryo Machi, Aoba-ku, Sendai, 980-8574, Japan
  2. Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan
  3. Department of Pediatrics and Developmental Biology, Graduate School of Medicine, Tokyo Medical and Dental University, Tokyo, Japan
  4. Department of Pediatrics, School of Medicine, University of Yamanashi, Yamanashi, Japan
  5. Department of Pediatrics, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan
  6. Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan
  

文摘

Patients with X-linked severe combined immunodeficiency (X-SCID) suffer from severe and persistent infections, and usually die early in life unless treated by hematopoietic stem cell transplantation. If a patient has an HLA-identical sibling donor, preparative conditioning is not necessary for T-cell engraftment and B-cell function. However, in the absence of such a donor, long-term reconstitution of full B-cell function is often problematic, leading in many cases to a lifetime requirement for immunoglobulin replacement therapy. Preparative myeloablative conditioning has been shown to improve long-term B-cell function, but may aggravate pre-existing infection and transplant-related toxicity. It is thus important to determine the minimum intensity of conditioning that assures immunoglobulin production. In the present study, we performed reduced-intensity conditioning (RIC), consisting of fludarabine 125?mg/m2 and melphalan 80?mg/m2, prior to unrelated umbilical cord blood transplantation (UCBT) for five patients with X-SCID, none of them had an HLA-identical donor. Four patients survived more than 4?years without sequelae, and none required long-term immunoglobulin replacement therapy. One patient succumbed to sepsis in conjunction with severe GVHD. Our result demonstrates that the RIC regimen described above in combination with UCBT is an effective and less toxic conditioning to correct B-cell function in patients with X-SCID.