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International Journal of Hematology

, Volume 98, Issue 3, pp 355–360 | Cite as

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

  • Satoru KumakiEmail author
  • Yoji Sasahara
  • Yoshiro Kamachi
  • Hideki Muramatsu
  • Tomohiro Morio
  • Kumiko Goi
  • Kanji Sugita
  • Tomonari Urabe
  • Hidetoshi Takada
  • Seiji Kojima
  • Shigeru Tsuchiya
  • Toshirou Hara
Original Article

Abstract

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.

Keywords

X-SCID  Reduced-intensity conditioning Umbilical cord blood transplantation Fludarabine/melphalan 

Notes

Acknowledgments

We thank Dr. David Cosman for critical reading of this manuscript. This study was supported by the Japanese Research Group on Primary Immunodeficiency Diseases, supported by the Ministry of Health, Labour and Welfare in Japan.

Conflict of interest

The authors declare no conflict of interest.

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Copyright information

© The Japanese Society of Hematology 2013

Authors and Affiliations

  • Satoru Kumaki
    • 1
    Email author
  • 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. 1.Department of PediatricsTohoku University Graduate School of MedicineSendaiJapan
  2. 2.Department of PediatricsNagoya University Graduate School of MedicineNagoyaJapan
  3. 3.Department of Pediatrics and Developmental Biology, Graduate School of MedicineTokyo Medical and Dental UniversityTokyoJapan
  4. 4.Department of Pediatrics, School of MedicineUniversity of YamanashiYamanashiJapan
  5. 5.Department of Pediatrics, Graduate School of Medical SciencesKumamoto UniversityKumamotoJapan
  6. 6.Department of Pediatrics, Graduate School of Medical SciencesKyushu UniversityFukuokaJapan

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