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

Satoru Kumaki, Yoji Sasahara, Yoshiro Kamachi, Hideki Muramatsu, Tomohiro Morio, Kumiko Goi, Kanji Sugita, Tomonari Urabe, Hidetoshi Takada, Seiji Kojima, Shigeru Tsuchiya, Toshirou Hara

研究成果: ジャーナルへの寄稿記事

1 引用 (Scopus)

抄録

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.

元の言語英語
ページ(範囲)355-360
ページ数6
ジャーナルInternational Journal of Hematology
98
発行部数3
DOI
出版物ステータス出版済み - 9 1 2013

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X-Linked Combined Immunodeficiency Diseases
Fetal Blood
B-Lymphocytes
Transplantation
Passive Immunization
Tissue Donors
Melphalan
Poisons
Hematopoietic Stem Cell Transplantation
Infection
Conditioning (Psychology)
Immunoglobulins
Siblings
Sepsis
T-Lymphocytes
Transplants

All Science Journal Classification (ASJC) codes

  • Hematology

これを引用

B-cell function after unrelated umbilical cord blood transplantation using a minimal-intensity conditioning regimen in patients with X-SCID. / Kumaki, Satoru; Sasahara, Yoji; Kamachi, Yoshiro; Muramatsu, Hideki; Morio, Tomohiro; Goi, Kumiko; Sugita, Kanji; Urabe, Tomonari; Takada, Hidetoshi; Kojima, Seiji; Tsuchiya, Shigeru; Hara, Toshirou.

:: International Journal of Hematology, 巻 98, 番号 3, 01.09.2013, p. 355-360.

研究成果: ジャーナルへの寄稿記事

Kumaki, S, Sasahara, Y, Kamachi, Y, Muramatsu, H, Morio, T, Goi, K, Sugita, K, Urabe, T, Takada, H, Kojima, S, Tsuchiya, S & Hara, T 2013, 'B-cell function after unrelated umbilical cord blood transplantation using a minimal-intensity conditioning regimen in patients with X-SCID', International Journal of Hematology, 巻. 98, 番号 3, pp. 355-360. https://doi.org/10.1007/s12185-013-1408-7
Kumaki, Satoru ; Sasahara, Yoji ; Kamachi, Yoshiro ; Muramatsu, Hideki ; Morio, Tomohiro ; Goi, Kumiko ; Sugita, Kanji ; Urabe, Tomonari ; Takada, Hidetoshi ; Kojima, Seiji ; Tsuchiya, Shigeru ; Hara, Toshirou. / B-cell function after unrelated umbilical cord blood transplantation using a minimal-intensity conditioning regimen in patients with X-SCID. :: International Journal of Hematology. 2013 ; 巻 98, 番号 3. pp. 355-360.
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