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Peer-reviewedCC BY 4.0International Journal of Hematology

Allogeneic hematopoietic cell transplantation with reduced-toxicity conditioning for X-linked hyper-IgM syndrome

  • Ryosuke Wakatsuki1
  • Satoshi Miyamoto1
  • Akihiro Hoshino2
  • Shuhei Takahashi1
  • Yusuke Ishibashi1
  • Ryohei Watanabe1
  • Tatsuya Magara1
  • Dan Tomomasa1
  • Akira Nishimura1
  • Kyogo Suzuki1
  • Takahiro Kamiya1
  • Takeshi Isoda1
5 more authors
  • Kohsuke Imai3
  • Michiko Kajiwara4
  • Tomohiro Morio5
  • Masatoshi Takagi1
  • Hirokazu Kanegane2
5 affiliations
  1. 1Department of Pediatrics and Developmental Biology, Graduate School of Medical and Dental Sciences, Institute of Science Tokyo, Tokyo, Japan
  2. 2Department of Child Health and Development, Graduate School of Medical and Dental Sciences, Institute of Science Tokyo, 1-5-45 Yushima, Bunkyo-ku, Tokyo 113-8519 Japan
  3. 3Department of Pediatrics, National Defense Medical College, Saitama, Japan
  4. 4Center for Transfusion Medicine and Cell Therapy, Institute of Science Tokyo Hospital, Tokyo, Japan
  5. 5Laboratory of Immunology and Molecular Medicine, Institute of Science Tokyo, Tokyo, Japan
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Abstract

X-linked hyper-IgM syndrome (XHIGM) is an inborn error of immunity caused by variants in the CD40LG gene, leading to disrupted T-cell function and impaired immunoglobulin class-switch recombination. Supportive care alone is linked to unfavorable outcomes, necessitating allogeneic hematopoietic cell transplantation (HCT) as a curative option. Although myeloablative conditioning is recommended for HCT, concerns remain regarding its toxicity. We reviewed ten patients with XHIGM who underwent HCT using reduced-toxicity conditioning (RTC) at our institution between 2010 and 2024. All patients underwent HCT by 5 years of age (range: 2–5 years; median: 3 years). Donor sources included unrelated bone marrow, unrelated cord blood, and haploidentical-related donors. Conditioning primarily consisted of fludarabine and busulfan (target area under the curve: 60–65 mg/L·h), with low-dose total body irradiation (TBI) administered in five patients. Two patients experienced graft failure, requiring a second transplantation, and one died from post-transplant complications. The remaining nine patients survived. The 5-year overall survival rate was 88.9%; the 5-year graft-versus-host disease-free, relapse-free, and second transplantation-free survival rate was 67.5%. The addition of low-dose TBI to fludarabine/busulfan was associated with favorable long-term donor chimerism, although this finding should be considered hypothesis-generating. RTC may represent a feasible conditioning strategy for XHIGM.

Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s12185-026-04245-8.

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Cite

BibTeXRIS
BibTeX
@article{wakatsuki2026allogeneic,
  title = {{Allogeneic hematopoietic cell transplantation with reduced-toxicity conditioning for X-linked hyper-IgM syndrome}},
  author = {Ryosuke Wakatsuki and Satoshi Miyamoto and Akihiro Hoshino and Shuhei Takahashi and Yusuke Ishibashi and Ryohei Watanabe and Tatsuya Magara and Dan Tomomasa and Akira Nishimura and Kyogo Suzuki and Takahiro Kamiya and Takeshi Isoda and Kohsuke Imai and Michiko Kajiwara and Tomohiro Morio and Masatoshi Takagi and Hirokazu Kanegane},
  journal = {International Journal of Hematology},
  year = {2026},
  month = jul,
  doi = {10.1007/s12185-026-04245-8},
  url = {https://doi.org/10.1007/s12185-026-04245-8},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Allogeneic hematopoietic cell transplantation with reduced-toxicity conditioning for X-linked hyper-IgM syndrome
AU  - Ryosuke Wakatsuki
AU  - Satoshi Miyamoto
AU  - Akihiro Hoshino
AU  - Shuhei Takahashi
AU  - Yusuke Ishibashi
AU  - Ryohei Watanabe
AU  - Tatsuya Magara
AU  - Dan Tomomasa
AU  - Akira Nishimura
AU  - Kyogo Suzuki
AU  - Takahiro Kamiya
AU  - Takeshi Isoda
AU  - Kohsuke Imai
AU  - Michiko Kajiwara
AU  - Tomohiro Morio
AU  - Masatoshi Takagi
AU  - Hirokazu Kanegane
PY  - 2026
DA  - 2026/07/07
JO  - International Journal of Hematology
DO  - 10.1007/s12185-026-04245-8
UR  - https://doi.org/10.1007/s12185-026-04245-8
AB  - X-linked hyper-IgM syndrome (XHIGM) is an inborn error of immunity caused by variants in the CD40LG gene, leading to disrupted T-cell function and impaired immunoglobulin class-switch recombination. Supportive care alone is linked to unfavorable outcomes, necessitating allogeneic hematopoietic cell transplantation (HCT) as a curative option. Although myeloablative conditioning is recommended for HCT, concerns remain regarding its toxicity. We reviewed ten patients with XHIGM who underwent HCT using reduced-toxicity conditioning (RTC) at our institution between 2010 and 2024. All patients underwent HCT by 5 years of age (range: 2–5 years; median: 3 years). Donor sources included unrelated bone marrow, unrelated cord blood, and haploidentical-related donors. Conditioning primarily consisted of fludarabine and busulfan (target area under the curve: 60–65 mg/L·h), with low-dose total body irradiation (TBI) administered in five patients. Two patients experienced graft failure, requiring a second transplantation, and one died from post-transplant complications. The remaining nine patients survived. The 5-year overall survival rate was 88.9%; the 5-year graft-versus-host disease-free, relapse-free, and second transplantation-free survival rate was 67.5%. The addition of low-dose TBI to fludarabine/busulfan was associated with favorable long-term donor chimerism, although this finding should be considered hypothesis-generating. RTC may represent a feasible conditioning strategy for XHIGM.  Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s12185-026-04245-8.
SN  - 0925-5710
SN  - 1865-3774
ER  -