Allogeneic hematopoietic cell transplantation for autoinflammatory disorders
Hirokazu Kanegane1, Satoshi Miyamoto2, Takahiro Kamiya2,3
1Department of Child Health and Development, Graduate School of Medical and Dental Sciences, Institute of Science Tokyo, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan. hkanegane.ped@tmd.ac.jp.
International Journal of Hematology
|June 11, 2025
Summary
Allogeneic hematopoietic cell transplantation (HCT) shows promise for treating refractory autoinflammatory disorders when other therapies fail. This review explores HCT as a potential treatment option for these complex immune system conditions.
Area of Science:
- Immunology
- Genetics
- Hematology
Background:
- Autoinflammatory disorders involve innate immune system dysregulation.
- New diseases and phenotypes are continually being identified.
- Current treatments include immunosuppressants, biologics, and targeted therapies, but some cases are refractory.
Purpose of the Study:
- To review the potential of allogeneic hematopoietic cell transplantation (HCT) in treating autoinflammatory disorders.
- To highlight HCT as a therapeutic option for treatment-refractory cases.
Main Methods:
- Literature review of existing reports on HCT for autoinflammatory disorders.
- Discussion of HCT's efficacy and safety based on previous findings.
Main Results:
- Allogeneic HCT has been reported to improve symptoms in refractory autoinflammatory disorders.
- HCT offers a potential solution for patients unresponsive to conventional treatments.
Conclusions:
- Allogeneic HCT is a viable therapeutic strategy for severe, refractory autoinflammatory disorders.
- Further research and clinical application of HCT are warranted for these conditions.
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