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Successful Allogeneic Hematopoietic Cell Transplantation for Patients with IL10RA Deficiency in Japan.
- Source :
-
Journal of clinical immunology [J Clin Immunol] 2024 Sep 12; Vol. 45 (1), pp. 6. Date of Electronic Publication: 2024 Sep 12. - Publication Year :
- 2024
-
Abstract
- Background: IL10RA (IL10 receptor subunit alpha) deficiency is an autosomal recessive disease that causes inflammatory bowel disease during early infancy. Its clinical course is often fatal and the only curative treatment is allogeneic hematopoietic cell transplantation (HCT). In Japan, only case reports are available, and there are no comprehensive reports of treatment outcomes.<br />Methods: We retrospectively analyzed patients with IL10RA deficiency in Japan.<br />Results: Two newly identified and five previously reported patients were included in this study. Five patients underwent HCT; one untransplanted patient survived to age 14, and one died of influenza encephalopathy before transplantation. All five HCT recipients underwent HCT at the age before 2 years. They all were conditioned with fludarabine/busulfan- or fludarabine /melphalan-based regimens. The donor source was human leukocyte antigen haploidentical donor bone marrow (BM) for two patients and unrelated umbilical cord blood (CB) for two patients. One patient experienced graft failure with unrelated CB and required a second transplant with unrelated BM. All patients who underwent HCT survived and demonstrated an improved performance status.<br />Conclusion: In cases of IL10RA deficiency, the need for transplantation should be promptly assessed, and early transplantation should be considered. (190/250).<br /> (© 2024. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)
- Subjects :
- Humans
Japan
Male
Female
Infant
Treatment Outcome
Retrospective Studies
Transplantation Conditioning methods
Adolescent
Child, Preschool
Interleukin-10 Receptor alpha Subunit genetics
Interleukin-10 Receptor alpha Subunit deficiency
Child
Inflammatory Bowel Diseases therapy
Hematopoietic Stem Cell Transplantation methods
Transplantation, Homologous
Subjects
Details
- Language :
- English
- ISSN :
- 1573-2592
- Volume :
- 45
- Issue :
- 1
- Database :
- MEDLINE
- Journal :
- Journal of clinical immunology
- Publication Type :
- Academic Journal
- Accession number :
- 39264505
- Full Text :
- https://doi.org/10.1007/s10875-024-01795-6