Zhongguo shi yan xue ye xue za zhi · 2026
[Clinical Analysis of Allogeneic Hematopoietic Stem Cell Transplantation for Very Early-Onset Inflammatory Bowel Disease Caused by IL-10RA Mutation].
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Wang Chao-Rong, Gao Hai-Li, Mao Yan-Na, Su Yu-Tai, Tian Liang, Ma Ping, Liu Wei, Wang Ya-Feng
Аннотация
OBJECTIVE: To investigate the clinical efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of very early-onset inflammatory bowel disease (VEO-IBD) caused by IL-10RA gene mutation.
METHODS: The clinical data of 6 pediatric patients with VEO-IBD caused by IL-10RA gene mutation who underwent allo-HSCT at Henan Children's Hospital Zhengzhou Children's Hospital from October 2019 to September 2023 were retrospectively analyzed.
RESULTS: Among the 6 patients, there were 3 girls and 3 boys. The median age at diagnosis was 9.0 (1.0-36.0) months, the median age at transplantation was 80.5 (4.3-96.0) months, and the median time from diagnosis to transplantation was 54.0 (2.3-95.0) months. Two cases received unrelated cord blood transplantation (UCBT), two cases received 10/10 HLA-matched unrelated donor (MUD) peripheral blood stem cell transplantation (PBSCT), and two cases received 10/10 HLA-matched sibling donor (MSD) PBSCT. All patients received myeloablative conditioning regimens primarily comprising busulfan, cyclophosphamide, and fludarabine. All 6 children achieved successful hematopoietic reconstitution. The median time for neutrophil engraftment was 13 (11-17) days, while the median time for platelet engraftment was 13.5 (11-28) days. One patient developed grade Ⅳ gastrointestinal acute graft-versus-host disease(aGVHD), and one patient had grade Ⅰ cutaneous aGVHD. None of the patients developed chronic graft-versus-host disease(cGVHD). Two patients experienced capillary leakage syndrome, and one patient developed hepatic veno-occlusive disease (VOD). Colonoscopy examinations after allo-HSCT in all patients showed evidence of mucosal healing.
CONCLUSION: Allo-HSCT is an efficacious therapeutic approach for children with VEO-IBD caused by IL-10RA mutation. The implementation of early HSCT is crucial for improving the prognosis and quality of life of children with VEO-IBD.
METHODS: The clinical data of 6 pediatric patients with VEO-IBD caused by IL-10RA gene mutation who underwent allo-HSCT at Henan Children's Hospital Zhengzhou Children's Hospital from October 2019 to September 2023 were retrospectively analyzed.
RESULTS: Among the 6 patients, there were 3 girls and 3 boys. The median age at diagnosis was 9.0 (1.0-36.0) months, the median age at transplantation was 80.5 (4.3-96.0) months, and the median time from diagnosis to transplantation was 54.0 (2.3-95.0) months. Two cases received unrelated cord blood transplantation (UCBT), two cases received 10/10 HLA-matched unrelated donor (MUD) peripheral blood stem cell transplantation (PBSCT), and two cases received 10/10 HLA-matched sibling donor (MSD) PBSCT. All patients received myeloablative conditioning regimens primarily comprising busulfan, cyclophosphamide, and fludarabine. All 6 children achieved successful hematopoietic reconstitution. The median time for neutrophil engraftment was 13 (11-17) days, while the median time for platelet engraftment was 13.5 (11-28) days. One patient developed grade Ⅳ gastrointestinal acute graft-versus-host disease(aGVHD), and one patient had grade Ⅰ cutaneous aGVHD. None of the patients developed chronic graft-versus-host disease(cGVHD). Two patients experienced capillary leakage syndrome, and one patient developed hepatic veno-occlusive disease (VOD). Colonoscopy examinations after allo-HSCT in all patients showed evidence of mucosal healing.
CONCLUSION: Allo-HSCT is an efficacious therapeutic approach for children with VEO-IBD caused by IL-10RA mutation. The implementation of early HSCT is crucial for improving the prognosis and quality of life of children with VEO-IBD.