Inflammatory Bowel Disease
Conditions
Interventions
Allogeneic Hematopoietic Stem Cell Transplantation for Monogenic Inflammatory Bowel Disease:NA
Sponsors
Children's Hospital of Fudan University
Eligibility
Sex/Gender
All
Age
0.2 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1 The age at transplant should be greater than 2 months and less than 18 years old. 2 The diagnosis of monogenic inflammatory bowel disease should be based on the following criteria: (1) Clinical manifestations: such as diarrhea, bloody stools, abdominal pain, and weight loss that persist for more than 4 weeks or recur more than 2 times within 6 months, often accompanied by systemic manifestations such as fever, growth retardation, malnutrition, anemia, and may be accompanied by perianal abscesses, dermatophytes, anal fissures, anal fistulas, and other perianal diseases. (2) Endoscopic examination: Crohn's disease is characterized by a segmental, asymmetric, and jumping distribution of lesions, with visible aphthous ulcers, fissure like ulcers, longitudinal ulcers, paving stone like intestinal mucosa, intestinal stenosis, and intestinal wall stiffness. Ulcerative colitis is characterized by granular mucosa, congestion, brittle texture, easy bleeding, blurred or disappeared vascular texture, diffuse punctate erosion, shallow or small ulcers. (3) Histopathology: The histopathological characteristics of Crohn's disease include full thickness intestinal wall lymphocyte proliferation, non-caseous granuloma, focal crypt structural abnormalities, aphthous ulcer, and fissure like ulcer. If the surgical specimen is used, the overall pathological characteristics are intestinal jumping lesions, fused linear ulcers, paving stone like changes, fistula formation, and intestinal stenosis. The histological manifestations of ulcerative colitis include changes in crypt structure and inflammatory infiltration. (4) Next-generation sequencing or Sanger sequencing showed mutations such as IL10, IL10R, FOXP3, CYBB, XIAP, LRBA, ZAP70, STAT3, IL2RA, CYBA, CD40LG. 3 Received allogeneic hematopoietic stem cell transplantation, including matched sibling donors, unrelated volunteers, unrelated umbilical cord blood, and haploid donors.
Exclusion criteria
Exclusion criteria: 1 Children who have severe organ damage during transplantation (such as requiring ventilator assisted ventilation, relying on vasoactive drugs, etc.) but still receive salvage transplantation. 2 Children undergoing secondary transplantation. 3 Children with highly suspected monogenic inflammatory bowel disease but no pathogenic genes found or the presence of related pathogenic genes cannot be ruled out as intestinal tuberculosis or other infectious enteritis.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 2-year disease-free surviva;2-year overall survival;Transplantation related complications; | — |
Secondary
| Measure | Time frame |
|---|---|
| The incidence of graft-versus-host disease; | — |
Countries
China
Contacts
Public ContactZhai Xiaowen
Children's Hospital of Fudan University
Outcome results
None listed