Inflammatory Bowel Diseases
Conditions
Brief summary
This is a single center, observational study to investigate the clinical outcomes of thalidomide treatment for very early onset inflammatory bowel disease
Detailed description
Very early onset inflammatory bowel disease (VEOIBD) refers to IBD diagnosed before 6 years of age. VEOIBD comprises a distinct subset of pediatric IBD characterized by stronger genetic predisposition, predominant colonic involvement, more extensive inflammation, more severe course, and peculiar response to treatments. Previous studies have been demonstrated that thalidomide, an oral molecule with immunomodulatory, antiangiogenic, and TNF-suppressing properties, is an effective and safe treatment for adults and children with IBD refractory to conventional therapies. This study is aimed to evaluate the efficacy and tolerance of thalidomide in VEOIBD.
Interventions
DRUGThalidomide
thalidomide, dose 1.5-2.5mg/kg.d, orally taken before bedtime.
Sponsors
Children's Hospital of Fudan University
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
28 Days to 6 Years
Healthy volunteers
No
Inclusion criteria
* diagnosed with inflammatory bowel disease * disease onset less than 6 years old * Pediatric Crohn's disease Activity Index (PCDAI)\>30 or Pediatric Ulcerative Colitis Activity Index (PUCAI)\>35 * patients and their legal guardians were willing to receive thalidomide treatment and participate in this study.
Exclusion criteria
* liver dysfunction * allergy to thalidomide * with Neuropathy * with thrombosis * thalidomide treatment in the previous 30 days * biologics treatment in the previous 8 weeks * not suitable participated in this study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| endoscopic response rate | week 52 | The Simple Endoscopic Score for Crohn Disease decreased 50% compared with baseline OR Mayo score 0-1 (higher scores mean a worse outcome) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| biomarker remission rate | week8, week26, week52 | biomarker remission is defined as not only C-reactive protein less than 8mg/L, but also fecal calprotectin less than 100ug/g. |
| growth development | week52 | weight and height Z score (higher scores mean a better outcome) |
| clinical response rate | week8, week26, week52 | Inflammatory bowel disease mainly contains Crohn's disease and ulcerative colitis. If the patient was diagnosed with Crohn's disease, we use the Pediatric Crohn's disease Activity Index score to evaluate the clinical response, which is defined as Pediatric Crohn's disease Activity Index decreased more than 12.5 compared with baseline. If the patient was diagnosed with ulcerative colitis, we use the Pediatric Ulcerative Colitis Activity Index score to evaluate the clinical response, which is defined as Pediatric Ulcerative Colitis Activity Index score decreased more than 20 compared with baseline. The Pediatric Crohn's disease Activity Index score varies from 0 to 100 and the Pediatric Ulcerative Colitis Activity Index score varies from 0 to 85 (higher scores mean a worse outcome). |
| adverse event | within one year | The rate of the potential adverse events were monitored during the intervention. |
| treatment persistence | within one year | the time between the start and the last dose of thalidomide |
| clinical remission rate with steroid-free | week26 | If the patient was diagnosed with Crohn's disease, this outcome is defined as the Pediatric Crohn's disease Activity Index score less than 10 without steroid treatment. If the patient was diagnosed with ulcerative colitis, this outcome is defined as the Pediatric Ulcerative Colitis Activity Index score less than 10 without steroid treatment. |
Countries
China
Contacts
Primary ContactYing Huang
Outcome results
None listed