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This is a single center, observational study to investigate the clinical outcomes of thalidomide treatment for very early onset inflammatory bowel disease
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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Thalidomide | Drug | thalidomide, dose 1.5-2.5mg/kg.d, orally taken before bedtime. |
| Measure | Description | Time Frame |
|---|---|---|
| endoscopic response rate | The Simple Endoscopic Score for Crohn Disease decreased 50% compared with baseline OR Mayo score 0-1 (higher scores mean a worse outcome) | week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| clinical response rate | 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). |
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Inclusion Criteria:
Exclusion Criteria:
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Children diagnosed with very early onset inflammatory bowel disease who were seen at the Children's hospital of Fudan university who met the inclusion and exclusion criteria would be enrolled in this study.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ying Huang | Contact | 021-64931727 | yhuang815@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Ying Huang | Children's Hospital of Fudan University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lin Wang | Recruiting | Shanghai | Shanghai Municipality | 201102 | China |
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| ID | Term |
|---|---|
| D015212 | Inflammatory Bowel Diseases |
| ID | Term |
|---|---|
| D005759 | Gastroenteritis |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
| D007410 | Intestinal Diseases |
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| ID | Term |
|---|---|
| D013792 | Thalidomide |
| ID | Term |
|---|---|
| D010797 | Phthalimides |
| D010795 | Phthalic Acids |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
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blood sample, stool samples and biopsy samples during endoscopy
| week8, week26, week52 |
| biomarker remission rate | biomarker remission is defined as not only C-reactive protein less than 8mg/L, but also fecal calprotectin less than 100ug/g. | week8, week26, week52 |
| growth development | weight and height Z score (higher scores mean a better outcome) | week52 |
| clinical remission rate with steroid-free | 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. | week26 |
| adverse event | The rate of the potential adverse events were monitored during the intervention. | within one year |
| treatment persistence | the time between the start and the last dose of thalidomide | within one year |
| D009930 |
| Organic Chemicals |
| D010881 | Piperidones |
| D010880 | Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D054833 | Isoindoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |