Efficacy of Infliximab as a Rescue Therapy in Pediatric Acute Severe Colitis

Study Overview
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Brief Summary
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Background and aims: Acute severe ulcerative colitis (ASC) is a potentially life-threatening event. Poor pediatric data are available about the success rates of Infliximab (IFX) as a second line therapy. This study was performed in consecutively observed pediatric patients with ASC, treated according to the 2011 European Crohn's colitis Organization (ECCO)- European Society for Pediatric Gastroenterology, Hepatology and nutrition (ESPGHAN) guidelines on pediatric ASC¹ and aim to assess the long-term efficacy of IFX and clinical predictors of poor outcome. Methods: Children hospitalized for an episode of ASC, defined as a Pediatric Ulcerative Colitis Activity Index (PUCAI) of at least 65 points, were enrolled. Clinical assessment through PUCAI and laboratory data (Erythrocyte Sedimentation Rate, C-Reactive Protein, hemoglobin, albumin, hematocrit, ferritin) was recorded at admission and at day 3 and 5. All patients were treated according to the above mentioned guidelines for ASC and received intravenous (iv) corticosteroids (CS) as first-line therapy. IFX was administered as second-line therapy in CS-refractory patients. In a 2-year follow up the overall colectomy rate and the efficacy of IFX in avoiding colectomy were evaluated.

Official Title
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Managing Pediatric Acute Severe Colitis According to the 2011 ECCO-ESPGHAN Guidelines: Efficacy of Infliximab as a Rescue Therapy

Conditions
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Pediatric Acute Severe Colitis

Intervention / Treatment
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* Other: Clinical and laboratory assessment


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of ASC Confirmed diagnosis of ulcerative colitis (UC) Age 1-18 years Exclusion Criteria: Not confirmed diagnosis of UC Contraindications for infliximab therapy

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| ASC<br>Consecutive children hospitalized for an episode of acute ASC, defined as a PUCAI > 65. All patients were treated according to the 2011 ECCO-ESPGHAN guidelines for ASC: all patients received intravenous (iv) corticosteroids (methylprednisolone 1.5-2 mg/Kg/day) for 5 days. Patients not responding to corticosteroids (i.e. PUCAI>65 at day 5) started Infliximab (IFX, 5 mg/Kg 0,2,6 then every 8 weeks) as second-line therapy. All therapies were decided at the discretion of the referral gastroenterologist and recorded on standardized case report forms. A follow-up of 2 years for the colectomy risk was evaluated for all patients. | Other: Clinical and laboratory assessment<br>* Clinical assessment through PUCAI and laboratory data (Erythrocyte Sedimentation Rate, C-Reactive Protein, hemoglobin, albumin, hematocrit, ferritin) were recorded at admission and at day 3 and 5. All patients were treated according to the 2011 ECCO-ESPGHAN guidelines for ASC: all patients received intravenous (iv) corticosteroids (methylprednisolone 1.5-2 mg/Kg/day) for 5 days. Patients not responding to corticosteroids (i.e. PUCAI>65 at day 5) started Infliximab (IFX, 5 mg/Kg 0,2,6 then every 8 weeks) as second-line therapy. All therapies were decided at the discretion of the referral gastroenterologist and recorded on standardized case report forms<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The number of patients with ASC treated with IFX requiring colectomy | The surgical need will be evaluated in the group of patients treated with IFX during a 24-month follow-up | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The number of patients with ASC requiring colectomy |  | 1,6,12,18,24 months | 
| To identify clinical predictive factors at the diagnosis of ASC of surgical need at follow-up | Clinical variables at the diagnosis of ASC (i.e. duration of the disease before the episode of SC, past and ongoing therapies, age, family history, Perinuclear Anti-Neutrophil Cytoplasmic Antibodies - pANCA, PUCAI) were evaluated in the group of children needed colectomy at follow-up versus those not requiring surgery. | First day of the hospitalization | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acute severe colitis, Children, Infliximab, colectomy