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

NCT02170714 | Completed

• 1 other identifier: 16062014
• Study Type: observational
• Target: 31
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Pediatric Acute Severe Colitis

Keywords

Acute severe colitis; Children; Infliximab; colectomy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• 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

  First day of the hospitalization

Study Arms (1)

ASC

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

Interventions

Clinical and laboratory assessment OTHER

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

ASC

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Consecutive children hospitalized for an episode of ASC, defined as a PUCAI\>65

You may qualify if:

• Diagnosis of ASC
• Confirmed diagnosis of ulcerative colitis (UC)
• Age 1-18 years

You may not qualify if:

• Not confirmed diagnosis of UC
• Contraindications for infliximab therapy

Sponsors & Collaborators

• University of Roma La Sapienza: lead

Study Sites (1)

Department of Pediatrics, Sapienza University of Rome

Rome, Italy

Location

Related Publications (3)

• Turner D, Travis SP, Griffiths AM, Ruemmele FM, Levine A, Benchimol EI, Dubinsky M, Alex G, Baldassano RN, Langer JC, Shamberger R, Hyams JS, Cucchiara S, Bousvaros A, Escher JC, Markowitz J, Wilson DC, van Assche G, Russell RK; European Crohn's and Colitis Organization; Porto IBD Working Group, European Society of Pediatric Gastroenterology, Hepatology, and Nutrition. Consensus for managing acute severe ulcerative colitis in children: a systematic review and joint statement from ECCO, ESPGHAN, and the Porto IBD Working Group of ESPGHAN. Am J Gastroenterol. 2011 Apr;106(4):574-88. doi: 10.1038/ajg.2010.481. Epub 2011 Jan 11.

  PMID: 21224839 BACKGROUND

• Turner D, Griffiths AM. Acute severe ulcerative colitis in children: a systematic review. Inflamm Bowel Dis. 2011 Jan;17(1):440-9. doi: 10.1002/ibd.21383.

  PMID: 20645317 BACKGROUND

• Turner D, Mack D, Leleiko N, Walters TD, Uusoue K, Leach ST, Day AS, Crandall W, Silverberg MS, Markowitz J, Otley AR, Keljo D, Mamula P, Kugathasan S, Hyams J, Griffiths AM. Severe pediatric ulcerative colitis: a prospective multicenter study of outcomes and predictors of response. Gastroenterology. 2010 Jun;138(7):2282-91. doi: 10.1053/j.gastro.2010.02.047. Epub 2010 Feb 26.

  PMID: 20193683 BACKGROUND

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: June 16, 2014
• First Posted: June 23, 2014
• Study Start: May 1, 2010
• Primary Completion: May 1, 2012
• Study Completion: May 1, 2014
• Last Updated: June 23, 2014

Record last verified: 2014-06

Locations

IT (1)