Open AccessThalidomide Use and Outcomes in Pediatric Patients With Crohn Disease Refractory to Infliximab and Adalimumab
Author(s) -
Felipez Lina M.,
Gokhale Ranjana,
Tierney Matthew P.,
Kirschner Barbara S.
Publication year - 2012
Publication title -
journal of pediatric gastroenterology and nutrition
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.206
H-Index - 131
eISSN - 1536-4801
pISSN - 0277-2116
DOI - 10.1097/mpg.0b013e318228349e
Subject(s) - medicine , thalidomide , infliximab , prednisone , azathioprine , adalimumab , mercaptopurine , gastroenterology , surgery , crohn's disease , adverse effect , discontinuation , disease , multiple myeloma
Objective: The aim of the study was to evaluate thalidomide as rescue therapy for pediatric patients with severe refractory Crohn disease (CD) who failed to respond to antitumor necrosis factor (TNF) biologic agents. Patients and Methods: A computerized database was used to identify children with CD who had failed conventional immunosuppression therapy and received thalidomide rescue therapy. Twelve patients, mean age at diagnosis 10 years, were identified. Eight children had disease localized to the ileum and colon and 4 to the gastroduodenal area and colon. Five cases were complicated by strictures and 7 by fistulae. Previous drug therapy included azathioprine/6‐mercaptopurine (11/12), methotrexate (7/12), and anti‐TNF biologics (12/12). Outcome measures were Harvey‐Bradshaw Index, change in prednisone dose, hospitalizations, bowel resections, and incision and drainage procedures. Laboratory evaluations were calculated before and after 1 to 6 months of thalidomide. Results: Mean Harvey‐Bradshaw Index score improved from 11.8 to 3.9 ( P = 0.0004), mean prednisone dose decreased from 13.9 to 2.3 mg/day ( P = 0.001), mean number of hospitalizations decreased from 6.3 to 1.3 ( P = 0.002), and erythrocyte sedimentation rate decreased from 35 to 14 mm/h ( P = 0.02). The surgery rate pre‐thalidomide was 0.031 and on thalidomide was 0.004. Of the 7 patients with fistulae, 5 had complete fistula closure, 1 had partial closure, and 1 showed no improvement. Adverse reactions that resulted in discontinuation of thalidomide are as follows: 42% peripheral neuropathy, 17% worsening of the CD, 8% dizziness, and 8% allergic reaction. All 5 patients who developed peripheral neuropathy had clinical resolution of the neurologic symptoms within 2 to 3 months after stopping thalidomide. Conclusions: Thalidomide is a potentially effective rescue therapy for severe refractory CD in children who fail to respond to anti‐TNF medications.