Are withdrawal trials in paediatric rheumatic disease helpful?

Abstract

In today's Lancet, Nicolino Ruperto and colleagues 1 Ruperto N Lovell DJ Quartier P et al. for the Paediatric Rheumatology International Trials Organization (PRINTO)the Pediatric Rheumatology Collaborative Study Group (PRCSG)Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trial. Lancet. 2008; (published online July 15.)https://doi.org/10.1016/S0140-6736(08)60998-8 Summary Full Text Full Text PDF PubMed Scopus (405) Google Scholar report on the efficacy and safety of abatacept in children with juvenile idiopathic arthritis. Their study shows acceptable safety but a surprisingly high rate of adverse events: the treatment group had an adverse event rate of 62% and the rate in the placebo recipients was 55%. However, before entering the double-blind phase of the study, all the children had first received abatacept in an open-label phase; the “withdrawal” design thus means the patients were withdrawn from active drug before being randomised to the same drug or placebo. This type of design makes it impossible to be sure that the high rate of adverse events in the placebo group was not related to their initial treatment with active drug, because of a carryover effect. Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trialSelective modulation of T-cell costimulation with abatacept is a rational alternative treatment for children with juvenile idiopathic arthritis. Full-Text PDF