Varying Doses of Epicutaneous Immunotherapy With Viaskin Milk vs Placebo in Children With Cow’s Milk Allergy
Authors: Petroni Daniel , Bégin Philippe , Bird J. Andrew , Brown-Whitehorn Terri , Chong Hey J. , Fleischer David M. , Gagnon Rémi , Jones Stacie M. , Leonard Stephanie , Makhija Melanie M. , Oriel Roxanne C. , Shreffler Wayne G. , Sindher Sayantani B. , Sussman Gordon L. , Yang William H. , Bee Katharine J. , Bois Timothée , Campbell Dianne E. , Green Todd D. , Rutault Karine , Sampson Hugh A. , Wood Robert A. 
Published: February 2024

ImportanceNo approved allergen-specific treatment exists for immunoglobulin E (IgE)–mediated cow’s milk allergy (CMA), a common childhood food allergy.ObjectiveTo assess dose, efficacy, and safety of epicutaneous immunotherapy with Viaskin Milk in children with IgE-mediated CMA.Design, Setting, and ParticipantsA phase 1/2, 2-part, randomized, double-blind, placebo-controlled dose-ranging clinical trial in children aged 2 to 17 years with IgE-mediated CMA was conducted between November 2014 through December 2017. It took place at 17 trial sites in the US and Canada. Current CMA was confirmed by double-blind, placebo-controlled food challenge at study entry. Part A assessed the short-term safety of 150 μg, 300 μg, or 500 μg of Viaskin Milk; part B evaluated the efficacy and safety of the 3 doses vs placebo over 12 months of treatment. Of the 308 screened participants with physician-diagnosed CMA, 198 met eligibility criteria (including an eliciting dose 300 mg or less) and were randomized.InterventionSafety of Viaskin Milk (150-μg, 300-μg, or 500-μg doses) was evaluated over a 3-week period (part A). In part B, 180 additional participants were randomized to receive Viaskin Milk at doses of 150 μg, 300 μg, or 500 μg or placebo (1:1:1:1) for 12 months.Main Outcomes and MeasuresThe primary outcome was the proportion of treatment responders, defined as a 10-fold or more increase in the cumulative reactive dose of cow’s milk protein (reaching at least 144 mg) or a cumulative reactive dose of cow’s milk protein at 1444 mg or more at the month 12 double-blind, placebo-controlled food challenge.ResultsA total of 95.5% of the randomized participants (mean [SD] age, 8 [4.17] years; 124 of 198 were male [62.6%]) completed treatment. The highest response rate was observed in participants who received Viaskin Milk at the 300-μg dose with 24 of 49 responders (49.0%) overall vs 16 of 53 responders (30.2%) in the placebo group (odds ratio, 2.19; 95% CI, 0.91-5.41; P = .09), highest in the 2 to 11 years age group (22 of 38 [57.9%] vs 13 of 40 [32.5%]; P = .04). Most treatment-emergent adverse events were mild or moderate application-site reactions. One participant in the 500-μg Viaskin Milk dose group experienced treatment-related anaphylaxis.Conclusions and RelevanceIn this randomized clinical trial, 12 months of daily epicutaneous immunotherapy with a dose of Viaskin Milk at 300 μg was associated with a statistically significant treatment response in 2- to 11-year-old children with IgE-mediated CMA. Treatment-related anaphylaxis and treatment-related discontinuation rates were low. Further research is needed to explore Viaskin Milk as a viable treatment option for children with IgE-mediated CMA.Trial RegistrationClinicalTrials.gov Identifier: NCT02223182