Anna R. Kahkoska|Michael T. Lawson|Jamie Crandell|Kimberly A. Driscoll|Jessica C. Kichler|Michael Seid|David M. Maahs|Michael R. Kosorok|Elizabeth J. Mayer?Davis
Assessment of a Precision Medicine Analysis of a Behavioral Counseling Strategy to Improve Adherence to Diabetes Self-management Among Youth
University of North Carolina at Chapel Hill|University of North Carolina at Chapel Hill|University of North Carolina at Chapel Hill|University of Colorado Denver|Cincinnati Children's Hospital Medical Center|University of Cincinnati|Cincinnati Children's Hospital Medical Center|University of Cincinnati|Stanford University|University of North Carolina at Chapel Hill|University of North Carolina at Chapel Hill
flexible lifestyles empowering change flex trial 18month randomized clinical trial testing adaptive behavioral intervention adolescents type 1 diabetes showed overall treatment effect primary outcome change hemoglobin a1c hba1c percentage total hemoglobin demonstrated benefit quality life qol prespecified secondary outcometo apply novel statistical method post hoc analysis derives individualized treatment rule itr identify flex participants may benefit intervention based changes hba1c percentage primary outcome qol body mass index z score bmiz secondary outcomes 18 monthsthis multisite clinical trial enrolled 258 adolescents aged 13 16 years type 1 diabetes 1 years literacy english hba1c percentage total hemoglobin 80 130 participating caregiver serious medical conditions january 5 2014 april 4 2016 258 adolescents recruited post hoc analysis excluded adolescents missing outcome measures 18 months 2 participants 08 continuous glucose monitoring data baseline 40 participants 155 data analyzed april december 2018the flex intervention included behavioral counseling strategy integrated motivational interviewing problemsolving skills training increase adherence diabetes selfmanagement control condition entailed usual diabetes caresubgroups flex participants derived itr estimating participants would benefit intervention would benefit control conditions would indifferent multiple imputation chained equations reinforcement learning trees used estimate itr subgroups based itr pertaining changes 18 months 3 univariate outcomes ie hba1c percentage qol bmiz composite outcome compared baseline demographic clinical psychosocial characteristicsdata 216 adolescents flex trial reanalyzed 166 769 nonhispanic white 108 teenaged girls 500 mean sd age 149 11 years mean sd diabetes duration 63 37 years univariate outcomes large proportion flex participants equivalent predicted outcomes intervention vs usual care settings regardless randomization assigned muted group hba1c 105 participants 486 qol 63 participants 292 bmiz 136 participants 630 regarding bmiz univariate outcome mean baseline bmiz participants assigned muted group lower assigned intervention control groups muted vs intervention mean difference 048 95 ci 021 075 p 002 muted vs control mean difference 086 95 ci 061 111 p 001 group also higher proportion individuals underweight normal weight using weight status cutoffs 95 699 muted group vs 24 546 intervention group 11 306 control group 24 2467 p 001 approach identified subgroups estimated benefit based hba1c percentage 54 participants 250 qol 89 participants 412 bmiz 44 participants 204 regarding hba1c percentage outcome participants expected benefit intervention significantly higher baseline hba1c percentages expected benefit usual care 94 vs 92 difference 02 95 ci 016 056 p 44 however participants muted group higher mean hba1c percentages baseline assigned intervention control groups muted vs intervention 99 vs 94 difference 05 95 ci 013 089 p 02 muted vs control 99 vs 92 difference 07 95 ci 034 108 p 001 significant differences found subgroups estimated benefit terms composite outcome flex intervention 91 participants 421 vs usual care 125 participants 579the precision medicine approach represents conceptually analytically novel approach post hoc subgroup identification work needed understand markers positive response flex interventionclinicaltrialgov identifier nct01286350