Adaptive Interventions: What are they? Why do we need them? and How - PowerPoint PPT Presentation

Adaptive Interventions: What are they? Why do we need them? and How can we study them? Daniel Almirall, Inbal Nahum-Shani, Susan A. Murphy Survey Research Center, Institute for Social Research University of Michigan October, 2016 Institute for Translational Research in Children’s Mental Health University of Minnesota Almirall, Nahum-Shani, Murphy Adaptive Interventions October 2016 1 / 66

Outine for the next 3 talks! 1 Introduction to Adaptive Interventions and SMART Daniel Almirall 2 From Adaptive to “Just in Time” Adaptive Interventions Inbal “Billie” Nahum-Shani 3 Micro-randomized Trials in Mobile Health Susan A. Murphy Almirall, Nahum-Shani, Murphy Adaptive Interventions October 2016 2 / 66

Introduction to Adaptive Interventions and (Cluster-level) SMART Designs Daniel Almirall Survey Research Center, Institute for Social Research University of Michigan October 7, 2016 Institute for Translational Research in Children’s Mental Health University of Minnesota Almirall Adaptive Interventions October 2016 3 / 66

I don’t do any of this research by myself. Colleagues Billie Nahum-Shani , Univ Mich (psychology, behavioral medicine) Susan A. Murphy , Univ Mich (statistician) Connie Kasari , UCLA (autism) Amy Kilbourne , Univ Mich (psychiatry) Ex-students Xi Lu , Google (statistician) Current Students Tim NeCamp , Univ Mich (statistician) And many others... Almirall Adaptive Interventions October 2016 4 / 66

Outline What is an Adaptive Intervention? Why do we need Adaptive Interventions? How can we study Adaptive Interventions? Three SMART Case Studies (2 in Autism, 1 in Adult Mood Disorder) ◮ Characterizing Cognition in Non-verbal Individuals with ASD (CCNIA) ◮ Getting SMART about Social & Academic Engagement (ASD Schools) ◮ Adaptive Implementation of Effective Programs (ADEPT, mood dx) Myths or Misconceptions about Adaptive Interventions and SMARTs Almirall Adaptive Interventions October 2016 5 / 66

Intervention often entails a sequential, individualized approach whereby treatment is adapted and re-adapted over time Almirall Adaptive Interventions October 2016 6 / 66

Q: Why do we need sequential, individualized treatments? A: Heterogeneity! This type of sequential decision-making is necessary when there is high level of individual heterogeneity in response to treatment. ◮ e.g., what works for one individual may not work for another ◮ e.g., what works now may not work later ◮ e.g., what appears not to work in the short-run has positive long-term consequences, and vice-versa ◮ e.g., adherence, engagement, and burden all have time-changing courses Adaptive Interventions help guide this type of individualized, sequential, treatment decision making Almirall Adaptive Interventions October 2016 7 / 66

What is an Adaptive Intervention? Almirall Adaptive Interventions October 2016 8 / 66

Definition of an Adaptive Intervention Sequence of decision rules that guide whether, how, or when (timing), and based on which measures, to alter the dosage (duration, freq or amount), type, or delivery of treatment(s) at decision stages in course of care. Intended to guide the strategies (eg, continue, augment, switch, step-down) leading to individualized treatment. a.k.a. dynamic treatment regimen/regime, adaptive treatment strategy, treatment policy, treatment algorithms Stepped care intervention models are a special case of adaptive interventions Almirall Adaptive Interventions October 2016 9 / 66

Example of an Adaptive Intervention in Autism Some Background First... Intervention efforts have overlooked older, school-aged children w/ASD who do not make signif. progress in spoken communication ≥ 50% of children with autism who received traditional interventions beginning at age 2 remained non-verbal at age 9 years of age. Failure to develop spoken language by age 5 increases likelihood of poor long-term prognosis of adaptive functioning Almirall Adaptive Interventions October 2016 10 / 66

Example of an Adaptive Intervention in Autism Some Background First... Intervention efforts have overlooked older, school-aged children w/ASD who do not make signif. progress in spoken communication ≥ 50% of children with autism who received traditional interventions beginning at age 2 remained non-verbal at age 9 years of age. Failure to develop spoken language by age 5 increases likelihood of poor long-term prognosis of adaptive functioning Evidence Based: Joint Attention, Symbolic Play, Engagement & Regulation (JASPER): two 1-hr sessions/week at clinic Promising: Augmentative, Alternative Communication (AAC) devices Almirall Adaptive Interventions October 2016 10 / 66

Example of an Adaptive Intervention in Autism Some Background First... Intervention efforts have overlooked older, school-aged children w/ASD who do not make signif. progress in spoken communication ≥ 50% of children with autism who received traditional interventions beginning at age 2 remained non-verbal at age 9 years of age. Failure to develop spoken language by age 5 increases likelihood of poor long-term prognosis of adaptive functioning Evidence Based: Joint Attention, Symbolic Play, Engagement & Regulation (JASPER): two 1-hr sessions/week at clinic Promising: Augmentative, Alternative Communication (AAC) devices However, AAC’s potentially costly & not all children may need it. Almirall Adaptive Interventions October 2016 10 / 66

Example of an Adaptive Intervention in Autism Some Background First... Intervention efforts have overlooked older, school-aged children w/ASD who do not make signif. progress in spoken communication ≥ 50% of children with autism who received traditional interventions beginning at age 2 remained non-verbal at age 9 years of age. Failure to develop spoken language by age 5 increases likelihood of poor long-term prognosis of adaptive functioning Evidence Based: Joint Attention, Symbolic Play, Engagement & Regulation (JASPER): two 1-hr sessions/week at clinic Promising: Augmentative, Alternative Communication (AAC) devices However, AAC’s potentially costly & not all children may need it. ◮ Research is limited. Mostly single-subject studies. No rigorous trials. Motivation for an adaptive intervention involving AAC’s in context of JASPER-EMT among older, minimally-verbal children with autism. Almirall Adaptive Interventions October 2016 10 / 66

‐ ‐ ‐ ‐ Example of an Adaptive Intervention in Autism For minimally verbal children with autism spectrum disorder Stage One JASP for 12 weeks; Stage Two At the end of week 12, determine early sign of response: ◮ IF slow responder: Augment JASP with AAC for 12 weeks; ◮ ELSE IF responder: Maintain JASP for 12 weeks. Almirall Adaptive Interventions October 2016 11 / 66

Example of an Adaptive Intervention in Autism For minimally verbal children with autism spectrum disorder Stage One JASP for 12 weeks; Stage Two At the end of week 12, determine early sign of response: ◮ IF slow responder: Augment JASP with AAC for 12 weeks; ◮ ELSE IF responder: Maintain JASP for 12 weeks. Continue: Responders JASP JASP Augment: Slow Responders JASP + AAC First ‐ stage Second ‐ stage End of Week 12 Treatment Treatment Responder Status (Weeks 1 ‐ 12) (Weeks 13 ‐ 24) Almirall Adaptive Interventions October 2016 11 / 66

How was response/slow-response defined? Percent change from baseline to week 12 was calculated for 7 variables: 7 variables: socially communicative utterances (SCU), percent SCU, mean length utterance, total word roots, words per minute, total comments, unique word combinations Fast Responder: if ≥ 25% change on 7 measures; Slower Responder: otherwise (this includes kids with no improvement, which is rare) Almirall Adaptive Interventions October 2016 12 / 66

But how did we come up with this adaptive intervention? Is it a good one? Do we really know how best to sequence and individualize treatments? Almirall Adaptive Interventions October 2016 13 / 66

Many Unanswered Questions when Building a High-Quality Adaptive Intervention. Is it better to provide AAC from the start? Who benefits from initial AAC versus who benefits from delayed AAC? For slow responders, what is the effect of providing the AAC vs intensifying JASP (not providing AAC)? Why begin treatment with JASP? Why not a high-quality course of core-principles of discrete trials training? Should we train parents following an initial successful course of treatment? If so, which families benefit most from this? Almirall Adaptive Interventions October 2016 14 / 66

Insufficient Empirical Evidence or Theories to Address such Questions In the past we have relied on Expert opinion Clinical expertise Piecing together an adaptive intevrention using results from separate RCTs Our group at Michigan has been developing novel randomized experimental design methods for answering many of these questions. Sequential Multiple Assignment Randomized Trials (SMARTs) address such questions empirically, using experimental design principles. Almirall Adaptive Interventions October 2016 15 / 66

Sequential Multiple Assignment Randomized Trials (SMART) Almirall Adaptive Interventions October 2016 16 / 66