Developing an Innovative Decision Support Tool for Pediatric Neuromuscular Scoliosis

Developing an Innovative Decision Support Tool for Pediatric Neuromuscular Scoliosis - Aims 2 and 3

The goal of this pilot hybrid type I efficacy/implementation trial is to assess a newly developed decision support tool patients, parents, and providers to use during surgical treatment decision making for neuromuscular scoliosis (NMS). Results from this pilot will inform the design of a future larger effectiveness trial of the decision support tool.

Participants will either receive usual care or receive the decision support tool. Researchers will assess the decision made, decision quality, individual affective, cognitive, and behavioral effects, and feasibility and acceptability of tool use. They will also collect potential barriers and facilitators to implementation and feedback about the tool and study design to maximize likelihood of successful deployment of the tool into clinical practice and inform the design of a future trial. The outcomes measures will be used to inform potential effect size estimates to inform a future trial.

Study Overview

• Status: Recruiting
• Conditions: Decision Support Systems, Clinical; Multiple Chronic Conditions; Shared Decision Making; Decision Aids; Neuromuscular Scoliosis; Children With Medical Complexity (CMC)
• Intervention / Treatment: Behavioral: Decision support tool

Detailed Description

Neuromuscular scoliosis (NMS) can result in severe disability for children. Non-operative management including bracing and physical therapy minimally slows scoliosis progression, but operative management with posterior spinal fusion (PSF) carries high risks of morbidity and mortality in part due to the multiple comorbid conditions seen in children with NMS. Decisions like PSF that have no clear best treatment option are best served by shared decision making (SDM). SDM is a collaborative process where patients, parents, and providers share their knowledge, preferences, and values to reach treatment plan agreement. Our prior qualitative study creating a framework of SDM for children with multiple comorbid conditions like children with NMS found that parents face immense decision related uncertainty that often goes unacknowledged by providers and that parents and providers struggle with identifying and communicating family preferences and values that are important for treatment decision making. Our prior work has identified optimal methods to communicate uncertainty and identified the preferences and values parents of children with NMS have when it comes to treatment decision making for NMS. This study will pilot test a decision support tool that contains these elements.

• Study Type: Interventional
• Enrollment (Estimated): 110
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Michelle Wilcox; Phone Number: 801-581-6410; Email: michelle.o.wilcox@hsc.utah.edu
• Study Contact Backup: Name: Angela Zhu, BS; Phone Number: 801-662-3675; Email: angela.zhu@hsc.utah.edu

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Recruiting
      • Children's Hospital Los Angeles
      • Contact: Tiffany Phan; Email: tphan@chla.usc.edu
      • Contact: Amberly Thai; Email: amthai@chla.usc.edu
      • Principal Investigator: Tamara Simon, MD, MSPH
      • Sub-Investigator: Lindsay Andras, MD
  • Utah
    • Salt Lake City, Utah, United States, 84114
      • Recruiting
      • University of Utah
      • Contact: Angela Zhu; Phone Number: 8016623675; Email: angela.zhu@hsc.utah.edu
      • Principal Investigator: Jody L Lin, MD, MS

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

• Parent-child dyads of children with neuromuscular scoliosis who speak English and Spanish.
• Child is between ages 8-21 years of age and they are coming into the pediatric orthopaedic surgery clinic for consultation about potential surgery for NMS.
• NMS is defined as having neurologic impairment (NI) and scoliosis using relevant ICD-9 or ICD-10 codes from Feudtner, et al. 2014 or Berry, et al. 2012. or a qualifying diagnosis per the Pediatric Spine Study Group definition of NMS.
• All pediatric orthopaedic surgeons and neurosurgeons who treat neuromuscular scoliosis at our study sites will be eligible participants.

Exclusion criteria:

• Families whose child with NMS is less than 8 years of age at time of orthopaedic consultation because surgery at a younger age usually indicates an atypical case.
• Children with the diagnosis of Duchenne's or Becker's muscular dystrophy due to potential disease modifying therapies that may alter curve progression.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Health Services Research
• Allocation: Non-Randomized
• Interventional Model: Crossover Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: Usual care; Participants receive usual care
Experimental: Decision support tool; Participants receive the novel decision support tool	Behavioral: Decision support tool; Decision support tool developed for patients, parents, and providers of children with neuromuscular scoliosis

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Intention for NMS treatment	Parent degree of decision preference (1: no surgery-9: surgery)	Immediately after clinic visit

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Intention for NMS treatment	Parent's desired treatment option: Binary (1- surgery, 0- no surgery)	Immediately after clinic visit
Intention for not delaying treatment	For parents who select surgery, the intention of treatment timing.	Immediately after clinic visit
Knowledge questions	Parent's response to five decision-related knowledge questions, scored as total answered correctly	Immediately after clinic visit
Acceptability of intervention	Measured quantitatively by the validated Acceptability of Intervention Measure for caregivers in the intervention group. The scale consists of 4 items rated on a 5-point Likert scale. A mean score is created with a higher score indicating higher acceptability.	Immediately after clinic visit
Physician trustworthiness	The Dugan Physician Trust Scale is a validated 5-item survey measure to assess the participant's trust in their physician. Each item is rated on a 5-point Likert scale and one item is reverse coded before all items are summed to create a composite score. Higher scores indicate stronger trust in the physician.	Immediately after clinic visit
Feasibility of intervention	Measured by the validated Feasibility of Intervention Measure. The scale consists of 4 items rated on a 5-point Likert scale. A mean score is created with a higher score indicating higher acceptability.	Immediately after clinic visit
Risk perception	2 unique items on perception of risk related to decision, scored individually. Each item is rated on a 5-point Likert scale. The items will be scored individually with higher responses indicating higher perceived decision-related risk.	Immediately after clinic visit
Decision conflict	Decisional conflict scale and 2 unique decision conflict items (individual items scored individually). Each item is rated on a 5-point Likert scale. The items will be scored individually with higher responses indicating higher decisional conflict.	Immediately after clinic visit
Decision readiness	Preparation for Decision Making (PrepDM): The PrepDM scale is a validated 10-item survey measure that evaluates the participant's perception of how helpful their clinic visit was in preparing them to make the decision. Each item is rated on a 5-point Likert scale, and scores are standardized to a 0-100 scale, with higher scores indicating greater perceived readiness for decision-making.	Immediately after clinic visit
Acceptability of Intervention- Qualitative	Qualitatively assessed via focus groups and individual interviews for caregivers in intervention group and provider participants.	Within 3 months of clinic visit (for parents) and at end of study, approximately within 18 months of consent (for providers).
Worry	Consists of 3 unique items to assess the participant's level of worry related to decision making. Each item is rated on a 5-point Likert scale. The items will be scored individually with higher responses indicating higher levels of decision-related worry.	Immediately after clinic visit
Quality of shared decision making	Quantitatively via the 9-item SDM-Q-9 (parent) and SDM-Q-Doc (provider) surveys. Each item is rated on a 6-point Likert scale. The total score is standardized to a scale from 0 to 100, with higher scores indicating greater perceived involvement in decision-making.	Immediately after clinic visit
Decisional self-efficacy	The Decisional Self-Efficacy Scale is a validated 11-item survey measure that evaluates the participant's ability to make informed health-related decisions. Items are rated on a 5-point Likert scale and scores are standardized to a 0-100 scale, with higher scores reflecting greater decisional self-efficacy.	Immediately after clinic visit
preference concordance for parents	Compare parent self-rated top three values/preferences driving decision making with decision they chose for consistency of decision with values/preferences. Will be scored based on number of values (out of 3) that match the treatment option selected.	Immediately after clinic visit
Appropriateness of intervention	Measured quantitatively by the validated Appropriateness of Intervention Measure. The scale consists of 4 items rated on a 5-point Likert scale. A mean score is created with a higher score indicating higher acceptability.	Immediately after clinic visit
Preference concordance between parents and providers	Correlation between top three parent preferences and values for decision identified by parents and by providers, calculated via a kappa score.	Immediately after clinic visit
Feasibility of Intervention- Qualitative	Qualitatively assessed via focus groups and individual interviews for caregivers in intervention group and provider participants.	Within 3 months of clinic visit (for parents) and at end of study, approximately within 18 months of consent (for providers).
Appropriateness of Intervention- Qualitative	Qualitatively assessed via focus groups and individual interviews for caregivers in intervention group and provider participants.	Within 3 months of clinic visit (for parents) and at end of study, approximately within 18 months of consent (for providers).
Quality of shared decision making- qualitative	Using the DEEP-SDM coding scheme on video- and audio-recorded clinical encounters	During clinic visit

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Barriers and facilitators to implementation	Qualitative analysis of interviews and focus groups from intervention group participants using the CFIR	Within 3 months of clinic visit (for parents) and at end of study, approximately within 18 months of consent (for providers).
Quality of interpretation	5-item survey questions based on Napoles 2015 and qualitative analysis of recorded clinic visits to assess quality of interpretation. The first item assesses the type and need for interpretation, with participants selecting from 7 categories. If an interpreter was used, participants will then answer 4 additional items about the quality of interpretation for the visit. Each of these subsequent items is rated on a 5-point Likert scale (1-poor to 5-excellent). A mean composite outcome of the completed items is used to calculate overall quality.	immediately after clinic visit
Usability of intervention	System Usability Scale (SUS): The SUS is a validated 10-item survey measure that evaluates the perceived usability of the intervention. Items are rated on a 5-point Likert scale and scores are calculated by converting responses to a 0-100 scale, with higher scores indicating better usability. Only parent participants assigned to the intervention group will answer this measure.	Immediately after clinic visit
Mental load of intervention	NASA Task Load Index (NASA-TLX): The NASA-TLX is a validated measure used to assess the perceived mental workload of using the intervention. It consists of 6-items to measure 6 core dimensions: mental demand, physical demand, temporal demand, performance, effort, and frustration. Each dimension is rated on a scale from 0 to 100, and scores are combined to produce an overall workload score. Higher scores indicate greater perceived workload. Only parent participants assigned to the intervention group will answer this measure.	Immediately after clinic visit
Tool use	Metadata on tool interaction, inputs, and use	End of study, approximately 18 months after first enrollment.
Severe pneumonia	Chart review of medical records to count number of admissions for pneumonia.	Within 18 months of clinic visit
Readability	Consists of 2 unique items to assess how clearly the intervention is presented. Each item is rated on a 5-point Likert scale. The items will be scored individually. Only parent participants assigned to the intervention group will answer these items.	Immediately after the clinic visit
provider preferences and values for decision making	provider free response of top three reasons for decisiion that was made	immediately after clinic visit
parent preferences and values	selection of top three parent preferences and values for decision making from a list of options including a free response "other" field; asked to parent/patient participants and provider participants	immediately after visit
Quality of interpretation	Qualitative analysis of quality of interpretation captured on video-recordings of clinic visits.	During clinic visit
Decision adherence	Chart review of medical records to assess adherence to decision made.	Within 18 months after clinic visit.
Pain symptoms	Chart review of patient record for changes in pain symptoms related to scoliosis. Options include: worsened, unchanged, improved, not mentioned.	Within 18 months of clinic visit
Surgical outcomes	Chart review of medical records to assess for presence surgical outcomes: minor complications, major complications.	Within 18 months of clinic visit
Sitting comfort	Chart review of patient record for changes in sitting comfort symptoms related to scoliosis. Options include: worsened, unchanged, improved, not mentioned.	Within 18 months of clinic visit
ICU length of stay	For those who undergo surgery, the days the patient spent in the ICU after surgery	Within 18 months of clinic visit
Total length of stay	For those who undergo surgery, the days the patient spent in the the hospital after surgery	Within 18 months of clinic visit
Length of ventilation	For those who undergo surgery, the days the patient spent on mechanical ventilation (if not ventilated at baseline) or days spent on higher than normal mechanical ventilation settings (if ventilated at baseline).	Within 18 months of clinic visit
Pneumonia hospital days	Chart review of medical records to count number of days admitted for pneumonia.	Within 18 months of clinic visit

Collaborators and Investigators

• Sponsor: University of Utah
• Collaborators: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Investigators: Principal Investigator: Jody Lin, MD, MS, University of Utah

Study record dates

Study Major Dates

• Study Start (Actual): October 21, 2025
• Primary Completion (Estimated): January 31, 2027
• Study Completion (Estimated): March 31, 2027

Study Registration Dates

• First Submitted: August 26, 2025
• First Submitted That Met QC Criteria: September 3, 2025
• First Posted (Estimated): September 11, 2025

Study Record Updates

• Last Update Posted (Actual): December 5, 2025
• Last Update Submitted That Met QC Criteria: December 4, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: shared decision making; children with medical complexity; decision support tool; values clarification; uncertainty communication
• Additional Relevant MeSH Terms: Pathologic Processes; Chronic Disease; Disease Attributes; Pathological Conditions, Signs and Symptoms; Multiple Chronic Conditions
• Other Study ID Numbers: IRB_00169930; 1K23AR079778 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No