The potential cost-effectiveness of novel cord blood therapies in children with autism spectrum disorder

To model the long-term clinical and economic outcomes of potential cord blood therapy in autism spectrum disorder (ASD). Markov microsimulation of ASD over the lifespan was used to compare two strategies: 1) standard of care (SOC), including behavioral and educational interventions, and 2) novel cor...

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Bibliographic Details
Published in:PloS one 2023-04, Vol.18 (4), p.e0282906-e0282906
Main Authors: Borre, Ethan D, Myers, Evan, Hamilton Lopez, Marianne, Kurtzberg, Joanne, Shaz, Beth, Troy, Jesse, Sanders Schmidler, Gillian D
Format: Article
Language:English
Subjects:
Adolescent
Adult
Age
Alternative approaches
Autism
Autism Spectrum Disorder - therapy
Behavior
Biology and Life Sciences
Blood
Care and treatment
Child
Child, Preschool
Children
Clinical trials
Communication
Cord blood
Cost analysis
Cost benefit analysis
Costs
Effectiveness
Efficacy
Efficiency
Evaluation
Fetal Blood
Health services
Humans
Impact analysis
Intellectual disabilities
Intervention
Life Expectancy
Life span
Medical care, Cost of
Medicine and Health Sciences
Mortality
Natural history
Parameter sensitivity
Patient outcomes
People and Places
Pervasive developmental disorders
Quality of life
Quality-Adjusted Life Years
Sensitivity analysis
Social Sciences
Socialization
Standard of care
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Summary:To model the long-term clinical and economic outcomes of potential cord blood therapy in autism spectrum disorder (ASD). Markov microsimulation of ASD over the lifespan was used to compare two strategies: 1) standard of care (SOC), including behavioral and educational interventions, and 2) novel cord blood (CB) intervention in addition to SOC. Input data reflecting behavioral outcomes included baseline Vineland Adaptive Behavior Scale (VABS-3), monthly VABS-3 changes, and CB intervention efficacy on adaptive behavior based on a randomized, placebo-controlled trial (DukeACT). Quality-adjusted life-years (QALYs) were correlated to VABS-3. Costs for children with ASD ($15,791, ages 2-17 years) and adults with ASD ($56,559, ages 18+ years), and the CB intervention (range $15,000-45,000) were incorporated. Alternative CB efficacy and costs were explored. We compared model-projected results to published data on life-expectancy, mean VABS-3 changes, and lifetime costs. Undiscounted lifetime QALYs in the SOC and CB strategies were 40.75 and 40.91. Discounted lifetime costs in the SOC strategy were $1,014,000, and for CB ranged from $1,021,000-$1,058,000 with CB intervention cost ($8,000-$45,000). At $15,000 cost, CB was borderline cost-effective (ICER = $105,000/QALY). In one-way sensitivity analysis, CB cost and efficacy were the most influential parameters on CB ICER. CB intervention was cost-effective at costs
ISSN:1932-6203
1932-6203
DOI:10.1371/journal.pone.0282906