Evidence map›Paper›PMID 37071655›Full record

Trial reportPloS one2023

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

Ethan D Borre, Evan Myers, Marianne Hamilton Lopez, Joanne Kurtzberg, Beth Shaz, Jesse Troy, Gillian D Sanders Schmidler

Open access · goldAbstract readRandomized Controlled Trial
In one paragraph

Trial report in PloS one, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact, top 96% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 0 citations in OpenAlex.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 2 institutions in 1 country.

Ethan D BorreDepartment of Population Health Sciences, Duke University School of Medicine, Durham, NC, United States of America.
Evan MyersDivision of Women's Community and Population Health, Department of Obstetrics & Gynecology, Duke University School of Medicine, Durham, NC, United States of America.
Marianne Hamilton LopezDuke-Margolis Center for Health Policy, Duke University, Durham, NC, United States of America.
Joanne KurtzbergMarcus Center for Cellular Cures, Duke University Medical Center, Durham, NC, United States of America.
Beth ShazMarcus Center for Cellular Cures, Duke University Medical Center, Durham, NC, United States of America.ORCID 0000-0002-2270-4821
Jesse TroyMarcus Center for Cellular Cures, Duke University Medical Center, Durham, NC, United States of America.
Gillian D Sanders SchmidlerDepartment of Population Health Sciences, Duke University School of Medicine, Durham, NC, United States of America.ORCID 0000-0003-0114-7568
Duke University · USDuke Medical Center · US

Funding

Economic Evaluation and Research Prioritization of Adult Hearing Screening in the USF30DC019846 · NIDCD · DUKE UNIVERSITY · PI BORRE, ETHAN D · 2021 to 2022
$88k
NIDCD NIH HHS F30 DC019846
6 · The paper itself

Abstract

objectiveTo model the long-term clinical and economic outcomes of potential cord blood therapy in autism spectrum disorder (ASD). STUDY

designMarkov 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.

resultsWe 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<$15,000 and efficacies ≥2.0. Five-year healthcare payer projected budgetary outlays at a $15,000 CB cost were $3.847B.

conclusionsA modestly effective intervention designed to improve adaptive behavior in autism can be cost-effective under certain circumstances. Intervention cost and efficacy most affected the cost-effectiveness results and should be targeted to increase economic efficiency.

Indexed as

Autism Spectrum DisorderAdolescentAdultChildChild, PreschoolCost-Benefit AnalysisFetal BloodHumansLife ExpectancyQuality-Adjusted Life Years

Identifiers

PMID37071655
PMCPMC10112778
OpenAlexW4366240679

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.