Evidence map›Paper›PMID 37041653›Full record

Trial reportOrphanet journal of rare diseases2023

Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series data.

Jef Van den Eynde, Bhargava Chinni, Hilary Vernon, W Reid Thompson, Brittany Hornby, Shelby Kutty, Cedric Manlhiot

Open access · goldAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Orphanet journal of rare diseases, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
0.2field-weighted citation impact, top 43% 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

3 citing papers in PubMed, 1 synthesis or guideline pooled it, 1 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
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 2 countries.

Jef Van den EyndeThe Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Department of Pediatrics, Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
Bhargava ChinniThe Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Department of Pediatrics, Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
Hilary VernonThe Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Department of Pediatrics, Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
W Reid ThompsonThe Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Department of Pediatrics, Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
Brittany HornbyDepartment of Physical Therapy, Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD, USA.
Shelby KuttyThe Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Department of Pediatrics, Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
Cedric ManlhiotThe Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Department of Pediatrics, Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA. cmanlhi1@jhmi.edu.ORCID 0000-0001-9419-7681
Johns Hopkins University · USKennedy Krieger Institute · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundBarth syndrome (BTHS) is a rare genetic disease that is characterized by cardiomyopathy, skeletal myopathy, neutropenia, and growth abnormalities and often leads to death in childhood. Recently, elamipretide has been tested as a potential first disease-modifying drug. This study aimed to identify patients with BTHS who may respond to elamipretide, based on continuous physiological measurements acquired through wearable devices.

resultsData from a randomized, double-blind, placebo-controlled crossover trial of 12 patients with BTHS were used, including physiological time series data measured using a wearable device (heart rate, respiratory rate, activity, and posture) and functional scores. The latter included the 6-minute walk test (6MWT), Patient-Reported Outcomes Measurement Information System (PROMIS) fatigue score, SWAY Balance Mobile Application score (SWAY balance score), BTHS Symptom Assessment (BTHS-SA) Total Fatigue score, muscle strength by handheld dynamometry, 5 times sit-and-stand test (5XSST), and monolysocardiolipin to cardiolipin ratio (MLCL:CL). Groups were created through median split of the functional scores into "highest score" and "lowest score", and "best response to elamipretide" and "worst response to elamipretide". Agglomerative hierarchical clustering (AHC) models were implemented to assess whether physiological data could classify patients according to functional status and distinguish non-responders from responders to elamipretide. AHC models clustered patients according to their functional status with accuracies of 60-93%, with the greatest accuracies for 6MWT (93%), PROMIS (87%), and SWAY balance score (80%). Another set of AHC models clustered patients with respect to their response to treatment with elamipretide with perfect accuracy (all 100%).

conclusionsIn this proof-of-concept study, we demonstrated that continuously acquired physiological measurements from wearable devices can be used to predict functional status and response to treatment among patients with BTHS.

Indexed as

Barth SyndromeCardiolipinsFatigueHumansOligopeptidesTime Factorsarginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamideCardiolipinsOligopeptidesBarth syndromeDigital healthHierarchical clustering

Identifiers

PMID37041653
PMCPMC10088720
OpenAlexW4364365286

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.