Evidence map›Paper›PMID 30282375›Full record

Trial reportJournal of neuromuscular diseases2018

Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular Dystroph.

Gunnar M Buyse, Christian Rummey, Thomas Meier, Mika Leinonen, Thomas Voit, Craig M McDonald, Oscar H Mayer

Open access · bronzeAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Journal of neuromuscular diseases, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 2 pooled it
0.7field-weighted citation impact, top 29% 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

9 citing papers in PubMed, 2 syntheses or guidelines pooled it, 14 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Article
  4. Review
  5. Review
  6. Review
  7. A comparison of clinic and home spirometry as longtudinal outcomes in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022
    Article
  8. Observational
  9. 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 4 institutions in 3 countries.

Gunnar M BuyseUniversity Hospitals Leuven, Leuven, Belgium.
Christian RummeyClinical Data Science, Basel, Switzerland.
Thomas MeierSanthera Pharmaceuticals, Pratteln, Switzerland.
Mika LeinonenSanthera Pharmaceuticals, Pratteln, Switzerland.
Thomas VoitUCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.
Craig M McDonaldUniversity of California Davis Medical Center, Sacramento, USA.
Oscar H MayerThe Children's Hospital of Philadelphia, Philadelphia, USA.
Santhera Pharmaceuticals (Switzerland) · CHChildren's Hospital of Philadelphia · USGreat Ormond Street Hospital · GBUniversity of California Davis Medical Center · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundLoss of pulmonary function is a main cause of early morbidity and mortality in patients with Duchenne muscular dystrophy (DMD). Standard of care guidelines recommend regular assessment of pulmonary function by hospital-based spirometry to detect onset and monitor progression of pulmonary function decline.

objectiveTo assess the feasibility of home-based monitoring of pulmonary function by a hand-held device (HHD) in adolescent and adult patients with DMD over a period of 12 months.

methodsIn the phase III randomized placebo-controlled DELOS trial in 10-18 year old DMD patients, peak expiratory flow (PEF) measurements were collected weekly at home by the patient (assisted by parent/caregiver) using a peak flow meter HHD. Adherence to the use of the HHD was assessed and 12-month changes in PEF as percent of predicted (PEF% p) for the idebenone (N = 31) and the placebo treatment groups (N = 33) from HHD-derived data were compared to results from hospital-based spirometry.

resultsA total of 2689 individual HHD assessments were analysed. Overall adherence to the use of the HHD over the course of the 12-month study duration was good (75.9%, SD 21.5%) and PEF% p data obtained at the same day by HHD and standard spirometry correlated well (Spearman's rho 0.80; p < 0.001). Several analysis methods of HHD-derived data for PEF% p consistently demonstrate that idebenone treatment slowed the decline in PEF% p compared to placebo, which supports the statistically significant difference in favour of idebenone for PEF% p measured by standard spirometry.

conclusionsThis study demonstrates that home-based monitoring of pulmonary function in adolescent patients with DMD using a HHD is feasible, provides reliable data compared to hospital-based spirometry and is therefore suitable for use in clinical practice and for clinical trials.

Indexed as

AdolescentAntioxidantsChildHumansMaleMuscular Dystrophy, DuchennePeak Expiratory Flow RateRespiratory Function TestsSelf CareUbiquinoneAntioxidantsidebenoneUbiquinoneDuchenne muscular dystrophyidebenonepeak expiratory flowPulmonary functionrespiration

Identifiers

PMID30282375
PMCPMC6218142
OpenAlexW2892601756

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.