Evidence map›Paper›PMID 34702655›Full record

ArticleNeuromuscular disorders : NMD2021

The impact of testosterone therapy on quality of life in adolescents with Duchenne muscular dystrophy.

C L Wood, J Page, J Foggin, M Guglieri, V Straub, T D Cheetham

Open access · hybridAbstract read
In one paragraph

Article in Neuromuscular disorders : NMD, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
0.6field-weighted citation impact, top 33% 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

11 citing papers in PubMed, 16 citations in OpenAlex.

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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

6 authors at 2 institutions in 1 country.

C L WoodTranslational and Clinical Research Institute, Newcastle University UK; Department of Paediatric Endocrinology, Newcastle upon Tyne Hospitals NHS Foundation Trust UK. Electronic address: Claire.wood@ncl.ac.uk.
J PageJohn Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust UK.
J FogginDepartment of Paediatric Endocrinology, Newcastle upon Tyne Hospitals NHS Foundation Trust UK.
M GuglieriTranslational and Clinical Research Institute, Newcastle University UK; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust UK.
V StraubTranslational and Clinical Research Institute, Newcastle University UK; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust UK.
T D CheethamTranslational and Clinical Research Institute, Newcastle University UK; Department of Paediatric Endocrinology, Newcastle upon Tyne Hospitals NHS Foundation Trust UK.
Newcastle upon Tyne Hospitals NHS Foundation Trust · GBNIHR Newcastle Biomedical Research Centre · GB

Funding

Medical Research Council MR/N020588/1
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy in childhood. It is associated with progressive muscle function decline and premature death. Long-term oral glucocorticoid use slows muscle weakness but is associated with several side effects including delayed puberty. This study assessed the impact of a 2-year incremental intramuscular testosterone regimen on quality of life (QoL) in a cohort of 15 adolescents with DMD. The Pediatric Quality of Life Inventory (PedsQL) Neuromuscular module was used to assess QoL and was completed by parent-child dyads. Semi-structured interviews were carried out to understand patient views on testosterone therapy. QoL scores increased in 10 of the 15 participants during treatment, with a mean total PedsQL score of 74.6 pre-treatment v 80.2 post treatment (p = 0.04). This was supported by comments in the semi-structured interviews. Parent-reported PedsQL scores were lower than their child's post treatment (p = 0.007). Testosterone therapy for pubertal induction was associated with an improvement in QoL and the observed physical changes during puberty played an important role. Low self-esteem was also a prevailing theme. This data supports the inclusion of testosterone therapy for pubertal induction as a Standard of Care.

Indexed as

Patient Reported Outcome MeasuresQuality of LifeAdolescentAndrogensChildDwarfismHumansMaleMuscular Dystrophy, DuchenneParentsPubertySelf ConceptTestosteroneAndrogensTestosteroneDuchenne muscular dystrophy: PubertyQuality of lifeShort statureTestosterone

Identifiers

PMID34702655
PMCPMC8721209
OpenAlexW3203063176

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.