Evidence map›Paper›PMID 40956566›Full record

Observational studyJournal of endocrinological investigation2026

Endocrinological complications of Duchenne muscular dystrophy and their subjective burden: observational study evaluating growth, puberty, and bone health.

Marie Rohlenova, Jana Haberlova, Petra Holotova, Marketa Kumhera, Lucie Simkova, Barbora Lauerova, Iveta Svabova, Ondrej Soucek

Abstract readObservational Study
In one paragraph

Observational study in Journal of endocrinological investigation, 2026. 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
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.

  1. 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

8 authors.

Marie RohlenovaDepartment of Pediatric Neurology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic. marie.rohlenova@fnmotol.cz.
Jana HaberlovaDepartment of Pediatric Neurology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic.
Petra HolotovaDepartment of Pediatric Neurology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic.
Marketa KumheraDepartment of Pediatric Neurology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic.
Lucie SimkovaDepartment of Pediatric Neurology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic.
Barbora LauerovaDepartment of Pediatric Neurology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic.
Iveta SvabovaDepartment of Rehabilitation and Sports Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic.
Ondrej SoucekDepartment of Pediatrics, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, 150 06, V Úvalu 84, Czech Republic.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeDuchenne muscular dystrophy (DMD) and its treatment by glucocorticoids are associated with secondary osteoporosis, short stature, and delayed puberty. We aimed at exploring the prevalence and subjective burden of these endocrinological complications after the implementation of recommended care protocols.

methodsA prospective cross-sectional medical report review of boys with DMD followed at the largest pediatric neuromuscular reference center. An online questionnaire survey was part of the study.

resultsWe included 35 boys with DMD, aged 5.7-19.7 years, most of them on long-term daily glucocorticoid therapy (91%). Vertebral fractures were present in 50% of boys, short stature in 74%, and pubertal delay in 56% of boys. The glucocorticoid treatment duration and cumulative doses were associated with a higher prevalence of short stature, but not with the presence of vertebral fractures or pubertal delay. Areal bone mineral density assessed by densitometry only poorly identified patients with osteoporosis, compared to clear evidence of vertebral fractures by lateral spine radiograph. The boys were most concerned about the risk of fractures. Those in the pubertal age, however, were troubled also by their childish looks. The boys tolerated the surveillance protocols and treatment of complications very well.

conclusionsVertebral fractures, short stature, and delayed puberty are very frequent among glucocorticoid-treated boys with DMD. Lateral spine radiograph is a crucial means for bone health assessment, with an even larger yield than densitometry. A questionnaire survey identifies patient needs and improves complex health care.

Indexed as

Endocrine System DiseasesGrowth DisordersMuscular Dystrophy, DuchenneOsteoporosisPubertyPuberty, DelayedSpinal FracturesAdolescentBone DensityChildChild, PreschoolCross-Sectional StudiesFollow-Up StudiesGlucocorticoidsHumansMaleGlucocorticoidsDelayed pubertyDuchenne muscular dystrophyFracturesOsteoporosisQuestionnaireShort stature

Identifiers

PMID40956566
PMCPMC12847132

What OpenQuestion holds

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LicenceCC BY
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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.