Evidence map›Paper›PMID 42178495›Full record

ArticleMuscle & nerve2026

Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD Consortium.

Claire L Wood, Funmbi Babalola, Robert W Benjamin, Carol Lam, Laura McAdam, Stefan Nicolau, Anne Marie Sbrocchi, Mena Scavina, Julia C Sorbara, Alexandra Ahmet and 19 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Muscle & nerve, 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

29 authors.

Claire L WoodDepartment of Paediatric Endocrinology, Great North Children's Hospital, Newcastle upon Tyne, UK.
Funmbi BabalolaDepartment of Pediatrics, Division of Endocrinology, Western University, Children's Hospital, London Health Sciences Centre, Ontario, Canada.
Robert W BenjaminDepartment of Pediatrics, Division of Endocrinology, Duke University Medical Center, Durham, North Carolina, USA.
Carol LamDepartment of Pediatrics, University of Toronto, Canada; Division of Endocrinology, Hospital for Sick Children, Toronto, Canada.
Laura McAdamBloorview Research Institute, Holland Bloorview Kids Rehabilitation Hospital, Toronto, Canada.
Stefan NicolauCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0001-5631-0559
Anne Marie SbrocchiDivision of Pediatrics and Metabolism, Montreal Children's Hospital, McGill University Health Center, Montreal, Canada.
Mena ScavinaParent Project Muscular Dystrophy, Washington, D.C, USA.
Julia C SorbaraDepartment of Pediatrics, University of Toronto, Canada; Division of Endocrinology, Hospital for Sick Children, Toronto, Canada.
Alexandra AhmetDivision of Endocrinology, Children's Hospital of Eastern Ontario (CHEO); Department of Pediatrics, University of Ottawa, Ottawa, Canada.
Susan ApkonDepartment of Physical Medicine and Rehabilitation, Children's Hospital Colorado and University of Colorado School of Medicine, Aurora, Colorado, USA.
Sonum BharillDivision of Endocrinology, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.ORCID https://orcid.org/0000-0003-2933-5579
Melissa FiscalettiDepartment of Pediatrics, Sainte Justine University Hospital, Université de Montréal, Montreal, Canada.
Kathi KinnettParent Project Muscular Dystrophy, Washington, D.C, USA.
Hugh J McMillanDivision of Neurology, Children's Hospital of Eastern Ontario (CHEO); Department of Pediatrics, University of Ottawa, Ottawa, Canada.
Nadia MerchantDivision of Pediatric Endocrinology, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Nat NasomyontDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center; College of Medicine, University of Cincinnati, Cincinnati, Ohio, USA.
Maria Fernanda Ochoa MolinaEndocrinology Unit, Division of Pediatrics, School of Medicine, Pontifical Catholic University of Chile, Santiago, CL, Chile.
Nora E RenthalDepartment of Pediatrics, Division of Endocrinology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0002-8186-4462
Pamela SmithDivision of Endocrinology, Phoenix Children's Hospital, Phoenix, Arizona, USA.
Prasanth N SurampudiDepartment of Internal Medicine, Division of Endocrinology, University of California, Davis Medical Center, Sacramento, California, USA.
Jaclyn G TamaroffDivision of Endocrinology and Diabetes, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, Tennessee, USA.ORCID https://orcid.org/0000-0002-3630-9937
Cuixia TianDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center; College of Medicine, University of Cincinnati, Cincinnati, Ohio, USA.
Leanne M WardDivision of Endocrinology, Children's Hospital of Eastern Ontario (CHEO); Department of Pediatrics, University of Ottawa, Ottawa, Canada.
David R WeberDivision of Endocrinology and Diabetes, the Children's Hospital of Philadelphia; Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA.ORCID https://orcid.org/0000-0001-6895-2372
Sze Choong Jarod WongDepartment of Pediatric Endocrinology, Royal Hospital for Children; Human Nutrition, School of Medicine, Dentistry and Nursing, University of Glasgow, Glasgow, Scotland, UK.ORCID https://orcid.org/0000-0003-2845-6555
Janet L CraneDepartment of Pediatrics, Division of Endocrinology and Department of Orthopedic Surgery, Center for Musculoskeletal Research, The Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Meilan M RutterDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center; College of Medicine, University of Cincinnati, Cincinnati, Ohio, USA.
OPTIMIZE DMD Consortium

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

INTRODUCTION/

aimOptimizing Management of Endocrine Complications in Duchenne Muscular Dystrophy (OPTIMIZE DMD) is an international consortium of clinicians created to advance endocrine and bone clinical care in DMD. The aim of this study was to better understand current views and practices regarding investigation and management of growth and puberty concerns in individuals with DMD, relative to the 2018 Care Considerations and to inform updated guidance around endocrine care.

methodsA survey was created and sent to 47 OPTIMIZE DMD Consortium members and allied clinicians between September and November 2024. Areas surveyed included evaluation of growth, puberty, and arrested puberty/hypogonadism, and related management including use of vamorolone, growth hormone and testosterone.

resultsSurvey responses were received from 37 clinicians (79%). Most individuals were referred to endocrinology for growth and puberty concerns, with referral patterns contingent upon the endocrinology/multidisciplinary clinic model. Management discussions for growth concerns involved continued monitoring (95% of clinicians), glucocorticoid adjustment (54%) including vamorolone (35%), and growth hormone (41%). For pubertal delay, 88% of endocrinologists offered testosterone for pubertal induction, most commonly intramuscular 4-weekly testosterone injections. Most endocrinologists (92%) offered testosterone supplementation for arrested puberty/hypogonadism, some (24%) on a case-by-case basis. Delivery routes were more varied, with subcutaneous injections prescribed by 52%. DISCUSSION: Compared with the 2018 Care Considerations, monitoring practices for growth and puberty remain largely consistent, while variability persists in management approaches. These findings provide important insights to inform future guidance and identify priorities for further education and research in endocrine management for individuals with DMD.

Indexed as

Growth DisordersMuscular Dystrophy, DuchennePubertyAdolescentChildHuman Growth HormoneHumansHypogonadismMalePuberty, DelayedSurveys and QuestionnairesTestosteroneHuman Growth HormoneTestosteroneDuchenneendocrinegrowthhypogonadismpuberty

Identifiers

PMID42178495
PMCPMC13332569

What OpenQuestion holds

Textmetadata
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.