Muscle & nerve · 2026
Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD Consortium.
СтатьяНазвание и аннотация: Перевод в очереди
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Wood Claire L, Babalola Funmbi, Benjamin Robert W, Lam Carol, McAdam Laura, Nicolau Stefan, Sbrocchi Anne Marie, Scavina Mena, Sorbara Julia C, Ahmet Alexandra, Apkon Susan, Bharill Sonum, Fiscaletti Melissa, Kinnett Kathi, McMillan Hugh J, Merchant Nadia, Nasomyont Nat, Molina Maria Fernanda Ochoa, Renthal Nora E, Smith Pamela, Surampudi Prasanth N, Tamaroff Jaclyn G, Tian Cuixia, Ward Leanne M, Weber David R, Wong Sze Choong Jarod, Crane Janet L, Rutter Meilan M, OPTIMIZE DMD Consortium
Аннотация
INTRODUCTION/AIM: Optimizing 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.
METHODS: A 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.
RESULTS: Survey 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.
METHODS: A 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.
RESULTS: Survey 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.