Bone density and bone health alteration in boys with Duchenne Muscular Dystrophy: a prospective observational study.


Journal

Journal of pediatric endocrinology & metabolism : JPEM
ISSN: 2191-0251
Titre abrégé: J Pediatr Endocrinol Metab
Pays: Germany
ID NLM: 9508900

Informations de publication

Date de publication:
26 May 2021
Historique:
received: 19 07 2020
accepted: 14 01 2021
pubmed: 11 4 2021
medline: 24 11 2021
entrez: 10 4 2021
Statut: epublish

Résumé

Boys with Duchenne Muscular Dystrophy (DMD) are at increased risk for compromised bone health, manifesting as low-impact trauma long bone fractures and vertebral compression fractures. In a prospective observational study, we studied bone health parameters in North Indian boys with DMD. We consecutively enrolled ambulatory boys with DMD on glucocorticoid therapy. Bone health was evaluated with X-ray spine, Dual-energy X-ray absorptiometry (DXA), serum calcium, vitamin D3 (25[OH]D), 1,25-dihyroxyvitamin D3 (1,25[OH] A total of 76 boys with DMD were enrolled. The median age was 8.5 (interquartile range [IQR] 7.04-10.77) years. Among these, seven (9.2%) boys had long bone fractures, and four (5.3%) had vertebral compression fractures. Fifty-four (71%) boys underwent DXA scan, and among these 31 (57%) had low bone mineral density (BMD, ≤-2 z-score) at the lumbar spine. The mean BMD z-score at the lumbar spine was -2.3 (95% confidence interval [CI] = -1.8, -2.8), and at the femoral neck was -2.5 (95% CI = -2, -2.9). 25(OH)D levels were deficient in 68 (89.5%, n=76) boys, and 1,25(OH) Bone health is compromised in North Indian boys with DMD. BMD at the lumbar spine is reduced in more than half of boys with DMD and nearly all had vitamin D deficiency on regular vitamin D supplements. Longer duration of glucocorticoid therapy is a risk factor for low BMD in our cohort.

Identifiants

pubmed: 33838091
pii: jpem-2020-0680
doi: 10.1515/jpem-2020-0680
doi:

Substances chimiques

Glucocorticoids 0

Types de publication

Journal Article Observational Study

Langues

eng

Sous-ensembles de citation

IM

Pagination

573-581

Informations de copyright

© 2021 Walter de Gruyter GmbH, Berlin/Boston.

Références

Emery, AE. Population frequencies of inherited neuromuscular diseases--a world survey. Neuromuscul Disord 1991;1:19–29. https://doi.org/10.1016/0960-8966(91)90039-u.
Moat, SJ, Bradley, DM, Salmon, R, Clarke, A, Hartley, L. Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK). Eur J Hum Genet 2013;21:1049–53. https://doi.org/10.1038/ejhg.2012.301.
Coratti, G, Brogna, C, Norcia, G, Ricotti, V, Abbott, L, D’Amico, A, et al.. Longitudinal natural history in young boys with Duchenne muscular dystrophy. Neuromuscul Disord 2019;29:857–62. https://doi.org/10.1016/j.nmd.2019.09.010.
Brogna, C, Coratti, G, Pane, M, Ricotti, V, Messina, S, D’Amico, A, et al.. Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53. PloS One 2019;14:e0218683. https://doi.org/10.1371/journal.pone.0218683.
Birnkrant, DJ, Bushby, K, Bann, CM, Apkon, SD, Blackwell, A, Brumbaugh, D, et al.. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol 2018;17:251–67. https://doi.org/10.1016/s1474-4422(18)30024-3.
Birnkrant, DJ, Bushby, K, Bann, CM, Alman, BA, Apkon, SD, Blackwell, A, et al.. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol 2018;17:347–61. https://doi.org/10.1016/s1474-4422(18)30025-5.
Birnkrant, DJ, Bushby, K, Bann, CM, Apkon, SD, Blackwell, A, Colvin, MK, et al.. Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Lancet Neurol 2018;17:445–55. https://doi.org/10.1016/s1474-4422(18)30026-7.
Matthews, E, Brassington, R, Kuntzer, T, Jichi, F, Manzur, AY. Corticosteroids for the treatment of Duchenne muscular dystrophy. Cochrane Database Syst Rev 2016;5:CD003725. https://doi.org/10.1002/14651858.CD003725.pub4.
McDonald, CM, Henricson, EK, Abresch, RT, Han, JJ, Escolar, DM, Florence, JM, et al.. The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used. Muscle Nerve 2013;48:32–54. https://doi.org/10.1002/mus.23807.
Vengalil, S, Preethish-Kumar, V, Polavarapu, K, Mahadevappa, M, Sekar, D, Purushottam, M, et al.. Duchenne muscular dystrophy and Becker muscular dystrophy confirmed by multiplex ligation-dependent probe amplification: genotype-phenotype correlation in a large cohort. J Clin Neurol 2017;13:91–7. https://doi.org/10.3988/jcn.2017.13.1.91.
Ward, LM, Weber, DR. Growth, pubertal development, and skeletal health in boys with Duchenne muscular dystrophy. Curr Opin Endocrinol Diabetes Obes 2019;26:39–48. https://doi.org/10.1097/med.0000000000000456.
Bianchi, ML, Biggar, D, Bushby, K, Rogol, AD, Rutter, MM, Tseng, B. Endocrine aspects of Duchenne muscular dystrophy. Neuromuscul Disord 2011;21:298–303. https://doi.org/10.1016/j.nmd.2011.02.006.
Bianchi, ML, Morandi, L, Andreucci, E, Vai, S, Frasunkiewicz, J, Cottafava, R. Low bone density and bone metabolism alterations in Duchenne muscular dystrophy: response to calcium and vitamin D treatment. Osteoporos Int 2011;22:529–39. https://doi.org/10.1007/s00198-010-1275-5.
Bianchi, ML, Mazzanti, A, Galbiati, E, Saraifoger, S, Dubini, A, Cornelio, F, et al.. Bone mineral density and bone metabolism in Duchenne muscular dystrophy. Osteoporos Int 2003;14:761–7. https://doi.org/10.1007/s00198-003-1443-y.
Pouwels, S, de Boer, A, Leufkens, HG, Weber, WE, Cooper, C, van Onzenoort, HA, et al.. Risk of fracture in patients with muscular dystrophies. Osteoporos Int 2014;25:509–18. https://doi.org/10.1007/s00198-013-2442-2.
Tian, C, Wong, BL, Hornung, L, Khoury, JC, Miller, L, Bange, J, et al.. Bone health measures in glucocorticoid-treated ambulatory boys with Duchenne muscular dystrophy. Neuromuscul Disord 2016;26:760–7. https://doi.org/10.1016/j.nmd.2016.08.011.
Mayo, AL, Craven, BC, McAdam, LC, Biggar, WD. Bone health in boys with Duchenne muscular dystrophy on long-term daily deflazacort therapy. Neuromuscul Disord 2012;22:1040–5. https://doi.org/10.1016/j.nmd.2012.06.354.
Bothwell, JE, Gordon, KE, Dooley, JM, MacSween, J, Cummings, EA, Salisbury, S. Vertebral fractures in boys with Duchenne muscular dystrophy. Clin Pediatr 2003;42:353–6. https://doi.org/10.1177/000992280304200408.
James, KA, Cunniff, C, Apkon, SD, Mathews, K, Lu, Z, Holtzer, C, et al.. Risk factors for first fractures among males with Duchenne or Becker muscular dystrophy. J Pediatr Orthop 2015;35:640–4. https://doi.org/10.1097/bpo.0000000000000348.
Crabtree, NJ, Roper, H, McMurchie, H, Shaw, NJ. Regional changes in bone area and bone mineral content in boys with Duchenne muscular dystrophy receiving corticosteroid therapy. J Pediatr 2010;156:450–5. https://doi.org/10.1016/j.jpeds.2009.09.010.
Crabtree, NJ, Adams, JE, Padidela, R, Shaw, NJ, Hogler, W, Roper, H, et al.. Growth, bone health & ambulatory status of boys with DMD treated with daily vs. intermittent oral glucocorticoid regimen. Bone 2018;116:181–6. https://doi.org/10.1016/j.bone.2018.07.019.
Vignos, PJJr, Spencer, GEJr, Archibald, KC. Management of progressive muscular dystrophy in childhood. J Am Med Assoc 1963;184:89–96. https://doi.org/10.1001/jama.1963.03700150043007.
Jung, IY, Chae, JH, Park, SK, Kim, JH, Kim, JY, Kim, SJ, et al.. The correlation analysis of functional factors and age with Duchenne muscular dystrophy. Ann Rehabil Med 2012;36:22–32. https://doi.org/10.5535/arm.2012.36.1.22.
Gordon, CM, Baim, S, Bianchi, ML, Bishop, NJ, Hans, DB, Kalkwarf, H, et al.. Special report on the 2007 Pediatric Position Development Conference of the International Society for Clinical Densitometry. South Med J 2008;101:740–3. https://doi.org/10.1097/smj.0b013e31817a8b55.
Khadilkar, AV, Sanwalka, NJ, Chiplonkar, SA, Khadilkar, VV, Mughal, MZ. Normative data and percentile curves for Dual Energy X-ray Absorptiometry in healthy Indian girls and boys aged 5-17 years. Bone 2011;48:810–9. https://doi.org/10.1016/j.bone.2010.12.013.
ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med 2002;166:111–7. https://doi.org/10.1164/ajrccm.166.1.at1102.
Joseph, S, Wang, C, Di Marco, M, Horrocks, I, Abu-Arafeh, I, Baxter, A, et al.. Fractures and bone health monitoring in boys with Duchenne muscular dystrophy managed within the Scottish Muscle Network. Neuromuscul Disord 2019;29:59–66. https://doi.org/10.1016/j.nmd.2018.09.005.
Khadilkar, AV, Ekbote, VH, Chiplonkar, SA, Mughal, MZ, Khadilkar, VV. Fractures in school going children. Indian J Pediatr 2015;82:871. https://doi.org/10.1007/s12098-015-1726-2.
King, WM, Ruttencutter, R, Nagaraja, HN, Matkovic, V, Landoll, J, Hoyle, C, et al.. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology 2007;68:1607–13. https://doi.org/10.1212/01.wnl.0000260974.41514.83.
Perera, N, Sampaio, H, Woodhead, H, Farrar, M. Fracture in Duchenne muscular dystrophy: natural history and vitamin D deficiency. J Child Neurol 2016;31:1181–7. https://doi.org/10.1177/0883073816650034.
Barzegar, M, Niknam, E, Habibi, P, Shiva, S, Tahmasebi, S. Bone mineral density and bone metabolism in patients with Duchenne muscular dystrophy. Iran J Child Neurol 2018;12:77–83.
Bian, Q, McAdam, L, Grynpas, M, Mitchell, J, Harrington, J. Increased rates of vitamin d insufficiency in boys with Duchenne muscular dystrophy despite higher vitamin d3 supplementation. Global Pediatr Health 2019;6:2333794X19835661. https://doi.org/10.1177/2333794x19835661.
Weber, DR, Thomas, S, Erickson, SW, Fox, D, Oleszek, J, Pandya, S, et al.. Bone health and endocrine care of boys with Duchenne muscular dystrophy: data from the MD STARnet. J Neuromuscul Dis 2018;5:497–507. https://doi.org/10.3233/jnd-180317.
Bushby, K, Finkel, R, Birnkrant, DJ, Case, LE, Clemens, PR, Cripe, L, et al.. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010;9:77–93. https://doi.org/10.1016/s1474-4422(09)70271-6.
Bushby, K, Finkel, R, Birnkrant, DJ, Case, LE, Clemens, PR, Cripe, L, et al.. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010;9:177–89. https://doi.org/10.1016/s1474-4422(09)70272-8.
Angurana, SK, Angurana, RS, Mahajan, G, Kumar, N, Mahajan, V. Prevalence of vitamin D deficiency in apparently healthy children in North India. J Pediatr Endocrinol Metab 2014;27:1151–6. https://doi.org/10.1515/jpem-2013-0387.
Ananda Kesavan, TM, Thuruthiyath, N, Thomas, EP. Subclinical vitamin D deficiency in children from Thrissur, Kerala. Indian Pediatr 2018;55:434–5.
Angurana, SK, Guglani, V. Severe vitamin d deficiency at admission and shock reversal in children with septic shock. J Intensive Care Med 2018;33:56–7. https://doi.org/10.1177/0885066617714771.
Davis, J, Samuels, E, Mullins, L. Nutrition considerations in Duchenne muscular dystrophy. Nutr Clin Pract 2015;30:511–21. https://doi.org/10.1177/0884533615586202.
Rufo, A, Del Fattore, A, Capulli, M, Carvello, F, De Pasquale, L, Ferrari, S, et al.. Mechanisms inducing low bone density in Duchenne muscular dystrophy in mice and humans. J Bone Miner Res 2011;26:1891–903. https://doi.org/10.1002/jbmr.410.
Tian, C, Wong, BL, Hornung, L, Khoury, JC, Rybalsky, I, Shellenbarger, KC, et al.. Oral bisphosphonate treatment in patients with Duchenne muscular dystrophy on long term glucocorticoid therapy. Neuromuscul Disord 2020;30:599–610. https://doi.org/10.1016/j.nmd.2020.06.005.

Auteurs

Renu Suthar (R)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

B V Chaithanya Reddy (BVC)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Manisha Malviya (M)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Titiksha Sirari (T)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Savita Verma Attri (SV)

Pediatric Biochemistry Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Ajay Patial (A)

Pediatric Biochemistry Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Minni Tageja (M)

Pediatric Biochemistry Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Gunjan Didwal (G)

Pediatric Biochemistry Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Niranjan K Khandelwal (NK)

Department of Radiodiagnosis, PGIMER, Chandigarh, India.

Arushi G Saini (AG)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Lokesh Saini (L)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Jitendra K Sahu (JK)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Devi Dayal (D)

Pediatric Endocrinology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

Naveen Sankhyan (N)

Pediatric Neurology Unit, Department of Pediatrics, APC, PGIMER, Chandigarh, India.

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