Abstract
Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle disease characterized by secondary osteoporosis and increased fractures. We describe the case of a 20-year-old boy with DMD suffering from back pain due to multiple vertebral fractures who was treated with teriparatide. Improvement of bone density, pain, and quality of life was achieved. DMD is an X-linked recessive muscle disease with secondary osteoporosis and related frequently occurring fractures. To date, only bisphosphonates have been used to treat osteoporosis in DMD. Black bear parathyroid hormone has been previously reported to enhance bone mass in the dystrophin-deficient mouse. This study reports the positive effect of osteoanabolic treatment with once-daily recombinant human parathyroid hormone 1–34 (rhPTH 1–34, teriparatide) in a 20-year-old DMD boy suffering from multiple vertebral fractures causing back pain. Bone formation and resorption markers (osteocalcin and C-telopeptide of type I collagen, respectively), as expected, increased within 6 months and intensity of back pain early decreased, with no pain reported after 6 months at visual analog scale. Over a 18-month period of treatment with teriparatide, bone mineral density and quality of life, assessed by the 36-item short-form questionnaire, considerably improved and no side effects were reported. Further studies on large cohorts are warranted to test the efficacy of this promising treatment for DMD related osteoporosis.
References
Eagle M, Baudouin SV, Chandler C, Giddings DR, Bullock R, Bushby K (2002) Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscul Disord 12:926–929
Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY (2016) Corticosteroids for the treatment of Duchenne muscular dystrophy. Cochrane Database Syst Rev 5(5):CD003725
Bianchi ML, Morandi L, Andreucci E, Vai S, Frasunkiewicz J, Cottafava R (2010) Low bone density and bone metabolism alterations in Duchenne muscular dystrophy: response to calcium and vitamin D treatment. Osteoporos Int 22:529–539
Pouwels S, de Boer A, Leufkens HG, et al. (2014) Risk of fracture in patients with muscular dystrophies. Osteoporos Int 25(2):509–518
Ferrari S, Bianchi ML, Eisman JA, et al. (2012) Osteoporosis in young adults: pathophysiology, diagnosis, and management. Osteoporos Int:2735–2748
Larson CM, Henderson RC (2000) Bone mineral density and fractures in boys with Duchenne muscular dystrophy. J Pediatr Orthop 20:71–74
Saag KG, Shane E, Boonen S, et al. (2007) Teriparatide or alendronate in glucocorticoid-induced osteoporosis. N Engl J Med 357(20):2028–2039
Gray SK, McGee-Lawrence ME, Sanders JL, Condon KW, Tsai CJ, Donahue SW (2012) Black bear parathyroid hormone has greater anabolic effects on trabecular bone in dystrophin-deficient mice than in wild type mice. Bone 51(3):578–585
Apolone G, Mosconi P (1998) The Italian SF-36 health survey: translation, validation and norming. J Clin Epidemiol 51:1025–1036
van Staa TP (2006) The pathogenesis, epidemiology and management of glucocorticoid-induced osteoporosis. Calcif Tissue Int 79:129–137
Lekamwasam S, Adachi JD, Agnusdei D, et al. (2012) A framework for the development of guidelines for the management of glucocorticoid-induced osteoporosis. Osteoporos Int 23(9):2257–2276
Weinstein RS, Jilka RL, Parfitt AM, Manolagas SC (1998) Inhibition of osteoblastogenesis and promotion of apoptosis of osteoblasts and osteocytes by glucocorticoids: potential mechanisms of their deleterious effects on bone. J Clin Invest 102:274–282
Neer RM, Arnaud CD, Zanchetta JR, et al. (2001) Effect of parathyroid hormone (1-34) on fractures and bone mineral density in postmenopausal women with osteoporosis. N Engl J Med 344(19):1434–1441
Finkelstein JS, Hayes A, Hunzelman JL, Wyland JJ, Lee H, Neer RM (2003) The effects of parathyroid hormone, alendronate, or both in men with osteoporosis. N Engl J Med 349(13):1216–1226
Deal C (2004) The use of intermittent human parathyroid hormone as a treatment for osteoporosis. Curr Rheumatol Rep 6(1):49–58
Arantes HP, Barros ER, Kunii I, Bilezikian JP, Lazaretti-Castro M (2011) Teriparatide increases bone mineral density in a man with osteoporosis pseudoglioma. J Bone Miner Res 26(12):2823–2826
Meriggioli MN, Roubenoff R (2015) Prospect for pharmacological therapies to treat skeletal muscle dysfunction. Calcif Tissue Int 96(3):234–242
Elis S, Courtland HW, Wu Y, et al. (2010) Elevated serum IGF-1 levels synergize PTH action on the skeleton only when the tissue IGF-1 axis is intact. J Bone Miner Res 25(9):2051–2058
Kimura S, Yoshioka K (2014) Parathyroid hormone and parathyroid hormone type-1 receptor accelerate myocyte differentiation. Sci Rep 4:5066
Nevitt MC, Chen P, Dore RK, et al. (2006) Reduced risk of back pain following teriparatide treatment: a metaanalysis. Osteoporos Int 17(2):273–280
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Conflicts of interest
None.
Additional information
Antonino Catalano and Gian Luca Vita contributed equally.
Nunziata Morabito and Sonia Messina both are senior authors.
Rights and permissions
About this article
Cite this article
Catalano, A., Vita, G.L., Russo, M. et al. Effects of teriparatide on bone mineral density and quality of life in Duchenne muscular dystrophy related osteoporosis: a case report. Osteoporos Int 27, 3655–3659 (2016). https://doi.org/10.1007/s00198-016-3761-x
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1007/s00198-016-3761-x