Bone mineral density;
Bone turnover;
Duchenne muscular dystrophy;
Fractures;
Osteoporosis;
Teriparatide;
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摘要:
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.
机构:
Univ Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
Zamani, Gholamreza
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Heidari, Morteza
Malamiri, Reza Azizi
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Ahvaz Jundishapur Univ Med Sci, Golestan Med Educ & Res Ctr, Dept Pediat Neurol, Ahvaz, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
Malamiri, Reza Azizi
Ashrafi, Mahmoud Reza
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Univ Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
Ashrafi, Mahmoud Reza
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Mohammadi, Mahmoud
Badv, Reza Shervin
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Univ Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
Badv, Reza Shervin
Hosseini, Seyed Ahmad
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Golestan Univ Med Sci, Dept Pediat, Gorgan, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
Hosseini, Seyed Ahmad
Salehi, Soodeh
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Univ Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
Salehi, Soodeh
Shahrokhi, Amin
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Univ Social Welf & Rehabil Sci, Pediat Neurorehabil Res Ctr, Tehran, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
Shahrokhi, Amin
Qorbani, Mostafa
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Alborz Univ Med Sci, Sch Med, Dept Community Med, Karaj, IranUniv Tehran Med Sci, Childrens Med Ctr, Pediat Ctr Excellence, Dept Pediat Neurol, Tehran, Iran
机构:
Ctr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, Mexico
Inst Politecn Nacl, Escuela Nacl Ciencias Biol, Dept Bioquim, Lab Biomembranas, Mexico City, DF, MexicoCtr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, Mexico
Del Rocio Cruz-Guzman, Oriana
Rodriguez-Cruz, Maricela
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Ctr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, MexicoCtr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, Mexico
Rodriguez-Cruz, Maricela
Almeida-Becerril, Tomas
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Ctr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, MexicoCtr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, Mexico
Almeida-Becerril, Tomas
Maldonado-Hernandez, Jorge
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Ctr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, MexicoCtr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, Mexico
Maldonado-Hernandez, Jorge
Wong Baeza, Carlos
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Inst Politecn Nacl, Escuela Nacl Ciencias Biol, Dept Bioquim, Lab Biomembranas, Mexico City, DF, MexicoCtr Med Nacl Siglo XXI IMSS, Hosp Pediat, Lab Nutr Mol, Unidad Invest Med Nutr, Ave Cuauhtemoc 330, Mexico City 06725, DF, Mexico