Therapeutics and Clinical Risk Management (Feb 2019)

Efficacy of denosumab therapy for a 21-year-old woman with Prader-Willi syndrome, osteoporosis and history of fractures: a case report

  • Uehara M,
  • Nakamura Y,
  • Takahashi J,
  • Suzuki T,
  • Iijima M,
  • Arakawa Y,
  • Ida K,
  • Kosho T,
  • Kato H

Journal volume & issue
Vol. Volume 15
pp. 303 – 307

Abstract

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Masashi Uehara,1 Yukio Nakamura,1 Jun Takahashi,1 Takako Suzuki,1 Mari Iijima,2 Yuko Arakawa,3 Koichi Ida,4 Tomoki Kosho,5,6 Hiroyuki Kato1 1Department of Orthopaedic Surgery, Shinshu University School of Medicine; 2Department of Clinical Nutrition, Shinshu University School of Medicine; 3Department of Dentistry and Oral Surgery, Shinshu University School of Medicine; 4Department of Obstetrics and Gynecology, Shinshu University School of Medicine; 5Department of Medical Genetics, Shinshu University School of Medicine; 6Center for Medical Genetics, Shinshu University Hospital, Matsumoto, Japan Abstract: Appropriate management for osteoporosis in adult patients with Prader-Willi syndrome (PWS) has not been established. We report on a 21-year-old woman with PWS, who underwent denosumab treatment for osteoporosis. She presented with fractures and was shown to have very low bone mineral density (BMD), while she had been treated with supplementation of growth hormone for 7–14 years of age and estrogen from 15 years of age. BMD was monitored in the total hip region by dual-energy X-ray absorptiometry. Laboratory tests included bone-specific alkaline phosphatase, urinary type I collagen amino-terminal telopeptide, tartrate-resistant acid phosphatase 5b, 1-alpha, 25-dihydroxyvitamin D3, and parathyroid hormone. BMD and laboratory data were evaluated before and at 4, 8, and 13 months of treatment. After 13 months of denosumab therapy, BMD increased by 4.5%, and bone turnover markers notably improved. No fractures occurred. To the best of our knowledge, this is the first report to describe the clinical outcomes of denosumab treatment for osteoporosis in patients with PWS. Based on our findings, denosumab could represent an effective treatment option for osteoporosis in PWS patients. Keywords: bone mineral density, denosumab, fracture, case report, osteoporosis

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