Long-term administration of oral alendronate in patients with severe motor and intellectual disabilities with bone fragility.

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  • Okamoto Yuko
    Department of Pediatrics, Hananoki Medical and Welfare Center, Kyoto, Japan Department of Pediatrics, Kyoto Prefectural University of Medicine, Kyoto, Japan
  • Matsui Fumihiro
    Department of Pediatrics, Hananoki Medical and Welfare Center, Kyoto, Japan
  • Nagai Hideyuki
    Department of Pediatrics, Hananoki Medical and Welfare Center, Kyoto, Japan
  • Kamiya Yasutaka
    Department of Pediatrics, Hananoki Medical and Welfare Center, Kyoto, Japan
  • Terada Naoto
    Department of Pediatrics, Hananoki Medical and Welfare Center, Kyoto, Japan
  • Nakajima Hisakazu
    Department of Pediatrics, Kyoto Prefectural University of Medicine, Kyoto, Japan

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Other Title
  • 骨脆弱性を伴う重症心身障害児（者）に対する経口アレンドロネートの長期投与の検討
  • ホネ ゼイジャクセイ オ トモナウ ジュウショウ シンシン ショウガイジ(モノ)ニ タイスル ケイコウ アレンドロネート ノ チョウキ トウヨ ノ ケントウ

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Abstract

In this study, we aimed to confirm clinical effectiveness and safety of oral bisphosphonate (alendronate) for patients with severe motor and intellectual disabilities (SMID) associated with bone fragility. We treated five patients with SMID who had a past history of bone fracture with oral alendronate for 72 months. We reviewed the incidence of bone fracture during 72 months before and after starting oral alendronate treatment on each patient. Cross-linked N-telopeptide of type I collagen in urine (urinary NTX), bone specific alkaline phosphatase in serum (serum BAP), and bone mineral density (BMD) of lumbar vertebrae (L2-L4) as well as the left femoral neck were monitored as indicators to evaluate the effects for bone metabolism. The incidence of bone fracture decreased in some patients after starting oral alendronate treatment compared to before the treatment. We found no improvement on BMD of lumbar vertebrae and left femoral neck. However, urinary NTX was relatively decreased in 6 months after starting the treatment. We detected improvement of urinary NTX in all patients with the minimum significant change exceeding 27.3 %, while serum BAP did not change in the observation period. The treatment was discontinued in one patient because of adverse effect of renal dysfunction. Esophageal ulcer was successfully avoided by keeping the head elevated for a while after taking the drug in all patients over the period. In conclusion, we suggest that oral alendronate treatment could be effective for preventing bone fractures in patients with SMID.

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