التفاصيل البيبلوغرافية
| العنوان: |
Individualized treatment with denosumab in children with osteogenesis imperfecta – follow up of a trial cohort |
| المؤلفون: |
Hoyer-Kuhn, Heike, Rehberg, Mirko, Netzer, Christian, Schoenau, Eckhard, Semler, Oliver |
| المساهمون: |
Wilsing Stiftung, Osteogenesis imperfecta Stiftung Baden-Wüttemberg |
| المصدر: |
Orphanet Journal of Rare Diseases ; volume 14, issue 1 ; ISSN 1750-1172 |
| بيانات النشر: |
Springer Science and Business Media LLC |
| سنة النشر: |
2019 |
| مصطلحات موضوعية: |
Pharmacology (medical), Genetics (clinical), General Medicine |
| الوصف: |
Background Osteogenesis imperfecta (OI) is a rare disease leading to hereditary bone fragility. Nearly 90% of cases are caused by mutations in the collagen genes COL1A1/A2 (classical OI) leading to multiple fractures, scoliosis, short stature and nonskeletal findings as blue sclera, hypermobility of joints, bone pain and delayed motor function development. Bisphosphonates are used in most moderate and severely affected patients assuming that an increase of bone mineral density might reduce fractures and bone pain in patients with OI. Denosumab as a RANK ligand antibody inhibiting osteoclast maturation has been approved for osteoporosis treatment in adults. First data from small clinical trials promised a high efficacy of Denosumab in children with OI. Aim of this analysis was a retrospective evaluation of an individualized biomarker-associated treatment regime with Denosumab in 10 children with classical OI which were followed for 1 year after their participation in a pilot trial with Denosumab. Therefore urinary deoxypyridinoline levels were evaluated frequently as an osteoclastic activity marker and depending on that levels Denosumab injections were scheduled individually. Methods Ten patients (age range: 6.16–12.13 years; all participated in the former OI-AK phase 2 trial (NCT01799798)) were included in the follow-up period. Denosumab was administered subcutaneously depending on the individual urinary excretion course of deoxypyridinoline (DPD/Crea) as osteoclastic activity marker with 1 mg/kg body weight. DPD/Crea levels were evaluated before denosumab administration and afterwards. If patients present after an initial decrease after injection with a re-increase up to the DPD/crea level before Denosumab injection next dosage was planned. Changes of areal bone mineral density (aBMD) using dual energy x-ray absorptiometry of the lumbar spine after 12 month was evaluated. Safety was assessed by bone metabolism markers and side effect reporting. Results During follow-up mean relative change of lumbar ... |
| نوع الوثيقة: |
article in journal/newspaper |
| اللغة: |
English |
| DOI: |
10.1186/s13023-019-1197-z |
| DOI: |
10.1186/s13023-019-1197-z.pdf |
| DOI: |
10.1186/s13023-019-1197-z/fulltext.html |
| الإتاحة: |
https://doi.org/10.1186/s13023-019-1197-zTest |
| حقوق: |
http://creativecommons.org/licenses/by/4.0Test/ ; http://creativecommons.org/licenses/by/4.0Test/ |
| رقم الانضمام: |
edsbas.20ABCFD2 |
| قاعدة البيانات: |
BASE |
