Cystic Fibrosis — Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis
Citation(s)
Anabtawi A, Le T, Putman M, Tangpricha V, Bianchi ML Cystic fibrosis bone disease: Pathophysiology, assessment and prognostic implications. J Cyst Fibros. 2019 Oct;18 Suppl 2:S48-S55. doi: 10.1016/j.jcf.2019.08.018.
Callaway DA, Jiang JX Reactive oxygen species and oxidative stress in osteoclastogenesis, skeletal aging and bone diseases. J Bone Miner Metab. 2015 Jul;33(4):359-70. doi: 10.1007/s00774-015-0656-4. Epub 2015 Mar 26.
Conway SP Impact of lung inflammation on bone metabolism in adolescents with cystic fibrosis. Paediatr Respir Rev. 2001 Dec;2(4):324-31. doi: 10.1053/prrv.2001.0167.
Elkin SL, Vedi S, Bord S, Garrahan NJ, Hodson ME, Compston JE Histomorphometric analysis of bone biopsies from the iliac crest of adults with cystic fibrosis. Am J Respir Crit Care Med. 2002 Dec 1;166(11):1470-4. doi: 10.1164/rccm.200206-578OC. Epub 2002 Sep 11.
Henderson RC, Madsen CD Bone density in children and adolescents with cystic fibrosis. J Pediatr. 1996 Jan;128(1):28-34. doi: 10.1016/s0022-3476(96)70424-9.
Hu L, Yin C, Zhao F, Ali A, Ma J, Qian A Mesenchymal Stem Cells: Cell Fate Decision to Osteoblast or Adipocyte and Application in Osteoporosis Treatment. Int J Mol Sci. 2018 Jan 25;19(2):360. doi: 10.3390/ijms19020360.
Javier RM, Jacquot J Bone disease in cystic fibrosis: what's new? Joint Bone Spine. 2011 Oct;78(5):445-50. doi: 10.1016/j.jbspin.2010.11.015. Epub 2011 Jan 12.
Moore SG, Dawson KL Red and yellow marrow in the femur: age-related changes in appearance at MR imaging. Radiology. 1990 Apr;175(1):219-23. doi: 10.1148/radiology.175.1.2315484.
Schellinger D, Lin CS, Lim J, Hatipoglu HG, Pezzullo JC, Singer AJ Bone marrow fat and bone mineral density on proton MR spectroscopy and dual-energy X-ray absorptiometry: their ratio as a new indicator of bone weakening. AJR Am J Roentgenol. 2004 Dec;183(6):1761-5. doi: 10.2214/ajr.183.6.01831761.
Tian X, Cong F, Guo H, Fan J, Chao G, Song T Downregulation of Bach1 protects osteoblasts against hydrogen peroxide-induced oxidative damage in vitro by enhancing the activation of Nrf2/ARE signaling. Chem Biol Interact. 2019 Aug 25;309:108706. doi: 10.1016/j.cbi.2019.06.019. Epub 2019 Jun 11.
Ullal J, Kutney K, Williams KM, Weber DR Treatment of cystic fibrosis related bone disease. J Clin Transl Endocrinol. 2021 Dec 21;27:100291. doi: 10.1016/j.jcte.2021.100291. eCollection 2022 Mar.
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.
