Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta
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J. Krischer | F. Glorieux | Mahim Jain | V. Sutton | Peter A. Smith | G. Harris | Brendan H. Lee | D. Eyre | D. Krakow | F. Rauch | E. Orwoll | S. Nagamani | D. Cuthbertson | L. Tosi | C. Murali | M. Bober | J. Retrouvey | P. Esposito | Brady Slater | Gerald Harris | E. Rush | C. Raggio | Dianne Nguyen | Alicia Turner | Frank Francis Jean-Marc Paul Eric Michael David Danielle Rauch Glorieux Retrouvey Esposito Rush Bobe | Danielle Gomez | T. Hart | David Eyre | Brendan Lee | Frank Francis Jean-Marc Paul Eric Michael David Danielle Rauch Glorieux Retrouvey Esposito Rush Bobe | Jean‐Marc Retrouvey
[1] A. Slade,et al. Patient reported outcome measures in rare diseases: a narrative review , 2018, Orphanet Journal of Rare Diseases.
[2] M H Liang,et al. The POSNA pediatric musculoskeletal functional health questionnaire: report on reliability, validity, and sensitivity to change. Pediatric Outcomes Instrument Development Group. Pediatric Orthopaedic Society of North America. , 1998, Journal of pediatric orthopedics.
[3] Including patient-reported outcomes and patient-reported resource-use questionnaires in studies. , 2014, Academic radiology.
[4] F. Rauch,et al. The psychosocial experience of individuals living with osteogenesis imperfecta: a mixed-methods systematic review , 2016, Quality of Life Research.
[5] F. Tarazona-Santabalbina,et al. Enfermedad por coronavirus 2019 (COVID-19) y edadismo: revisión narrativa de la literatura , 2020, Revista Española de Geriatría y Gerontología.
[6] K. White,et al. Musculoskeletal Functional Outcomes in Children With Osteogenesis Imperfecta: Associations With Disease Severity and Pamidronate Therapy , 2014, Journal of pediatric orthopedics.
[7] P. Waber,et al. Impact of Alendronate on Quality of Life in Children With Osteogenesis Imperfecta , 2005, Journal of pediatric orthopedics.
[8] Ruta Bajorunaite,et al. Ankle Strength and Functional Limitations in Children and Adolescents With Type I Osteogenesis Imperfecta , 2010, Pediatric physical therapy : the official publication of the Section on Pediatrics of the American Physical Therapy Association.
[9] U. S. Department of Health and Human Services FDA Cen Research,et al. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance , 2006, Health and quality of life outcomes.
[10] J. Krischer,et al. Assessing disease experience across the life span for individuals with osteogenesis imperfecta: challenges and opportunities for patient-reported outcomes (PROs) measurement: a pilot study , 2019, Orphanet Journal of Rare Diseases.
[11] R. Haynes,et al. The Pediatric Orthopaedic Society of North America Pediatric Orthopaedic Functional Health Questionnaire: An Analysis of Normals , 2001, Journal of pediatric orthopedics.
[12] A. Gnanasakthy,et al. A Review of Patient-Reported Outcome Labeling in the United States (2011-2015). , 2017, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.
[13] L. Idolazzi,et al. Treatment with neridronate in children and adolescents with osteogenesis imperfecta: Data from open-label, not controlled, three-year Italian study. , 2017, Bone.
[14] B. Hero,et al. Safety and efficacy of denosumab in children with osteogenesis imperfecta - a first prospective trial , 2016, Journal of musculoskeletal & neuronal interactions.
[15] R. Steiner,et al. COL1A1/2-Related Osteogenesis Imperfecta , 2013 .
[16] J. Ablon. Personality and stereotype in osteogenesis imperfecta: Behavioral phenotype or response to life's hard challenges? , 2003, American journal of medical genetics. Part A.
[17] J. Krischer,et al. A cross‐sectional multicenter study of osteogenesis imperfecta in North America – results from the linked clinical research centers , 2015, Clinical genetics.
[18] Charlotte Kingsley,et al. Patient-reported outcome measures and patient-reported experience measures , 2017 .
[19] K. Siegel,et al. Brief Assessment of Motor Function: Reliability and Concurrent Validity of the Gross Motor Scale , 2003, American journal of physical medicine & rehabilitation.
[20] Sheila Unger,et al. Nosology and classification of genetic skeletal disorders: 2015 revision , 2015, American journal of medical genetics. Part A.
[21] Peter A. Smith,et al. Gait characteristics and functional assessment of children with Type I Osteogenesis Imperfecta , 2009, Journal of orthopaedic research : official publication of the Orthopaedic Research Society.
[22] M. Stockler,et al. The importance of patient-reported outcomes in clinical trials and strategies for future optimization , 2018, Patient related outcome measures.
[23] B. Biesecker,et al. Quality of life in rare genetic conditions: A systematic review of the literature , 2010, American journal of medical genetics. Part A.
[24] R. McCall,et al. Alendronate for the treatment of pediatric osteogenesis imperfecta: a randomized placebo-controlled study. , 2011, The Journal of clinical endocrinology and metabolism.
[25] A. Gnanasakthy,et al. Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014). , 2016, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.
[26] L. Gerber,et al. Brief Assessment of Motor Function: Content Validity and Reliability of the Fine Motor Scale , 2007, Pediatric physical therapy : the official publication of the Section on Pediatrics of the American Physical Therapy Association.
[27] A. Gnanasakthy,et al. Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006-2010. , 2012, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.
[28] S. Walters,et al. Quality of life in children and adolescents with Osteogenesis Imperfecta: a qualitative interview based study , 2014, Health and Quality of Life Outcomes.
[29] Jay J. Han,et al. The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year. , 2013, PLoS currents.
[30] J. Krischer,et al. A multicenter study to evaluate pulmonary function in osteogenesis imperfecta , 2018, Clinical genetics.
[31] John J. Mitchell,et al. Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study. , 2017, Molecular genetics and metabolism.
[32] M. Warman,et al. Mechanistic and therapeutic insights gained from studying rare skeletal diseases. , 2015, Bone.
[33] R. Engelbert,et al. Skeletal effects and functional outcome with olpadronate in children with osteogenesis imperfecta: a 2-year randomised placebo-controlled study , 2004, The Lancet.
[34] G. Gorton,et al. Outcome tools used for ambulatory children with cerebral palsy: responsiveness and minimum clinically important differences , 2008, Developmental medicine and child neurology.
[35] M. Brundage,et al. Added value of health-related quality of life measurement in cancer clinical trials: the experience of the NCIC CTG , 2010, Expert review of pharmacoeconomics & outcomes research.
[36] A. Schott,et al. Effects of Oral Alendronate on BMD in Adult Patients With Osteogenesis Imperfecta: A 3‐Year Randomized Placebo‐Controlled Trial , 2005, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.
[37] Theresa Weldring,et al. Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs) , 2013, Health services insights.
[38] T. Félix,et al. Health-related quality of life of children and adolescents with osteogenesis imperfecta: a cross-sectional study using PedsQL™ , 2018, BMC Pediatrics.