Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta

[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for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance , 2006, Health and quality of life outcomes.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.