title: Improving patient outcomes in fibrous dysplasia/McCune-Albright syndrome: an international multidisciplinary workshop to inform an international partnership.
abstract: To develop consensus on improving the management of patients, we convened an international workshop involving patients, clinicians, and researchers. Key findings included the diagnostic delay and variability in subsequent management with agreement to develop an international natural history study. We now invite other stakeholders to join the partnership.
  
  
  OBJECTIVE
  The aim of this study was develop a consensus on how to improve the management of patients with fibrous dysplasia and prioritize areas for research METHODS: An international workshop was held over 3 days involving patients, clinicians, and researchers. Each day had a combination of formal presentations and facilitated discussions that focused on clinical pathways and research.
  
  
  RESULTS
  The patient workshop day highlighted the variability of patients' experience in getting a diagnosis, the knowledge of general clinical staff, and understanding long-term outcomes. The research workshop prioritized collaborations that improved understanding of the contemporary natural history of fibrous dysplasia/McCune-Albright syndrome (FD/MAS). The clinical workshop outlined the key issues around diagnostics, assessment of severity, treatment and monitoring of patients.
  
  
  CONCLUSIONS
  In spite of advances in understanding the genetic and molecular underpinnings of fibrous dysplasia/McCune-Albright syndrome, clinical management remains a challenge. From the workshop, a consensus was reached to create an international, multi-stakeholder partnership to advance research and clinical care in FD/MAS. We invite other stakeholders to join the partnership.
authors.affiliation: Skeletal Clinical Studies Unit, Craniofacial and Skeletal Diseases Branch, National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD, USA.|Bone Health Program, Division of Orthopedics and Sports Medicine, Children's National Medical Center, Washington, DC, USA.|Division of Endocrinology and Diabetes, Children's National Medical Center, Washington, DC, USA.
authors.forename: A M
authors.initials: AM
authors.lastname: Boyce
authors.affiliation: NIHR Musculoskeletal Biomedical Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Nuffield Orthopaedic Centre, Windmill Road, Oxford, OX3 7LD, UK.
authors.forename: A
authors.initials: A
authors.lastname: Turner
authors.affiliation: NIHR Musculoskeletal Biomedical Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Nuffield Orthopaedic Centre, Windmill Road, Oxford, OX3 7LD, UK.
authors.forename: L
authors.initials: L
authors.lastname: Watts
authors.affiliation: NIHR Musculoskeletal Biomedical Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Nuffield Orthopaedic Centre, Windmill Road, Oxford, OX3 7LD, UK.
authors.forename: L
authors.initials: L
authors.lastname: Forestier-Zhang
authors.affiliation: , Chandlers Ford, Eastleigh, Hampshire, SO53 1TQ, UK.
authors.forename: A
authors.initials: A
authors.lastname: Underhill
authors.affiliation: NIHR Musculoskeletal Biomedical Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Nuffield Orthopaedic Centre, Windmill Road, Oxford, OX3 7LD, UK.
authors.forename: R
authors.initials: R
authors.lastname: Pinedo-Villanueva
authors.affiliation: Bristol Royal Hospital for Children, Paul O'Gorman Building, Upper Maudlin Street, Bristol, BS2 8BJ, UK.
authors.forename: F
authors.initials: F
authors.lastname: Monsell
authors.affiliation: Department of Pediatric Endocrinology and Diabetology, Regina Margherita Children Hospital, University of Turin, Piazza Polonia 94, 10126, Turin, Italy.
authors.forename: D
authors.initials: D
authors.lastname: Tessaris
authors.affiliation: Bristol Royal Hospital for Children, Paul O'Gorman Building, Upper Maudlin Street, Bristol, BS2 8BJ, UK.
authors.forename: C
authors.initials: C
authors.lastname: Burren
authors.affiliation: Department of Internal Medicine, University of Florence, Florence, Viale Pieraccini 6, 50134, Florence, Italy.
authors.forename: L
authors.initials: L
authors.lastname: Masi
authors.affiliation: Department of Endocrinology and Metabolic Diseases, Leiden University Medical Centre, Leiden, 2333 ZA, The Netherlands.
authors.forename: N
authors.initials: N
authors.lastname: Hamdy
authors.affiliation: Department of Internal Medicine, University of Florence, Florence, Viale Pieraccini 6, 50134, Florence, Italy.
authors.forename: M L
authors.initials: ML
authors.lastname: Brandi
authors.affiliation: NSERM UMR 1033, Department of Rheumatology, Université de Lyon, Hospices Civils de Lyon, Lyon, France.
authors.forename: R
authors.initials: R
authors.lastname: Chapurlat
authors.affiliation: Skeletal Clinical Studies Unit, Craniofacial and Skeletal Diseases Branch, National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, MD, USA.
authors.forename: M T
authors.initials: MT
authors.lastname: Collins
authors.affiliation: NIHR Musculoskeletal Biomedical Research Unit, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Nuffield Orthopaedic Centre, Windmill Road, Oxford, OX3 7LD, UK. kassim.javaid@ndorms.ox.ac.uk.
authors.forename: Muhammad Kassim
authors.initials: MK
authors.lastname: Javaid
mesh_terms: D000328:Adult; D057210:Delayed Diagnosis / Q000009:adverse effects / Q000517:prevention & control; D019468:Disease Management; D005260:Female; D005359:Fibrous Dysplasia, Polyostotic / Q000175:diagnosis / Q000453:epidemiology / Q000628:therapy; D006801:Humans; D007391:International Cooperation; D008297:Male; D018802:Patient-Centered Care / Q000379:methods / Q000458:organization & administration; D058996:Quality Improvement; D012720:Severity of Illness Index; D063189:Symptom Assessment / Q000379:methods
keywords: Epidemiology; Fibrous dysplasia; McCune-Albright syndrome; Patient-centered care