Priority 1 from the Rare Musculoskeletal Diseases in Adulthood PSP
| UNCERTAINTY: What is considered a good outcome of treatment in rare bone metabolic disorders? How can this be measured in studies of new treatments? (JLA PSP Priority 1) | |
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| Overall ranking | 1 |
| JLA question ID | 0076/1 |
| Explanatory note | Not available for this PSP |
| Evidence |
Determinants of impaired quality of life in patients with fibrous dysplasia.Majoor BCJ, Andela CD, Bruggemann J, van de Sande MAJ, Kaptein AA, Hamdy NAT, Dijkstra PDS, Appelman-Dijkstra NM.Orphanet J Rare Dis. 2017 Apr 27;12(1):80. Physical function is impaired but quality of life preserved in patients with fibrous dysplasia of bone.Kelly MH, Brillante B, Kushner H, Gehron Robey P, Collins MT. Bone. 2005 Sep;37(3):388-94 Health-related quality of life and a cost-utility simulation of adults in the UK with osteogenesis imperfecta, X-linked hypophosphatemia and fibrous dysplasia. Forestier-Zhang L, Watts L, Turner A, Teare H, Kaye J, Barrett J, Cooper C, Eastell R, Wordsworth P, Javaid MK, Pinedo-Villanueva R https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3235275/ http://www.thebonejournal.com/article/S8756-3282(08)00897-1/fulltext https://www.ncbi.nlm.nih.gov/pubmed/12794240 |
| Health Research Classification System category | Musculoskeletal |
| Extra information provided by this PSP | |
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| Original uncertainty examples | For FD:Correlation between markers and QOL? ~ In each of these areas there are is little by way of high quality outcome data as to clinically relevant end points. Where data is available it tends to be predicated on laboratory or imaging data rather than clinically relevant data ~ X: anti-FGF23 treatments in XLH-how will outcomes in trials be measured meaningfully? |
| Submitted by | Individual survey submissions categorised by Health or Social Care Professionals, Organisations representing people with rare musculoskeletal diseases, people with rare musculoskeletal diseases, relatives/carers/friends, Other. For full details of the type of submitter for each individual question, please see the spreadsheet of data held on the JLA website. |
| PSP information | |
|---|---|
| PSP unique ID | 0076 |
| PSP name | Rare Musculoskeletal Diseases in Adulthood |
| Total number of uncertainties identified by this PSP. | 39 (To see a full list of all uncertainties identified, please see the detailed spreadsheet held on the JLA website) |
| Date of priority setting workshop | 18 June 2018 |