The top 10 research priorities in bleeding disorders: a James Lind Alliance Priority Setting Partnership

Shapiro, Susan; Stephensen, David; Camp, Charlotte; Carroll, Liz; Collins, Peter William; Elston, Derek; Gallagher, Patrick J.; Khair, Kate; McKeown, William; O’Hara, Jamie; Stanworth, Simon; Waterman, Amanda H.; Woollard, Laurence; Upadhyaya, Sheela; Laffan, Michael

doi:10.1111/bjh.15928

Cited by 9 publications

(10 citation statements)

References 9 publications

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“…Our findings support the findings from James Lind Alliance partnerships between researchers and patient organization underlining fatigue as one of the 10 most important research questions in several rare disorders [27][28][29][30]. There may be reason to investigate experienced fatigue more extensively both in diseases where we found studies and in diseases where we did not.…”

Section: Characteristics Of Included Articles and Study Populationssupporting

confidence: 88%

“…Experienced fatigue has been described as a challenge in everyday life for people with some rare diseases [1,[24][25][26]. In research setting priority partnerships between patients and researchers, experienced fatigue has also been determined as one of the ten most important research questions in several rare disorders: rare inherited anemias [27], rare bleeding disorders [28], rare musculoskeletal disorders [29], and mitochondrial diseases [30]. This indicates a need for more knowledge about the status of fatigue research in rare diseases.…”

Section: Introductionmentioning

confidence: 99%

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Experienced fatigue in people with rare disorders: a scoping review on characteristics of existing research

Bathen

Johansen

Strømme

et al. 2022

Orphanet J Rare Dis

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Background Experienced fatigue is an under-recognized and under-researched feature in persons with many different rare diseases. A better overview of the characteristics of existing research on experienced fatigue in children and adults with rare diseases is needed. The purpose of this review was to map and describe characteristics of existing research on experienced fatigue in a selection of rare diseases in rare developmental defects or anomalies during embryogenesis and rare genetic diseases. Furthermore, to identify research gaps and point to research agendas. Methods We applied a scoping review methodology, and performed a systematic search in March 2020 in bibliographic databases. References were sorted and evaluated for inclusion using EndNote and Rayyan. Data were extracted on the main research questions concerning characteristics of research on experienced fatigue (definition and focus on fatigue, study populations, research questions investigated and methods used). Results This review included 215 articles on ten different rare developmental defects/anomalies during embryogenesis and 35 rare genetic diseases. Of the 215 articles, 82 had investigation of experienced fatigue as primary aim or outcome. Included were 9 secondary research articles (reviews) and 206 primary research articles. A minority of articles included children. There were large differences in the number of studies in different diseases. Only 29 of 215 articles gave a description of how they defined the concept of experienced fatigue. The most common research-question reported on was prevalence and/ -or associations to fatigue. The least common was diagnostics (development or validation of fatigue assessment methods for a specific patient group). A large variety of methods were used to investigate experienced fatigue, impeding comparisons both within and across diagnoses. Conclusion This scoping review on the characteristics of fatigue research in rare diseases found a large variety of research on experienced fatigue. However, the minority of studies had investigation of experienced fatigue as a primary aim. There was large variation in how experienced fatigue was defined and also in how it was measured, both within and across diagnoses. More research on experienced fatigue is needed, both in children and adults with rare diseases. This review offers a basis for further research.

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Section: Characteristics Of Included Articles and Study Populationssupporting

confidence: 88%

Section: Introductionmentioning

confidence: 99%

Experienced fatigue in people with rare disorders: a scoping review on characteristics of existing research

Bathen

Johansen

Strømme

et al. 2022

Orphanet J Rare Dis

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“…This Pain is an issue that many PWH state is one of their major concerns, 30 Only two studies included quality of life assessment, 22,25 and three included an assessment of function. [26][27][28] The minimal evalua- intervention, thereby making it difficult to evaluate them in their practical effectiveness or to identify how they may be exerting their effects.…”

Section: Discussionmentioning

confidence: 99%

“…Pain is an issue that many PWH state is one of their major concerns, 30 yet no study included in this review defined pain as a specific inclusion criteria or ascertained if the presence of pain was of concern to participants. This may indicate assumptions made on the presence of pain based only on having haemophilia and/or arthropathy.…”

Section: Discussionmentioning

confidence: 99%

Physiotherapy interventions for pain management in haemophilia: A systematic review

et al. 2020

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“…Management of acute and ongoing pain as well as improved differentiation between acute and chronic joint damage symptoms was identified by PWH among the most important research objectives that mattered to them. 7 Physiotherapists can help with the aforementioned issues, as long as they are adequately represented in the haemophilia team with respect to dedicated time, training and resources.…”

Section: Dear Editormentioning

confidence: 99%

Comprehensive care on paper only? The challenge for physiotherapy provision in day to day haemophilia practice

et al. 2020

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The top 10 research priorities in bleeding disorders: a James Lind Alliance Priority Setting Partnership

Cited by 9 publications

References 9 publications

Experienced fatigue in people with rare disorders: a scoping review on characteristics of existing research

Experienced fatigue in people with rare disorders: a scoping review on characteristics of existing research

Physiotherapy interventions for pain management in haemophilia: A systematic review

Comprehensive care on paper only? The challenge for physiotherapy provision in day to day haemophilia practice

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