Trial reportQuality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation2019
Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency.
Trial report in Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
6 citing papers in PubMed.
- Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial.Journal of patient-reported outcomes · 2023Trial
- Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency.Blood advances · 2022Article
- Experienced fatigue in people with rare disorders: a scoping review on characteristics of existing research.Orphanet journal of rare diseases · 2022Article
- Pyruvate Kinase Deficiency: Current Challenges and Future Prospects.Journal of blood medicine · 2022Review
- The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment.The patient · 2021Review
- Development of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment: Disease-specific assessments.European journal of haematology · 2020Article
Corrections and comments
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Authors and funding
4 authors.
Funding
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Abstract
purposePyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining adequate hemoglobin levels through transfusion and subsequent support, but with frequent complications. Treatment goals are to maintain/improve the patient's quality of life. With new therapies, reliable, valid, and relevant patient-reported outcome (PRO) tools are required for use in clinical trials.
methodsSystematic literature search identified no current PRO tools for capturing/measuring the impact of PKD and treatments in clinical trials. Therefore, the search strategy was revised to consider conditions analogous to PKD in terms of symptoms and impacts that might serve as parallels to the experience in PKD; this included sickle cell anemia, thalassemia, and hemolytic anemia. Psychometric properties, strengths, and weakness of selected appropriate PRO instruments were compared, and recommendations made for choice of PRO tools.
resultsIn adult populations, EORTC QLQ C30 and SF-36v2 are recommended, the former being a basic minimum, covering generic HRQoL, and core symptoms such as fatigue. In pediatric populations, PedsQL Generic Core Scale to measure HRQoL and PedsQL MFS scale to measure fatigue are recommended.
conclusionsSome symptoms/life impacts may be unique to PKD and not observable in analogous conditions. A 'Physico-Psychosocial Model' derived from the 'Medical Model' is proposed to form the basis for a hypothesized conceptual framework to address the development of PKD-specific PRO instruments.
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