Comparative responsiveness of generic and specific quality-of-life instruments

Wiebe, Samuel; Guyatt, Gordon; Weaver, Bruce; Matijevic, Suzan; Sidwell, Casey

doi:10.1016/s0895-4356(02)00537-1

Cited by 479 publications

(357 citation statements)

References 93 publications

Supporting

Mentioning

333

Contrasting

Unclassified

Order By: Relevance

“…Since the DISABKIDS specifically addresses a population with health conditions, conceptualization of HrQoL was closer to the MetabQoL 1.0. Nevertheless, due to its more specific content, we 26); not bad at all, slightly bad, medium bad, bad, very bad (item 27); never, once, twice, three to five times, six times or more (item 28) hypothesise that the MetabQoL 1.0 will be more responsive to disease-related changes than the chronic-generic DIS-ABKIDS (Wiebe et al 2003) and thus most valuable for clinical practice and research settings. This hypothesis will be followed up in long-term studies.…”

Section: Discussionmentioning

confidence: 99%

“…Considering research, disease-specific HrQoL measures are a most interesting additional approach for measuring outcome in clinical trials (Wiebe et al 2003). The MetabQoL 1.0 is a promising tool for assessing diseaserelated HrQoL changes in IT-IEM.…”

Section: Discussionmentioning

confidence: 99%

“…Disease-specific tools such as the PKU-QOL (Regnault et al 2015) investigate the impact of a particular disease or disease group on patients' life. They have shown high responsiveness to change of HrQoL (Wiebe et al 2003) and are therefore the method of choice for measuring this outcome parameter in clinical trials or long-term patient management.…”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Development and Psychometric Evaluation of the MetabQoL 1.0: A Quality of Life Questionnaire for Paediatric Patients with Intoxication-Type Inborn Errors of Metabolism

et al. 2017

View full text Add to dashboard Cite

Introduction: This study is part of the "European network and registry for intoxication type metabolic diseases" (E-IMD) project. Intoxication-type inborn errors of metabolism (IT-IEM) such as urea cycle disorders (UCD) and organic acidurias (OA) have a major impact on patients' lives. Patients have to adhere to strict diet and medication and may suffer from metabolic crises and neurocognitive impairment. Disease-specific health-related quality of life (HrQoL) assessment questionnaires are the method of choice to estimate the subjective burden of a Communicated by: Pascale de Lonlay "Peter Burgard," "Markus A. Landolt," and "Martina Huemer" are contributed equally to this project. Electronic supplementary material:The online version of this chapter

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Development and Psychometric Evaluation of the MetabQoL 1.0: A Quality of Life Questionnaire for Paediatric Patients with Intoxication-Type Inborn Errors of Metabolism

et al. 2017

View full text Add to dashboard Cite

show abstract

“…Second, it is often recommended to combine a generic instrument and a disease-specific instrument. For most diseases, the generic instruments have lower responsiveness compared to specific ones [145], but the generic measures are useful to compare the patient cohort against cohorts with other diseases or with the normal population. Third, the proof of superior QoL after one type of surgery is a strong but not a sufficient argument to use this type of surgery.…”

Section: Discussionmentioning

confidence: 99%

Evaluation of quality of life after laparoscopic surgery: evidence-based guidelines of the European Association for Endoscopic Surgery

et al. 2004

View full text Add to dashboard Cite

Background: Measuring health-related quality of life (QoL) after surgery is essential for decision making by patients, surgeons, and payers. The aim of this consensus conference was twofold. First, it was to determine for which diseases endoscopic surgery results in better postoperative QoL than open surgery. Second, it was to recommend QoL instruments for clinical research. Methods: An expert panel selected 12 conditions in which QoL and endoscopic surgery are important. For each condition, studies comparing endoscopic and open surgery in terms of QoL were identified. The expert panel reached consensus on the relative benefits of endoscopic surgery and recommended generic and disease-specific QoL instruments for use in clinical research. Results: Randomized trials indicate that QoL improves earlier after endoscopic than open surgery for gastroesophageal reflux disease (GERD), cholecystolithiasis, colorectal cancer, inguinal hernia, obesity (gastric bypass), and uterine disorders that require hysterectomy. For spleen, prostate, malignant kidney, benign colorectal, and benign non-GERD esophageal diseases, evidence from nonrandomized trials supports the use of laparoscopic surgery. However, many studies failed to collect long-term results, used nonvalidated questionnaires, or measured QoL components only incompletely.

show abstract

“…It has been shown, at least for RCT in which a therapeutic effect was demonstrated, that specific instruments are more responsive than generic tools, supporting the idea that a combination of specific and generic instruments are useful. 13 While there are specific HRQOL instruments for several neurological diseases (e.g., epilepsy, migraine, and neuromuscular disorders), none specifically address function during an episode of illness (J. Langfitt, personal communication).…”

Section: Skeletal Musclementioning

confidence: 99%

Challenges in the Design and Conduct of Therapeutic Trials in Channel Disorders

2007

View full text Add to dashboard Cite

Summary: Neurologic channelopathies are rare, inherited paroxysmal disorders of muscle (e.g., the periodic paralyses and nondystrophic myotonias) and brain (e.g., episodic ataxias, idiopathic epilepsies, and familial hemiplegic migraine). Mutation is necessary but not sufficient for phenotypic expression and there are no simple phenotype-genotype relationships. Attacks may be spontaneous or triggered, with affected individuals often asymptomatic and neurologically normal between attacks. Performance of daily activities may be affected by the unpredictable nature; often late-onset degenerative changes cause permanent disability; for example, muscle atrophy and fixed weakness in periodic paralysis and cerebellar atrophy and progressive ataxia in the episodic ataxias. Currently, the natural history of these disorders is being defined. Clearly, the established methodologies for randomized controlled clinical trials are not feasible for rare diseases and innovative trial design is essential. There is a requirement for clinically relevant outcome measures for episodic disorders. Increasing our knowledge of the pathophysiology will help in targeting and designing rational therapeutic approaches. We will use the current understanding of the neurological channelopathies to illustrate some of the opportunities, challenges, and strategies in bringing safe and effective treatments to patients. There are reasons for optimism that new partnerships between clinical investigators, government, patient advocacy groups, and industry will prevent symptoms and progression of the neurological channelopathies.

show abstract

Comparative responsiveness of generic and specific quality-of-life instruments

Cited by 479 publications

References 93 publications

Development and Psychometric Evaluation of the MetabQoL 1.0: A Quality of Life Questionnaire for Paediatric Patients with Intoxication-Type Inborn Errors of Metabolism

Development and Psychometric Evaluation of the MetabQoL 1.0: A Quality of Life Questionnaire for Paediatric Patients with Intoxication-Type Inborn Errors of Metabolism

Evaluation of quality of life after laparoscopic surgery: evidence-based guidelines of the European Association for Endoscopic Surgery

Challenges in the Design and Conduct of Therapeutic Trials in Channel Disorders

Contact Info

Product

Resources

About