Endoscopic sinus surgery in adult patients with Chronic  Rhinosinusitis with nasal polyps (PolypESS) - statistical  analysis plan for a multicentre randomised controlled trial

Background: Chronic rhinosinusitis with nasal polyps (CRSwNP) afflicts 2-4% of the population and comes with a long time burden of disease and high societal costs. The current treatment consists of medical treatment alone or in combination with endoscopic sinus surgery. No consensus exists on the right timing and extent of disease that warrants surgery. Furthermore, there is lack of clinical knowledge about the benefit of surgery over medication only. The current study evaluates the clinical effectiveness and cost-effectiveness of endoscopic sinus surgery in addition to medical treatment versus medication alone in the adult patient group with nasal polyps (CRSwNP). Methods: The PolypESS trial is designed as a prospective, randomised, multicentre trial in adult patients with CRSwNP selected for primary or revision endoscopic sinus surgery by their otorhinolaryngologist. Patients are randomly assigned to endoscopic sinus surgery in addition to medication or medical therapy only. This paper details the statistical analysis plan (SAP) of this trial and was submitted before outcome data were available. Results: The primary outcome of the trial is disease-specific Health-Related Quality of Life quantified by the SNOT-22 at 12-months follow-up. Secondary outcomes consist of generic and disease-specific Health-Related Quality of Life, objective signs of disease and adverse events of treatment. Subgroup analyses will be performed to verify if treatment effects differ among patient phenotypes. Analyses will be completed according to this pre-specified SAP. The main analysis will be performed as a standard ITT analysis. Discussion: The PolypESS trial will show whether addition of endoscopic sinus surgery to medical treatment improves the disease-specific Health-Related Quality of Life quantified by the SNOT-22 at 12-months follow-up. Unforeseen deviations from the SAP at the time of analysis will be motivated and discussed in the final publication of the primary outcome of this study.

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Explanatory Factors for Disease-Specific Health-Related Quality of Life in Women with Anorexia Nervosa

Journal of Clinical Medicine ◽

10.3390/jcm10081592 ◽

2021 ◽

Vol 10 (8) ◽

pp. 1592

Author(s):

Laura Al-Dakhiel Winkler ◽

Claire Gudex ◽

Mia Beck Lichtenstein ◽

Michael Ejnar Røder ◽

Carol E. Adair ◽

...

Keyword(s):

Quality Of Life ◽

Eating Disorder ◽

Normal Weight ◽

Health Related Quality ◽

Explanatory Factors ◽

Related Quality ◽

Health Related ◽

Disease Specific ◽

Specific Health

A better understanding of explanatory factors for disease-specific health-related quality of life (HRQoL) in anorexia nervosa (AN) could help direct treatment providers to aspects of the most relevance for patient wellbeing and recovery. We aimed to investigate whether factors associated with HRQoL are the same for women with AN and normal-weight controls. The participants in this study were women with AN recruited from specialized eating disorder centers in Denmark and healthy, normal-weight controls invited via online social media. Participants completed online questionnaires on medical history, disease-specific HRQoL (Eating Disorders Quality of Life Scale, EDQLS) and generic HRQoL (SF-36), eating disorder symptomatology, depression, psychological wellbeing, and work and social adjustment. Questionnaires were fully completed by 211 women with AN (median age 21.7 years) and 199 controls (median age 23.9 years). Women with AN had poorer scores on all measures, i.e., worse HRQoL, psychological health, and work/social functioning. Eating disorder symptomatology affected EDQLS score in both groups, but poorer HRQoL in women with AN was also significantly associated with worse scores on bulimia, maturity fears, depression, vitality, and with older age. The factors investigated together explained 79% of the variance in EDQLS score. Management of disordered self-assessment and thought processes may be of particular importance to women with AN. Greater emphasis on these aspects alongside weight gain could enhance patient–clinician alliance and contribute to better treatment outcomes.

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