Clostridium difficile Infection–Daily Symptoms (CDI-DaySyms™) questionnaire: psychometric characteristics and responder thresholds

Abstract

Background The purpose of the current study was to determine the final content validation, psychometric characteristics, clinically meaningful improvement, and responder thresholds of the Clostridium difficile infection (CDI)–Daily Symptoms (CDI-DaySyms™) patient-reported outcome (PRO) questionnaire. Methods This validation study was part of two phase III studies (NCT01987895 and NCT01983683) conducted in patients with mild-to-moderate or severe CDI who completed the CDI-DaySyms™ daily throughout the treatment period. The questionnaire was evaluated in three stages: final PRO item content validation (Stage I); psychometric evaluation of reliability and construct validity (Stage II); and determination of clinically meaningful improvement and responder thresholds using distribution-based methods (Stage III). Results The analysis included 168 patients. Most patients were female and Caucasian with mild-to-moderate CDI. The mean age was 57.1 years. Initial item analysis supported by confirmatory factor analysis demonstrated the relevance of 10 items grouped into three distinct domains (Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms). Domain scores demonstrated acceptable internal consistency and test-retest reliability, were sensitive to change, and correlated in expected directions with other relevant symptom and disease-severity measures. Responder thresholds were defined as score changes of − 1.00, − 0.80, and − 0.70 in the Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms domains, respectively. Conclusions The CDI-DaySyms™ is a valid measure of patient-reported CDI symptoms, with good measurement properties, which supports its utility as an endpoint in clinical studies. Further studies confirming responder thresholds based on anchor-based methods are required. Trial registration NCT01987895 , registered November 20, 2013; NCT01983683 , registered November 14, 2013. Electronic supplementary material The online version of this article (10.1186/s12955-019-1142-9) contains supplementary material, which is available to authorized users.

Recurrent Clostridioides difficile infection worsens anxiety-related patient-reported quality of life

“…Consequently, health-related quality of life (HrQOL) measures seem particularly important in patients with CDI [ 3 , 5 , 10 ]. There are different generic tools created for patient-reported outcomes [ 11 ] and quality of life [ 4 – 7 ], however, Cdiff32 is the only instrument specifically developed to assess HrQOL in patients with CDI [ 3 ]. A recently conducted real-world experience study of bezlotoxumab in outpatient infusion centers [ 9 ] provided an opportunity to assess CDI patient-reported quality of life changes from baseline to follow-up using an adapted Cdiff32 survey.…”

Patient Experiences with Clostridioides difficile Infection and Its Treatment: A Systematic Literature Review

“…Across the 21 studies, humanistic CDI burden was assessed using four generic and two CDI disease-specific PRO measures designed for use in clinical trials. The generic HRQoL measures were EuroQol EQ-5D-3L (index and visual analogue scale [EQ VAS]) [ 25 , 26 , 30 , 37 ], 36-Item Short Form Health Survey (SF-36)/RAND-36 [ 22 , 29 , 38 – 40 ], Short Form 6-Dimension (SF-6D) [ 29 ], and National Health Institute’s Patient-Reported Outcomes Measurement Information System—Global Health (PROMIS-GH) [ 25 , 27 , 28 , 41 ]. The CDI disease-specific PROs included the C. difficile Health-Related Quality-of-Life Questionnaire (Cdiff32) [ 16 , 22 , 25 ] and the Clostridium difficile Infection–Daily Symptoms (CDI-DaySyms) [ 27 , 28 ].…”

“…The decrement increased significantly with CDI severity, worse for women, and those > 65 years of age. The QALY loss for CDI episode, not integrating excess mortality, was 0.028 (SD 0.053) Paul et al [ 30 ] 2019 69 USA ED-5D-3L Current CDI Antibiotics: ridinilazole vs vancomycin Patients receiving ridinilazole had significant improvement in EQ-5D-3L mean change from baseline in index and EQ VAS scores as early as day 5, but no significant improvements with vancomycin (mean scores not reported) Talbot et al [ 27 ] 2019 168 USA, Canada, Australia, Korea, and Europe CDI-DaySyms ADL, GSRS, PROMIS global items Patients with mild to moderate or severe CDI: pCDI or first recurrence Psychometric validation of the newly developed CDI-DaySyms, a 10-item, 3-domain, CDI disease-specific PRO assessing abdominal symptoms, pain, and systemic/other symptoms Heinrich et al [ 29 ] 2018 2411 USA, EU (UK, France, Germany, Italy, and Spain), China, and Brazil SF-36, SF-6D, WPAI ≥ 18 years old either currently being treated ( n = 299), previously treated ( n = 2111), or never diagnosed with CDI ( n = 350,370) (NHWS data) Current CDI and prior CDI had significantly lower HRQoL relative to never having CDI for SF-36 PCS and MCS and SF-6D (utility). Current CDI and prior CDI missed more work and greater impairment while working and more activity impairment than never having CDI.…”

“…For the purposes of this review, the term CDI encompasses primary CDI (pCDI), rCDI, or current history of CDI. HRQoL data differentiated by CDI recurrence have been presented where available, see for example Han et al [ 25 ] and Garey et al [ 22 ]; however, data were frequently pooled without differentiating by CDI recurrence (see for example, Wilcox et al [ 26 ], Talbot et al [ 27 ], Kleinman et al [ 28 ]) or CDI status (current, prior, or no CDI) (see Heinrich et al [ 29 ] and Paul et al [ 30 ]). Excluded were papers solely measuring treatment efficacy, economic outcomes, clinician-reported outcomes, reviews, or commentaries.…”

Genomic characterization of nine Clostridioides difficile strains isolated from Korean patients with Clostridioides difficile infection

“…NAP1/ribotype 027) synthesize the actin-ADP-ribosylating toxin known as binary toxin C. difficile transferase (CDT), which leads to the depolymerization of actin filaments, disrupting the actin cytoskeleton in the cytosol [ 16 – 19 ]. Therefore, the toxins A, B, and CDT can cause severe CDI symptoms [ 20 ].…”