Patient-reported outcomes to assess the efficacy of extended-release guaifenesin for the treatment of acute respiratory tract infection symptoms

Respiratory Research

Gail Solomon¹  Margaret Vernon³  Helmut Albrecht² 

[1] Reckitt Benckiser Inc., 399 Interpace Parkway, Parsippany, NJ, 07054-0225, USA;H2A Associates, LLC, 3350 SW 27th Ave, Ste 1803, Miami, FL, 33133, USA;United BioSource, 7101 Wisconsin Ave, Ste 600, Bethesda, MD, 20814, USA

关键词: Thickened mucus;    Upper respiratory tract infections;    Content validity;    Psychometric;    Validation;    Symptom improvement;    Patient-reported outcomes;    Clinical methods development;    Extended-release guaifenesin;    Expectorant;   

Others  :  796570
DOI  :  10.1186/1465-9921-13-118

 received in 2012-09-10, accepted in 2012-12-13,  发布年份 2012

【 摘 要 】

Background

Guaifenesin is a component of medicines used to improve symptoms associated with upper respiratory tract infections. Patient-reported outcome instruments are valuable for evaluating symptom improvements; however, a validated tool to assess efficacy of mucoactive drugs does not exist. We compared the efficacy of extended-release guaifenesin with placebo for treatment of symptoms of upper respiratory tract infection using subjective efficacy assessments in a pilot study and confirmed precision of assessments in a validation study.

Methods

The pilot study was a randomized, double-blind study where patients were dosed with either 1200 mg extended-release guaifenesin (n = 188) or placebo (n = 190), every 12 hours for 7 days. Efficacy was assessed using subjective measures including the Daily Cough and Phlegm Diary, the Spontaneous Symptom Severity Assessment and the Wisconsin Upper Respiratory Symptom Survey. End-of-study assessments were completed by patients and investigator. The validation study consisted of two phases. In Phase I, subjects completed interviews to gather evidence to support the content validity of the Daily Cough and Phlegm Diary, the Spontaneous Symptom Severity Assessment and Patient’s End-of-Treatment Assessment. Phase II examined the psychometric properties of assessments evaluated in Phase I of the validation study using data from the pilot study.

Results

Subjective measures of efficacy at Day 4 showed the most prominent difference between treatment groups, in favor of guaifenesin. The 8-symptom related questions (SUM8) in the Daily Cough and Phlegm Diary, analyzed as a composite score appeared to be the strongest candidate endpoint for further evaluation. Results from the interviews in Phase I supported the content of the assessments which were validated during Phase II. Treatments were well tolerated.

Conclusions

Results from the clinical pilot and validation studies showed that the SUM8 diary scores were robust and reliable for use as efficacy endpoints in studies of mucoactive drugs.

Trial registration

The study was registered with clinicaltrials.gov (NCT01046136).

【 授权许可】

   
2012 Albrecht et al.; licensee BioMed Central Ltd.

【 图 表 】

Figure 1.

Figure 2.

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