Evaluation of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) in Patients With Bronchiectasis

An Open-Label, Multicenter Trial to Validate the Quality of Life Questionnaire-Bronchiectasis and to Evaluate Perception of Symptom Improvement Following One Course of Aztreonam for Inhalation Solution (AZLI) in Subjects With Bronchiectasis and Gram-negative Bacteria in the Airways

Over the 70-day study period, eligible patients visited the study clinic every 2 weeks (total of 6 visits) and received a 28-day course of aztreonam for inhalation solution (AZLI). The Quality of Life-Bronchiectasis (QOL-B) questionnaire was completed at several time points during the study, in additional to pulmonary function testing and other standard procedures.

Study Overview

• Status: Completed
• Conditions: Bronchiectasis
• Intervention / Treatment: Drug: AZLI

• Study Type: Interventional
• Enrollment (Actual): 89
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35209
  • Arizona
    • Peoria, Arizona, United States, 85381
    • Phoenix, Arizona, United States, 85006
  • California
    • Orange, California, United States, 92868
  • Colorado
    • Denver, Colorado, United States, 80206
  • Connecticut
    • Farmington, Connecticut, United States, 06030
    • New Haven, Connecticut, United States, 06519
  • District of Columbia
    • Washington, District of Columbia, United States, 20007
  • Florida
    • Miami, Florida, United States, 33136
  • Georgia
    • Atlanta, Georgia, United States, 30342
    • Marietta, Georgia, United States, 30060
  • Illinois
    • Chicago, Illinois, United States, 60637
  • Kansas
    • Olathe, Kansas, United States, 66061
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
  • Missouri
    • Chesterfield, Missouri, United States, 63017
  • New York
    • Mineola, New York, United States, 11501
    • New York, New York, United States, 10032
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
  • Oregon
    • Portland, Oregon, United States, 97239
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
  • South Carolina
    • Charleston, South Carolina, United States, 29425
  • Texas
    • Tyler, Texas, United States, 75708
  • Virginia
    • Charlottesville, Virginia, United States, 22908
  • Wisconsin
    • Madison, Wisconsin, United States, 53792

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Written informed consent prior to any study-related procedures
• Ability to read and understand the English language
• Bronchiectasis confirmed by CT scan of the chest
• Previous treatment with antibiotics for bronchiectasis
• Documented history of positive sputum culture for a gram-negative organism within 5 years
• Positive sputum culture for a gram-negative organism at first visit (Day -14)

Exclusion Criteria:

• Hospitalization or hemoptysis > 30 mL within 14 days of first visit (Day -14)
• Antibiotic use for respiratory symptoms within 14 days of first visit (Day -14), excluding chronic, stable azithromycin use
• Change in corticosteroid or bronchodilator regimen within 14 days of first visit (Day -14)
• Forced expiratory volume in 1 second (FEV1) < 25% predicted approximately 15 minutes following use of a bronchodilator at first visit (Day -14)
• Cigarette smoking within 6 months of first visit (Day -14)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: AZLI; Participants were evaluated beginning 14 days prior to starting a 28-day course of AZLI (Day 0 to Day 28), followed by post-treatment assessments every 14 days through Day 56, for a total of 70 days of participation in the study.	Drug: AZLI; 75 mg aztreonam for inhalation solution (AZLI), administered 3 times daily using the PARI Investigational eFlow® Nebulizer System (with a minimum of 4 hours between doses) following administration of a short-acting bronchodilator; Other Names: Cayston®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reliability of the Respiratory Domain of the Quality of Life Questionnaire-Bronchiectasis (QOL-B)	Test-retest reliability is a measure of the stability or reproducibility of a measure over a period of time during which status on the underlying construct has not changed, and is measured by the intraclass correlation of scores obtained at 2 time points within that period. Test-retest reliability of respiratory symptoms was calculated for response at Day -14 and Day 0. Reliability of the participants' QOL-B responses was assessed from an Intraclass Correlation Coefficient (ICC). A score of ≥ 0.70 would indicate strong reliability. The QOL-B respiratory symptoms score was transformed onto a scale of 0-100, with higher scores representing a better quality of life.	Day -14 to Day 0
Convergent Validity of the Respiratory Domain of the QOL-B	Convergent validity was assessed at Day -14 by examining the correlations between relevant QOL-B domains and other indicators of health status: a bronchiectasis severity score based on high-resolution computerised tomography (HRCT) scan results, forced expiratory volume in 1 second (FEV1) percent predicted, 6-minute walk test (6MWT) results, and St. George's Respiratory Questionnaire (SGRQ) symptoms scores. Correlations with absolute values of 0.30 to 0.50 indicated moderate evidence of convergent validity.	Day -14

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Responsiveness of the Respiratory Domain of the QOL-B as Assessed by the Anchor-based Minimal Clinically Important Difference (MCID) Following Categorization of Level of Change Using the Global Rating of Change Questionnaire (GRCQ)	The anchor-based method measured the participant's perception of change at Day 28 using the GRCQ, which assesses improving/worsening symptoms. Distribution of ratings was categorized as no change (-1 to 1), minimal change (≥ 1.1 to < 3.1 or ≤ -1.1 to > -3.1), moderate change (≥ 3.1 to < 5.1 or ≤ -3.1 to > -5.1) or large change (≥ 5.1, ≤ -5.1). The GRCQ evaluated change in respiratory symptoms on a visual analog scale from -7 (worsening) to +7 (improvement). The following algorithm was used to obtain the mean change: If the corresponding GRCQ score was in the "no change" group, then change from baseline QOL-B = Observed QOL-B change from baseline score; if > 1, then change from baseline QOL-B score = Observed QOL-B change from baseline score; if < -1, the change from baseline QOL-B score = (-1) * Observed QOL-B change from baseline score. Then the mean change from baseline of QOL-B respiratory symptoms score of the minimal change category group is the anchor-based MCID.	Day 0 to Day 28

Collaborators and Investigators

• Sponsor: Gilead Sciences
• Investigators: Study Director: Steve Lamola, MD, Gilead Sciences

Publications and helpful links

General Publications

• Quittner AL, Marciel KK, Salathe MA, O'Donnell AE, Gotfried MH, Ilowite JS, Metersky ML, Flume PA, Lewis SA, McKevitt M, Montgomery AB, O'Riordan TG, Barker AF. A preliminary quality of life questionnaire-bronchiectasis: a patient-reported outcome measure for bronchiectasis. Chest. 2014 Aug;146(2):437-448. doi: 10.1378/chest.13-1891. Erratum In: Chest. 2014 Nov;146(5):1422.

Study record dates

Study Major Dates

• Study Start: December 1, 2008
• Primary Completion (ACTUAL): October 1, 2009
• Study Completion (ACTUAL): October 1, 2009

Study Registration Dates

• First Submitted: December 5, 2008
• First Submitted That Met QC Criteria: December 8, 2008
• First Posted (ESTIMATE): December 9, 2008

Study Record Updates

• Last Update Posted (ESTIMATE): March 20, 2014
• Last Update Submitted That Met QC Criteria: February 3, 2014
• Last Verified: February 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Bronchial Diseases; Bronchiectasis
• Other Study ID Numbers: GS-US-219-0102