PMID- 19447923
OWN - NLM
STAT- MEDLINE
DCOM- 20090707
LR  - 20221129
IS  - 1931-3543 (Electronic)
IS  - 0012-3692 (Print)
IS  - 0012-3692 (Linking)
VI  - 135
IP  - 6
DP  - 2009 Jun
TI  - Determination of the minimal clinically important difference scores for the 
      Cystic Fibrosis Questionnaire-Revised respiratory symptom scale in two 
      populations of patients with cystic fibrosis and chronic Pseudomonas aeruginosa 
      airway infection.
PG  - 1610-1618
LID - S0012-3692(09)60367-6 [pii]
LID - 10.1378/chest.08-1190 [doi]
AB  - BACKGROUND: The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a validated 
      patient-reported outcome (PRO) containing both generic scales and scales specific 
      to cystic fibrosis (CF). The minimal clinically important difference (MCID) score 
      for a PRO corresponds to the smallest clinically relevant change a patient can 
      detect. MCID scores for the CFQ-R respiratory symptom (CFQ-R-Respiratory) scale 
      were determined using data from two 28 day, open-label, tobramycin inhalation 
      solution (TIS) studies in patients with CF and chronic Pseudomonas aeruginosa 
      airway infection. At study enrollment, patients in the study 1-exacerbation had 
      symptoms indicative of pulmonary exacerbation (n = 84; < 14 years of age, 31 
      patients; > or = 14 years of age, 53 patients); patients in study 2-stable had 
      stable respiratory symptoms (n = 140; < 14 years of age, 14 patients; > or = 14 
      years, 126 patients). METHODS: The anchor-based method utilized a global 
      rating-of-change questionnaire (GRCQ) that assessed patients' perceptions of 
      change in their respiratory symptoms after TIS treatment. The mean change from 
      baseline CFQ-R-Respiratory scores were mapped onto the GRCQ to estimate the MCID. 
      The two distribution-based methods were as follows: (1) 0.5 SD of mean change in 
      CFQ-R-Respiratory scores (baseline to end of TIS treatment); and (2) 1 SEM for 
      baseline CFQ-R-Respiratory scores. Triangulation of these three estimates defined 
      the MCIDs. RESULTS: MCID scores were larger for patients in study 1-exacerbation 
      (8.5 points) than for those in study 2-stable (4.0 points), likely reflecting 
      differences in patient disease status (exacerbation/stable) between these 
      studies. CONCLUSIONS: Patient benefit from new and current CF therapies can be 
      evaluated using changes in CFQ-R-Respiratory scores. Using the MCID provides a 
      systematic way to interpret these changes, and facilitates the identification of 
      CF treatments that improve both symptoms and physiologic variables, potentially 
      leading to better treatment adherence and clinical outcomes. Trial registration 
      (study 1-exacerbation): Australian-New Zealand Clinical Trials Registry 
      Identifier: ACTRN 12605000602628 Trial registration (study 2-stable): 
      ClinicalTrials.gov Identifier: NCT00104520.
FAU - Quittner, Alexandra L
AU  - Quittner AL
AD  - University of Miami, Coral Gables, FL. Electronic address: aquittner@miami.edu.
FAU - Modi, Avani C
AU  - Modi AC
AD  - Cincinnati Children's Hospital Medical Center and the University of Cincinnati, 
      Cincinnati, OH.
FAU - Wainwright, Claire
AU  - Wainwright C
AD  - Royal Children's Hospital and University of Queensland, Brisbane, QLD, Australia.
FAU - Otto, Kelly
AU  - Otto K
AD  - Kelly Otto Consulting, Seattle, WA.
FAU - Kirihara, Jean
AU  - Kirihara J
AD  - JKirihara Consulting Services, Seattle, WA.
FAU - Montgomery, A Bruce
AU  - Montgomery AB
AD  - Gilead Sciences Inc, Seattle, WA.
LA  - eng
SI  - ClinicalTrials.gov/NCT00104520
GR  - M01 RR000400/RR/NCRR NIH HHS/United States
GR  - M01 RR00065/RR/NCRR NIH HHS/United States
GR  - M01 RR000034/RR/NCRR NIH HHS/United States
GR  - M01 RR000082/RR/NCRR NIH HHS/United States
GR  - R01 FD003016/FD/FDA HHS/United States
GR  - M01 RR00400/RR/NCRR NIH HHS/United States
GR  - M01 RR000065/RR/NCRR NIH HHS/United States
GR  - M01 RR000043/RR/NCRR NIH HHS/United States
GR  - M01 RR10710/RR/NCRR NIH HHS/United States
GR  - M01 RR00037/RR/NCRR NIH HHS/United States
GR  - M01 RR00070/RR/NCRR NIH HHS/United States
GR  - M01 RR00039/RR/NCRR NIH HHS/United States
GR  - M01 RR000046/RR/NCRR NIH HHS/United States
GR  - M01 RR00188/RR/NCRR NIH HHS/United States
GR  - M01 RR000069/RR/NCRR NIH HHS/United States
GR  - M01 RR01066/RR/NCRR NIH HHS/United States
GR  - M01 RR002172/RR/NCRR NIH HHS/United States
GR  - M01 RR000039/RR/NCRR NIH HHS/United States
GR  - M01 RR000188/RR/NCRR NIH HHS/United States
GR  - M01 RR000037/RR/NCRR NIH HHS/United States
GR  - M01 RR00827/RR/NCRR NIH HHS/United States
GR  - M01 RR00046/RR/NCRR NIH HHS/United States
GR  - M01 RR000750/RR/NCRR NIH HHS/United States
GR  - M01 RR000489/RR/NCRR NIH HHS/United States
GR  - M01 RR00043/RR/NCRR NIH HHS/United States
GR  - M01 RR010710/RR/NCRR NIH HHS/United States
GR  - M01 RR001066/RR/NCRR NIH HHS/United States
GR  - M01 RR00750/RR/NCRR NIH HHS/United States
GR  - M01 RR023940/RR/NCRR NIH HHS/United States
GR  - UL1 TR000454/TR/NCATS NIH HHS/United States
GR  - M01 RR00069/RR/NCRR NIH HHS/United States
GR  - M01 RR00064/RR/NCRR NIH HHS/United States
GR  - M01 RR00042/RR/NCRR NIH HHS/United States
GR  - M01 RR02172/RR/NCRR NIH HHS/United States
GR  - M01 RR10733/RR/NCRR NIH HHS/United States
GR  - M01 RR000042/RR/NCRR NIH HHS/United States
GR  - M01 RR00034/RR/NCRR NIH HHS/United States
GR  - M01 RR001070/RR/NCRR NIH HHS/United States
GR  - M01 RR00082/RR/NCRR NIH HHS/United States
GR  - M01 RR000064/RR/NCRR NIH HHS/United States
GR  - M01 RR000070/RR/NCRR NIH HHS/United States
GR  - M01 RR000827/RR/NCRR NIH HHS/United States
PT  - Comparative Study
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, Non-P.H.S.
PT  - Review
DEP - 20090515
PL  - United States
TA  - Chest
JT  - Chest
JID - 0231335
RN  - 0 (Anti-Bacterial Agents)
SB  - IM
MH  - Administration, Inhalation
MH  - Adolescent
MH  - Age Factors
MH  - Anti-Bacterial Agents/therapeutic use
MH  - Child
MH  - Clinical Trials as Topic
MH  - Cystic Fibrosis/*diagnosis/epidemiology/therapy
MH  - Female
MH  - Follow-Up Studies
MH  - Humans
MH  - Male
MH  - Pneumonia, Bacterial/*diagnosis/drug therapy/epidemiology
MH  - Pseudomonas Infections/*diagnosis/drug therapy/epidemiology
MH  - Pseudomonas aeruginosa/*isolation & purification
MH  - Quality of Life
MH  - Respiratory Function Tests
MH  - Risk Assessment
MH  - Severity of Illness Index
MH  - Sex Factors
MH  - *Surveys and Questionnaires
MH  - Treatment Outcome
MH  - Young Adult
PMC - PMC2821291
EDAT- 2009/05/19 09:00
MHDA- 2009/07/08 09:00
PMCR- 2010/06/01
CRDT- 2009/05/19 09:00
PHST- 2009/05/19 09:00 [entrez]
PHST- 2009/05/19 09:00 [pubmed]
PHST- 2009/07/08 09:00 [medline]
PHST- 2010/06/01 00:00 [pmc-release]
AID - S0012-3692(09)60367-6 [pii]
AID - 08-1190 [pii]
AID - 10.1378/chest.08-1190 [doi]
PST - ppublish
SO  - Chest. 2009 Jun;135(6):1610-1618. doi: 10.1378/chest.08-1190. Epub 2009 May 15.