PMID- 20926506
OWN - NLM
STAT- MEDLINE
DCOM- 20110222
LR  - 20231213
IS  - 1753-4666 (Electronic)
IS  - 1753-4658 (Linking)
VI  - 4
IP  - 6
DP  - 2010 Dec
TI  - Retinoid treatment of Emphysema in Patients on the Alpha-1 International 
      Registry. The REPAIR study: study design, methodology and quality control of 
      study assessments.
PG  - 319-32
LID - 10.1177/1753465810379617 [doi]
AB  - Emphysema is characterized by the destruction of alveolar wall and enlargement of 
      alveolar airspaces, resulting in a reduction of the total lung gas exchange area, 
      loss of lung elastic recoil and hyperinflation. The REPAIR study (Retinoid 
      treatment of Emphysema in Patients on the Alpha-1 International Registry) is the 
      first proof-of-concept study of a new potential disease-modifying drug, 
      Palovarotene(c), an orally active, gamma selective retinoid agonist in patients 
      with emphysema secondary to alpha-1-antitrypsin deficiency (AATD) as a model 
      population for the general smoke-induced emphysema population. This article 
      describes the study design as well as the effectiveness of the quality control 
      that was implemented on the key efficacy endpoints, based on data derived from 
      the placebo-treated subjects. In this multicentre, multinational study the 
      implementation of standardized procedures included: careful site selection, use 
      of trained staff, regular monitoring and machine calibration, use of biological 
      controls and regular feedback to sites by an independent quality control centre. 
      All of these procedures resulted in high-quality measurements of lung density, 
      spirometry, static lung volumes and gas transfer. It was also confirmed that CT 
      lung density was the most sensitive endpoint followed by TLco, FEV(1) and RV 
      measured by body box.
FAU - Stolk, Jan
AU  - Stolk J
AD  - Leiden University Medical Center, P.O. Box 9600, 2300 RC Leiden, The Netherlands.
FAU - Cooper, Brendan G
AU  - Cooper BG
FAU - Stoel, Berend
AU  - Stoel B
FAU - Rames, Alexis
AU  - Rames A
FAU - Rutman, Olga
AU  - Rutman O
FAU - Soliman, Sherif
AU  - Soliman S
FAU - Stockley, Robert
AU  - Stockley R
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20101006
PL  - England
TA  - Ther Adv Respir Dis
JT  - Therapeutic advances in respiratory disease
JID - 101316317
RN  - 0 (Pyrazoles)
RN  - 0 (Receptors, Retinoic Acid)
RN  - 0 (Stilbenes)
RN  - 28K6I5M16G (Palovarotene)
SB  - IM
MH  - Administration, Oral
MH  - Double-Blind Method
MH  - Emphysema
MH  - Female
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Pulmonary Emphysema/*drug therapy/etiology/physiopathology
MH  - Pyrazoles/pharmacology/*therapeutic use
MH  - Quality Control
MH  - Receptors, Retinoic Acid/*agonists
MH  - Research Design
MH  - Respiratory Function Tests
MH  - Stilbenes/pharmacology/*therapeutic use
MH  - alpha 1-Antitrypsin Deficiency/*complications
MH  - Retinoic Acid Receptor gamma
EDAT- 2010/10/12 06:00
MHDA- 2011/02/23 06:00
CRDT- 2010/10/08 06:00
PHST- 2010/10/08 06:00 [entrez]
PHST- 2010/10/12 06:00 [pubmed]
PHST- 2011/02/23 06:00 [medline]
AID - 1753465810379617 [pii]
AID - 10.1177/1753465810379617 [doi]
PST - ppublish
SO  - Ther Adv Respir Dis. 2010 Dec;4(6):319-32. doi: 10.1177/1753465810379617. Epub 
      2010 Oct 6.