Progress in cystic fibrosis and the CF Therapeutics Development Network

被引：50

作者：

Rowe, Steven M. ^{[1
]}

Borowitz, Drucy S. ^{[2
]}

Burns, Jane L. ^{[3
,4
]}

Clancy, John P. ^{[5
,6
]}

Donaldson, Scott H. ^{[7
]}

Retsch-Bogart, George ^{[7
]}

Sagel, Scott D. ^{[8
,9
]}

Ramsey, Bonnie W. ^{[3
,4
]}

机构：

[1] Univ Alabama Birmingham, Dept Med, Birmingham, AL 35294 USA

[2] SUNY Buffalo, Dept Pediat, Buffalo, NY 14260 USA

[3] Seattle Childrens Hosp, Seattle, WA USA

[4] Univ Washington, Seattle, WA 98195 USA

[5] Cincinnati Childrens Hosp, Med Ctr, Dept Pediat, Cincinnati, OH USA

[6] Univ Cincinnati, Cincinnati, OH USA

[7] Univ N Carolina, Chapel Hill, NC USA

[8] Childrens Hosp Colorado, Dept Pediat, Aurora, CO USA

[9] Univ Colorado, Aurora, CO USA

来源：

THORAX | 2012年 / 67卷 / 10期

关键词：

TRANSMEMBRANE CONDUCTANCE REGULATOR; PSEUDOMONAS-AERUGINOSA INFECTION; NASAL POTENTIAL DIFFERENCE; EXOCRINE PANCREATIC INSUFFICIENCY; INHALED HYPERTONIC SALINE; DRY POWDER MANNITOL; HIGH-DOSE IBUPROFEN; CLINICAL-TRIALS; DOUBLE-BLIND; YOUNG-CHILDREN;

D O I：

10.1136/thoraxjnl-2012-202550

中图分类号：

R56 [呼吸系及胸部疾病];

学科分类号：

摘要：

Cystic fibrosis (CF), the most common life-shortening genetic disorder in Caucasians, affects approximately 70 000 individuals worldwide. In 1998, the Cystic Fibrosis Foundation (CFF) launched the CF Therapeutics Development Network (CF-TDN) as a central element of its Therapeutics Development Programme. Designed to accelerate the clinical evaluation of new therapies needed to fulfil the CFF mission to control and cure CF, the CF-TDN has conducted 75 clinical trials since its inception, and has contributed to studies as varied as initial safety and proof of concept trials to pivotal programmes required for regulatory approval. This review highlights recent and significant research efforts of the CF-TDN, including a summary of contributions to studies involving CF transmembrane conductance regulator (CFTR) modulators, airway surface liquid hydrators and mucus modifiers, anti-infectives, anti-inflammatories, and nutritional therapies. Efforts to advance CF biomarkers, necessary to accelerate the therapeutic goals of the network, are also summarised.

引用

页码：882 / 890

页数：9

共 125 条

[1] Denufosol Tetrasodium in Patients with Cystic Fibrosis and Normal to Mildly Impaired Lung Function [J].

Accurso, Frank J. ;

Moss, Richard B. ;

Wilmott, Robert W. ;

Anbar, Ran D. ;

Schaberg, Amy E. ;

Durham, Todd A. ;

Ramsey, Bonnie W. .

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2011, 183 (05) :627-634

[2] Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation [J].

Accurso, Frank J. ;

Rowe, Steven M. ;

Clancy, J. P. ;

Boyle, Michael P. ;

Dunitz, Jordan M. ;

Durie, Peter R. ;

Sagel, Scott D. ;

Hornick, Douglas B. ;

Konstan, Michael W. ;

Donaldson, Scott H. ;

Moss, Richard B. ;

Pilewski, Joseph M. ;

Rubenstein, Ronald C. ;

Uluer, Ahmet Z. ;

Aitken, Moira L. ;

Freedman, Steven D. ;

Rose, Lynn M. ;

Mayer-Hamblett, Nicole ;

Dong, Qunming ;

Zha, Jiuhong ;

Stone, Anne J. ;

Olson, Eric R. ;

Ordonez, Claudia L. ;

Campbell, Preston W. ;

Ashlock, Melissa A. ;

Ramsey, Bonnie W. .

NEW ENGLAND JOURNAL OF MEDICINE, 2010, 363 (21) :1991-2003

[3]

Aherns R, 2011, PED PULM S, V46, pA198

[4]

Ahrens R, 2011, PEDIATR PULM, V34, P283

[5] Use of nasal potential difference and sweat chloride as outcome measures in multicenter clinical trials in subjects with cystic fibrosis [J].

Ahrens, RC ;

Standaert, TA ;

Launspach, J ;

Han, SH ;

Teresi, ME ;

Aitken, ML ;

Kelley, TJ ;

Hilliard, KA ;

Milgram, LJH ;

Konstan, MW ;

Weatherly, MR ;

McCarty, NA .

PEDIATRIC PULMONOLOGY, 2002, 33 (02) :142-150

[6] Long-Term Inhaled Dry Powder Mannitol in Cystic Fibrosis An International Randomized Study [J].

Aitken, Moira L. ;

Bellon, Gabriel ;

De Boeck, Kris ;

Flume, Patrick A. ;

Fox, Howard G. ;

Geller, David E. ;

Haarman, Eric G. ;

Hebestreit, Helge U. ;

Lapey, Allen ;

Schou, I. Manjula ;

Zuckerman, Jonathan B. ;

Charlton, Brett .

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2012, 185 (06) :645-652

[7]

[Anonymous], J CYSTIC FIBROSIS S1

[8]

[Anonymous], 2011, J CYST FIBROS, V10, P491

[9] ACUTE AND LONG-TERM AMILORIDE INHALATION IN CYSTIC-FIBROSIS LUNG-DISEASE - A RATIONAL APPROACH TO CYSTIC-FIBROSIS THERAPY [J].

APP, EM ;

KING, M ;

HELFESRIEDER, R ;

KOHLER, D ;

MATTHYS, H .

AMERICAN REVIEW OF RESPIRATORY DISEASE, 1990, 141 (03) :605-612

[10] Suppression of a CFTR premature stop mutation in a bronchial epithelial cell line [J].

Bedwell, DM ;

Kaenjak, A ;

Benos, DJ ;

Bebok, Z ;

Bubien, JK ;

Hong, J ;

Tousson, A ;

Clancy, JP ;

Sorscher, EJ .

NATURE MEDICINE, 1997, 3 (11) :1280-1284

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