PTC Therapeutics Announces Data Supporting Cough Frequency as a New Outcome Measure in Evaluating Treatments for Cystic Fibrosis
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"Cough is one of the major symptomatic manifestations of the underlyingdisease process in CF," stated Eitan Kerem, M.D., head of the Department ofPediatrics and Cystic Fibrosis Center, Hadassah University Hospital. "Chronicexcessive coughing is a burden on CF patients and a source of anxiety forcaregivers and loved ones. Frequent and intense coughing has a profoundeffect on the overall quality of life of the patient -- compromising work,school, sleep and social interactions."
CF is among the most common life-threatening genetic disorders worldwideand affects nearly 70,000 adults and children. Patients with CF lack thecystic fibrosis transmembrane conductance regulator (CFTR) protein, a chlorideion channel that maintains proper hydration of epithelial cells in the lungs,pancreas, and liver. Loss of the CFTR protein leads to chronic airwayinflammation in association with obstruction and mucopurulent secretions, allof which provoke persistent coughing. While there are approved palliativetreatments for CF, clinical development efforts would benefit from reliablequantitative endpoints that are clinically meaningful because they directlymeasure changes in how a patient feels and functions.
"The ability to quantify cough represents a significant step forward inour understanding of one of the most prominent symptoms of CF," added PrestonCampbell, III, M.D., Executive Vice President of Medical Affairs at the CysticFibrosis Foundation. "A significant reduction in cough frequency would be ofreal value to patients."
The study was designed to assess cough frequency as a measure of clinicalbenefit. Quantitative measurement was achieved using a new technology, theVivoMetrics(R) LifeShirt(R), which integrates cough signals from chest wallmotion transducers and a throat microphone and stores the data for computeranalysis. FEV1 (Forced Expiratory Volume in the first second) and FVC (forcedvital capacity) -- key measures of lung function -- were assessed byspirometry, and patients completed a symptom survey.
Patients included 19 adults not in CF exacerbation. Although there wasoccasional neck pressure due to the throat microphone, compliance wasexcellent, with cough data collected for a median of 24 hours. Coughfrequency averaged 643 coughs per day (ranging from 324 to 1,569 coughs perday) and tended to increase with lower FEV1 and greater age.
"Anecdotal information concerning cough frequency in CF patients led us toassess cough quantitatively using the LifeShirt(R)," said Langdon Miller,M.D., Chief Medical Officer of PTC. "The cough frequency data announced todayhighlights the potential for cough assessment as an outcome measure that maybe used in the development of new therapies for CF. We look forward topresenting data on the effect of PTC124 on cough frequency at the 31st annualEuropean Cystic Fibrosis Society Conference in June, and to initiatinglonger-term trials later this year to further evaluate the clinical efficacyof PTC124 in patients with CF."
About Cystic Fibrosis
Cystic fibrosis (CF) is among the most common life-threatening geneticdisorders worldwide. According to the Cystic Fibrosis Founda
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