: There is a significant and positive relationship between the lower- and higher-level goals. Healthcare providers can work with their patients to achieve their goals. Both good communication with the prescriber and the effectiveness of the drug product were identified as the most important facilitator by one-third of the respondents. Future research should study if relating the impact of good symptom control or the reduction of future health risks to QOL or longevity, as deemed relevant by the patient, influences medication adherence behavior.
Therapy for cystic fibrosis (CF) has progressed during the past several decades. Much of this progress is because of advances in genetic testing to precisely identify the underlying cause of CF transmembrane regulator (CFTR) dysfunction. However, with more than 1900 mutations that can produce a faulty CFTR, the management of CF can remain a challenge. Several innovative drugs recently approved by the Food and Drug Administration, termed genetic modulators, target the underlying disease by modulating the CFTR defect. This review provides physicians with an established simple classification scheme to guide their use of these drugs. The treatment challenge of 1900 CFTR mutations has been simplified into 6 physiologic classes, each paired with an available therapy to offer patients the most functional improvement. Drug therapy monitoring, adverse effects, and indications for discontinuation must also be considered.
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