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Dr. Paul Robinson, Queensland Children’s Hospital, Australia, spoke about transforming the Cystic Fibrosis clinical care landscape at the ERS Congress 2023 in Milan.
Cystic fibrosis in the era of highly effective cystic fibrosis transmembrane conductance regulator modulator therapy
Dr Robinson gave an introduction on Cystic Fibrosis, focusing on CF and modulators for the whole audience. Among others, he provided these facts:
- CF affects at least 100,000 people worldwide, a figure that is likely to be significantly underestimated, as in most of Africa and in many countries in Latin America and Asia there is no available information on cases.
- At least 700 of the more than 2000 variants identified in this gene cause Cystic Fibrosis. (www.cftr2.org).
- The modulators have been developed using cutting-edge technology such as high-throughput screening, capable of assaying the biological or biochemical activity of thousands of compounds to identify those that are active.
- The development of CFTR modulators in both single therapy (ivacaftor or kalydeco) and dual therapy (orkambi and simkevi) or triple therapy (kaftrio or trikafta), especially the latter, is changing the landscape of this disease, with significant improvements in lung function, weight gain, lung exacerbations, etc.
- Good monitoring of long-term effects will be very important.
- More sensitive techniques than spirometry (uninformative in children), such as the lung clearance index (LCI), are incorporated to measure primary outcomes in clinical trials.
- No one should be left behind, so new approaches are needed for people who do not have access to modulators, either because of their mutations, the countries they live in or because they cannot tolerate them.
More information in the Lancet magazine.