ArticleScientific reports2025
Blood platelet reduction after elexacaftor/tezacaftor/ivacaftor treatment in people with cystic fibrosis may depend on systemic inflammation reduction.
Article in Scientific reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
The elexacaftor/tezacaftor/ivacaftor (ETI) combination for cystic fibrosis transmembrane regulator modulators is a safe and effective treatment in both adults and children who are homozygous or compound heterozygous for the F508del variant. However, few cases involving a significant reduction in blood platelets and an increase in the alanine aminotransferase/platelet ratio have been described in adult and pediatric patients receiving ETI therapy. In the present study, we describe 272 people with cystic fibrosis (pwCF) (166 adult and 106 pediatric pwCF) who were independently followed at two centers; moreover, these individuals were homozygous or compound heterozygous for the F508del variant, were treated with ETI for at least one year, and exhibited monitored platelet and leukocyte counts (together with liver and inflammatory biochemical indices). As controls, 272 healthy subjects (HCs) matched for sex and age were evaluated. At baseline, both adult and pediatric pwCF demonstrated significantly (p < 0.01) greater blood platelet and leukocyte counts compared with HCs. One year of treatment significantly reduced blood platelet counts (adults: 248*10
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