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Key clinical point: Lumacaftor-ivacaftor treatment for 120 weeks improved exocrine pancreatic insufficiency and pancreatic damage in children as young as 2-5 years of age with cystic fibrosis (CF) homozygous for F508del-CF transmembrane conductance regulator gene.
Major finding: At baseline, 100% of participants had fecal elastase-1 (FE-1) less than 200 µg/g, of which 41 participants had FE-1 less than 15 µg/g. Treatment with lumacaftor-ivacaftor increased FE-1 (mean absolute change [D], 132.6±174.2 µg/g) and decreased immunoreactive trypsinogen (D, −108.5±306.6 ng/mL) from baseline to 120 weeks.
Study details: This phase 3 rollover trial (study 116) included 57 children with CF who completed 24 weeks of lumacaftor-ivacaftor treatment in previous study 115. Patients received weight- and age-based doses of lumacaftor-ivacaftor for additional 96 weeks, totaling 120 weeks of treatment.
Disclosures: This study was funded by Vertex Pharmaceuticals Incorporated (VPI). VPI was funded by CF Foundation for lumacaftor development. Some investigators including the lead author reported ties with VPI and CF Foundation.
Source: Hoppe JE et al. Lancet Respir Med. 2021 May 6. doi: 10.1016/S2213-2600(21)00069-2.
Key clinical point: Lumacaftor-ivacaftor treatment for 120 weeks improved exocrine pancreatic insufficiency and pancreatic damage in children as young as 2-5 years of age with cystic fibrosis (CF) homozygous for F508del-CF transmembrane conductance regulator gene.
Major finding: At baseline, 100% of participants had fecal elastase-1 (FE-1) less than 200 µg/g, of which 41 participants had FE-1 less than 15 µg/g. Treatment with lumacaftor-ivacaftor increased FE-1 (mean absolute change [D], 132.6±174.2 µg/g) and decreased immunoreactive trypsinogen (D, −108.5±306.6 ng/mL) from baseline to 120 weeks.
Study details: This phase 3 rollover trial (study 116) included 57 children with CF who completed 24 weeks of lumacaftor-ivacaftor treatment in previous study 115. Patients received weight- and age-based doses of lumacaftor-ivacaftor for additional 96 weeks, totaling 120 weeks of treatment.
Disclosures: This study was funded by Vertex Pharmaceuticals Incorporated (VPI). VPI was funded by CF Foundation for lumacaftor development. Some investigators including the lead author reported ties with VPI and CF Foundation.
Source: Hoppe JE et al. Lancet Respir Med. 2021 May 6. doi: 10.1016/S2213-2600(21)00069-2.
Key clinical point: Lumacaftor-ivacaftor treatment for 120 weeks improved exocrine pancreatic insufficiency and pancreatic damage in children as young as 2-5 years of age with cystic fibrosis (CF) homozygous for F508del-CF transmembrane conductance regulator gene.
Major finding: At baseline, 100% of participants had fecal elastase-1 (FE-1) less than 200 µg/g, of which 41 participants had FE-1 less than 15 µg/g. Treatment with lumacaftor-ivacaftor increased FE-1 (mean absolute change [D], 132.6±174.2 µg/g) and decreased immunoreactive trypsinogen (D, −108.5±306.6 ng/mL) from baseline to 120 weeks.
Study details: This phase 3 rollover trial (study 116) included 57 children with CF who completed 24 weeks of lumacaftor-ivacaftor treatment in previous study 115. Patients received weight- and age-based doses of lumacaftor-ivacaftor for additional 96 weeks, totaling 120 weeks of treatment.
Disclosures: This study was funded by Vertex Pharmaceuticals Incorporated (VPI). VPI was funded by CF Foundation for lumacaftor development. Some investigators including the lead author reported ties with VPI and CF Foundation.
Source: Hoppe JE et al. Lancet Respir Med. 2021 May 6. doi: 10.1016/S2213-2600(21)00069-2.