{
  "abstract": "Introduction The TRAJECTORY Study, an ongoing real-world observational study, previously showed that elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) improves clinical outcomes and quality of life (QoL) in people with CF aged ≥12 years. We examine the effect of ELX/TEZ/IVA on health outcomes among children aged 6–11 years after 12 months of treatment.Methods Medical record data was collected from 42 sites (United Kingdom, Canada, Germany, Italy, Portugal, Spain) for 12 months prior to (baseline) and up to 5 years after ELX/TEZ/IVA initiation (follow-up). Outcomes included body mass index (BMI) BMI z-score, percent predicted forced expiratory volume in one second (ppFEV 1), annualized event rates of pulmonary exacerbations (PEx) and hospitalizations, and QoL as measured by the Cystic Fibrosis Questionnaire-Revised (CFQ-R; Children Ages 6–11 [Interviewer-administered], Parents/Caregivers [proxy for children ages 6–11]). Descriptive analyses were stratified by prior CF transmembrane conductance regulator modulator (CFTRm) use and baseline ppFEV1.Results Among the 163 children aged 6–11 years who initiated ELX/TEZ/IVA between August 2019–June 2023 and had 12 months of follow-up, 49.7% (81/163) were female, 49.1% (80/163) were homozygous for F508del-CFTR, and 23.3% (38/163) had prior CFTRm use. At ELX/TEZ/IVA initiation, the mean (SD) age, BMI z-score, and ppFEV1 were 8.2 (1.6) years, -0.25 (0.85), and 95.2 (14.9). Most had ppFEV1 >90 (62.0%, 101/163) and ppFEV1 ≥70 to ≤90 (31.9%, 52/163). At 12 months, ELZ/TEZ/IVA treatment led to mean (SD) increases in BMI z-score (+0.14 [0.45]) and ppFEV1 (+8.7 percentage points [10.8]), and decreased annualized event rates of PEx (1.1 to 0.6) and hospitalizations (0.6 to 0.2).All CFQ-R domains showed improvement through 12 months regardless of prior CFTR modulator use. Domains with the most improvement observed in mean (SD) change for parents/caregivers were Weight (14.3 [36.0]), Respiratory Symptoms (11.7 [20.6]), Digestive Symptoms (10.0 [8.9]), Eating Problems (9.4 [25.6]), and Treatment Burden (9.4 [21.0]) (table 1); consistent improvements were reported for children ages 6–11 in all domains.Abstract P133 Table 1Summary of Mean CFQ-R Domains at Baseline and Change from BaselineConclusions ELX/TEZ/IVA treatment showed improvement in function and nutritional status, and respiratory and non-respiratory CFQ-R domains for children aged 6–11 years after 12 months of treatment.",
  "authors": [
    {
      "affiliations": [
        "British Columbia Children’s Hospital, University of British Columbia, Vancouver, Canada"
      ],
      "name": "JH Rayment"
    },
    {
      "affiliations": [
        "Ospedale Maggiore Policlinico, Fondazione IRCCS Ca, Milano, Italy"
      ],
      "name": "V Daccò"
    },
    {
      "affiliations": [
        "University of Toronto, Hospital for Sick Children, Toronto, Canada"
      ],
      "name": "F Ratjen"
    },
    {
      "affiliations": [
        "University Hospital Llandough, Penarth, UK"
      ],
      "name": "J Duckers"
    },
    {
      "affiliations": [
        "Hospital Universitario Y Politécnico La Fe, University of Valencia, Valencia, Spain"
      ],
      "name": "A Solé"
    },
    {
      "affiliations": [
        "H. Santa Maria. Centro Hospitalar Lisboa Norte, Lisboa, Portugal"
      ],
      "name": "P Azevedo"
    },
    {
      "affiliations": [
        "Borgo Trento, Ospedale Civile Maggiore, AOU Integrata Verona, Verona, Italy"
      ],
      "name": "M Cipolli"
    },
    {
      "affiliations": [
        "Centre Hospitalier de l, Quebec, Canada"
      ],
      "name": "P Daigneault"
    },
    {
      "affiliations": [
        "University Hospitals of Leicester, Leicestershire, UK"
      ],
      "name": "MI Ahmed"
    },
    {
      "affiliations": [
        "Vertex Pharmaceuticals, Incorporated, Boston, USA"
      ],
      "name": "QN Do"
    },
    {
      "affiliations": [
        "Vertex Pharmaceuticals, Incorporated, Boston, USA"
      ],
      "name": "E Muller"
    },
    {
      "affiliations": [
        "Vertex Pharmaceuticals, Incorporated, Boston, USA"
      ],
      "name": "M Jennings"
    },
    {
      "affiliations": [
        "Department of Pulmonary Medicine, University Hospital Essen – Ruhrlandklinik, Essen, Germany"
      ],
      "name": "S Sutharsan"
    },
    {
      "affiliations": [
        "UK"
      ],
      "name": "TST the TRAJECTORY Study Team"
    }
  ],
  "title": "P133 Impact of elexacaftor/tezacaftor/ivacaftor on clinical outcomes and quality of life in children with cystic fibrosis aged 6–11 years in the real-world setting",
  "uid": "27783fa4-f417-5e70-a87d-ad6f17e11a6d"
}