Compassionate use triple therapy CFTR modulation (Kaftrio®) in severe disease. Single-centre, real-world clinical outcomes, safety and tolerability
Journal of Cystic Fibrosis
; 20:S50-S51, 2021.
Article
Dans Anglais
| EMBASE | ID: covidwho-1368823
ABSTRACT
Objective:
Patients (F508del/F508del or F508del/MF) with severe CF (ppFEV1<40%) were not represented in the Phase 3 VX445 (Kaftrio®) [Table presented] clinical trials. We aimed to assess real-world efficacy, safety and tolerability in this cohort.Method:
All patients with severe lung disease (ppFEV1<40%) attending our clinic eligible for Vertex's managed access programme who received treatment were assessed prospectively at 3 monthly intervals.Results:
7 of 8 patients remain on therapy. Irrespective of prior CFTR modulator therapy, notable improvements were observed (Table 1). After adjusting for time, there was a relative reduction of 68.89% IV and 21.43% PO antibiotic usage as well as a 66.9% reduction in hospital bed days. Improvements in CFQR-R and D domains and FACIT fatigue scores were observed. Patient 4 developed an erythematous diffuse rash on day 7 which resolved with antihistamines. No liver function abnormalities have been seen to date. Patient 6 died while on therapy due to multimorbidities including pulmonary embolism (Covid PCR and antibody negative), persistent pneumothorax and sepsis.Conclusion:
This cohort demonstrates preliminary clinically meaningful real-world efficacy, safety and tolerability for patients (F/F, F/MF) with severe disease (ppFEV1<40%).
Texte intégral:
Disponible
Collection:
Bases de données des oragnisations internationales
Base de données:
EMBASE
Type d'étude:
Étude pronostique
langue:
Anglais
Revue:
Journal of Cystic Fibrosis
Année:
2021
Type de document:
Article
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