REAL‐LIFE EXPERIENCE WITH A GENERIC FORMULATION OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR IN CHILDREN WITH CYSTIC FIBROSIS��
Authors: Oneglia M1; Giugno H1; Agustinho A2; Plubatsch P3; Crespi N4; Castaños C1
�1Department of Pulmonology, 2Department of Clinical Nutrition, 3Department of Physical therapy, 4Department of Nursing�Hospital de Pediatria Dr Prof JP Garrahan, Buenos Aires, Argentina
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INTRODUCTION�Cystic Fibrosis (CF) is an autosomal recessive disease. Triple combination therapy, such as elexacaftor/tezacaftor/ivacaftor (ETI), could�change the disease course and life of people with CF (pwCF). Argentina has provided a generic ETI formulation since 2021.
OBJECTIVE�To report real-life effects of a generic ETI formulation in pwCF.
MATERIALS AND METHODS�PwCF, ≥ 6 years, receiving ETI for ≥12 months at the CF center of J. P. Garrahan Hospital. Variables analyzed: lung function (ppFEV1), nutritional status (BMI z-score), sweat test (ST), respiratory exacerbations, microbiology findings in sputum samples, and quality of life �(CFQ-R); comparisons were made before ETI initiation and after 12 months of treatment.
RESULTS�A total of 40 pwCF were included; 65% were female. The median age at the start of treatment was 12 years (9.9-14.7). Overall, 21 (52.5%), patients were F508del heterozygotes, 18 (45%) F508del homozygotes, and one patient (2.5%) had another gene mutation included in ETI indications. In 25 patients no previous combination therapy was used (62.5%) (Table 1).�The median ppFEV1 improved from 79.75% (62 - 90) to 100.5% (76 - 107) (p=0.00) (change of +26.01%) after 12 months (Figure1). �The BMI z-score was -0.32 (-0.91 to 0.27), increasing to -0.07 (-0.76 to 0.30) (p=0.11) (Figure 2).
The median decrease in the ST after treatment was 49.5 meq/L. Severe and mild respiratory exacerbations reduced in the first year. Sputum cultures showed a decrease in Pseudomonas aeruginosa and Burkholderia cepacia. The CFQ-R respiratory domain improved post-ETI.
Adverse events were reported in only four patients: 1 skin rash, 1 mood changes, 1 CPK increase, 1 increased liver enzymes; in the latter two
treatment was discontinued until laboratory values normalized and, subsequently, they were able to continue ETI without complications.
Figure 2. Individual changes in nutritional status after receiving a generic formulation ETI
CONCLUSION�As previously reported for the triple combination therapy, the Argentine generic formulation was found to be effective and safe.
Figure 1. Individual changes in lung function after receiving a generic formulation of ETI
Table 1. Results