Impact of interruption of CFTR modulator therapies.
J Cyst Fibros
; 23(5): 947-949, 2024 Sep.
Article
in En
| MEDLINE
| ID: mdl-38762388
ABSTRACT
Novel drug therapy targeting the defective cystic fibrosis transmembrane conductance regulator protein has the potential to significantly enhance the quality of life for numerous patients with cystic fibrosis. However, in some countries social insurance does not pay for modulators because these drugs are extremely expensive. This study sought to understand the impact on the health of children whose modulator treatments were interrupted because of legal procedures and delivery processes. Our study identified that the significant increase in percent-predicted forced expiratory volume levels (FEV1) and BMI z-score values associated with modulator therapies decreased sharply with their discontinuation. Significant worsening in FEV1, BMI z-scores, and BW z-scores were detected in the first follow-up visit after therapy discontinuation within 1 month. Eight patients had a reduction of FEV1 of more than 10%. The findings suggest that modulatory treatment continuation is important, and it is crucial that treatment is not interrupted.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Cystic Fibrosis Transmembrane Conductance Regulator
/
Cystic Fibrosis
Limits:
Adolescent
/
Child
/
Female
/
Humans
/
Male
Language:
En
Journal:
J Cyst Fibros
/
J. cyst. fibros
/
Journal of cystic fibrosis
Year:
2024
Document type:
Article
Country of publication: