Losses in the gains of children with cystic fibrosis who had to
interrupt their modulator therapies: Time flies for half taken breaths!
Abstract
Background: In this study, we aimed to evaluate the losses in
the gains of children who had to discontinue their modulator therapies
due to drug delivery procedures. Methods: Demographic,
clinical, microbiologic, radiologic, and pulmonary function test
parameters of twelve CF children, were evaluated. Parameters were
divided into three groups as ‘before treatment’ (BT), ‘during treatment’
(DT) and ‘after interruption of treatment’ (AT) to show differences
between. Results: There was a significant increase in forced
expiratory volume in 1 s (FEV1) z-score, body mass index (BMI) z-score
and Cystic Fibrosis Questionnaire-Revised respiratory domain score
(CFQR-RS) of DT compared with the values BT (p=0.001, p=0.012,
p<0.001; respectively). It was found that FEV1 z-score, BMI
z-score and CFQR-RS of DT decreased significantly compared with the
values AT (p=0.003, p=0.01, and p<0.001, respectively). When
post and pre-treatment levels were compared, there was no significant
difference between FEV1 z-score (p=0.07), BMI z-score (p=0.56), CFQR-RS
(p=0.7). Half of patients had percent-predicted forced expiratory volume
levels with a drop of more than 20%. It was also detected that
Pseudomonas aeruginosa colonization was a significant factor in
degradation of FEV1 z score to the lower pre-treatment levels.
Conclusion: This is the first retrospective detailed study
about discontinuation of modulatory therapies in children. Our study
shows the importance of treatment continuation as well as the patients
access to these drugs. We hope that this study will raise awareness
about the regular long-term use of modulator therapies. To make these
therapies available worldwide, immediate action is required.