Šest-minutni test hoda u djece s cističnom fibrozom prije i tijekom liječenja modulatorima transmebranskog regulatora provodljivosti za cističnu fibrozu

master's thesis

• Title (english): Six minute walk test in children with cystic fibrosis before and during treatment with cystic fibrosis transmembrane conductance regulator modulators
• Author: Lovro Hrvoić
• Mentor: Duška Tješić-Drinković (mentor)
• Committee member: Nevena Krnić (predsjednik povjerenstva)
• Committee member: Marija Jelušić (član povjerenstva)
• Committee member: Duška Tješić-Drinković (član povjerenstva)
• Granter: University of Zagreb; School of Medicine; (Department of Pediatrics); Zagreb
• Defense date and country: 2023-07-13, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Pediatrics

Abstract

Uvod. Narušena tjelesna spremnost bitno negativno utječe na kvalitetu života oboljelih od cistične fibroze (CF). Može se procijeniti šest-minutnim testom hoda (6MWT).

Cilj istraživanja. Istražiti povezanost primjene modulatora transmembranskog regulatora provodljivosti za CF (CFTR) s poboljšanjem tjelesne spremnosti.

Metode. Ovo retrospektivno istraživanje provedeno je s podatcima prikupljenim od maloljetnih bolesnika ≥ 5 god. koji su u razdoblju od 15. 10. 2021. do 30. 10. 2022. započeli liječenje eleksakaftorom/tezakaftorom/ivakaftorom (ELX/TEZ/IVA) ili lumakaftorom/ivakaftorom (LUM/IVA) u Centru za cističnu fibrozu djece i odraslih Kliničkog bolničkog centra Zagreb. Ispitanici su praćeni prvih 12 mjeseci liječenja te im je svaka tri mjeseca određen indeks tjelesne mase (BMI), učinjena spirometrija i 6MWT. Praćena je promjena navedenih parametara u tom razdoblju te međuodnos rezultata 6MWT-a i BMI-ja, odnosno plućne funkcije. Nedostajali podatci nadomješteni su primjenom imputacije.

Rezultati. 36 bolesnika (medijan dobi 14 god.) uključeno je u istraživanje; njih 27 liječeno s ELX/TEZ/IVA, a 9 s LUM/IVA. Prosječna duljina hodne pruge na početku liječenja iznosila je 522,1 ± 99,9 m, a nakon 12 mjeseci 592,0 ± 106,3 m (p < 0,001). Pritom je najveće povećanje duljine hodne pruge bilo zabilježeno u prvih šest mjeseci liječenja (p = 0,023). Promjena z-vrijednosti BMI-ja (p = 0,006) i postotka prediktivne vrijednosti FEV1 (ppFEV1) (p < 0,001) tijekom 12 mjeseci praćenja također je bila značajna. Duljina hodne pruge nakon 12 mjeseci liječenja pozitivno je korelirala sa z-vrijednošću BMI-ja (p = 0,002) i ppFEV1 (p < 0,001).

Zaključak. Liječenje CFTR modulatorima praćeno je i klinički i statistički značajnim poboljšanjem tjelesne spremnosti uz pozitivnu korelaciju s poboljšanjem uhranjenosti i plućne funkcije.

Abstract (english)

Background. Exercise tolerance is compromised in many patients with cystic fibrosis (CF) and negatively affects their quality of life. The six-minute walk test (6MWT) can be used to assess exercise tolerance.

Aim. To investigate whether cystic fibrosis transmembrane conductance regulator (CFTR) modulators are associated with an improvement in exercise tolerance.

Methods. This is a retrospective study including children aged ≥ 5 years who began treatment with lumacaftor/ivacaftor (LUM/IVA) or elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) at the Cystic Fibrosis Centre for Children and Adults at the University Hospital Centre Zagreb in the period between 15. 10. 2021. and 30. 10. 2022. Patients were monitored during the first 12 months of treatment; every 3 months body mass index (BMI) was calculated and spirometry and 6MWT were performed. Collected data were analysed for a change in 6MWT results, BMI and percent-predicted FEV1 (ppFEV1) as well as correlation between these parameters. Missing values were replaced using imputation methods.

Results. 36 patients (median age 14 years) were included; 27 received ELX/TEZ/IVA and 9 received LUM/IVA. Average 6MWT walking distance (6MWD) prior to treatment and after 12 months were 522.1 ± 99.9 and 592.0 ± 106.3 m respectively (p < 0.001). Average increase in 6MWD in the first half-year was significantly higher than in the following 6 months (p = 0.023). Changes in BMI z-score (p = 0.006) and ppFEV1 (p < 0.001) during treatment were also significant. 6MWD at 12 months positively correlated with BMI z-score (p = 0.002) and ppFEV1 (p < 0.001).

Conclusions. Both clinically and statistically significant improvement in 6MWT results was visible following initiation of CFTR modulator treatment. A positive correlation with lung function improvement and improvement in BMI was observed.

• Keywords: cistična fibroza; modulatori transmembranskog regulatora provodljivosti za cističnu fibrozu; šest-minutni test hoda
• Keywords (english): cystic fibrosis; cystic fibrosis transmembrane conductance regulator modulators; six-minute walk test
• Language: croatian
• URN:NBN: urn:nbn:hr:105:766956
• Study programme: Title: Medicine; Study programme type: university; Study level: integrated undergraduate and graduate; Academic / professional title: doktor/doktorica medicine (doktor/doktorica medicine)
• Type of resource: Text
• File origin: Born digital
• Access conditions: Open access
• Created on: 2023-10-24 11:34:26