• Something wrong with this record ?

Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor

N. Stastna, L. Kunovsky, M. Svoboda, E. Pokojova, L. Homola, M. Mala, Z. Gracova, B. Jerabkova, J. Skrickova, J. Trna

. 2024 ; 42 (4) : 361-368. [pub] 20240403

Language English Country Switzerland

Document type Journal Article

Persistent link   https://www.medvik.cz/link/bmc24020045

INTRODUCTION: Cystic fibrosis transmembrane conductance regulator modulator therapy improves nutritional status and quality of life. Clinical trials have shown pancreatic insufficiency conversion, mostly in pediatric patients treated with ivacaftor. Studies with elexacaftor/tezacaftor/ivacaftor (ETI) in older patients have not suggested restoration of exocrine pancreas function, but quality data in adults are lacking. Our aim was to show the effect of ETI in adults with cystic fibrosis (CF) on nutritional status and digestive function. We hypothesized improvement of nutritional parameters and gastrointestinal symptoms, reduction of pancreatic enzyme replacement therapy, but uncertain improvement in exocrine pancreatic function. METHODS: We prospectively enrolled adults with CF treated with ETI from August 2021 to June 2022. We measured anthropometric parameters, laboratory nutritional markers, change of fecal elastase, pancreatic enzymes replacement therapy needs, and gastrointestinal symptoms. RESULTS: In the cohort of 29 patients (mean age 29.1 years), 82.8% suffered exocrine pancreatic insufficiency. After ETI, mean BMI increased by 1.20 kg/m2 (p < 0.001), mean body weight by 3.51 kg (p < 0.001), albumin by 2.81 g/L, and prealbumin by 0.06 (both p < 0.001). Only 1 patient, initially pancreatic insufficient (4.5%, p < 0.001), developed pancreatic sufficiency, indicated by increased fecal elastase from 45 μg/g to 442.1 μg/g. Mean change in lipase substitution decreased by 1,969 units/kg/day (p < 0.001) and stools frequency by 1.18 per day (p < 0.001). CONCLUSION: Our data suggest increased nutritional parameters, lower pancreatic substitution requirements, and improved defecation in adult CF patients on ETI. Improvement in exocrine pancreatic function might be mutation-specific and needs further study.

References provided by Crossref.org

000      
00000naa a2200000 a 4500
001      
bmc24020045
003      
CZ-PrNML
005      
20241024110900.0
007      
ta
008      
241015s2024 sz f 000 0|eng||
009      
AR
024    7_
$a 10.1159/000538606 $2 doi
035    __
$a (PubMed)38569478
040    __
$a ABA008 $b cze $d ABA008 $e AACR2
041    0_
$a eng
044    __
$a sz
100    1_
$a Stastna, Nela $u Department of Pneumology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czechia $u Faculty of Medicine, Masaryk University, Brno, Czechia
245    10
$a Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor / $c N. Stastna, L. Kunovsky, M. Svoboda, E. Pokojova, L. Homola, M. Mala, Z. Gracova, B. Jerabkova, J. Skrickova, J. Trna
520    9_
$a INTRODUCTION: Cystic fibrosis transmembrane conductance regulator modulator therapy improves nutritional status and quality of life. Clinical trials have shown pancreatic insufficiency conversion, mostly in pediatric patients treated with ivacaftor. Studies with elexacaftor/tezacaftor/ivacaftor (ETI) in older patients have not suggested restoration of exocrine pancreas function, but quality data in adults are lacking. Our aim was to show the effect of ETI in adults with cystic fibrosis (CF) on nutritional status and digestive function. We hypothesized improvement of nutritional parameters and gastrointestinal symptoms, reduction of pancreatic enzyme replacement therapy, but uncertain improvement in exocrine pancreatic function. METHODS: We prospectively enrolled adults with CF treated with ETI from August 2021 to June 2022. We measured anthropometric parameters, laboratory nutritional markers, change of fecal elastase, pancreatic enzymes replacement therapy needs, and gastrointestinal symptoms. RESULTS: In the cohort of 29 patients (mean age 29.1 years), 82.8% suffered exocrine pancreatic insufficiency. After ETI, mean BMI increased by 1.20 kg/m2 (p &lt; 0.001), mean body weight by 3.51 kg (p &lt; 0.001), albumin by 2.81 g/L, and prealbumin by 0.06 (both p &lt; 0.001). Only 1 patient, initially pancreatic insufficient (4.5%, p &lt; 0.001), developed pancreatic sufficiency, indicated by increased fecal elastase from 45 μg/g to 442.1 μg/g. Mean change in lipase substitution decreased by 1,969 units/kg/day (p &lt; 0.001) and stools frequency by 1.18 per day (p &lt; 0.001). CONCLUSION: Our data suggest increased nutritional parameters, lower pancreatic substitution requirements, and improved defecation in adult CF patients on ETI. Improvement in exocrine pancreatic function might be mutation-specific and needs further study.
650    _2
$a lidé $7 D006801
650    12
$a cystická fibróza $x farmakoterapie $x komplikace $7 D003550
650    _2
$a mužské pohlaví $7 D008297
650    _2
$a dospělí $7 D000328
650    _2
$a ženské pohlaví $7 D005260
650    12
$a nutriční stav $7 D009752
650    12
$a exokrinní pankreatická insuficience $x farmakoterapie $x etiologie $x komplikace $7 D010188
650    12
$a indoly $x terapeutické užití $7 D007211
650    12
$a fixní kombinace léků $7 D004338
650    _2
$a benzodioxoly $x terapeutické užití $7 D052117
650    _2
$a prospektivní studie $7 D011446
650    _2
$a aminofenoly $x terapeutické užití $7 D000627
650    _2
$a mladý dospělý $7 D055815
650    _2
$a pyridiny $x terapeutické užití $7 D011725
650    _2
$a chinolony $x terapeutické užití $7 D015363
650    _2
$a pyrazoly $x terapeutické užití $7 D011720
650    _2
$a výsledek terapie $7 D016896
650    _2
$a pyrrolidiny $x terapeutické užití $7 D011759
650    _2
$a gastrointestinální nemoci $x farmakoterapie $7 D005767
650    _2
$a pankreatická elastasa $x metabolismus $7 D010196
650    _2
$a chinoliny $7 D011804
655    _2
$a časopisecké články $7 D016428
700    1_
$a Kunovsky, Lumir $u Faculty of Medicine, Masaryk University, Brno, Czechia $u Department of Surgery, University Hospital Brno, Brno, Czechia $u Faculty of Medicine and Dentistry, Palacky University Olomouc, Olomouc, Czechia $u 2nd Department of Internal Medicine - Gastroenterology and Geriatrics, University Hospital Olomouc, Olomouc, Czechia $u Department of Gastroenterology and Digestive Endoscopy, Masaryk Memorial Cancer Institute, Brno, Czechia
700    1_
$a Svoboda, Michal $u Faculty of Medicine, Masaryk University, Brno, Czechia $u Institute of Biostatistics and Analyses s.r.o., Brno, Czechia
700    1_
$a Pokojova, Eva $u Faculty of Medicine, Masaryk University, Brno, Czechia $u Department of Respiratory Diseases, University Hospital Brno, Brno, Czechia
700    1_
$a Homola, Lukas $u Faculty of Medicine, Masaryk University, Brno, Czechia $u Department of Children's Infectious Diseases, University Hospital Brno, Brno, Czechia
700    1_
$a Mala, Miriam $u Faculty of Medicine, Masaryk University, Brno, Czechia $u Department of Children's Infectious Diseases, University Hospital Brno, Brno, Czechia
700    1_
$a Gracova, Zaneta $u Department of Children's Infectious Diseases, University Hospital Brno, Brno, Czechia
700    1_
$a Jerabkova, Barbora $u Department of Children's Infectious Diseases, University Hospital Brno, Brno, Czechia
700    1_
$a Skrickova, Jana $u Faculty of Medicine, Masaryk University, Brno, Czechia $u Department of Respiratory Diseases, University Hospital Brno, Brno, Czechia
700    1_
$a Trna, Jan $u Faculty of Medicine, Masaryk University, Brno, Czechia $u Department of Gastroenterology and Digestive Endoscopy, Masaryk Memorial Cancer Institute, Brno, Czechia
773    0_
$w MED00173707 $t Digestive diseases (Basel, Switzerland) $x 1421-9875 $g Roč. 42, č. 4 (2024), s. 361-368
856    41
$u https://pubmed.ncbi.nlm.nih.gov/38569478 $y Pubmed
910    __
$a ABA008 $b sig $c sign $y - $z 0
990    __
$a 20241015 $b ABA008
991    __
$a 20241024110854 $b ABA008
999    __
$a ok $b bmc $g 2202339 $s 1232018
BAS    __
$a 3
BAS    __
$a PreBMC-MEDLINE
BMC    __
$a 2024 $b 42 $c 4 $d 361-368 $e 20240403 $i 1421-9875 $m Digestive diseases (Basel, Switzerland) $n Dig Dis $x MED00173707
LZP    __
$a Pubmed-20241015

Find record

Citation metrics

Archiving options