Background: Modulator therapies improve weight and body mass index (BMI) in cystic fibrosis (CF) patients. We aimed to compare the nutritional risk index (NRI) in adult CF patients receiving modulator (MT) or only non-modulator (conventional) therapies (non-MT). Methods: A single-center prospective cohort study was conducted between June and December 2023. The NRI based on weight gain and albumin was calculated at beginning and end of a 12-week period in both groups. This design was pragmatic, since it was based on individual patient access to MT for 12 weeks. Results: In total, 107 patients were included [mean (SD) age: 23.85 (4.98) years, 54.7% male, 46.7% MT]. In the MT group, mean (SD) weight (kg) and albumin (g/dL) increased significantly [changes: +3.09 (2.74) and +0.17 (0.37); p < 0.001]. In the non-MT group, weight and albumin decreased significantly [changes: −0.99 (1.73) and −0.12 (0.30); p < 0.001]. Compared to the MT group, baseline mean (SD) NRI in the non-MT group was significantly higher [100.65 (11.80) vs. 104.10 (10.10); p = 0.044]. At the end of the 12 weeks, mean (SD) NRI in the MT group was higher than in the non-MT group [104.18 (10.40) vs. 102.58 (12.39); p = 0.145]. In the MT group, the NRI category improved in 22 (44%), and worsened in 3 (6%) patients (p < 0.001). In the non-MT group, the NRI category improved in 2 (3.5%), and worsened in 10 (17.5%) patients (p < 0.001). Conclusions: This is the first study reporting on a positive effect of MT on NRIs, based on weight gain and albumin. Personalized nutrition and routine follow-up of adults with CF based on NRI is recommended prior to MT initiation.
Background: Modulator therapies improve weight and body mass index (BMI) in cystic fibrosis (CF) patients. We aimed to compare the nutritional risk index (NRI) in adult CF patients receiving modulator (MT) or only non-modulator (conventional) therapies (non-MT). Methods: A single-center prospective cohort study was conducted between June-December 2023. The NRI based on weight gain and albumin was calculated at beginning and end of a 12 weeks period in both groups. This design was pragmatic, since based on individual patient access to MT for 12 weeks. Results: 107 patients were included [mean (SD) age: 23.85 (4.98) years, 54.7% male, 46.7% MT]. In the MT group, mean (SD) weight (kg) and albumin (g/dL) increased significantly [changes: +3.09 (2.74) and +0.17 (0.37); p
Background: The increase in treatment burden of adult patients with cystic fibrosis (pwCF), might have influence on their treatment satisfaction. Aim: We aimed to evaluate treatment satisfaction of adult pwCF and to determine influencing factors. Methods: This was a prospective cohort study conducted between June and December, 2023. Demographical data and the percentage of forced expiratory flow rate in 1 second (FEV1%) of the patients were recorded. All patients completed the treatment satisfaction questionnaire for medication (TSQM 1.4), adult version quality of life questionnaires (CFQR) and medication adherence report scale (MARS). This study was approved by local ethics committee. Results: A total of 107 patients aged ≥18 were included in this study [mean (SD) age: 23.85 (4.98) years, %54.7 male]. Also, 50 (%46.7) of the patients were used modulator therapies. A mean (SD) of 4.29 (2.10) medications per patient were prescribed. FEV1% was weak positively correlated with TSQM effectiveness (p=0.004) and global satisfaction (p=0.028). TSQM effectiveness, convenience, global satisfaction was moderate positively correlated with CFQR treatment burden (p0.05). The number of hospitalizations within the last year was weak negatively correlated with TSQM global satisfaction (p=0.031). Modulator users were correlated with higher TSQM all domains’ scores than non-modulator users (p<0.001). Conclusion: PwCF treatment satisfaction is related to treatment burden, number of treatments, and modulator therapies.
12 April 2025 in Orphanet Journal of Rare Diseases
Background: Treatment satisfaction can be described as the patient’s experience in patients with cystic fibrosis (CF). It can be influenced using modulators and clinical characteristics. The aims of this study were to evaluate and compare adult CF patients with and without modulators regarding treatment satisfaction, related factors and to manage their drug related problems (DRPs). Methods: A single-center prospective cohort study was conducted between June 2023 and January 2024. Treatment Satisfaction Questionnaire for Medication (TSQM 1.4), including effectiveness, side effects, convenience, global satisfaction domains, CF Questionnaire-Revised (CFQ-R), and Medication Adherence Report Scale were applied and assessed with and without modulator therapy groups. The relationship between clinical characteristics and TSQM was analyzed by correlations and regression analysis. Recommendations on DRPs identified by clinical pharmacists were made to the physicians and patients and classified according to Pharmaceutical Care Network Europe (PCNE v9.1). Results: A total of 110 patients with 51 modulator therapy and 59 without modulator therapy were included. The mean global satisfaction score of modulator users was found to be 19.733 (p < 0.001) points higher than non-users. When the CFQ-R treatment burden score improved by 1point, global satisfaction score increased by 0.233 points (p < 0.001). When the number of hospitalizations increased by 1 day, the global satisfaction score decreased by 4.751 points (p < 0.001). A total of 84 DRPs were identified, and 69 (82.1%) of them were resolved. Conclusions: Treatment satisfaction in adult CF patients is influenced by modulators, treatment burden, and clinical factors, so access to modulators is important. This is the first study to classify DRPs according to PCNE in CF. Clinical pharmacists contribute to the management of CF.
10 March 2026 in Pulmonary Pharmacology & Therapeutics