Nutritional status, nutrient intake and use of enzyme supplements in paediatric patients with Cystic Fibrosis; a European multicentre study with reference to current guidelines

Calvo-Lerma, Joaquim; Hulst, Jessie M.; Asseiceira, I.; Claes, I.; Garriga, M.; Colombo, Carla; Fornés-Ferrer, Victoria; Woodcock, Sandra; Martins, Tiago Freire; Boon, Mieke; Ruperto, Mar; Walet, Sylvia; Speziali, Chiara; Witters, Peter; Masip, Etna; Barreto, Celeste; Boeck, K. De; Ribes-Koninckx, Carmen

doi:10.1016/j.jcf.2017.03.005

Cited by 39 publications

(20 citation statements)

References 17 publications

(36 reference statements)

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“…In Spain and Portugal, some informants were recommended much higher doses than usual, and thus expected. Dosage differences between Northern Europe and Southern Europe were earlier found in a previous study conducted in MyCyFAPP [ 31 ]. HCPs recommended patients to adjust high doses to one-half or one-third, but informants handled recommendations differently, either giving it a try, adjusting to an intermediate dose, or ignoring it (E7).…”

Section: Resultsmentioning

confidence: 55%

Users’ Experiences of a Mobile Health Self-Management Approach for the Treatment of Cystic Fibrosis: Mixed Methods Study

Floch¹,

Vilarinho²,

Zettl³

et al. 2020

JMIR Mhealth Uhealth

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Background Despite a large number of clinical trials aiming at evaluating the digital self-management of chronic diseases, there is little discussion about users’ experiences with digital approaches. However, a good user experience is a critical factor for technology adoption. Understanding users’ experiences can inform the design of approaches toward increased motivation for digital self-management. Objective This study aimed to evaluate the self-management of cystic fibrosis (CF) with a focus on gastrointestinal concerns and the care of young patients. Following a user-centered design approach, we developed a self-management app for patients and parents and a web tool for health care professionals (HCPs). To evaluate the proposed solutions, a 6-month clinical trial was conducted in 6 European CF competence centers. This paper analyzes the user acceptance of the technology and the benefits and disadvantages perceived by the trial participants. Methods A mixed methods approach was applied. Data were collected through 41 semistructured qualitative interviews of patients, parents, and HCPs involved in the clinical trial. In addition, data were collected through questionnaires embedded in the self-management app. Results Support for enzyme dose calculation and nutrition management was found to be particularly useful. Patients and parents rapidly strengthened their knowledge about the treatment and increased their self-efficacy. Reported benefits include reduced occurrence of symptoms and enhanced quality of life. Patients and parents had different skills, requiring follow-up by HCPs in an introductory phase. HCPs valued obtaining precise information about the patients, allowing for more personalized advice. However, the tight follow-up of several patients led to an increased workload. Over time, as patient self-efficacy increased, patient motivation for using the app decreased and the quality of the reported data was reduced. Conclusions Self-management enfolds a collaboration between patients and HCPs. To be successful, a self-management approach should be accepted by both parties. Through understanding behaviors and experiences, this study defines recommendations for a complex case—the demanding treatment of CF. We identify target patient groups and situations for which the app is most beneficial and suggest focusing on these rather than motivating for regular app usage over a long time. We also advise the personalized supervision of patients during the introduction of the approach. Finally, we propose to develop guidance for HCPs to facilitate changes in practice. As personalization and technology literacy are factors found to influence the acceptance of digital self-management of other chronic diseases, it is relevant to consider the proposed recommendations beyond the case of CF.

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Section: Resultsmentioning

confidence: 55%

Users’ Experiences of a Mobile Health Self-Management Approach for the Treatment of Cystic Fibrosis: Mixed Methods Study

Floch¹,

Vilarinho²,

Zettl³

et al. 2020

JMIR Mhealth Uhealth

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“…Currently patients with CF have to decide the dose of enzymatic supplement to take every single time they eat. As a matter of fact, large differences in dosing criteria were previously documented between European countries, evidencing the lack of a common consensus [10]. The newly developed evidence-based method to adjust PERT could be implemented in the clinical practice to support clinicians and patients’ decision on the dose of the enzymatic supplement, possibly contributing to a more efficient nutrient digestion and absorption, and consequently, to a better nutritional status.…”

Section: Discussionmentioning

confidence: 99%

“…Recently, an enormous variability in the response to doses used in PERT among patients has been confirmed, with no clear association between CFA and the dose of the enzymatic supplement [8,9]. Moreover, wide intra-and inter- patient ranges of the dose of enzymatic supplement (LU/g fat) in a multicentre observational study were showed [10].…”

Section: Introductionmentioning

confidence: 99%

Clinical validation of an evidence-based method to adjust Pancreatic Enzyme Replacement Therapy through a prospective interventional study in paediatric patients with Cystic Fibrosis

et al. 2019

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BackgroundA method to adjust Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis is not currently available.ObjectivesTo assess the in vivo efficacy of a method to adjust the dose of enzymatic supplement in CF extrapolated from previous in vitro digestion studies (theoretical optimal dose, TOD). Secondly, to assess how individual patient characteristics influence the expected coefficient of fat absorption (CFA) and thus to identify an individual correction factor to improve TOD.MethodsA prospective interventional study in 43 paediatric patients with CF from 5 European centres. They followed a 24h fixed diet with the theoretical optimal dose for each meal. Faecal collection was carried out between colorimetric markers in order to include all the faeces corresponding to the fixed diet. Beta regression models were applied to assess the associations of individual patient characteristics with the CFA.ResultsMedian CFA was 90% (84, 94% 1st, 3rd Q.) with no significant differences among centres. Intestinal transit time was positively associated with CFA (p = 0.007), but no statistical associations were found with and age, gender, phenotype or BMI. Regression model showed no improvement of the in vitro predicted theoretical optimal dose when taking individual patient characteristics into account.ConclusionStrict adherence to the theoretical optimal dose of enzymatic supplement for a prescribed meal, led to median CFA levels at the clinical target of 90% with a low variability between patients. The proposed method can be considered as a first approach for an evidence-based method in PERT dosing based on food characteristics. Results have to be confirmed in free dietary settings.

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“…A 100-g serving of the peanut-RUSF-CF provides nearly 24.7% kcal, 18% of recommended protein intake (100 g/d), and 31.9% of recommended lipid intake (89 g/d) for a 2000-kcal diet (8, 11). This represents an increase in nutritional density from currently available nutritional supplements, an important consideration because intakes of protein (12% of dietary calories) and fat (34–35% of dietary calories) are persistently inadequate in children with CF despite recommendations for dietary protein and fat to comprise 20% and 40% of total calories, respectively (6, 8, 11, 29).…”

Section: Discussionmentioning

confidence: 99%

Ready-to-Use Supplemental Food for Nutritional Supplementation in Cystic Fibrosis

Pitman

Mui

Michelson

et al. 2019

Current Developments in Nutrition

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Undernutrition is common in cystic fibrosis (CF) and is correlated with long-term outcomes, yet current nutritional interventions have not demonstrated consistent improvements in energy intake, and subsequently, growth. Development of novel nutritional interventions to increase energy intake is essential to improve clinical outcomes of individuals with CF. Ready-to-use supplemental food (RUSF) is a modifiable, inexpensive, palatable, safe, and nutrient-dense food for treatment or prevention of acute malnutrition in developing countries. Utilizing a linear-programming tool we identified 6 RUSF formulations with sufficient nutrient density (495 kcal/100 g), protein, and fat for children with CF. Palatability was established by a taste-trial and affirmed by a 2-wk tolerability assessment that demonstrated consistent consumption and tolerance of the RUSF. Although preliminary, this study demonstrates the potential for developing RUSF as a nutritional supplement for increasing energy intake in children with CF.

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Nutritional status, nutrient intake and use of enzyme supplements in paediatric patients with Cystic Fibrosis; a European multicentre study with reference to current guidelines

Cited by 39 publications

References 17 publications

Users’ Experiences of a Mobile Health Self-Management Approach for the Treatment of Cystic Fibrosis: Mixed Methods Study

Users’ Experiences of a Mobile Health Self-Management Approach for the Treatment of Cystic Fibrosis: Mixed Methods Study

Clinical validation of an evidence-based method to adjust Pancreatic Enzyme Replacement Therapy through a prospective interventional study in paediatric patients with Cystic Fibrosis

Ready-to-Use Supplemental Food for Nutritional Supplementation in Cystic Fibrosis

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