Treatment of Infants and Toddlers With Cystic Fibrosis–related Pancreatic Insufficiency and Fat Malabsorption With Pancrelipase MT

Vijver, Els Van de; Desager, Kristine; Mulberg, Andrew E.; Staelens, Sofie; Verkade, Henkjan J.; Bodewes, Frank; Malfroot, Anne; Hauser, Bruno; Sinaasappel, M.; Biervliet, S. Van; Behm, Martin O.; Pelckmans, Paul; Callens, Dirk; Veereman-Wauters, G.

doi:10.1097/mpg.0b013e31820e208e

Cited by 45 publications

(43 citation statements)

References 14 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Pancreatic elastases constitute around 6% of the secretion, with fecal levels being approximately five times that of the pancreatic juice [16]. Fecal levels of elastase-1 correlate well with its pancreatic output, as well as the output of other pancreatic enzymes, such as amylase, lipase, and trypsin [17,18]. Furthermore, elastase-1 is highly stable in feces for up to 1 week at room temperature, and for 1 month when stored at 4°C, thus removing the requirement for specialist testing facilities or storage conditions [16].…”

Section: The Fecal Elastase-1 Testmentioning

confidence: 96%

“…Clinically, the most common cause of PEI is CP, although symptoms of PEI may not appear until several years after disease onset [17]. In contrast, PEI is present at birth in 85% of infants with CF [18]. The high prevalence of PEI in these disorders creates a great need for a reliable, simple diagnostic tool to screen patients and determine whether they require PERT.…”

Section: Introductionmentioning

confidence: 96%

See 1 more Smart Citation

Potential for Screening for Pancreatic Exocrine Insufficiency Using the Fecal Elastase-1 Test

et al. 2017

View full text Add to dashboard Cite

The early diagnosis of pancreatic exocrine insufficiency (PEI) is hindered because many of the functional diagnostic techniques used are expensive and require specialized facilities, which prevent their widespread availability. We have reviewed current evidence in order to compare the utility of these functional diagnostic techniques with the fecal elastase-1 (FE-1) test in the following three scenarios: screening for PEI in patients presenting with symptoms suggestive of pancreatic disease, such as abdominal pain or diarrhea; determining the presence of PEI in patients with an established diagnosis of pancreatic disease, such as chronic pancreatitis or cystic fibrosis; determining exocrine status in disorders not commonly tested for PEI, but which have a known association with this disorder. Evidence suggests the FE-1 test is reliable for the evaluation of pancreatic function in many pancreatic and non-pancreatic disorders. It is non-invasive, is less time-consuming, and is unaffected by pancreatic enzyme replacement therapy. Although it cannot be considered the gold-standard method for the functional diagnosis of PEI, the advantages of the FE-1 test make it a very appropriate test for screening patients who may be at risk of this disorder.

show abstract

Section: The Fecal Elastase-1 Testmentioning

confidence: 96%

Section: Introductionmentioning

confidence: 96%

Potential for Screening for Pancreatic Exocrine Insufficiency Using the Fecal Elastase-1 Test

et al. 2017

View full text Add to dashboard Cite

show abstract

“…The selection of an oral dosage form and the pharmaceutical aspects of the formulation, such as the palatability of an oral suspension or the size of a tablet, are important factors in the overall acceptability of an oral paediatric medicine 6 10. As adequate child and parent acceptability are prerequisites for good drug adherence, paediatric treatment outcomes may be enhanced by a careful selection of the formulation including the type of the dosage form.…”

Section: Introductionmentioning

confidence: 99%

Acceptability of different oral formulations in infants and preschool children

Riet‐Nales

Neef

Schobben

et al. 2013

Archives of Disease in Childhood

108

View full text Add to dashboard Cite

ObjectiveLiquid medicines are easy to swallow. However, they may have disadvantages, such as a bad taste or refrigerated storage conditions. These disadvantages may be avoided by the use of oral solid medicines, such as powders or tablets. The aim of this study was to investigate the acceptability of and preference among four oral formulations in domiciliary infants and preschool children in The Netherlands.MethodsParents administered four oral placebo dosage forms that were aimed at a neutral taste, at home, to their child (1–4 years of age) twice on one day following a randomised cross-over design: small (4 mm) tablet, powder, suspension and syrup. They were asked to report the child's acceptability by a score on a 10 cm visual analogue scale (VAS score) and by the result of the intake. At the end of the study, they were asked to report the preference of the child and themselves.Results183 children were included and 148 children were evaluated. The data revealed a period/cross-over effect. The estimate of the mean VAS score was significantly higher for the tablet than for the suspension (tablet 9.39/9.01; powder 8.84/8.20, suspension 8.26/7.90, syrup 8.35/8.19; data day 1/all days). The estimate of the mean number of intakes fully swallowed was significantly higher for the tablet than for the other formulations (all p values <0.05). Children and parents preferred the tablet and syrup over the suspension and the suspension over the powder (all p values <0.05).ConclusionsAll formulations were well accepted. The tablets were the best accepted formulation; the tablets and syrup the most preferred.Trial Registration numberISRCTN63138435.

show abstract

“…8 In cystic fibrosis, PEI is present in 85% of infants at birth. 9 A typical symptom of advanced PEI is steatorrhea. Steatorrhea, however, may not be present or may have another cause.…”

Section: Background and Objectivementioning

confidence: 99%

“…Patients are commenced on the minimum dose and then up titrated based on weight gain and bowel signs to ascertain the lowest effective dose. 9,20 A maximum dose recommendation of 10,000 units of lipase per kilogram per day is used because of the possible risk of fibrosing colonopathy. 2 In case of insufficient response to PERT, the pancreatin dose should be doubled or tripled (Evidence 5; recommendation B) and/or a proton pump inhibitor should be administered (Evidence 1b; recommendation B) (Figure 1).…”

Section: General Measurementsmentioning

confidence: 99%

Synopsis of recent guidelines on pancreatic exocrine insufficiency

Löhr

Oliver²,

Frulloni

2013

UEG Journal

View full text Add to dashboard Cite

Background: In recent years, three national gastroenterology societies established guidelines for the diagnosis and therapy of pancreatic exocrine insufficiency (PEI). In addition, the Cochrane Collaboration issued a review. Objective: The purpose of this paper is to present an overview of the recommendations and concordance between the four recent published guidelines and stimulate further discussion. Methods: A review of the Australian, German and Italian guidelines and the Cochrane review was conducted, and a synthesis was made of common statements.Results: There is a high degree of agreement on almost all items within these guidelines, both in the diagnosis of PEI and in terms of therapy and approach to management of PEI. In addition, novel emerging developments are highlighted, such as the fecal elastase-1 test, which is widely used but is not suitable for measuring mild-to-moderate PEI despite its ability to positively establish the diagnosis of severe PEI. One of the few novel tests proving to be useful is the 13 C mixed-chain triglycerides (MCT) breath test. This test, albeit an excellent quantitative test, is not widely used and is rarely available. The use of this test is making it apparent that there is a difference between treating the symptoms of PEI and treating malnutrition, the broader underlying defect. This may have direct consequences for the dosing of pancreatic enzymes (pancreatin), in that the consensus starting dose of all guidelines may be too low for some patients. Although chronic pancreatitis in adults and cystic fibrosis in children account for the main evidence base used for PEI, other indications are also discussed. Conclusions: There is good concordance between recommendations provided by international groups. More prospective studies are required in many areas, including the use of pancreatic enzymes in other gastrointestinal disorders, such as celiac disease and irritable bowel syndrome (IBS). We also need to assess the feasibility of the 13 C MCT breath test. At the same time, it needs to be confirmed that higher doses of pancreatic enzymes are really necessary to not only relieve the symptoms of PEI but also treat malnutrition appropriately.

show abstract

Treatment of Infants and Toddlers With Cystic Fibrosis–related Pancreatic Insufficiency and Fat Malabsorption With Pancrelipase MT

Cited by 45 publications

References 14 publications

Potential for Screening for Pancreatic Exocrine Insufficiency Using the Fecal Elastase-1 Test

Potential for Screening for Pancreatic Exocrine Insufficiency Using the Fecal Elastase-1 Test

Acceptability of different oral formulations in infants and preschool children

Synopsis of recent guidelines on pancreatic exocrine insufficiency

Contact Info

Product

Resources

About