Pancreatic enzyme replacement therapy for people with cystic fibrosis

Somaraju, Usha Rani; Solís-Moya, Arturo

doi:10.1002/14651858.cd008227.pub4

Cited by 26 publications

(28 citation statements)

References 98 publications

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“…[20][21][22][23] Exocrine pancreatic insufficiency (PI) arises in CF due to the thick secretions that block the pancreatic ducts. This leads to decreased digestion and absorption of fats, protein and the fat-soluble vitamins A, D, E and K. 24 Up to 85% of the people with CF have PI and require long-term pancreatic enzyme replacement therapy and replacement of fat soluble vitamins. 2,25 Careful monitoring of growth and nutrition is therefore essential in PI patients.…”

Section: Clinical Features Diagnosis and Management Of Cfmentioning

confidence: 99%

“…26,27 Accumulation of viscous material within the bowel can cause obstruction, presenting as meconium ileus in the neonatal period or leading to distal intestinal obstruction syndrome (DIOS) in older children and adults. 24 In the liver, CFTR is expressed in the bile ducts, regulating the fluid and electrolyte content of the bile. A wide spectrum of liver disease may occur in people with CF, from steatosis and neonatal cholestasis to more severe multilobular cirrhosis necessitating liver transplantation.…”

Section: Clinical Features Diagnosis and Management Of Cfmentioning

confidence: 99%

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Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis

Haq¹,

Almulhem²,

Soars³

et al. 2022

PGPM

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Cystic fibrosis (CF) is an autosomal recessive genetic condition that is caused by variants in the cystic fibrosis transmembrane conductance regulator gene. This causes multisystem disease due to dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel at the apical surface of epithelia. Until recently, treatment was directed at managing the downstream effects in affected organs, principally improving airway clearance and treating infection in the lungs and improving malabsorption in the gastrointestinal tract. Care delivered by multidisciplinary teams has yielded incremental improvements in outcomes. However, the development of small-molecule CFTR modulator drugs over the last decade has heralded a new era of CF therapeutics. Modulators target the underlying defect and improve CFTR function. Either monotherapy or a combination of modulators is used depending on the specific genotype and class of CFTR disease-causing variants that an individual has. Both ivacaftor and the ivacaftor/tezacaftor/elexacaftor combination have been demonstrated to be associated with clinically very significant benefits in randomised trials and have rapidly been made available as part of standard care in many countries. CFTR modulators represent one of the best examples of precision medicine to date. They are expensive, however, and equity of access to them worldwide remains an issue. Studies and approvals are also ongoing for children under the age of 6 years for ivacaftor/tezacaftor/elexacaftor. Furthermore, no modulators are available for around 10% of the people with CF. In this review, we firstly summarise the genetics, pathophysiology and clinical problems associated with CF. We then discuss the development of CFTR modulators and key clinical trials to support their use along with other potential future therapeutic approaches.

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Section: Clinical Features Diagnosis and Management Of Cfmentioning

confidence: 99%

Section: Clinical Features Diagnosis and Management Of Cfmentioning

confidence: 99%

Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis

Haq¹,

Almulhem²,

Soars³

et al. 2022

PGPM

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“…Human data on the safety of pancreatic enzymes are available from their therapeutic use. Pancreatic enzymes of porcine origin have been used for decades in drugs used to treat patients with pancreatic insufficiency, including infants, with the diagnosis of cystic fibrosis (Brady et al, 1991;Graff et al, 2010;Whitcomb et al, 2010;Gubergritis et al, 2011;Littlewood et al, 2011;Sander-Struckmeier et al, 2013;Kashirskaya et al, 2015;Somaraju and Solis-Moya, 2020).…”

Section: Toxicological Datamentioning

confidence: 99%

Safety evaluation of the food enzyme trypsin from porcine pancreas

Lambré¹,

Baviera²,

Bolognesi³

et al. 2022

EFS2

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The food enzyme trypsin (EC 3.4.21.4) is extracted from porcine pancreas by Ningbo Linzyme Biosciences Co., Ltd. It is intended to be used for the hydrolysis of whey proteins for use in infant formulae and follow‐on formulae. Based on maximum use levels and the maximum permitted protein content in infant formula, dietary exposure to the food enzyme–total organic solids (TOS) was estimated to be 16.8 mg TOS/kg body weight (bw) per day for infants. In the toxicological evaluation, clinical studies with pancreatic enzymes were considered. Hypersensitivity to the pharmaceuticals was identified as the major side effect. However, allergic reactions to porcine pancreatic enzymes in hydrolysed foods have not been reported. The Panel considered that a risk of allergic sensitisation to this food enzyme after consumption of products prepared by hydrolysis of milk proteins could not be excluded in infants, but it considered the likelihood to be low. Based on the origin of the food enzyme from an edible tissue of pigs, the data provided by the applicant, the information from the evaluation of clinical studies based on pancreatic enzymes and the estimated dietary exposure, the Panel concluded that the trypsin from porcine pancreas does not give rise to safety concerns under the intended conditions of use.

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“…The consequent maldigestion and malabsorption of nutrients leads to several nutritional deficiencies. To improve patients' quality of life, pancreatic ERT represents a valid approach [36,37]. However, nutrient malabsorption has also been observed in acquired immunodeficiency syndrome (AIDS), whose related experimental studies have shown promising results for the use of pancreatic ERT in the improvement of this condition [38].…”

Section: Further Metabolic Deficienciesmentioning

confidence: 99%

Enzyme Therapy: Current Challenges and Future Perspectives

Fuente

Lombardero²,

Gómez-González

et al. 2021

IJMS

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In recent years, enzymes have risen as promising therapeutic tools for different pathologies, from metabolic deficiencies, such as fibrosis conditions, ocular pathologies or joint problems, to cancer or cardiovascular diseases. Treatments based on the catalytic activity of enzymes are able to convert a wide range of target molecules to restore the correct physiological metabolism. These treatments present several advantages compared to established therapeutic approaches thanks to their affinity and specificity properties. However, enzymes present some challenges, such as short in vivo half-life, lack of targeted action and, in particular, patient immune system reaction against the enzyme. For this reason, it is important to monitor serum immune response during treatment. This can be achieved by conventional techniques (ELISA) but also by new promising tools such as microarrays. These assays have gained popularity due to their high-throughput analysis capacity, their simplicity, and their potential to monitor the immune response of patients during enzyme therapies. In this growing field, research is still ongoing to solve current health problems such as COVID-19. Currently, promising therapeutic alternatives using the angiotensin-converting enzyme 2 (ACE2) are being studied to treat COVID-19.

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Pancreatic enzyme replacement therapy for people with cystic fibrosis

Cited by 26 publications

References 98 publications

Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis

Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis

Safety evaluation of the food enzyme trypsin from porcine pancreas

Enzyme Therapy: Current Challenges and Future Perspectives

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