**1.4 Treatment**


3.**Ursodeoxycholic acid (UDCA).** UDCA has a cytoprotective effect on the cell membranes of cholangiocytes. It stimulates the secretion of chloride ions through calcium-dependent chloride channel. It reduces the ratio of cholic acid in bile (less than 5%), reduces its synthesis, and lowers its overall volume. It also stimulates cholangiocytes and hepatocytes to secrete. It has antiapoptotic effect and reduces the toxic effects of hydrophobic bile acids.

The dose is 15–20 mg/kg b.m. in two divided doses over 24 hours.

There are only a few clinical trials assessing the effectiveness of ursodeoxycholic acid. There is insufficient evidence to justify its routine use in cystic fibrosis as a preventative measure. Present data suggesting that UDCA should be started before severe liver damage is present as it might be able to prevent the progression of CFLD and has the potential to induce a reversal of fibrosis [1, 2, 4, 40, 49–51].

4. The treatment of portal hypertension should include:


**5. Liver transplant.** Advancing dysfunction of the liver, progressing ascites and jaundice, recurrent bleeding from esophageal varices and hepatopulmonary syndrome, and recurring peritonitis and hepatocarcinoma are indications for liver transplantation. MELD/PELD scores are helpful to evaluate the eligibility for liver transplantation. Model for End-Stage Liver Disease (MELD) includes bilirubin level, albumin, and creatinine concentration. Pediatric end-stage liver disease (PELD) consists of age, bilirubin level, INR index, albumin concentration. PELD/ MELD score obtained upon admission may be of help to establish the optimal timing for LT evaluation and listing. A higher score correlates with a more critical condition and worse survival.

In patients with cystic fibrosis following isolated liver transplantation, there is an increased risk of pulmonary complications (severe infections). It seems that an FEV1 < 50% was associated with poor outcomes in isolated liver transplantation, and thus patients with poor lung function should be considered for combined lung-liver transplantation. For isolated liver transplantation, if the FEV1 is <40%, patients are listed with their MELD/PELD score plus a 10% mortality equivalence. If listed for a combined liver-lung transplantation with an FEV1 < 40%, the liver listing starts with a MELD of 40 [54, 55]. Simultaneous liver-pancreas transplantation restores exocrine and endocrine pancreatic function in patients with CFLD and enables improved nutritional outcomes concurrent with the potential for discontinuation of insulin and pancreatic enzyme supplementation therapies. Diabetes has been reported to exert a negative effect on the already decreased pulmonary function, observed in CF patients. FEV1 in CF patients with diabetes is markedly reduced in all age groups compared to CF patients without diabetes. Simultaneous liver-pancreas transplantation is associated with an improved BMI in the posttransplant course [56, 57].

The outcome for combined heart/lung/liver grafting in adult people with CF was poor, whereas liver transplantation alone had acceptable waiting times and good survival outcome. High incidence of renal impairment in this group, and in contrast to previous studies, largely in pediatric patients, respiratory function can decline dramatically [55, 58–61].

European recommendations for the treatment of cystic fibrosis and hepatic lesions:

**85**

*Liver Changes in the Course of Cystic Fibrosis DOI: http://dx.doi.org/10.5772/intechopen.89306*

alternatively annual CT or MR.

nary shunts as they intensify hypoxemia.

complex and not yet fully explained.

is often symptomless or limited.

lets) every 6 months.

effective.

every 6 months.

**2. Summary**

1.Biochemical tests (AlAT, AspAT, GGTP, FA, prothrombin time, blood plate-

2.Imaging tests—abdominal ultrasound with Doppler option and elastography,

4.Panendoscopy performed every 2–3 years is necessary in patients with cirrho-

5.Assessment of the hepatopulmonary syndrome—assessment of intrapulmo-

6.In the case of cirrhosis—assessment of the levels of alpha-fetoprotein (AFP)

7.Mild esophageal varices—nonselective beta blockers? Level 2–3 varices—

1.The etiopathogenesis of hepatic lesions in the course of cystic fibrosis is very

2.The clinical symptoms of CFLD are not characteristic, and the clinical picture

3.Further studies into the causes of hepatic lesions in cystic fibrosis are necessary, which will contribute to the reduction in the number of deaths, extended

survival rate, and improvement in patients' quality of life.

endoscopic treatment or intrahepatic portosystemic shunts.

8.Prevention of undernutrition (via feeding tube or PEG).

3.Ursodeoxycholic acid at 20 mg/ g daily with divided doses being more

sis and or splenomegaly in order to exclude esophageal varices.

*Cystic Fibrosis - Heterogeneity and Personalized Treatment*

3.**Ursodeoxycholic acid (UDCA).** UDCA has a cytoprotective effect on the cell membranes of cholangiocytes. It stimulates the secretion of chloride ions through calcium-dependent chloride channel. It reduces the ratio of cholic acid in bile (less than 5%), reduces its synthesis, and lowers its overall volume. It also stimulates cholangiocytes and hepatocytes to secrete. It has antiapoptotic

There are only a few clinical trials assessing the effectiveness of ursodeoxycholic acid. There is insufficient evidence to justify its routine use in cystic fibrosis as a preventative measure. Present data suggesting that UDCA should be started before severe liver damage is present as it might be able to prevent the progression of CFLD

• Beta blockers—mild oesophageal varices in patiens without contrindications

**5. Liver transplant.** Advancing dysfunction of the liver, progressing ascites and jaundice, recurrent bleeding from esophageal varices and hepatopulmonary syndrome, and recurring peritonitis and hepatocarcinoma are indications for liver transplantation. MELD/PELD scores are helpful to evaluate the eligibility for liver transplantation. Model for End-Stage Liver Disease (MELD) includes bilirubin level, albumin, and creatinine concentration. Pediatric end-stage liver disease (PELD) consists of age, bilirubin level, INR index, albumin concentration. PELD/ MELD score obtained upon admission may be of help to establish the optimal timing for LT evaluation and listing. A higher score correlates with a more critical

In patients with cystic fibrosis following isolated liver transplantation, there is an increased risk of pulmonary complications (severe infections). It seems that an FEV1 < 50% was associated with poor outcomes in isolated liver transplantation, and thus patients with poor lung function should be considered for combined lung-liver transplantation. For isolated liver transplantation, if the FEV1 is <40%, patients are listed with their MELD/PELD score plus a 10% mortality equivalence. If listed for a combined liver-lung transplantation with an FEV1 < 40%, the liver listing starts with a MELD of 40 [54, 55]. Simultaneous liver-pancreas transplantation restores exocrine and endocrine pancreatic function in patients with CFLD and enables improved nutritional outcomes concurrent with the potential for discontinuation of insulin and pancreatic enzyme supplementation therapies. Diabetes has been reported to exert a negative effect on the already decreased pulmonary function, observed in CF patients. FEV1 in CF patients with diabetes is markedly reduced in all age groups compared to CF patients without diabetes. Simultaneous liver-pancreas transplantation is associated with an improved BMI in the post-

The outcome for combined heart/lung/liver grafting in adult people with CF was poor, whereas liver transplantation alone had acceptable waiting times and good survival outcome. High incidence of renal impairment in this group, and in contrast to previous studies, largely in pediatric patients, respiratory function can

European recommendations for the treatment of cystic fibrosis and hepatic lesions:

• Endoscopic methods for the treatment of severe oesophageal varices [37, 51–53]

effect and reduces the toxic effects of hydrophobic bile acids. The dose is 15–20 mg/kg b.m. in two divided doses over 24 hours.

and has the potential to induce a reversal of fibrosis [1, 2, 4, 40, 49–51].

4. The treatment of portal hypertension should include:

condition and worse survival.

transplant course [56, 57].

decline dramatically [55, 58–61].

**84**

