This review presents current updates of pancreatic enzyme replacement therapy in children with cystic fibrosis based on literature published in the last decade and some special considerations regarding pancreatic enzyme replacement therapy in the era of new therapies, such as cystic fibrosis transmembrane conductance regulator modulator therapies. Few articles evaluate the efficacy of pancreatic enzyme replacement therapy in the pediatric population, and most studies also included children and adults with cystic fibrosis. Approximately 85% of cystic fibrosis patients have exocrine pancreatic insufficiency and need pancreatic enzyme replacement therapy. Fecal elastase is the most commonly used diagnostic test for exocrine pancreatic insufficiency, although this value can fluctuate over time. While it is used as a diagnostic test, it cannot be used for monitoring the effectiveness of pancreatic enzyme replacement therapy and for adjusting doses. Pancreatic enzyme replacement therapy, the actual treatment for exocrine pancreatic insufficiency, is essential in children with cystic fibrosis to prevent malabsorption and malnutrition and needs to be urgently initiated. This therapy presents many considerations for physicians, patients, and their families, including types and timing of administration, dose monitoring, and therapy failures. Based on clinical trials, pancreatic enzyme replacement therapy is considered effective and well-tolerated in children with cystic fibrosis. An important key point in cystic fibrosis treatment is the recent hypothesis that cystic fibrosis transmembrane conductance regulator modulators could improve pancreatic function, further studies being essential. Pancreatic enzyme replacement therapy is addressed a complication of the disease (exocrine pancreatic insufficiency), while modulators target the defective cystic fibrosis transmembrane conductance regulator protein. Exocrine pancreatic insufficiency in cystic fibrosis remains an active area of research in this era of cystic fibrosis transmembrane conductance regulator modulator therapies. This new therapy could represent an example of personalized medicine in cystic fibrosis patients, with each class of modulators being addressed to patients with specific genetic mutations.

One of the affected organs in cystic fibrosis (CF) is the pancreas, mainly translated into exocrine pancreatic insufficiency. Although rare, acute pancreatitis (AP) has been described, mostly in pancreatic sufficient patients. Objectives. Estimating AP prevalence in CF pediatric population of the I.N.S.M.C. „AlessandrescuRusescu” CF centre. Material and methods. 5 year retrospective study (2011-2016) including INSMC CF centre pediatric population aged 3-18 years. PA diagnosis was established on the presence of minimum 2 criteria between: characteristic abdominal pain, x3 normal value for age elevation of amylase/lipase, medical imaging evaluation suggestive for AP. Patients were evaluated with different criteria including PIP score of predicting AP risk. Outcomes. 48 patients were included with a slight female predominance (54%). 3 of them presented diagnostic criteria for AP (6,25%), all heaving pancreatic insufficiency and high PIP score (low risk of developing AP); 2 of them have low compliance with pancreatic enzymes substitution therapy; one of them presented recurrent episodes of AP. Conclusions. AP is a rare CF complication. It can be easily missed due to a large number of abdominal pain and emesis causes in CF patients. Although it is usually associated with pancreatic sufficiency, all our patients were pancreatic insufficient.

One of the most frequent benign tumor pathology in children is represented by infantile hemangiomas (IHs). Although they are mostly cutaneous, sometimes they can develop at visceral level, the liver being the most common localization. Objectives. Estimating visceral hemangiomas (VHs) prevalence, and identification of risk factors for VHs in patients with infantile cutaneous hemangiomas (ICHs). Materials and methods. 6 years cross-sectional study (2012-2017) including children diagnosed with ICHs, admitted in I.N.S.M.C “Alfred-Rusescu“. All patients underwent an ultrasound screening for the detection of VHs. In order to identify possible risk factors, we collected demographic and perinatal data. Outcomes. 138 patients diagnosed with infantile cutaneous hemangiomas (ICHs) were included, with a slight predominance of girls (58%). The prevalence of the VHs in our study was 7,24% (10 patients). The liver was the most common visceral localization (7 patients). Conclusions. Female gender, preterm birth, low birth weight, and multiple gestations were described as potential risk factors for IHs. In our study, only multiple gestations tend to be associated with visceral hemangiomas, but without a significant statistical correlation.