Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting.

Background: Ivacaftor has been shown to improve lung function and body weight in patients with CF and a gating mutation. Real-world evaluation is warranted to examine its safety and effectiveness over the long term.

Methods: A retrospective observational multicentre study collected clinical data in the year before and the 2years after ivacaftor initiation in patients with CF and a Gly551Asp-CFTR mutation.

Outcome of home parenteral nutrition in 251 children over a 14-y period: report of a single center.

Background: Parenteral nutrition (PN) is the main treatment for intestinal failure.

Objective: We aimed to review the indications for home parenteral nutrition (HPN) in children and describe the outcome over a 14-y period from a single center.

Design: We conducted a retrospective study that included all children who were referred to our institution and discharged while receiving HPN between 1 January 2000 and 31 December 2013.

[New therapies for cystic fibrosis targeting the CFTR gene or the CFTR protein].

Background: The treatment of cystic fibrosis has been symptom-based for a number of years. New therapies that aim to improve CFTR protein function are now emerging.

Current Scientific Knowledge: The results of gene therapy has been modest but a recent clinical trial shows a positive effect on FEV1.

Assessment and outcome of children with intestinal failure referred for intestinal transplantation.

Background & Aims: Chronic intestinal failure (CIF) requires long term parenteral nutrition (PN) and, in some patients, intestinal transplantation (ITx). Indications and timing for ITx remain poorly defined. In the present study we aimed to analyze causes and outcome of children with CIF.

Intestinal absorption rate in children after small intestinal transplantation.

Background: Small bowel transplantation has now become a recognized treatment of irreversible, permanent, and subtotal intestinal failure.

Objective: The aim of this study was to assess intestinal absorption at the time of weaning from parenteral nutrition in a series of children after intestinal transplantation.

Design: Twenty-four children (age range: 14-115 mo) received intestinal transplantation, together with the liver in 6 children and the colon in 16 children.

A new intravenous fat emulsion containing soybean oil, medium-chain triglycerides, olive oil, and fish oil: a single-center, double-blind randomized study on efficacy and safety in pediatric patients receiving home parenteral nutrition.

Background: SMOFlipid 20% is an intravenous lipid emulsion (ILE) containing soybean oil, medium-chain triglycerides, olive oil, and fish oil developed to provide energy, essential fatty acids (FAs), and long-chain ω-3 FAs as a mixed emulsion containing α-tocopherol. The aim was to assess the efficacy and safety of this new ILE in pediatric patients receiving home parenteral nutrition (HPN) compared with soybean oil emulsion (SOE).

Methods: This single-center, randomized, double-blind study included 28 children on HPN allocated to receive either SMOFlipid 20% (n = 15) or a standard SOE (Intralipid 20%, n = 13).

Some new insights in intestinal failure-associated liver disease.

Purpose Of Review: To point new insights in the cholestasis that is a complication of both intestinal failure and parenteral nutrition. View on liver disease has recently evolved with the onset of fish oil-based intravenous lipid emulsions (ILE).

Recent Findings: Focused on the role of ILE in causing liver disease.

Role of the colon in short bowel syndrome and intestinal transplantation.

Colon is a crucial partner for small intestinal adaptation and function in patients who underwent extensive small intestinal resection or transplantation. This short review deals with the different properties and roles of the colon in these settings, involving fluid and electrolytes absorption, absorption of medium-chain triglycerides, and production of short-chain fatty acids for malabsorbed energy salvage. The colon may adapt after small intestinal resection, whereas it hosts the most important part of the intestinal microbiota, which plays a crucial role in intestinal function and health.