Cirrhosis for biliary atresia (BA) is associated with risk of gastrointestinal bleeding (GB) from gastroesophageal varices due to portal hypertension. Primary prophylaxis of GB is controversial in ...children who are candidates for liver transplantation (LT). The aim of the study was to define the management of gastroesophageal varices and to identify the benefit of primary prophylaxis for GB in BA children waiting for LT.
A retrospective single-center study including all BA children listed for LT in 2008–2016. Clinical, endoscopical, and biochemical data were analyzed.
Of 82 children, 50 (61%) did not receive primary prophylaxis and did not present any episode of bleeding, 16 (19.5%) underwent primary prophylaxis, and 16 (19.5%) presented spontaneous GB and received secondary prophylaxis. Children without primary prophylaxis and GB were younger than patients with primary prophylaxis and those with GB (7.7 years range, 4.1–37.9 years vs 11.2 years range, 5.1–43 years; P = .03 vs 10.7 years range, 6.9–39.9 years, respectively; P = .004). Seventy-five percent of GB occurred in children older than 8 months. Fifteen (93.8%) children with GB presented esophageal varices (grade III = 10 62.5%) and 10 (62.5%) required endoscopic treatments, consisting mainly of sclerotherapy. Median time to LT was similar for children with or without bleeding (2 months range, 0–17.7 months vs 2.2 months 0–17.9 months, respectively; P = .89). After 45.5 months (range, 13.7–105.5 months) of follow-up, the overall patient survival was 97.6%. At the intention-to-treat analysis, the survival rate was 100% for patients without bleeding episode and 87.5% for children with GB (P = .16).
Despite the risk of GB being not clinically predictable in children with BA waiting for LT, our experience suggests that primary prophylaxis of GB might be unnecessary in children younger than 6 months, while it should be considered in older children. Thus, the occurrence of GB does not delay the timing of transplantation.
•Risk of gastrointestinal bleeding is not clinically predictable in biliary atresia.•Esophageal varices screening in children younger than 6 months who are waiting for liver transplant is unnecessary.•Primary prophylaxis should be always considered in selected children older than 6 months.•Occurrence of gastrointestinal bleeding does not delay the time of transplantation.
The low prevalence of European paediatric transplanted patients and scarcity of resources and expertise led to the need for a multidisciplinary network able to improve the quality of life of ...paediatric patients and families requiring a solid organ or haematopoietic stem cell transplantation. The European Reference Network (ERN) TransplantChild is one of the 24 ERNs established in a European legal framework to improve the care of patients with rare diseases. ERN TransplantChild is the only ERN focused on both solid organ and haematopoietic stem cell paediatric transplantation, based on the understanding of paediatric transplantation as a complex and highly specialised process where specific complications appear regardless the organ involved, thus linking the skills and knowledge of different organ disciplines. Gathering European centres of expertise in paediatric transplantation will give access to a correct and timely diagnosis, share expertise and knowledge and collect a critical mass of patients and data that increases the speed and value of clinical research outcomes. Therefore, the ERN TransplantChild aims for a paediatric Pan-European, Pan-transplant approach.
G protein‐coupled receptor (GPCR) associated sorting protein‐1 (GASP‐1) is suspected to play a key role in recycling and degradation of several GPCRs. In a previous study, we have shown that ...GASP‐1‐knock‐out (GASP‐1‐KO) mice displayed deficits in acquiring a cocaine self‐administration task, associated with an exacerbated down‐regulation of striatal dopaminergic and cholinergic receptors. Among several possibilities, GASP‐1 deficiency could have impaired memory processes underlying the acquisition of the operant conditioning task. Therefore, the present study investigated cognitive performances of GASP‐1‐KO mice and their wild‐type littermates (WT) in a broad variety of memory tasks. Consistent with a deficit in procedural memory, GASP‐1‐KO mice showed delayed acquisition of a food‐reinforced bar‐press task. During water‐maze training in hidden‐ or visible‐platform paradigms, mutant and WT mice acquired the tasks at the same rate. However, GASP‐1 mice exhibited persistent thigmotaxic swimming, longer distance to the platform, and reduced swim speed. There was no deficit in several tasks requiring simple behavioral responses (Barnes maze, object recognition and passive avoidance tasks). Thus, the ability to acquire and/or express complex responses seems affected in GASP‐1‐deficient mice. Hippocampal functions were preserved, as the retention of an acquired memory in spatial tasks remained unaffected. The pattern of behavioral deficits observed in GASP‐1‐KO mice is coherent with current knowledge on the role of striatal GPCRs in acquisition/expression of skilled behavior and in motivation. Together with the previous findings, the so far established phenotype of GASP‐1‐KO mice makes them a potentially exciting tool to study striatal functions.
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