Three projects on pediatric liver transplantation recognized at the Congress of the Spanish Society of Pediatric Gastroenterology, Hepatology and Nutrition

Two studies on pediatric liver transplantation led by Dr. Jesús Quintero, head of the Pediatric Hepatology and Liver Transplantation Unit at Vall d'Hebron University Hospital and researcher at the Vall d'Hebron Research Institute (VHIR) received the first and second prizes for the best oral communication in Hepatology at the Congress of the Spanish Society of Pediatric Gastroenterology, Hepatology and Nutrition (SEGHNP). In addition, within the framework of the conference, a new project of the same team received the "Sira Carrasco" research grant to study the risk of rejection in transplanted patients.

Sira Carrasco" research grant: Assessment of the risk of rejection in pediatric liver transplant patients

Firstly, a project of the Vall d'Hebron team received the "Sira Carrasco" research grant, a 14,000€ grant awarded by the SEGHNP to promote research in the field of pediatric gastroenterology, hepatology and nutrition. The aim of the project is to identify pediatric patients at increased risk of rejection after liver transplantation by assessing their evolution with non-invasive techniques.

Specifically, the study will analyze the expression in blood of microRNAs (miRNAs), small molecules that regulate certain genes and signaling pathways. It has previously been suggested that some types of miRNAs promote or reduce liver fibrosis and may therefore play a role in organ rejection. Together with clinical, analytical and imaging data, it will allow monitoring patients after transplantation and assessing their evolution.

miRNA analysis would be a less invasive technique than, for example, biopsy, and would make it possible to select patients at lower risk of fibrosis or liver rejection from whom immunosuppressant treatment could be withdrawn. The ultimate goal is to improve the quality of life of patients, since it would make it possible to reduce invasive procedures and treatments that until now have been considered to be lifelong.

First prize for the best oral communication in Hepatology: Odevixibat for the treatment of progressive familial intrahepatic cholestasis

The first prize for the best oral communication in Hepatology at the SEGHNP congress went to an international paper with the participation of Dr. Jesús Quintero. Positive results of phase III clinical trials (PEDFIC 1 and PEDFIC 2) with odevixibat for the treatment of children with progressive familial intrahepatic cholestasis (PFIC) were presented. This rare disease is based on a defect in the transport of bile acids out of the liver, so that they accumulate in the organ itself and in the bloodstream with severe progressive effects.

In the study, the drug odevixibat was administered to 77 pediatric patients with CIFP and proved to be effective: the levels of bile acids in the blood and pruritus (itching associated with decreased bile excretion) were reduced, as well as the need for painkillers and sleep aids. Despite the occurrence of adverse effects in some patients, no serious ones were reported.

Second prize for the best oral communication in Hepatology: Liver transplantation in patients with protein metabolism disorders

Vall d'Hebron also leads the study that won the second prize for the best oral communication in Hepatology. In this case, it is a joint research with the Hospital Sant Joan de Déu that aimed to describe the hepatic, neurological and metabolic-nutritional evolution in children with protein metabolism disorders after liver transplantation. Protein metabolism disorders are a group of pathologies that prevent the correct assimilation by the organism of some amino acid from the diet, the basic molecules that constitute proteins.

The researchers reviewed the medical records of 22 patients with liver transplantation due to protein metabolism disorders over the last 9 years and analyzed the clinical data before and after transplantation, the neurological and nutritional evolution and possible complications. The underlying pathologies were organic acidemias, tyrosinemia, urea cycle disorders and maple syrup urine disease.

The results show that liver transplantation is a useful tool for the treatment of patients with protein metabolism disorders. In this regard, survival is 100% and only two retransplantations were necessary. Among the transplanted patients, a decrease in the frequency of metabolic decompensations is observed, which makes it possible to increase protein intake and even to withdraw treatment in some cases. The transplanted children also improved their neurological symptoms, mainly spasticity and hypotonia.