Group leader: Itxaso Martí Carrera, M.D., Ph.D.

Donostialdea IHO mariaitxaso.marticarrera@osakidetza.eus

Strategic objectives

The Paediatrics Group coordinates the research carried out by both the Donostialdea IHO Paediatrics Service and its paediatric outpatient facility in such a way as to obtain a structure that provides cover for the research work and projects carried out asymmetrically and individually by the units making up the Service. The idea is that other units with less research experience can also participate in these studies.

We believe that clinical care and research must not be separated from the activity of a University Hospital service, and that, to achieve a high degree of excellence, research and care activity must proceed hand-in-hand; it is impossible to provide good care without the backing of good research.

Paediatrics have special characteristics within research. Children have their own particular physiological characteristics, and don’t even form a homogenous group, given that their characteristics may vary depending on the different age groups. Thus, only 30% of the medicines commercialised in Europe include an authorisation for paediatric use, and fewer than 50% of the medicines authorised for use with children have been studied in the infant population. The proportion of drugs used despite not being recommended for children stands at some 90% in Neonatal Intensive Care Units, 70% in Paediatric Intensive Care, 50% in Digestive, 47% in Cardiology and 36% in General Paediatrics. While the use of drugs not approved for such use can have ethical and legal consequences, failing to use said drugs may mean denying the child a potentially beneficial intervention.

Even more vulnerable groups exist, such as extremely premature newborns, where practically all drugs are used outside their technical indications and where there is a great deal of ignorance as regards physiology and pharmacodynamics; the same applies to the case of children with rare diseases, where it is very often impossible to extrapolate the data of adults due to these being diseases of a purely paediatric nature and very low incidence.

Main research lines

Paediatrics is a very wide-ranging area with myriad specialities, but there are certain common areas into which we can classify the majority of the research lines underway or proposals for this group.

• Research into rare or minority diseases in children (RD):

The ultimate aim of this research line is to improve knowledge of the aetiology, physiopathological mechanisms and treatment of this group of diseases. Despite not being exclusive to the infant stage, 80% of diseases start to form in this period and the majority are genetic in origin. Research into rare diseases is complex given the small number of patients suffering from each pathology, meaning that to improve our knowledge of them it is essential to work in collaboration. The main aims of this line are therefore to:

• • • Foster collaboration with other national and/or international research institutes and health centres in order to create registries and clinical trials in these pathologies. Today the majority of the research developed by the members of this group is carried out within this framework with the core objective of promoting participation in this type of multi-centre registries and trials.
    • Promote the creation of a Biobank section containing biological samples of different low-incidence paediatric pathologies.
    • Lead research in the diagnosis/physiopathology and treatment of certain specific diseases:
      • Duchenne muscular dystrophy
      • Cystic fibrosis
    • Included in rare children’s diseases are oncology diseases. This group of diseases plays an essential role in the group, given that the majority of their treatments are carried out in collaborative fashion based on multi-centre research work.

• Research line in child development:

Another of the characteristic and specific paediatrics research lines is child development and its disorders. Childhood is a period of constant change where the person develops. Many adult diseases start to gestate during this period, meaning that it is important to know the environmental and genetic factors that affect human development. Projects in this line encompass the following areas:

• • Neonatology and prematurity. Specific characteristics of development in the premature child.
  • Childhood obesity.
  • Study of the environmental factors associated to sleep problems and disorders
  • Dimensional characterisation of neurodevelopmental disorders.
  •  “INMA (infancy and environment) project”: creation of the Donostialdea cohort.

   

• Principally paediatric diseases:
  • Infant asthma
  • Paediatric infectious diseases

Several research lines also currently exist which were essentially launched to encompass the following areas: meningococcal meningitis and sepsis, vaccinations, emerging viruses such as ZIKA, respiratory viruses and Covid).