Primjena direktnih oralnih antikoagulansa u pedijatrijskoj populaciji

master's thesis

• Title (english): Use of direct oral anticoagulants in the pediatric population
• Author: Matea Didović
• Mentor: Sandra Šupraha Goreta (mentor)
• Committee member: Sandra Šupraha-Goreta (predsjednik povjerenstva)
• Committee member: Ivana Perković (član povjerenstva)
• Committee member: Lovorka Vujić (član povjerenstva)
• Granter: University of Zagreb; Faculty of Pharmacy and Biochemistry; (Department of biochemistry and molecular biology); Zagreb
• Defense date and country: 2024-09-30, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Pharmacy; Pharmacy

Abstract

Incidencija venske tromboembolije (VTE) u pedijatrijskoj populaciji značajno je porasla u posljednjih dvadeset godina, posebno među hospitaliziranom djecom. Ovaj porast se može pripisati boljoj dijagnostici, ali i povećanju rizičnih čimbenika od kojih je najčešći korištenje centralnog venskog katetera. Terapija VTE-e kod djece može se podijeliti na standardnu terapiju, koja uključuje primjenu nefrakcioniranog heparina (UFH), niskomolekularnog heparina (LMWH) i antagonista vitamina K (VKA). Ovi lijekovi imaju brojne nedostatke, heparini (UFH, LMWH) se primjenjuju parenteralno, a njihov antikoagulacijski učinak je nepredvidiv jer je ovisan o antitrombinu (AT), koji je kod novorođenčadi i bolesne djece često smanjen. Donedavno jedina oralna opcija su bili VKA, koji imaju spor početak i završetak djelovanja, brojne interakcije s hranom i lijekovima, što zahtjeva često praćenje i prilagodbu doze. Odobrenje direktnih oralnih antikoagulansa (DOAK) predstavlja značajan korak naprijed u liječenju VTE-e kod pedijatrijske populacije. Ovi lijekovi u odnosu na standardnu terapiju imaju brojne prednosti, poput jednostavnije primjene, manje potrebe za redovitim praćenjem te potencijalno niži rizik od interakcija i nuspojava. No unatoč prednostima, primjena DOAK-a kod djece nosi sa sobom ograničenja i izazove. Iako kliničke studije pokazuju sličnu djelotvornost i sigurnost kao standardna terapija, primjena kod djece mlađe od 1 godine, onkoloških pacijenata te osoba s oštećenjem funkcije jetre i bubrega je ograničena. Glavni nedostatak DOAK-a je nemogućnost primjene antidota kod djece, budući da nema dovoljno podataka o njihovoj sigurnosti i djelotvornosti.

Abstract (english)

The incidence of venous thromboembolism (VTE) in the pediatric population has increased significantly over the past twenty years, especially among hospitalized children. This increase can be attributed to improved diagnostics, but also to an increase in risk factors, the most common being use of a central venous catheter. Treatment of VTE in children can be divided into standard therapy, which includes use of unfractionated heparin (UFH), low molecular weight heparin (LMWH) and vitamin K antagonists (VKA). These drugs have numerous disadvantages: heparins are administered parenterally, and their anticoagulation effect is unpredictable because it depends on antithrombin (AT), which is often reduced in neonates and sick children. Until recently, the only oral option was VKA, which have a slow onset and end of action, numerous interactions with food and drugs, which requires frequent monitoring and dose adjustment. The approval of direct oral anticoagulants (DOACs) represents a significant step forward in pediatric VTE treatment. Compared to standard therapy, these drugs have several advantages, easier application, less need for regular monitoring, and a potentially lower risk of interactions and side effects. Despite the advantages, the use of DOACs in children comes with limitations and challenges. Although clinical studies show similar efficacy and safety as standard therapy, their use in children under 1 year of age, oncology patients, and those with impaired liver and kidney function is limited. The main disadvantage of DOACs is the lack of an antidote in children, as there is insufficient dana on their safety and efficacy.

• Keywords: DOAK; pedijatrijska populacija; venska tromboembolija
• Keywords (english): DOAC; pediatric population; venous thromboembolism
• Language: croatian
• URN:NBN: urn:nbn:hr:163:788242
• Study programme: Title: Pharmacy - integrated undergraduate and graduate - university study; Study programme type: university; Study level: integrated undergraduate and graduate; Academic / professional title: sveučilišni magistar farmacije (sveučilišni magistar farmacije)
• Type of resource: Text
• File origin: Born digital
• Access conditions: Open access
• Created on: 2025-01-09 13:04:48