Novi lijekovi u liječenju akutne mijeloične leukemije i njihov utjecaj na ishode liječenja

master's thesis

• Title (english): New drugs in the treatment of acute myeloid leukemia and their influence on treatment outcomes
• Author: Lara Divjak
• Mentor: Nadira Duraković (mentor)
• Committee member: Radovan Vrhovac (predsjednik povjerenstva)
• Committee member: Jasenka Markeljević (član povjerenstva)
• Committee member: Nadira Duraković (član povjerenstva)
• Granter: University of Zagreb; School of Medicine; (Department of Internal Medicine); Zagreb
• Defense date and country: 2022-09-02, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Internal Medicine

Abstract

Akutna mijeloična leukemija (AML) je kompleksna heterogena skupina bolesti koja uglavnom pogađa osobe starije životne dobi. U tih je pacijenata učestalija pojava bolesti rezistentne na standardnu terapiju uz znatno veći mortalitet vezan uz nju. Stope izlječenja od 30-40% primjenom standardne terapije te kratko ukupno preživljenje stvorilo je potrebu za pronalaskom novih strategija liječenja. Napreci napravljeni u razumijevanju patogeneze AML-a omogućili su razvoj novih ciljanih terapija koje zahvaljujući svojoj specifičnosti i dobrom sigurnosnom profilu sve više pronalaze svoje mjesto u kliničkoj praksi kako u starijih tako i u mlađih pacijenata. Niz novih lijekova pokazao je kliničku učinkovitost u izazivanju odgovora te produljenju ukupnog preživljenja u pacijenata s novodijagnosticiranom AML, ali i relapsom te refraktornim oblikom bolesti. U tijeku su brojna klinička ispitivanja koja procjenjuju ishode liječenja novim lijekovima primjenjivanih u obliku monoterapije, u kombinaciji sa standardnim indukcijskim/konsolidacijskim te spasonosnim protokolima, kao i nakon alogene transplantacije krvotvornih matičnih stanica. Ovaj rad donosi pregled dosadašnjih saznanja o trima skupinama novih lijekova, FLT3 inhibitorima, BCL-2 inhibitorima te IDH inhibitorima, te njihovim utjecajima na ishode liječenja.

Abstract (english)

Acute myeloid leukemia (AML) is a complex heterogeneous disease that mainly occurs in elderly patients. This patient group frequently has a disease resistant to standard therapy and therapy-related mortality is common. Cure rates of 30-40% using standard therapy and short overall survival resulted in the need to create new treatment strategies. Advances in the understanding of the pathogenesis of AML have enabled the development of new targeted therapies, that thanks to their specificity and good safety profile, are increasingly finding their place in clinical practice in both older and younger patients. Several new drugs have shown clinical effectiveness in inducing responses and prolonging overall survival in patients with newly diagnosed AML, as well as relapsed and refractory forms of the disease. Numerous clinical trials are underway that evaluate the outcomes of treatment with new drugs applied as monotherapy, in combination with standard induction/consolidation and salvage protocols, as well as after allogeneic hematopoietic stem cell transplantation. This paper provides an overview of the current knowledge about three groups of new drugs, FLT3 inhibitors, BCL-2 inhibitors and IDH inhibitors, and their impact on treatment outcomes.

• Keywords: AML; novi lijekovi; ishodi liječenja; FLT3; BCL-2; IDH
• Keywords (english): AML; new drugs; treatment outcomes; FLT3; BCL-2; IDH
• Language: croatian
• URN:NBN: urn:nbn:hr:105:053630
• Study programme: Title: Medicine; Study programme type: university; Study level: integrated undergraduate and graduate; Academic / professional title: doktor/doktorica medicine (doktor/doktorica medicine)
• Type of resource: Text
• File origin: Born digital
• Access conditions: Open access
• Created on: 2023-01-05 09:33:03