5-azacitidin u liječenju mijelodisplastičnog sindroma

master's thesis

• Title (english): 5-azacitidine in the treatment of myelodysplastic syndrome
• Author: Mihael Grzelja
• Mentor: Inga Mandac Smoljanović (mentor)
• Committee member: Slobodanka Ostojić-Kolonić (predsjednik povjerenstva)
• Committee member: Ivana Vuković Brinar (član povjerenstva)
• Committee member: Inga Mandac Smoljanović (član povjerenstva)
• Granter: University of Zagreb; School of Medicine; (Department of Internal Medicine); Zagreb
• Defense date and country: 2023-07-13, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Internal Medicine

Abstract

Uvod: Mijelodisplastični sindrom (MDS) predstavlja skupinu malignih klonalnih hematoloških poremećaja s displazijom jedne ili više hematopoetskih loza u koštanoj srži, koji se očituje citopenijama i povećanim rizikom za razvoj akutne mijeloične leukemije. Za liječenje visokorizičnog MDS, od 2013.godine u Hrvatskoj je odobren 5-azacitidin (5-AZA).

Ciljevi istraživanja: Cilj ovog istraživanja je odrediti utjecaj 5-AZA na ishod liječenja pacijenata s visokorizičnim MDS u razdoblju od 1.1.2013. do 31.12.2021. u Kliničkoj bolnici Merkur.

Metode: Retrospektivno su analizirani podaci pacijenata s visokorizičnim MDS. U studiji je sudjelovalo 38 pacijenata (M:Ž=25:13), do završetka istraživanja 34 pacijenta su umrla. Analizirani su laboratorijski i morfološki podaci i ukupno preživljenje pacijenata. Podaci su analizirani Kaplan-Meierovom metodom, Log-rank testom i Mann-Whitney U testom.

Rezultati: Dokazano je duže ukupno preživljenje kod pacijenata liječenih s više od 12 ciklusa 5-AZA. Medijan preživljenja skupine s više od 12 ciklusa 5-AZA bio je 24 mjeseca, dok je medijan preživljenja pacijenata s manje od 12 ciklusa 5-AZA bio 11 mjeseci (p=0.023). Također je uočena viša razina kreatinina kod dijagnoze i niža vrijednost LDH prije prvog ciklusa u skupini preživjelih pacijenata.

Zaključak: Rezultati ovog istraživanja upućuju na učinkovitost 5-AZA u prvoj liniji liječenja MDS pacijenata visokog rizika, uz statistički značajnu razliku u produljenju preživljenja kod pacijenata koji su dobili najmanje 12 ciklusa 5-AZA.

Abstract (english)

Introduction: Myelodysplastic syndrome (MDS) represents a group of malignant clonal hematological disorders characterized by dysplasia in one or more hematopoietic lineages in the bone marrow, leading to cytopenias and an increased risk of developing acute myeloid leukemia. One therapeutic option for high-risk MDS is 5-azacitidine (5-AZA).

Aim: The aim of this study was to determine the impact of 5-AZA on the treatment outcome of patients with high-risk MDS from January 1, 2013, to December 31, 2021, at the Clinical Hospital Merkur.

Methods: Retrospective analysis was performed on data from patients with high-risk MDS. The study included 38 patients (M:F=25:13), at time of study conclusion, 34 patients died. Laboratory data and overall survival of the patients were analyzed. Data were analyzed using the Kaplan-Meier method, Log-rank test, and Mann-Whitney U test.

Results: A longer overall survival was observed in patients treated with more than 12 cycles of 5-AZA. The median survival of the group receiving more than 12 cycles of 5-AZA was 24 months, while the median survival of patients receiving less than 12 cycles was 11 months (p=0.023). Higher creatinine levels at diagnosis and lower LDH levels before the first cycle were observed in the group of surviving patients.

Conclusion: The results of this study highlight the efficacy of 5-AZA in the first-line treatment of high-risk MDS patients, with a statistically significant difference in overall survival extension observed in patients receiving at least 12 cycles of 5-AZA.

• Keywords: MDS; 5-azacitidin; preživljenje
• Keywords (english): MDS; 5-azacitidine; survival
• Language: croatian
• URN:NBN: urn:nbn:hr:105:813100
• 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: 2023-10-24 10:49:02