20.500.12556/RUL-170019 Vpliv profilaktičnega zdravljenja z emicizumabom na kakovost življenja bolnikov s težko obliko hemofilije A magistrsko delo The effect of prophylactic emicizumab treatment on the quality of life of patients with severe haemophilia A master thesis Uvod: Hemofilija A je redka dedna motnja strjevanja krvi, za katero je značilna nagnjenost h krvavitvam zaradi pomanjkanja koagulacijskega faktorja VIII. Težko obliko hemofilije A spremljajo spontane krvavitve v sklepe in mišice, najpogostejši zaplet pa je hemofilična artropatija. Ti bolniki se zdravijo profilaktično ali po potrebi s faktorjem VIII, od leta 2019 pa tudi profilaktično z monoklonskim protitelesom emicizumabom. Podatki o z zdravjem povezani kakovosti življenja pri odraslih bolnikih s težko obliko hemofilije A in hemofilično artropatijo, zdravljenih z emicizumabom v redni klinični praksi, so še vedno omejeni. Namen: Namen raziskave je bil oceniti kakovost življenja odraslih bolnikov s težko obliko hemofilije A in hemofilično artropatijo brez inhibitorjev pred uvedbo profilaktičnega zdravljenja z emicizumabom in po enem letu zdravljenja z emicizumabom v redni klinični praksi. Metode dela: Izvedli smo prospektivno raziskavo z uporabo kvantitativne metodologije. Raziskava je potekala na Kliničnem oddelku za hematologijo Interne klinike Univerzitetnega kliničnega centra Ljubljana med letoma 2020–2022. Z zdravjem povezano kakovost življenja pred profilakso z emicizumabom in po enem letu le-te smo ocenili s slovensko priredbo mednarodno validiranega vprašalnika SF-36v2 in s parnim t-testom ali Wilcoxonovim testom z rangi. Rezultati: Na začetku raziskave so anketirani bolniki svojo kakovost življenja v primerjavi s splošno populacijo ocenili kot slabo, predvsem na področju telesnega delovanja, saj so bile povprečne vrednosti točk za dimenzije telesne komponente pod 50 (od 39,3 do 45,9 točke), kjer 50 predstavlja normo za splošno populacijo. Po enem letu profilakse z emicizumabom so anketirani bolniki pokazali boljše rezultate v vseh dimenzijah, statistično pomembno pa v dimenziji telesne zmogljivosti (44,5 ± 10,2 po emicizumabu v primerjavi z 39,3 ± 6,8 pred emicizumabom, p = 0,018), telesni komponenti (46,6 ± 9,6 v primerjavi z 42,4 ± 7,1, p = 0,017) in socialnem funkcioniranju (50,9 ± 9,7 v primerjavi s 46,4 ± 9,4, p = 0,014). Razprava in zaključek: Rezultati naše raziskave kažejo izboljšano telesno in socialno delovanje odraslih bolnikov s hemofilijo A in hemofilično artropatijo po enem letu profilakse z emicizumabom. Za izboljšanje zdravstvene oskrbe in učinkovito spremljanje uvajanja novih zdravil pri hemofiliji bi bilo smiselno vzpostaviti sistematično spremljanje kakovosti življenja in duševnega zdravja, vključno s simptomi depresije, pri vseh bolnikih s hemofilijo. Introduction: Haemophilia A is a rare, inherited bleeding disorder characterised by a tendency to bleed due to a lack of coagulation factor VIII. The severe form of haemophilia A is accompanied by spontaneous bleeding in the joints and muscles, and the most common complication is haemophilic arthropathy. These patients are treated prophylactically or as needed with factor VIII, and since 2019, also prophylactically with the monoclonal antibody emicizumab. In routine clinical practice, data on health-related quality of life in adult patients with severe haemophilia A and haemophilic arthropathy treated with emicizumab are still limited. Purpose: The study aimed to assess the quality of life of adult patients with severe haemophilia A and haemophilic arthropathy without inhibitors before the introduction of prophylactic treatment with emicizumab and after one year of treatment with emicizumab in routine clinical practice. Methods: We conducted a prospective study using quantitative methodology at the Clinical Department of Haematology of the Internal Clinic of the University Medical Centre Ljubljana from 2020 to 2022. Health-related quality of life before and after one year of emicizumab prophylaxis was assessed using the Slovenian adaptation of the internationally validated SF-36v2 questionnaire and a paired t-test or Wilcoxon rank-sum test. Results: At the beginning of the research, the surveyed patients assessed their quality of life as poor compared to the general population, especially in the field of physical functioning, as the average points for the dimensions of the physical component were below 50 (from 39.3 to 45.9 points), where 50 presents norm for the general population. After one year of prophylaxis with emicizumab, the surveyed patients showed better results in all dimensions, and statistically significant in the dimension of physical functioning (44.5 ± 10.2 after emicizumab vs. 39.3 ± 6.8 before emicizumab, p=0.018), physical component summary (46.6 ± 9.6 vs. 42.4 ± 7.1, p=0.017), and social functioning (50.9 ± 9.7 vs. 46.4 ± 9.4, p=0.014). Discussion and conclusion: The results of our study suggest improved physical and social functioning in adult patients with haemophilia A and haemophilic arthropathy after one year of emicizumab prophylaxis. To improve healthcare and effectively monitor the introduction of new medications in haemophilia, it would be reasonable to establish systematic monitoring of quality of life and mental health, including symptoms of depression, in all patients with haemophilia. magistrska dela zdravstvena nega motnje strjevanja krvi nefaktorska terapija redna klinična praksa zdravstvena vzgoja izidi zdravljenja master theses nursing care haemostatic disorders non-factor therapy routine clinical practice patient education treatment outcomes true false false [T. Sobotič] Slovenski jezik Angleški jezik Magistrsko delo/naloga 2025-07-02 12:30:15 2025-07-02 12:30:18 2025-07-04 09:15:16 0000-00-00 00:00:00 2025 0 Ljubljana 0 Ljubljana 48 str., [16] str. pril. 0000-00-00 NiDoloceno NiDoloceno NiDoloceno 0000-00-00 0000-00-00 0000-00-00 616-083 146467 241386499 50175.pdf 50175.pdf 1 F0AFB8247839711A52F65A21D43A19A4 9d11ee868ba5d379544937b850ee7421ccfbbb1fb2386d3044a1c91e4e065793 a78570c2-572d-11f0-b232-0050569b8976 https://repozitorij.uni-lj.si/Dokument.php?lang=slv&id=211928 Zdravstvena fakulteta 0 0 0