o:1774 Odnos troškova i efekata terapijskih alternativa u lečenju alfa manozidoze Cost/effectiveness of therapeutic alternatives in the treatment of alpha mannosidosis : doctoral dissertation sr Uvod: Postoje tri mogućnosti za lečenje blage do umerene alfa manozidoze: nadoknadaenzima velmanaze alfa, transplantacija kostne srži i simptomatska terapija.Cilj: Upoređenje troškova i efekata velmanaze alfa sa troškovima i efektima transplantacijekostne srži i simptomatske terapije u lečenju blage do umerene alfa manozidoze.Metod: Istraživanje je sprovedeno pomoću modela simulacije diskretnih događaja izgrađenogu tabelarnom kalkulativnom softveru. Ulazni parametri u model su dobijeni sistematskompretragom naučne literature, a ishodi su bili inkrementalni odnos troškova i efekata, netonovčani benefit, verovatnoća prihvatljivosti velmanaze alfa od strane Republičkog fondazdravstvenog osiguranja i procenjena vrednost savršene informacije. Ishodi su dobijenipomoću Monte Karlo simulacije na kohorti od 1000 virtuelnih bolesnika sa alfamanozidozom.Rezultati: Nadoknada enzima alfa manozidaze primenom velmanaze alfa nije isplativa upoređenju sa simptomatskom terapijom ili sa transplantacijom kostne srži (neto novčanibenefit je iznosio -366,179,427.48 RSD odnosno -358,074,387.57 RSD, po redosledu).Transplantacija kostne srži može biti isplativa opcija lečenja blage do umerene alfamanozidoze u poređenju sa simptomatskom terapijom, ako se spremnosti na plaćanje Fondazdravstvenog osiguranja u Srbiji poveća na 3 - 9 bruto nacionalnih dohodaka po glavistanovnika po dodatnoj godini života prilagođenoj za kvalitet (neto novčani benefit: -6,849,191.70 RSD). Ulaganje u dodatna istraživanja kako bi se ispitala tačnost ovihzaključaka nije isplativo.Zaključak: Farmakoekonomski najpovoljnija strategija lečenja blage do umerene alfamanozidoze je transplantacija kostne srži. Introduction: There are three treatment options for mild to moderate alpha mannosidosis:replacement of the enzyme velmanase alfa, bone marrow transplantation, and symptomatictherapy.Objective: To compare the costs and effects of velmanase alfa with the costs and effects ofbone marrow transplantation and symptomatic therapy in the treatment of mild to moderatealpha mannosidosis.Method: The research was conducted using a discrete event simulation model built in aspreadsheet software. The input parameters to the model were obtained by a systematic searchof the scientific literature, and the outcomes were the incremental cost-effectiveness ratio, thenet monetary benefit, the probability of acceptance of velmanase alfa by the Republic HealthInsurance Fund and the estimated value of perfect information. The results were obtainedusing Monte Carlo simulation on a cohort of 1000 virtual patients with alpha mannosidosis.Results: Alpha mannosidase enzyme replacement using velmanase alfa is not cost-effectivecompared to symptomatic therapy or bone marrow transplantation (net monetary benefit was -366,179,427.48 RSD and -358,074,387.57 RSD, respectively). Bone marrow transplantationmay be a cost-effective treatment option for mild to moderate alpha-mannosidosis comparedto symptomatic therapy, if the willingness to pay of the Health Insurance Fund in Serbia isincreased to 3 - 9 gross domestic products per capita per additional quality-adjusted life year(net monetary benefit: -6,849,191.70 RSD). Investing in additional research to test theaccuracy of these conclusions is not worthwhile.Conclusion: The most pharmacoeconomically favorable treatment strategy for mild tomoderate alpha mannosidosis is bone marrow transplantation. - 91552100 156876553 91552101 8807 2024-11-19T09:30:01.053Z 45 no 46 Ana, 1987- Antanasković 79747593 2024 63 mentor Slobodan, 1961- Janković 13565031 2024 63 predsednik komisije Miloš, 1977- Milosavljević 28270695 2024 63 član komisije Ana, 1992- Pejčić 21714791 2024 63 član komisije Gorana, 1985- Nedin-Ranković 14372711 2024 75 listova 3162034 http://phaidrakg.kg.ac.rs/o:1774 no yes 10 2024-11-19T09:30:01.320Z 70 alfa manozidaza; alfa manozidoza; bolest lizozomalnih depoa; velmanaza alfa;transplantacija kostne srži; simptomatska terapija; odnos troškova i efekata; neto novčanibenefit; probabilistička analiza senzitivnosti; procenjena vrednost savršene informacije. alpha mannosidase; alpha mannosidosis; lysosomal depot disease; velmanasealfa; bone marrow transplantation; symptomatic therapy; the cost/effectiveness ratio; netmonetary benefit; probabilistic sensitivity analysis; the estimated value of perfect information. 615 Farmakoekonomija 1738 34A05 2024