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    <ns1:title language="sr">Farmakoekonomska analiza kauzalne terapije cerebralne adrenoleukodistrofije</ns1:title>
    <ns2:alt_title language="sr">Pharmacoeconomic analysis of the causal therapy of cerebral adrenoleukodystrophy : doctoral dissertation</ns2:alt_title>
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    <ns1:description language="sr">SažetakUvod: Cerebralna adrenoleukodistrofija je težak oblik X vezane adrenoleukodistrofije, retkebolesti koja nastaje usled poremećaja razgradnje masnih kiselina veoma dugačkog lanca uperoksizomima. Za lečenje ove bolesti danas na raspolaganju imamo tri terapijske opcije:simptomatsku terapiju, alogenu transplantaciju hematopoetskih matičnih ćelija (HSCT) igensku terapiju u vidu elivaldogen autotemcela. Cilj ovog istraživanja je bio da se utvrdi kojaod ovih terapijska opcija ima najbolji farmakoekonomski profil.Materijal i metod: Studija je sprovedena konstruisanjem farmakoekonomskih modelasimulacije diskretnih događaja. Sprovedena je iz perspektive Republičkog fonda zazdravstveno osiguranje Republike Srbije kao institucije, tako da su uračunavani samo direktnimedicinski troškovi. Efekti su izražavani kroz broj dobijenih godina života prilagođenih zakvalitet.Rezultati: Elivaldogen autotemcel nije farmakoekonomski isplativa opcija za lečenjecerebralne adrenoleukodistrofije u odnosu na alogenu HSCT i simptomatsku terapiju koja setakođe koristi za lečenje ove retke metaboličke bolesti, budući da je njegova primena praćenaneprihvatljivo visokim vrednostima ICER-a i negativnim neto novčanim benefitom. AlogenaHSCT je farmakoekonomski isplativija terapijska opcija za lečenje pacijenata sa cerebralnomadrenoleukodistrofijom u poređenju sa simptomatskom terapijom koja se koristi za lečenjeove retke metaboličke bolesti.Zaključak: Rezultati ove farmakoekonomske analize ukazuju da alogena HSCT predstavljafarmakoekonomski najisplativiju opciju za lečenje pacijenata obolelih od cerebralneadrenoleukodistrofije.</ns1:description>
    <ns1:description language="en">AbstractIntroduction: Cerebral adrenoleukodystrophy is a severe form of X-linkedadrenoleukodystrophy, a rare disease that results from a disorder in the breakdown of verylong-chain fatty acids in peroxisomes. For the treatment of this disease, we have threetherapeutic options available today: symptomatic therapy, allogeneic hematopoietic stem celltransplantation (HSCT) and gene therapy in the form of elivaldogen autotemcel. The aim ofthis research was to determine which of these therapeutic options has the bestpharmacoeconomic profile.Material and methods: The study was conducted by constructing discrete event simulationpharmacoeconomic models. It was conducted from the perspective of the Republic HealthInsurance Fund of the Republic of Serbia as an institution, so only direct medical costs wereincluded. Effects are expressed in terms of quality-adjusted life years gained.Results: Elivaldogen autotemcel is not a cost-effective option for the treatment of cerebraladrenoleukodystrophy compared to allogeneic HSCT and symptomatic therapy that is alsoused for the treatment of this rare metabolic disease, since its use is accompanied byunacceptably high ICER values and a negative net monetary benefit. Allogeneic HSCT is amore cost-effective therapeutic option for treating patients with cerebraladrenoleukodystrophy compared with symptomatic therapy used to treat this rare metabolicdisease.Conclusion: The results of this pharmacoeconomic analysis indicate that allogeneic HSCTrepresents the most cost-effective option for the treatment of patients with cerebraladrenoleukodystrophy.</ns1:description>
    <ns1:description language="sr">-</ns1:description>
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        <ns3:firstname> Minela, 1991-</ns3:firstname>
        <ns3:lastname>Vapljanin Muratović</ns3:lastname>
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        <ns3:firstname> Slobodan, 1961-</ns3:firstname>
        <ns3:lastname>Janković</ns3:lastname>
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        <ns3:firstname> Miloš, 1991-</ns3:firstname>
        <ns3:lastname>Milosavljević</ns3:lastname>
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        <ns3:firstname> Ana, 1992-</ns3:firstname>
        <ns3:lastname>Pejčić</ns3:lastname>
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        <ns3:firstname> Gorana, 1985-</ns3:firstname>
        <ns3:lastname>Nedin-Ranković</ns3:lastname>
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    <ns7:keyword language="sr" seq="1">cerebralna adrenoleukodistrofija, elivaldogen autotemcel, alogenatransplantacija hematopoetskih matičnih ćelija, farmakoekonomija</ns7:keyword>
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