Cost and effectiveness comparison of sirolimus versus standard treatment in Kasabach-Merritt phenomenon: a real-world evidence study in Thailand.

IF 1.2 4区 医学 Q4 HEMATOLOGY
Pediatric Hematology and Oncology Pub Date : 2024-04-01 Epub Date: 2024-01-18 DOI:10.1080/08880018.2023.2300485
Chanikarn Tongruang, Siriwan Wananukul, Susheera Chatproedprai, Nattee Narkbunnam, Rattanavalai Nitiyarom, Nongnuch Sirachainan, Rungrote Natesirinilkul, Phumin Chaweephisal, Darintr Sosothikul
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引用次数: 0

Abstract

The conventional treatment of Kasabach-Merritt Phenomenon (KMP) consists of corticosteroids with vincristine/vinblastine or others. The aim of the study is to compare the first-year direct costs and effectiveness between sirolimus and conventional treatment. A retrospective case-control study of KMP patients was conducted at a mean age of 9 months (1 day to 12 years) between 2000 and 2022 from four tertiary centers in Thailand. The direct costs, hematologic and clinical complete response (HCR, CCR), hospitalization, length of stay, and complications were compared. Of 29 patients, 13 underwent sirolimus (four upfront and nine were refractory to the conventional). The first-year total cost had no statistically significant difference between sirolimus VS conventional treatment (8,852.63 VS 9,083.56 USD: p value: 0.94). The therapeutics achievement was the same in both HCR (244.75 VS 168.94 days; p value: 0.60) and CCR (419.77 VS 399.87 days; p value: 0.90). The subgroup analysis of the first-line sirolimus (n = 4) compared with the conventional (n = 25) showed a more reduced total cost (4,907.84 VS 9,664.05 USD; p value: 0.26) rendered net total cost of -4,756.21 USD per patient (cost saving). A more significant contrast of therapeutic achievement by reduction of both HCR (11.67 VS 224.20 days; p value: 0.36) and CCR (38.50 VS 470.88 days; p value: 0.04) was shown. The sirolimus had no difference in hospitalization, length of stay, and complications. Even though, it was unable to identify significant differences in cost-effectiveness. Sirolimus is suitable for all patients who have diagnosis of KMP either for rescue therapy or first-line treatment.

西罗莫司与标准疗法治疗卡萨巴赫-梅里特现象的成本和效果比较:泰国真实世界证据研究。
卡萨斯巴赫-梅里特现象(KMP)的传统治疗方法包括皮质类固醇与长春新碱/长春花碱或其他药物。本研究的目的是比较西罗莫司和传统治疗的第一年直接费用和疗效。2000 年至 2022 年期间,泰国四家三级医疗中心对平均年龄为 9 个月(1 天至 12 岁)的 KMP 患者进行了一项回顾性病例对照研究。研究比较了直接费用、血液学和临床完全反应(HCR、CCR)、住院时间、住院时间和并发症。在29名患者中,13名接受了西罗莫司治疗(4名前期患者和9名常规治疗无效患者)。西罗莫司与常规治疗的第一年总费用差异无统计学意义(8852.63 美元对 9083.56 美元:P 值:0.94)。HCR(244.75 天 VS 168.94 天;P 值:0.60)和 CCR(419.77 天 VS 399.87 天;P 值:0.90)的治疗效果相同。对一线西罗莫司(n = 4)与常规西罗莫司(n = 25)进行的亚组分析显示,每位患者的总费用(4,907.84 美元 VS 9,664.05美元;P 值:0.26)减少了-4,756.21美元(节省费用)。通过减少 HCR(11.67 天 VS 224.20 天;P 值:0.36)和 CCR(38.50 天 VS 470.88 天;P 值:0.04),治疗效果对比更为明显。西罗莫司在住院时间、住院时间和并发症方面没有差异。尽管如此,西罗莫司在成本效益方面仍无法找出显著差异。西罗莫司适用于所有确诊为 KMP 的患者,无论是作为抢救治疗还是一线治疗。
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来源期刊
CiteScore
2.60
自引率
5.90%
发文量
71
审稿时长
6-12 weeks
期刊介绍: PHO: Pediatric Hematology and Oncology covers all aspects of research and patient management within the area of blood disorders and malignant diseases of childhood. Our goal is to make PHO: Pediatric Hematology and Oncology the premier journal for the international community of clinicians and scientists who together aim to define optimal therapeutic strategies for children and young adults with cancer and blood disorders. The journal supports articles that address research in diverse clinical settings, exceptional case studies/series that add novel insights into pathogenesis and/or clinical care, and reviews highlighting discoveries and challenges emerging from consortia and conferences. Clinical studies as well as basic and translational research reports regarding cancer pathogenesis, genetics, molecular diagnostics, pharmacology, stem cells, molecular targeting, cellular and immune therapies and transplantation are of interest. Papers with a focus on supportive care, late effects and on related ethical, legal, psychological, social, cultural, or historical aspects of these fields are also appreciated. Reviews on important developments in the field are welcome. Articles from scientists and clinicians across the international community of Pediatric Hematology and Oncology are considered for publication. The journal is not dependent on or connected with any organization or society. All submissions undergo rigorous peer review prior to publication. Our Editorial Board includes experts in Pediatric Hematology and Oncology representing a wide range of academic and geographic diversity.
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