米多司他林
医学
全身性肥大细胞增多症
内科学
肿瘤科
危险系数
临床试验
白血病
置信区间
骨髓
作者
H. Pilkington,Sarah Smith,Neil Roskell,Sergio Iannazzo
出处
期刊:Future Oncology
[Future Medicine]
日期:2022-04-01
卷期号:18 (13): 1583-1594
被引量:9
标识
DOI:10.2217/fon-2021-1509
摘要
Objective: This research aimed to compare the relative efficacy of avapritinib versus midostaurin for patients with advanced systemic mastocytosis. Method: A systematic literature review was performed to identify relevant evidence. Unanchored matching-adjusted indirect comparisons were conducted for overall survival (OS), overall response rate (ORR) and complete remission (CR). Results: The systematic literature review identified the clinical trials EXPLORER and PATHFINDER (investigating avapritinib) and D2201 and A2213 (investigating midostaurin). The avapritinib versus midostaurin adjusted hazard ratio for OS was 0.44 (95% CI: 0.25-0.76), and the adjusted odds ratios for ORR and CR were 4.06 (95% CI: 3.09-5.33) and 9.56 (95% CI: 0.97-93.81), respectively. Conclusion: The results suggest that avapritinib improves survival and response (ORR and CR) compared with midostaurin.Systemic mastocytosis is a rare blood disorder caused by the build-up of too many abnormal mast cells, a type of white blood cell, in the skin and organs. Patients with advanced systemic mastocytosis have a low life expectancy and limited treatment options. This research aimed to compare the effectiveness of two recent and innovative treatments (called avapritinib and midostaurin) in extending life expectancy and decreasing mast cells and organ damage. As avapritinib and midostaurin were not investigated in the same clinical studies, it was necessary to compare the two treatments using the results from studies of each individual treatment. The published evidence used to support this comparison was systematically searched for and consisted of four clinical studies: the EXPLORER and PATHFINDER studies (investigating avapritinib) and D2201 and A2213 studies (investigating midostaurin). An indirect comparison between the studies was made that adjusted for differences in key patient characteristics. The results suggest that compared with midostaurin, avapritinib has the potential to extend life expectancy and decrease disease burden.
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