ABSTRACT
BACKGROUND: Von Hippel-Lindau (VHL) disease is a rare autosomal dominant disorder that predisposes patients to develop multiple cysts and tumors, such as hemangioblastomas (HBs) and clear cell renal cell carcinoma (ccRCC), due to mutations in the VHL tumor suppressor gene. While treatment of HBs varies based on their characteristics and has improved patient survival, it still involves high morbidity and mortality, leading to ongoing debates and studies to refine therapy strategies. Recent developments include the emergence of Belzutifan, a novel inhibitor targeting hypoxia-inducible factor 2α (HIF-2α), which has shown promising results in ongoing trials, particularly for patients not immediately requiring surgery. METHODS: This systematic review and meta-analysis aimed to comprehensively evaluate the efficacy and safety of Belzutifan for treating HBs associated with VHL disease. Search was conducted across Medline, Embase, Cochrane, and Web of Science databases. Statistical Analysis was performed, with proportions and 95 % confidence intervals. Statistical analyses were carried out using R Studio. RESULTS: Ten studies were selected, comprising 553 patients. The population mean age was 40 (24-65), and 50 % of the population was formed by males. In terms of proportion, 6 analyses were performed: Disease Stability of 31 % [95 %CI:14 %-47 %; I2 = 2 %]; Disease Progression of 2 %[95 %CI:0 %-9 %; I2 = 0 %]; Partial Response of 75 % [95 %CI:54 %-96 %; I2 = 58 %]. Complete response of 1 % [95 %CI:0 %-7 %; I2 = 0 %];and Side effects, anemia 81 % rate [95 % CI:54 %-100 %; I2 = 94 %], and fatigue rate of 79 % [95 % CI:54 %-100 %;I2 = 94 %]. CONCLUSION: Results indicate that Belzutifan effectively stabilizes disease, reduces tumor progression, and achieves significant therapeutic responses, although side effects like anemia and fatigue were noted.
ABSTRACT
The use of invasive or noninvasive intracranial pressure (ICP) monitoring post-decompressive craniectomy (DC) has been a continuous matter of debate. Accordingly, this meta-analysis aims to examine the existing evidence of both approaches and compare their impact among patients undergoing DC, guiding clinical decision-making in the management of elevated ICP. The databases used were Pubmed, Cochrane, Web of Science, and Embase. Inclusion criteria included: (1) English studies; (2) randomized and nonrandomized studies; (3) reporting on invasive OR noninvasive ICP monitoring after DC; (4) with at least one of the outcomes of interest: incidence of mortality, new cerebral hemorrhages, and the Glasgow Outcome Scale. The study followed the Cochrane and Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines. Thirty-six studies were included in this meta-analysis, resulting in a sample of 1624 patients. One thousand two hundred eighty-six underwent invasive monitoring, and 338 underwent noninvasive methods. In the invasive group, a mortality rate of 17% (95% confidence interval [CI]: 12%-22%), a good outcome rate of 58% (95% CI: 38%-49%), a poor outcome rate of 42% (95% CI: 21%-62%), and an overall incidence of new hemorrhages of 4% (95% CI: 0%-8%) were found. Whereas in the noninvasive sample, a mortality rate of 20% (95% CI: 15%-26%) and a good outcome rate of 38% (95% CI: 25%-52%) were obtained. It seems that the effectiveness of invasive and noninvasive ICP monitoring methods are comparable in post-DC patients. While invasive monitoring remains gold standard, noninvasive methods offer a safer and cost-effective alternative, potentially improving post-DC patient care, and can mostly be used simultaneously with invasive methods.