Summary

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• This summary is based on the review of four systematic review(s)/meta-analysis(es). ^[1-4]
• Caplacizumab significantly reduced all-cause mortality in thrombotic thrombocytopenic purpura (TTP) patients in some systematic reviews, although one review reported a nonsignificant reduction in mortality risk (relative risk (RR) 0.21; 95% confidence interval (CI) 0.05-1.74 in randomized controlled trials (RCTs)). Another review found no significant difference in mortality between caplacizumab and placebo (RR 0.56; 95% CI 0.18-1.72).
• Caplacizumab was associated with a significant reduction in refractory immune thrombotic thrombocytopenic purpura (iTTP) and exacerbation risks, while shortening the time to platelet count normalization (weighted mean difference (WMD) -1.18; 95% CI -2.55 to -0.19), plasma exchange time (WMD -2.97; 95% CI -4.44 to -1.50), and hospital stay (WMD -2.88; 95% CI -4.56 to -1.21). However, it increased the risk of relapse in some studies.
• Caplacizumab was associated with an increased risk of bleeding in randomized controlled trials (RR 1.37, 95% CI 1.06-1.77), though this risk was not significantly elevated in observational studies (RR 7.10, 95% CI 0.90-56.14).
• There were no significant differences in major thrombotic events compared to standard treatment (RR 1.01, 95% CI 0.65-1.57).
• The studies focused on patients with TTP, including acquired and immune types, and demonstrated that caplacizumab was effective in reducing refractory TTP and improving time to response. No specific subgroup differences related to age, gender, or comorbid conditions were reported.