Blood: Nano monoantigen Caplacizumab is actually used in real clinical data in TTP patients

The plasma exchange (PEX) joint immunomodulation strategy is the basis for life-saving treatment of immuno-mediated plateplate reduction cyanosis (iTTP).
Caplacizumab is a new type of anti-von Willebrand factor nanosome, and the results of two multi-center, randomized, placebo-controlled clinical trials have been approved by the European Union and the FDA and have been listed in the UK through the Patient Access Program.
and others collected retrospective data from 22 UK hospitals on 85 patients treated with Caplacizumab in 2018-2020, including four children.
compare patient characteristics and prognostics in practical clinical applications with the end of the Caplacizumab clinical trial and the historic prognostics of the pre-Caplacizumab era.
84 patients who received Caplacizumab treatment for TTP were treated with steroids and lytoxifen at the same time as PEX;
the average time (3 days), PEX medium duration (7 days) and medium hospital stay (12 days) were all comparable to RCT data.
the medium duration of the PEX and the normal time from the start of the PEX to the plateplate count were better than the historical prognosis (p.lt;0.05).
TTP relapse in five patients, who continued to have a below 5iu/dL activity of ADAMTS13.
31 adverse reactions occurred in 26 patients, of which 17 (55%) were bleeding and 5 (16%) were thrombosis events (two were not related to Caplacizumab).
mortality rate was 6 per cent (5/85) and no deaths were attributed to Caplacizumab, which in four cases did not begin to be used until 48 hours after the start of PEX (3-21 days).
data are the first and largest in clinical trials, including pediatric patients, that Caplacizumab actually used in clinical practice in TTP patients.
results represent real clinical practice and provide valuable experience for clinicians around the world in treating TTP.