Abstract: TH-PO121
Use of Caplacizumab in Germany to Treat Acquired Thrombotic Thrombocytopenic Purpura in a Real-World Setting
Session Information
- AKI: Biomarkers, Drugs, Onco-Nephrology
November 07, 2019 | Location: Exhibit Hall, Walter E. Washington Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Acute Kidney Injury
- 102 AKI: Clinical, Outcomes, and Trials
Authors
- Volker, Linus A., University Hospital Cologne, Cologne, Germany
- Kaufeld, Jessica Katharina, Medical School of Hannover, Hannover, Germany
- Brinkkoetter, Paul T., University Hospital Cologne, Cologne, Germany
- Menne, Jan, Medical School Hannover, Hannover, Germany
Background
Acquired thrombotic thrombocytopenic purpura (aTTP), a form of thrombotic microangiopathy, is a rare but life-threatening disease, which is characterized by microangiopathic hemolysis and thrombocytopenia with and without end-organ damage. It results from an autoantibody mediated inhibition of the metalloproteinase ADAMTS13 that is required for the degradation of ultra-large von-Willebrand-factor multimeres. Two recent seminal trials (HERCULES and TITAN) demonstrate the efficacy of the new nanobody caplacizumab for the treatment of this condition. To assess the utility and role of caplacizumab in the treatment of aTTP patients, we analyzed the first 22 German patients that had been treated with the new drug since Mai 2018.
Methods
Retrospective analysis of epidemiologic and treatment related data of the first 23 German aTTP patients undergoing caplacizumab treatment in 12 German medical institutions between Mai 2018 and April 2019.
Results
Between Mai 2018 and April 2019, 22 patients (age range 26-83 years) were treated with caplacizumab in addition to pulsed steroids, plasma exchange and rituximab. On average, patients received 32 daily doses of caplacizumab, and treatment was initiated on day 7 after disease onset. Patients received an average of 12 plasma exchanges. 36 percent of all patients relapsed, however, most patients received caplacizumab only as treatment of refractory disease or relapse and not as front-line therapy. This resulted in only 20% of patients being treated strictly according to the HERCULES trial protocol. One patient died from microangiopathic complications despite early caplacizumab treatment. One patient suffered major bleeding complications, one patient an allergic reaction to the drug; in general, minor bleeding complications were reported but scarce.
Conclusion
Caplacizumab appears to be efficacious and reduced the time to platelet normalization in a real-world setting. Caplacizumab augments therapy options for patients with aTTP. The data presented here and future experience will help address pending questions about patient selection for caplacizumab treatment, therapy monitoring, timing of treatment cessation, and drug safety.