Abstract
Abstract
Objectives
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare hematological disease whose clinical management includes caplacizumab along with plasma exchange and immunosuppression, according to international guidelines. Caplacizumab has been available in Colombia since 2022. This study seeks to determine the therapeutic classification of caplacizumab according to the methodology of the Instituto de Evaluación Tecnológica en Salud.
Methods
The classification was carried out through a deliberative process following the modified Delphi technique, with a panel of experts, made up of four hemato-oncologists, a pharmaceutical chemist, and a patient. The results of effectiveness and safety obtained through a systematic review, therapeutic thresholds (clinical significance), and degree of acceptability (willingness to use the technology) were used for the classification.
Results
Fourteen effectiveness and safety outcomes were submitted for the classification process. Caplacizumab showed clinical significance for some effectiveness outcomes, was not considered inferior in terms of safety, and displayed acceptability of use. Through consensus, the panel determined that caplacizumab plus the standard regimen is superior to the standard regimen in terms of treatment response and composite outcome, and no different for the other effectiveness and safety outcomes. Likewise, in overall terms, the panel determined that caplacizumab together with the standard regimen is superior to the standard regimen.
Conclusion
Treatment with caplacizumab together with the standard regimen was considered superior to the standard regimen for the treatment of patients with aTTP, as it showed clinically significant benefits in critical outcomes for decision making, and a safety profile no different to its comparator.
Publisher
Cambridge University Press (CUP)
Reference40 articles.
1. 29. Brożek, J , Guyatt, G , Oxman, A. GRADE handbook; 2013.
2. ISTH guidelines for treatment of thrombotic thrombocytopenic purpura
3. 21. European Medicines Agency (EMA). Anexo I ficha técnica o resumen de las características del producto: Caplacizumab. European Medicines Agency (EMA). 2021. Available from: https://www.ema.europa.eu/en/documents/product-information/cablivi-epar-product-information_es.pdf.
4. Efficacy of Caplacizumab in Patients with aTTP in the HERCULES Study According to Baseline Disease Severity
5. Caplacizumab: a change in the paradigm of thrombotic thrombocytopenic purpura treatment