OBJECTIVES: Internationally, caplacizumab has been included as part of the initial treatment of acquired thrombotic thrombocytopenic purpura (aTTP) along with plasma exchange and immunosuppression. In Colombia, caplacizumab was approved in January 2022. This research aims to determine the therapeutic qualification of caplacizumab in conjunction with plasma exchange and immunosuppression, compared with the pre-caplacizumab standard regimen (plasma exchange and immunosuppression), using the methodology of the local HTA agency (Colombian Institute of Technological Evaluation in Health).

METHODS: Qualification was performed following the modified Delphi technique with a panel of experts composed of four haemato-oncologists, a pharmaceutical chemist, and one patient. For the qualification, the results of effectiveness and safety obtained through a systematic review of the literature (statistical significance), therapeutic threshold values (clinical significance), and degree of acceptability (willingness to use the technology) were considered. The threshold and acceptability were previously established in a deliberative process with the same panel of experts.

CONCLUSIONS: Treatment using caplacizumab together with plasma exchange and immunosuppression was considered superior to plasma exchange and immunosuppression for the treatment of patients with aTTP, since it showed clinically significant benefits in critical outcomes for decision making and has a safety profile which is not different from its comparator.