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Impact of first-line use of caplacizumab on treatment outcomes in immune thrombotic thrombocytopenic purpura
Ist Teil von
Journal of thrombosis and haemostasis, 2023-03, Vol.21 (3), p.559-572
Ort / Verlag
England: Elsevier Inc
Erscheinungsjahr
2023
Link zum Volltext
Quelle
MEDLINE
Beschreibungen/Notizen
The von Willebrand factor–directed nanobody caplacizumab has greatly changed the treatment of immune thrombotic thrombocytopenic purpura (iTTP) in recent years. Data from randomized controlled trials established efficacy and safety.
This study aims to address open questions regarding patient selection, tailoring of therapy duration, obstacles in prescribing caplacizumab in iTTP, effect on adjunct treatment, and outcomes in the real-world setting.
We report retrospective, observational cohorts of 113 iTTP episodes treated with caplacizumab and 119 historical control episodes treated without caplacizumab. We aggregated data from the caplacizumab phase II/III trials and real-world data from France, the United Kingdom, Germany, and Austria (846 episodes, 396 treated with caplacizumab, and 450 historical controls).
Caplacizumab was efficacious in iTTP, independent of the timing of therapy initiation, but curtailed the time of active iTTP only when used in the first-line therapy within 72 hours after diagnosis and until at least partial ADAMTS13-activity remission. Aggregated data from multiple study populations showed that caplacizumab use resulted in significant absolute risk reduction of 2.87% for iTTP-related mortality (number needed to treat 35) and a relative risk reduction of 59%.
Caplacizumab should be used in first line and until ADAMTS13-remission, lowers iTTP-related mortality and refractoriness, and decreases the number of daily plasma exchange and hospital stay. This trial is registered at www.clinicaltrials.gov as #NCT04985318.
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•It is unclear whether caplacizumab should be used in the first-line therapy or be reserved for severe cases of iTTP.•Observational, retrospective cohort study comparing 113 iTTP patients treated with caplacizumab with 119 historical controls in Germany and Austria.•Caplacizumab is efficacious independent from the time of therapy start, but benefits may be diminished if not used in first line.•Aggregated data from randomized, controlled trials and real-world cohorts indicate a relative risk reduction of 2.87% for mortality with a number needed to treat of 35.