Drug updated on 4/18/2024
| Dosage Form | Injection (intravenous, subcutaneous; 11 mg/vial) |
| Drug Class | Von Willebrand factor-directed antibody fragments |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.
Summary
- Caplacizumab-yhdp (Cablivi) is indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy. It significantly improves treatment outcomes when compared to standard care alone, reducing all-cause mortality, time to response, exacerbation rates, and refractoriness.
- The findings were derived from four systematic reviews/meta-analyses on caplacizumab's safety and effectiveness for treating aTTP.
- While caplacizumab has been shown to reduce the time needed for platelet count recovery and length of plasma exchange treatment, it also carries an increased risk of bleeding. This necessitates careful patient selection and monitoring during its use.
- Compared to other therapies such as plasma exchange or immunosuppressive therapy without caplacizumab, this drug provides clinical benefits including reduced mortality rate, and a shortened disease remission period but at the expense of an elevated risk for bleeding incidents.
- Although detailed subgroup analyses were not provided, the studies suggest that Cablivi is particularly beneficial among adults suffering from aTTP. Future research should focus on identifying specific subgroups that could benefit the most while minimizing adverse effects like excessive bleeding risks associated with this medication's usage.
- In conclusion, despite some concerns about potential side effects such as a higher incidence of bleeding, overall findings indicate significant improvements across various endpoints such as lower death rates and faster response times, which make it worth considering, especially given its proven efficacy against complex life-threatening conditions like aTTP where individual patient characteristics must be taken into account before deciding upon therapeutic strategies due to the inherent risks versus benefits trade-offs involved.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Cablivi (caplacizumab-yhdp) Prescribing Information. | 2023 | Ablynx N.V., Ghent (Zwijnaarde), Belgium |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. | 2020 | The International Society on Thrombosis and Haemostasis |