Add-on Cablivi improves treatment for iTTP patients, study finds
Data analysis shows early treatment could reduce time patients spend in hospital
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Treatment with Cablivi (caplacizumab-yhdp) as an add-on to standard therapy significantly reduced the number of daily plasma exchange (PEX) treatments among people with immune-mediated thrombotic thrombocytopenic purpura (iTTP) compared with standard therapy alone in real-world clinical settings.
That’s according to a meta-analysis of pooled data from 13 real-world studies involving nearly 3,000 patients. Data also showed that platelet counts normalized more quickly with Cablivi, with fewer exacerbations, shorter hospital stays, and a lower risk of death.
The researchers said starting Cablivi as soon as possible, ideally at diagnosis and/or at the first plasma exchange, may significantly reduce potential hospitalization time. The findings “support incorporating [Cablivi] into standard management of iTTP across clinical settings,” they wrote.
The study, “An updated systematic review and meta-analysis of Caplacizumab for immune thrombotic thrombocytopenic purpura: Insights into efficacy and safety,” was published in Blood Reviews.
iTTP, also called acquired TTP, develops when self-directed antibodies target the enzyme ADAMTS13 and trigger the widespread aggregation of platelets, the clot-forming fragments in blood. This reduces platelet counts, damages red blood cells, and can cause clots to block blood flow to organs, resulting in a broad and often severe range of TTP symptoms.
Many relapse despite plasma exchange
PEX, a standard TTP treatment, involves replacing a patient’s blood plasma (blood lacking cells that contain self-directed antibodies) with healthy donor plasma. Corticosteroids are typically administered alongside PEX to suppress the underlying autoimmune response.
Nevertheless, about half of patients see their symptoms come back after initial recovery. Persistent or recurrent ADAMTS13 deficiency is a major risk factor for these relapses.
Cablivi, marketed by Sanofi, is approved to treat iTTP patients 12 and older in combination with PEX and immunosuppressive therapy. The clinical trials that supported its approval showed that Cablivi, when added to standard treatment, led to faster platelet count normalization and shorter hospital stays than standard therapy alone.
Despite these benefits, evidence on Cablivi’s efficacy and safety in real-world clinical settings has yielded variable results, mainly due to differences in study design and enrollment sizes.
When faced with variable findings in the medical literature, scientists can conduct a meta-analysis, which pools data from multiple studies that address a similar question.
The research team, in Qatar, said their meta-analysis was designed “to update the available evidence and provide adequate real-world evidence regarding the role of [Cablivi] in treating iTTP.”
A search of the medical literature identified 13 real-world studies, involving 2,956 people with iTTP. The mean patient age was 45.3, and the average follow-up was 12 months. Cablivi was usually started early, within three days of TTP diagnosis or PEX initiation. Most patients also received standard therapies, including PEX, corticosteroids, and rituximab.
According to the meta-analysis, Cablivi treatment significantly reduced the number of daily PEX sessions and the rate of exacerbations (sudden worsening of symptoms) compared with those not treated with Cablivi. Cablivi-treated patients achieved normal platelet counts more quickly, had a lower risk of death, and had shorter hospital stays.
In studies comparing timing, early initiation of Cablivi significantly reduced hospital length of stay without increasing the risk of death compared with late initiation.
“These observations underscore the unpredictability of early deterioration and emphasize the importance of initiating [Cablivi] as early as possible, ideally at the time of iTTP diagnosis and [PEX] initiation,” the team wrote.
Cablivi showed no significant benefit or concerns with respect to relapse rates, major bleeding, blood-clotting events, or time in the intensive care unit.
“This comprehensive meta-analysis supports the efficacy of [Cablivi] as an adjunctive treatment for iTTP,” the researchers concluded. “[Cablivi] should be initiated as early as possible in an acute iTTP episode (ideally at diagnosis with first plasma exchange) to speed platelet recovery and shorten hospital stay.”
