Management of immune thrombotic thrombocytopenic purpura with caplacizumab: a Canadian, single-centre, real-world experience

Tse, Brandon; Buchholz, Megan; Pavenski, Katerina

doi:10.1080/09537104.2022.2157807

Cited by 9 publications

(8 citation statements)

References 28 publications

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“…However, after matching the groups with or without caplacizumab for baseline covariates, the post-PSM analysis confirmed the results on main clinical endpoints, as caplacizumab-treated patients still had tendentially more favorable outcomes in terms of all-cause mortality and iTTP-related hospitalizations. These data are in line with the clinical trial HERCULES [ 9 ] and other European RWE analyses [ 13 , 14 ]. In the HERCULES trial, where iTTP-related death was assessed during the treatment period (30 days), the iTTP-related mortality of caplacizumab-treated patients was 0% (0/72), compared to 4% (3/73) in the placebo group [ 9 ].…”

Section: Discussionsupporting

confidence: 87%

“…These results were also confirmed by Izquierdo et al, who reported a statistically significant difference in hospitalization days between patients treated and untreated with caplacizumab (12 days vs. 19 days, respectively, p < 0.001) [ 12 ]. Moreover, Völker et al described a mean hospitalization length of 21.6 days in patients treated with caplacizumab [ 14 ]. Similarly, the analysis of the Milan TTP Registry indicated a median length of hospital stays for patients treated with caplacizumab of 18 days [ 17 ].…”

Section: Discussionmentioning

confidence: 99%

“…Similarly, the analysis of the Milan TTP Registry indicated a median length of hospital stays for patients treated with caplacizumab of 18 days [ 17 ]. The therapy with caplacizumab implied a mean consumption of around 31 vials per patient during the overall observation period; data from the Milan Registry and German RWE analyses showed a median duration of caplacizumab treatment of 26 and 34 days, respectively [ 14 , 17 , 18 ].…”

Section: Discussionmentioning

confidence: 99%

“…The data from clinical trials have been successively corroborated by real-world evidence (RWE) studies that confirmed the significant clinical and economic benefits of caplacizumab over traditional therapy [ 13 , 14 ].…”

Section: Introductionmentioning

confidence: 97%

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Real-World Data Analysis of Patients Affected by Immune-Mediated Thrombotic Thrombocytopenic Purpura in Italy

Angelucci,

Artoni,

Fianchi

et al. 2024

JCM

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Background: The therapeutic management of immune-mediated thrombotic thrombocytopenic purpura (iTTP) has recently benefited from the introduction of caplacizumab, an agent directed at the inhibition of platelet aggregation. This real-world analysis investigated the epidemiology and the demographic and clinical characteristics of iTTP patients in Italy before and after caplacizumab introduction in 2020. Methods: Hospitalized adults with iTTP were included using the administrative databases of healthcare entities covering 17 million residents. Epidemiological estimates of iTTP considered the 3-year period before and after caplacizumab introduction. After stratification by treatment with or without caplacizumab, iTTP patients were characterized for their baseline features. Results: The annual incidence before and after 2020 was estimated in the range of 4.3–5.8 cases/million and 3.6–4.6 cases/million, respectively. From 2018 to 2022, 393 patients with iTTP were included, and 42 of them were treated with caplacizumab. Caplacizumab-treated patients showed better clinical outcomes, with tendentially shorter hospital stays and lower mortality rates (no treated patients died at either 1 month or 3 months after caplacizumab treatment initiation, compared to 10.5% and 11.1% mortality rates at 1 and 3 months, respectively, of the untreated ones). Conclusions: These findings may suggest that caplacizumab advent provided clinical and survival benefits for patients with iTTP.

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Section: Discussionsupporting

confidence: 87%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 97%

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Real-World Data Analysis of Patients Affected by Immune-Mediated Thrombotic Thrombocytopenic Purpura in Italy

Angelucci,

Artoni,

Fianchi

et al. 2024

JCM

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“…While caplacizumab is approved in the US for the treatment of all patients with iTTP alongside initiation of TPE and immunosuppressants, previous literature shows that certain patients may receive caplacizumab in a delayed manner. 21 , 22 This analysis similarly found that, after the caplacizumab approval until February 2021, a large proportion of patients in the US received delayed caplacizumab treatment. Patients with delayed start of caplacizumab treatment had longer duration of hospital stay by approximately 7 days, higher TPE burden, and 40% higher mean total cost of care (inclusive of caplacizumab cost) than patients who received caplacizumab front-line alongside initiation of TPE and immunosuppressants.…”

Section: Discussionmentioning

confidence: 83%

Outcomes and Costs in Patients with Immune Thrombotic Thrombocytopenic Purpura Receiving Front-Line Versus Delayed Caplacizumab: A US Hospital Database Study

Arnaud,

Schilsky,

Lucia

et al. 2024

Clin Appl Thromb Hemost

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European real-world data indicate that front-line treatment with caplacizumab is associated with improved clinical outcomes compared with delayed caplacizumab treatment. The objective of the study was to describe the characteristics, treatment patterns, and outcomes in hospitalized patients with an immune-mediated thrombotic thrombocytopenic purpura (iTTP) episode treated with front-line versus delayed caplacizumab in the US. This retrospective cohort analysis of a US hospital database included adult patients (≥18 years) with an acute iTTP episode (a diagnosis of thrombotic microangiopathy and ≥1 therapeutic plasma exchange [TPE] procedure) from January 21, 2019, to February 28, 2021. Unadjusted baseline characteristics, treatment patterns, healthcare resource utilization, and costs were compared between patients who received front-line versus delayed (<2 vs ≥2 days after TPE initiation) caplacizumab treatment. Out of 39 patients, 16 (41.0%) received front-line and 23 (59.0%) received delayed treatment with caplacizumab. Baseline characteristics and symptoms were similar between the two groups. Patients who received front-line caplacizumab treatment had significantly fewer TPE administrations (median: 5.0 vs 12.0); and a significantly shorter hospital stay (median: 9.0 days vs 16.0 days) than patients receiving delayed caplacizumab therapy. Both of these were significantly lower in comparison of means (t-test P < .01). Median inpatient costs (inclusive of caplacizumab costs) were 54% higher in the delayed treated patients than in the front-line treated patients (median: $112 711 vs $73 318). TPE-specific cost was lower in the front-line treated cohort (median: $6 989 vs $10 917). In conclusion, front-line treatment with caplacizumab had shorter hospitalizations, lower healthcare resource utilization, and lower costs than delayed caplacizumab treatment after TPE therapy.

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Caplacizumab

2024

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Management of immune thrombotic thrombocytopenic purpura with caplacizumab: a Canadian, single-centre, real-world experience

Cited by 9 publications

References 28 publications

Real-World Data Analysis of Patients Affected by Immune-Mediated Thrombotic Thrombocytopenic Purpura in Italy

Real-World Data Analysis of Patients Affected by Immune-Mediated Thrombotic Thrombocytopenic Purpura in Italy

Outcomes and Costs in Patients with Immune Thrombotic Thrombocytopenic Purpura Receiving Front-Line Versus Delayed Caplacizumab: A US Hospital Database Study

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