Impact of first-line use of caplacizumab on treatment outcomes in immune thrombotic thrombocytopenic purpura

Völker, Linus A.; Kaufeld, Jessica; Balduin, Gesa; Merkel, Lena; Kühne, Lucas; Eichenauer, Dennis A.; Osterholt, Thomas; Hägele, Holger; Kann, Martin; Grundmann, Franziska; Kolbrink, Benedikt; Schulte, Kevin L.; Gäckler, Anja; Kribben, Andreas; Boss, Kristina; Potthoff, Sebastian A.; Rump, Lars Christian; Schmidt, Tilman; Mühlfeld, Anja; Schulmann, Karsten; Hermann, Matthias; Gaedeke, Jens; Sauerland, Kristin; Bramstedt, Jörn; Hinkel, Ulrich P.; Miesbach, Wolfgang; Bauer, Frederic; Westhoff, Timm H.; Bruck, Heike; Buxhofer‐Ausch, Veronika; Müller, Tobias; Wendt, Ralph T.; Harth, Ana; Schreiber, Adrian; Seelow, Evelyn; Tölle, Markus; Gohlisch, Christopher; Bieringer, Markus; Geuther, Gesa; Jabs, Wolfram J.; Fischereder, Michael; Bergwelt‐Baildon, Anke von; Schönermarck, Ulf; Knoebl, Paul; Menne, Jan; Brinkkoetter, Paul T.

doi:10.1016/j.jtha.2022.11.010

Cited by 13 publications

(6 citation statements)

References 27 publications

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“…The results of this analysis are consistent with previously published conclusions from Spanish and German real-world studies 18 , 19 and suggestions from ISTH guidelines which state that caplacizumab benefit is greatest when given earlier. 23 Similarly to previous publications, adverse outcomes were likely a consequence of delayed initiation of caplacizumab.…”

Section: Discussionsupporting

confidence: 90%

“…These two studies found that front-line use of caplacizumab alongside initiation of TPE and immunosuppression was associated with shorter time to clinical response, significantly shorter time to platelet normalization, significantly lower rates of exacerbations, significantly lower rates of refractoriness, significantly fewer TPE treatments, and significantly shorter hospitalization duration as opposed to delayed start. 18 , 19 …”

Section: Introductionmentioning

confidence: 99%

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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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Section: Discussionsupporting

confidence: 90%

Section: Introductionmentioning

confidence: 99%

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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“…Then, after removing duplicates, followed by the title and abstract screening, the full text of 41 studies was assessed to fulfill the eligibility criteria. Finally, two RCTs [1–4] and seven observational studies review [15–19] nine studies were selected for review.…”

Section: Resultsmentioning

confidence: 99%

Does Caplacizumab for the management of thrombotic thrombocytopenic purpura increase the risk of relapse, exacerbation, and bleeding? An updated systematic review and meta‐analysis based on revised criteria by the International Working Group for thrombotic thrombocytopenic purpura

Neupane,

Thapa,

Mahmoud

et al. 2023

eJHaem

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Thrombotic thrombocytopenic purpura (TTP) is a rare and life‐threatening condition marked by abnormal blood clotting and organ damage. Caplacizumab is a potential treatment for the TTP management. This systematic review and meta‐analysis aimed to assess Caplacizumab's effectiveness and safety in the TTP management. A comprehensive database search identified nine studies, including randomized controlled trials and observational studies. Primary outcomes included TTP exacerbation, relapse, and major bleeding. Major bleeding risk was evaluated using updated definitions recommended by the International TTP Working Group in 2021. Revised criteria proposed by the IWG for TTP recurrence were employed for a comprehensive assessment of Caplacizumab's impact on relapse and exacerbation. Analysis revealed Caplacizumab significantly reduced all‐cause mortality in TTP patients. Some studies raised concerns about bleeding risk, but overall, it did not significantly differ from standard treatment. Likewise, there was no significant difference in TTP relapse rates between Caplacizumab and standard care. This study supports Caplacizumab as a potential adjunct therapy for TTP. However, careful consideration of its advantages and risks is crucial in clinical practice. Further research is needed to address concerns related to adverse effects like bleeding risk and relapse rates associated with Caplacizumab in the TTP management. The findings emphasize the importance of weighing potential benefits and risks when considering Caplacizumab as an adjunct therapy for TTP.

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“…Positive results were provided by two randomized controlled trials where unfavourable outcomes including mortality, refractoriness and exacerbations were less commonly observed when caplacizumab was added. Based on these results, also confirmed by real‐world experience studies, 5,6 caplacizumab was approved for iTTP treatment by the European Medicines Agency in Europe (September 2018) and the US Food and Drug Agency (February 2019) and mentioned as part of frontline treatment by the International Society on Thrombosis and Haemostasis guidelines 7 . In these trials, however, pregnancy was a non‐inclusion criterion as caplacizumab induces a von Willebrand disease‐like syndrome and may have exposed both the mother and the foetus to bleeding complications.…”

mentioning

confidence: 79%

Caplacizumab: A game changer also in pregnancy‐associated immune‐mediated thrombotic thrombocytopenic purpura?

Coppo

Joly

2023

Br J Haematol

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Therapeutic options in immune‐mediated thrombotic thrombocytopenic purpura (iTTP) during pregnancy are limited besides therapeutic plasma exchange (TPE) and corticosteroids. The report by Odetola et al. suggests that caplacizumab represents a reasonable option in iTTP during pregnancy, especially when the disease is not rapidly controlled with the standard TPE–corticosteroid association.Commentary on: Odetola et al. Safe and effective use of caplacizumab in pregnancy‐related acquired thrombotic thrombocytopenic purpura. Br J Haematol 2023 (Online ahead of print). doi: 10.1111/bjh.18888.

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Impact of first-line use of caplacizumab on treatment outcomes in immune thrombotic thrombocytopenic purpura

Cited by 13 publications

References 27 publications

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

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

Does Caplacizumab for the management of thrombotic thrombocytopenic purpura increase the risk of relapse, exacerbation, and bleeding? An updated systematic review and meta‐analysis based on revised criteria by the International Working Group for thrombotic thrombocytopenic purpura

Caplacizumab: A game changer also in pregnancy‐associated immune‐mediated thrombotic thrombocytopenic purpura?

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