Real-world effectiveness of caplacizumab vs the standard of care in immune thrombotic thrombocytopenic purpura

Article Hematology

Mortality in acquired thrombotic thrombocytopenic purpura in the pre-caplacizumab era

Julio del Rio-Garma et al.

Summary: The effectiveness of drug treatments plays a crucial role in the prognosis of patients with aTTP, with early symptoms and treatment response being key factors in predicting mortality risk.

ANNALS OF HEMATOLOGY (2022)

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Article Hematology

Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura

George Goshua et al.

Summary: The addition of caplacizumab to standard of care in acquired TTP treatment is not cost-effective due to the high cost of the medication and lack of improvement in relapse rates. Further study on the long-term impact of caplacizumab on health system cost is warranted.

BLOOD (2021)

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Editorial Material Hematology

TTP: the evolution of clinical practice Comment

James N. George

Summary: The study shows that caplacizumab is an effective targeted immunotherapy for TTP, with the potential to alter established clinical practices.

BLOOD (2021)

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Article Hematology

Redefining outcomes in immune TTP: an international working group consensus report

Adam Cuker et al.

Summary: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a potentially fatal disease caused by severe deficiency of ADAMTS13, and standardized definitions of response and exacerbation primarily based on platelet count have been modified to incorporate ADAMTS13 activity and the effects of anti-VWF therapy. The revised definitions distinguish between clinical and ADAMTS13 remission and relapse, and are benchmarked against not only the timing of discontinuation of therapeutic plasma exchange (TPE) but also that of anti-VWF therapy. Validation of the revised definitions is retrospective, with discussion on the clinical implications of the updated outcome definitions.

BLOOD (2021)

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Article Hematology

Real-world experience with caplacizumab in the management of acute TTP

Tina Dutt et al.

Summary: In a real-world study of 85 patients receiving caplacizumab, it was found that caplacizumab in combination with PEX and other treatments effectively shortened the time to platelet count normalization in a clinical setting, demonstrating favorable outcomes compared to historical controls.

BLOOD (2021)

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Article Hematology

Incidence, diagnosis, and outcome of immune-mediated thrombotic thrombocytopenic purpura: A nationwide survey by the Spanish registry of thrombotic thrombocytopenic purpura

Cristina Pascual-Izquierdo et al.

Summary: A cross-sectional survey conducted in Spanish hospitals revealed that the incidence and prevalence of iTTP in Spain were slightly higher than neighboring countries. Most patients underwent testing for ADAMTS13 activity and anti-ADAMTS13 autoantibody at diagnosis. Although mortality rate was low, a portion of treated episodes were refractory and required improved diagnosis and treatment.

JOURNAL OF CLINICAL APHERESIS (2021)

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Article Hematology

Successful management of three patients with autoimmune thrombotic thrombocytopenic purpura with paradigm-changing therapy: Caplacizumab, steroids, plasma exchange, rituximab, and intravenous immunoglobulins (CASPERI)

Joan Cid et al.

Summary: The combination of standard care with caplacizumab, steroids, PE, upfront rituximab therapy, and intravenous immunoglobulins was successful in inducing remission in cases of acute aTTP.

TRANSFUSION AND APHERESIS SCIENCE (2021)

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Letter Hematology