Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP

Study Overview
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Brief Summary
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The objective of this national, prospective, multi-centre observational study is to describe the prescription rational and practice in Germany, confirm the efficacy of caplacizumab in a real-world setting, and identify predicting factors in iTTP-patients with regard to persistent autoimmune activity, therapy guidance and risk of complications. The rational is to develop new treatment algorithms that optimize overall patient outcome and reduce treatment cost.

Official Title
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Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura (aTTP)- REACT-2020

Conditions
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Acquired Thrombotic Thrombocytopenic Purpura

Intervention / Treatment
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* Drug: Cablivi®


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed diagnosis of an acute episode of acquired thrombotic thrombocytopenic purpura Treatment with at least one single dose of caplacizumab (10 mg i.v. or s.c.) Male or female patients ≥ 18 years of age signed written informed consent Exclusion Criteria: Hereditary thrombotic thrombocytopenic purpura disability to give informed consent

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Registergroup<br>Patients with acquired Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®) | Drug: Cablivi®<br>* Intervention with Cablivi® take place outside of the study<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment of aTTP with caplacizumab | Description of the prescription rationale and practice of caplacizumab in the treatment of aTTP | Enrollment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Stabilization of thrombocyte | Documentation of the efficacy of caplacizumab in the treatment of aTTP outside of clinical trials (real-world data) using by means of stabilization of thrombocyte. Thrombocyte stabilization is defined as thrombocyte > 150 x 10E9/L | Enrollment | 
| Normalization of LDH | Documentation of the efficacy of caplacizumab in the treatment of aTTP outside of clinical trials (real-world data) using by means of normalization of Lactate dehydrogenase (LDH). Normalization of LDH is defined as LDH below upper limit of normal | Enrollment | 
| Normalization of haptoglobin | Documentation of the efficacy of caplacizumab in the treatment of aTTP outside of clinical trials (real-world data) using by means of normalization of haptoglobin. Normalization of haptoglobin is defined as haptoglobin above lower limit of normal | Enrollment | 
| Risk factors for complications | Identification of risk factors for complications | Enrollment | 
| Risk factors for adverse outcomes | Identification of risk factors for adverse outcomes | Enrollment | 
| Risk factors for persistent autoimmune activity | Identification of risk factors for persistent autoimmune activity | Enrollment | 
| Parameters for therapy guidance | Identification of parameters that guide duration and regimen of caplacizumab treatment | Enrollment | 
| Effect of caplacizumab on adjunct treatments with PEX | Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of therapeutic plasma exchange therapy (PEX) | Enrollment | 
| Effect of caplacizumab on adjunct treatments | Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of glucocorticoids | Enrollment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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aTTP, caplacizumab