Acquired Thrombotic Thrombocytopenic Purpura
Conditions
Keywords
aTTP, caplacizumab
Brief summary
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.
Interventions
DRUGCablivi®
Intervention with Cablivi® take place outside of the study
Sponsors
University of Cologne
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment of aTTP with caplacizumab | Enrollment | Description of the prescription rationale and practice of caplacizumab in the treatment of aTTP |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Stabilization of thrombocyte | Enrollment | 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 |
| Normalization of LDH | Enrollment | 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 |
| Normalization of haptoglobin | Enrollment | 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 |
| Risk factors for complications | Enrollment | Identification of risk factors for complications |
| Risk factors for adverse outcomes | Enrollment | Identification of risk factors for adverse outcomes |
| Risk factors for persistent autoimmune activity | Enrollment | Identification of risk factors for persistent autoimmune activity |
| Parameters for therapy guidance | Enrollment | Identification of parameters that guide duration and regimen of caplacizumab treatment |
| Effect of caplacizumab on adjunct treatments with PEX | Enrollment | Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of therapeutic plasma exchange therapy (PEX) |
| Effect of caplacizumab on adjunct treatments | Enrollment | Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of glucocorticoids |
Countries
Germany
Contacts
PRINCIPAL_INVESTIGATORLinus Völker, MD
Department II of Internal Medicine, University of Cologne
Outcome results
None listed