Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura (PREDICT2020)

University of Cologne

Status: Completed

Conditions: Acquired Thrombotic Thrombocytopenic Purpura

Treatments: Drug: Cablivi

Study type: Observational

Funder types: Other

Identifiers

NCT05876221

2020-11-17

Details and patient eligibility

About

The interpretation of platelet counts has to be revaluated in the light of caplacizumab. By effectively blocking platelet binding sites on VWF-multimers, the nanobody leads to a rapid normalization of the platelet count within 3 to 4 days. Most importantly, caplacizumab uncouples platelet counts from ADAMTS13 activity and thereby launches unprecedented thrombocyte dynamics, with potential pitfalls for over- and undertreatment.

A relevant number of patients responds to caplacizumab with a brisk increase in platelet count, followed by a marked dip of platelets (patient on the left). This may mislead treating physicians into re-intensifying therapy, with a respective risk for adverse side-effects and complications. Taken together, these observations call for reliable descriptions and the identification of predictive parameters to predict the platelet response upon administration of caplacizumab in a large patient cohort. Here, PREDICT-2020 is designed as a retrospective study to specifically address the following aspects:

• Identifying and describing clusters of platelet responses to caplacizumab
• Identifying potential pitfalls for treating physicians
• Predicting the individual thrombocyte response
• Correlating platelet responses with individual patient outcome

Enrollment: 223 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of autoimmune thrombotic thrombocytopenic purpura
• Treatment with at least a single dose of caplacizumab, either i.v. or s.c.
• Male or female patients older than 18 years of age

Exclusion criteria

• Hereditary thrombotic thrombocytopenic purpura

Trial design

223 participants in 1 patient group

aTTP-Patients

Description:

Patients with immune Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®)

Treatment:

Drug: Cablivi