Positive Results for aTTP Announced by New England Journal of Medicine

Patients with acquired thrombotic thrombocytopenic purpura (aTTP) showed a significantly improved platelet count response after treatment with Cablivi® according to the New England Journal of Medicine‘s Phase 3 trial results.

A Rare Life-Threatening Blood Disorder

Acquired thrombotic thrombocytopenic purpura (aTTP) affects clot formation in small blood vessels and thus results in a severely low platelet count, anemia and the restriction of blood supply, together with organ damage (especially in the heart and brain).

Despite the current treatment for aTTP (the daily removal of blood plasma and replacement with donor plasma) patients are at risk of blood clotting that could lead to a stroke or heart attack.

The Phase 3 Study of Cablivi (caplacizumab)

The time to platelet count normalization was significantly reduced by the administration of Cablivi.  In comparison to the placebo cohort, normal platelet counts increased an average of 1.55 for Cablivi patients.  In addition, the drug was associated with a 74 percent reduction in aTTP-related deaths or recurrence of the disease.

Results were also encouraging due to the reduction of patients requiring daily plasma exchange or time in the intensive care unit.


At least one adverse event was experienced during the trial period by the majority of patients in both groups (96% Cablivi; 90% placebo).  Mucocutaneous bleeding, involving skin and mucous membrane, was the most common adverse event experienced by both cohorts (65% Cablivi; 48% placebo).

The Biologics License Application for Cablivi has been accepted for priority review by the Food and Drug Administration for treatment of aTTP patients 18 years of age and older with a target date of February 6, 2019.


















he European Commission approved Cablivi in August 2018.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email

What are your thoughts on being a rare disease advocate? Share your stories, thoughts, and hopes with the Patient Worthy community!

Close Menu