Thrombotic thrombocytopenic purpura (TTP), also called Moschcowitz disease, is described in the packet definition as an aggressive and life-threatening form of thrombotic microangiopathy, characterized by profound peripheral thrombocytopenia, microangiopathic hemolytic anemia, and organ failure of variable severity. It comprises congenital TTP and acquired TTP, which are listed as its two subtypes. Prevalence is not certified in this packet.

Per the packet definition, the characteristic findings are profound peripheral thrombocytopenia, microangiopathic hemolytic anemia, and organ failure whose severity varies. Individual phenotypes and age-of-onset categories are not certified in this packet.

The packet divides the condition into congenital TTP and acquired TTP. Causal genes, inheritance patterns, and molecular mechanism are not certified in this packet, so the genetic basis is not described here.

Diagnostic criteria and testing methods are not certified in this packet. The definition names the combination of thrombocytopenia, microangiopathic hemolytic anemia, and organ involvement as defining features.

The packet lists two FDA-approved therapies with ACTIVE market status. Adzynma (ADAMTS13, recombinant-krhn) was approved under a BLA on 2023-11-09; its orphan record gives the indication as TTP including congenital, acquired idiopathic, and secondary forms. Cablivi (caplacizumab) was approved under a BLA on 2019-02-06 for treatment of TTP. Several other products, including anfibatide, defibrotide, and S-59 treated plasma, carry FDA orphan drug designation for TTP; designation does not indicate approval. Two orphan records, an anti-von Willebrand aptamer and a solvent/detergent treated plasma, have WITHDRAWN status. Management guidance such as GeneReviews is not included in this packet.

Prognosis is not certified in this packet. The definition describes the condition as aggressive and life-threatening.

ClinicalTrials.gov records in the packet include NCT07353099, a recruiting Takeda study in children and adults with congenital TTP treated with Adzynma; NCT04683003, a Phase 3 study of TAK-755 in congenital TTP, listed as active, not recruiting; and NCT06831058, a Phase 2 pilot study of efgartigimod in immune-mediated TTP, sponsored by the University of Minnesota and recruiting. Other recruiting or ongoing studies include daratumumab in immune-mediated TTP. No patient organizations are certified in this packet.