GlobalResearch and clinical trials
Recombinant ADAMTS13 enzyme in congenital TTP: final results of a phase 3 study published
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
A total of 48 people aged 3–68 with congenital TTP took part in the study. Each person received the recombinant ADAMTS13 enzyme for six months and plasma treatment for six months. There were no acute TTP episodes during the enzyme period. One episode occurred during the plasma period. Treatment-related side effects were 4.3% with the enzyme and 45.8% with plasma.
What does it mean for patients and families?
These results confirm the benefit of enzyme treatment in congenital TTP seen in the interim analysis. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes; the decision is made with a hematologist.
Source
Scientific paper: Recombinant ADAMTS13 in congenital thrombotic thrombocytopenic purpura: final analysis from a randomized phase 3 trial. Blood, 2026. DOI: 10.1182/blood.2026034361 · PubMed 42728013
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Thrombotic thrombocytopenic purpura (TTP)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.