Clinical Research Directory

Inclusion Criteria: 1. Those who voluntarily signed the informed consent form and were able to comply with the study protocol. 2. Subjects diagnosed with severe ADAMTS13 deficiency, defined as ADAMTS13 activity \<10%, documented in the patient's medical history or at screening. Note: In patients receiving fresh frozen plasma (FFP) or other prophylactic treatments containing ADAMTS13 products, plasma ADAMTS13 activity levels at screening may exceed 10%. hTTP will be documented by ADAMTS13 activity \<10% and biallelic pathogenic ADAMTS13 mutations. Patients with hTTP may be asymptomatic. iTTP will be diagnosed by ADAMTS13 activity \<10% and the presence of an ADAMTS13 activity inhibitor (or comparable test for anti-ADAMTS13 antibodies). The diagnosis of hTTP may be supported by the recovery of ADAMTS13 activity to \>10% during clinical remission. 3. Subjects do not exhibit any severe symptoms of TTP at the time of screening. At screening, patients with mild but stable laboratory abnormalities (LDH not higher than three times the upper limit of normal; platelet count not less than 100,000/microliter) are eligible for enrollment. 4. No stroke was detected on cranial MRI and there was no previous history of stroke. 5. The subject is willing and able to comply with the requirements of this protocol. Exclusion Criteria: 1. Subject has a history of significant neurological events, such as a major stroke, indicating that a relapse may have serious consequences, as judged by the investigator 2. Subject has increased risk for bleeding (e.g., platelet count \<30,000/µL, severe coagulopathy, gastrointestinal disease) 3. Subject has a history of drug and/or alcohol abuse within six months before enrollment. 4. Subject has a life expectancy of fewer than three months. 5. The investigator considers the subject unable or unwilling to cooperate with the study procedures. 6. The subject is a family member or employee of the investigator. 7. The patient is pregnant or breast-feeding.