Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by inherited changes in the ADAMTS13 gene that reduce the body's ability to produce the ADAMTS13 enzyme. ADAMTS13 normally cleaves ultra-large multimers of a protein called von Willebrand factor (VWF). In cTTP, low ADAMTS13 activity allows these ultra-large VWF multimers to build up and promote blood clot formation in small blood vessels. These clots can restrict blood flow to vital organs and lead to serious complications. Recombinant ADAMTS13 (rADAMTS13) is a manufactured form of human ADAMTS13 designed to replace the missing enzyme and restore ADAMTS13 activity. This study aims to describe the impact of cTTP on participants before and after treatment with rADAMTS13. It will also evaluate participants' health outcomes after treatment and describe treatment patterns before and after rADAMTS13, including whether treatment was used to prevent or treat TTP episodes, how often it was given, the amount received, and others. In addition, the study will describe pregnancies and outcomes for the mother and baby before and during treatment with rADAMTS13. Only data already available in the medical records of the people who received rADAMTS13 through Takeda's early access program (EAP) for cTTP will be collected and reviewed in this study.
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Number of cTTP Acute and Subacute Episodes Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Hematological Measures (Platelet Count) Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Hematological Measures (Microangiopathic Hemolytic Anemia [MAHA]) Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical Measures (Lactate Dehydrogenase [LDH]) Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical Measures (Proteinuria) Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical Measures (Serum creatinine) Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biomarker Measures Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Number of Participants with cTTP Symptoms Before and During Treatment with rADAMTS13
Timeframe: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration