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.
Participants of any age can participate who have a confirmed diagnosis of severe congenital ADAMTS13 deficiency or cTTP.
Participants must be on preventative or prophylactic treatment for cTTP or must have had at least one TTP event in the past.
Participants must have no other treatment options available (this includes other clinical studies for cTTP).
Pediatric and adult participants (no age restrictions) with cTTP, treated with rADAMTS13 via the EAP, who received at least two administrations of rADAMTS13 and who have provided consent (or the legal guardians) to participate in this retrospective chart review.
None
Adult and pediatric participants treated with rADAMTS13 via the EAP at participating centers will be included in this study.
Cohort
Retrospective
No Intervention
Other interventionThis is a non-interventional study.
Number of cTTP Acute and Subacute Episodes Before and During Treatment with rADAMTS13
Acute episodes will be defined as those with clinically overt features of thrombotic microangiopathy, including thrombocytopenia, microangiopathic hemolytic anemia, and/or organ dysfunction, often requiring urgent therapeutic intervention. Subacute or non-overt episodes will be defined as episodes with laboratory evidence of thrombocytopenia or hemolysis without significant clinical symptoms or organ involvements, often detected on routine monitoring.
Changes in Hematological Measures (Platelet Count) Before and During Treatment with rADAMTS13
Changes in hematological measures like platelet count (thrombocytopenia) will be reported.
Changes in Hematological Measures (Microangiopathic Hemolytic Anemia [MAHA]) Before and During Treatment with rADAMTS13
Changes in hematological measures like MAHA will be reported.
Changes in Biochemical Measures (Lactate Dehydrogenase [LDH]) Before and During Treatment with rADAMTS13
Changes in Biochemical measures like LDH will be reported.
Number of Reasons for rADAMTS13 Early Access Request
Number of reasons for early access request will be described.
Number of Treatment Initiation Characteristics at the First Dose of rADAMTS13
Treatment initiation characteristics includes first rADAMTS13 treatment, participants weight at initiation of treatment, starting dose of rADAMTS13, other dose than specified in summary of product characteristics for prophylaxis or acute treatment, starting prophylaxis dose, frequency and duration will be reported.
Number of Treatment Changes During rADAMTS13 Treatment
Treatment changes during rADAMTS13 treatment (for each treatment episode) includes treatment purpose, treatment dates, location, doses and duration, any change(s) to dose, dosing frequency or duration, including dates and the reason(s), additional cTTP prophylaxis treatments will be reported.
Peak Activity Level of ADAMTS13
Peak ADAMTS13 activity level i.e., highest level post-infusion will be reported.
This trial is not recruiting at the moment. You can still explore other options:
