Long-term vascular health in patients with thrombotic thrombocytopenic purpura

A Prospective Study on the Long-Term Vascular Burden in Thrombotic Thrombocytopenic Purpura Patients

This study is looking at how patients with a rare blood condition called TTP do over a year to see if it affects their blood vessels and overall health in the long run.

Quick facts

• Study type: Observational
• Enrollment: 35 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's (other)
• Locations: 1 site (London, Ontario)
• Trial ID: NCT03187652 on ClinicalTrials.gov

What this trial studies

This observational study follows patients diagnosed with idiopathic thrombotic thrombocytopenic purpura (TTP) and atypical hemolytic uremic syndrome who are in remission. Participants will be monitored for 12 months after their initial scan, with assessments including laboratory tests, biomarker evaluations, cardiovascular and neurocognitive function tests, and non-invasive imaging. The goal is to understand the long-term vascular effects of TTP and develop strategies to prevent further vascular injuries.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older who have recently experienced their first episode of idiopathic TTP and are currently in remission.

Not a fit: Patients with typical hemolytic uremic syndrome or those who do not meet the diagnostic criteria for idiopathic TTP may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved monitoring and prevention strategies for vascular complications in TTP patients.

How similar studies have performed: While there have been studies on TTP, this specific long-term observational approach focusing on vascular burden is novel.

Eligibility criteria

Inclusion Criteria:

* Adults (=18 years) with a recent diagnosis of their first episode of idiopathic TTP, who have been treated with plasma exchange with or without other therapies, and who are in remission. Remission is defined as the normalization of platelet counts and lactate dehydrogenase levels with no clinical signs or symptoms of microvascular injury for more than 30 days. The patients will be included in the study within 30 days of remission of their symptoms. The patients will all meet the following diagnostic criteria for idiopathic TTP: (1) thrombocytopenia with platelet count \<150 x 109 /L, (2) microangiopathic hemolytic anaemia (presence of red blood cell fragmentation by peripheral blood smear), (3) elevation of lactate dehydrogenase (LDH)\> 1.25 X of the upper limit of normal, and (4) ADAMTS13 Activity \< 10%

Exclusion Criteria:

* Patients will be excluded if they have diagnoses of typical HUS (diarrhea-associated HUS), atypical HUS, and disseminated intravascular coagulation, have abnormal international normalized ratio at the time of presentation, have diagnoses of malignant hypertension at the time of presentation, are on the following drugs within 90 days prior to their presentation: ticlopidine, clopidogrel, mitomycin C, gemcitabine, cyclosporine, and quinine, have a history of hematopoietic stem cell transplantations prior to their presentation, have a history and/or diagnosis of vasculitis, systemic lupus erythematosus, scleroderma, rheumatoid arthritis, antiphospholipid antibody syndrome, or HIV/AIDS, have a history of solid organ malignancy within 5 years prior to presentation, i.e. lung, breast, gastric, colon, pancreatic, prostate, or liver, are pregnant at the time of presentation, have severe bronchospasm, unstable angina, and severe ischemic heart disease, have advanced kidney failure (estimated Glomerular Filtration Rate \< 30 mL/min/1.73m2), and have history of allergic reaction to contrast dye.

Where this trial is running

London, Ontario

• London Health Sciences Centre — London, Ontario, Canada (RECRUITING)

Study contacts

• Principal investigator: Huang — London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
• Study coordinator: Huang

Conditions: Thrombotic Thrombocytopenic Purpura