Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Caplacizumab.
- Who it may be relevant to
- Registry conditions: Immune Thrombotic Thrombocytopenia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
1. We expect to find that the silent cerebral infarct (SCI) rate is two fold higher in patients treated without caplacizumab. We also expect to find that the rate of mild and major cognitive impairment in patients treated with caplacizumab within 3 days of starting plasma exchange will be lower than patients treated without caplacizumab. 2. We expect that the differences in cognitive impairment in cases (caplacizumab) versus controls (no caplacizumab) will persist on serial evaluation 1 year later. We also expect that there will be differences in these groups even after adjusting for time since episode and severity of presentation. 3. We expect to find that SCI and cognitive impairment is associated with worse scores on the health related quality of life instrument (SF-36) 4. Based on studies in non-TTP populations, we expect to find that the rate of incident stroke over the period of follow up is at least 2 fold higher in patients that have SCI compared with patients who do not have SCI
Interventions
- Drug Caplacizumab
Early Caplacizumab use
Primary outcome measures
- Silent cerebral infarction [Time frame: 12 months]
- Cognitive impairment [Time frame: 12 months]
Secondary outcome measures (3)
- Depression scores on BDI-II [Time frame: 12 months]
- SF-36 scores for quality of life [Time frame: 12 months]
- Patient reported cognitive performance [Time frame: 12 months]
Eligibility criteria
Inclusion criteria
- Age >= 18 years
- Confirmed iTTP based on ADAMS13 activity < 10 % (or 10-20% with positive inhibitor or antibody) during an acute iTTP episode
- Only 1 episode of iTTP that was treated with plasma exchange and caplacizumab started within 3 days of diagnosis (or if more than 1 episode, then all episodes treated with plasma exchange and caplacizumab started within 3 days of diagnosis)
Exclusion criteria
- Any contraindication for MRI (metallic implants, shrapnel, MRI incompatible stents, etc)
- Unable to speak, read or understand instructions in English (for NIH ToolBox)
- Combination of iTTP episodes treated without caplacizumab or with caplacizumab.
- Caplacizumab started at >= 4 days from diagnosis or for refractory iTTP
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-control
Study locations
United States · 1 center
- USTMA — Groveport
Identifiers
NCT: NCT06358703 · P400