Study Objectives
Primary objective: To determine the safety of therapies used in the treatment of participants with ________ or ________ non-neoplastic blood disorders and connective issue disorders with bleeding tendency.
Congenital or Acquired
The study has a target of _______ participants.
3000
Participants who meet the following inclusion criteria are eligible for enrollment into this cohort:
1. Meeting the definition of VWD or low VWF per most recent international guidelines
Von Willebrand Disease Cohort
Study visit will be timed to coincide ________ whenever possible.
routine or scheduled care
Inhibitor testing will be performed at a central lab for all participants in this cohort.
Hemophilia Cohort
According to the primary objective, safety will be measured by those events in the ______________ (EUHASS)
European Haemophilia Safety Surveillance
Participants will be followed for a minimum of ____ years.
15
Participants who meet the following inclusion criteria are eligible for enrollment into this cohort:
1. Having any congenital or acquired non-neoplastic hematologic disorder not included in any other cohort
Non-Neoplastic Hematologic Conditions Cohort
Visit windows will be +1 week for baseline visit, +/-2 weeks for quarterly visits, and +/- ___ weeks for annual visit.
Eight
Any inhibitor test that results in a positive titer should be repeated by the central lab within _______ of result receipt for confirmation.
10 days
A subject has experienced a drug induced liver injury. This information is collected by the study as an _______.
AESI
Each participant will be assigned in _____ cohort(s).
Participants who meet the following inclusion criteria are eligible for enrollment into this cohort:
1. Have a bleeding phenotype as indicated by an age-adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; OR
2. Connective tissue disorder with bleeding tendency as indicated by an age-adjusted abnormal ISTH Bleeding Assessment Tool score
Bleeding NOS Cohort
If a participant enrolls in an Arm within _______ of being enrolled in the Base, then the baseline assessments from the Cohort can be used for the baseline of assessments in the Arm if the assessment was already completed.
3 months
All participants will have the option of having specimens drawn (about 5mL each) at baseline to be stored in the ARB. ARB stands for ____________.
ATHN Research Biorepository
The following is considered an AESI: The development of __________ antibodies, to be measured and confirmed, if feasible
anti-drug
Patients of this age are eligible to participate.
Any age
A patient with a Thromboxane Receptor Defect would be eligible to participate in this cohort.
Congenital Platelet Disorder Cohort
1. Study enrollment/Baseline - Study activities will begin after consent and relevant authorizations are obtained.
2. Quarterly visits-phone contact or clinic visit
3. _______ follow-up for study specific safety events and treatment switches
4. Annual Study Visit
5.Study Exit
Ad hoc
For participants receiving non-factor products (e.g., emicizumab), if available, this type of testing will be performed by an appropriate laboratory, at baseline, annually, and at any timepoint of clinical suspicion of poor response.
Anti-drug Antibodies Testing
To describe bleeding events, changes in overall bleeding, and annualized bleeding rate (ABR) as measured by individual bleeding components, calculated per ISTH Bleeding Assessment Tool (ISTH BAT), and if applicable, the ____________________, for applicable diagnoses
Pictorial Bleeding Assessment Chart (PBAC)
This tool may be used to determine eligibility with an age adjusted score.
ISTH Bleeding Assessment Tool
Participants who meet the following inclusion criteria are eligible for enrollment into this cohort:
Have an established diagnosis of one of the following:
a.PAI-1 deficiency
b.Factor I, II, V, VII, X, XI, XIII deficiencies
c.Combined FV and FVIII deficiency
Rare Bleeding Disorders Cohort
This visit may be performed in-office or by phone.
Quarterly
Genetic testing will be optional and provided by central labs, as funding allows, across all cohorts. Genetic testing will be performed at this frequency, for applicable participants.
Once