Purpura Fulminans: Proposal for an International Epidemiological Study of Neonatal Purpura Fulminans

Why are we doing this research?

The aim of this study is to learn about neonatal purpura fulminans (NPF), a rare blood clot condition (when blood becomes solid and blocks a vein) in babies that is not well understood. Gathering information about your child’s experience with NPF, along with those of children from other countries, we can conduct future research that will help us better understand the nature of this condition. As NPF is a rare condition, international collaboration is important for improving our current understanding of this condition. The gathered information will be entered into the Neonatal Purpura Fulminans Registry, which is a medical database at the Hospital for Sick Children (SickKids) and supported by the International Society of Thrombosis and Hemostasis (ISTH).

Who can participate?

Inclusion criteria:

1. Birth to 18-year-old patients who fulfill the diagnostic criteria for congenital NPF, regardless of their outcome, including death, as stated below:
2. Protein C or protein S circulating levels below 0.2 U/ml
3. Typical cutaneous purpura fulminans lesion with confirmed diagnosis previously made on parents.
4. For a genetic diagnosis, both double heterozygosity and homozygosity for either protein C or S gene mutation will be accepted.
5. Patients who fulfil these criteria, but diagnosed prior to the commencement of the registry may also be included provided they are aged 5 years or less at the time of data entry

Exclusion criteria:

1. Any case in which acquired purpura fulminans is possible (i.e., post-infectious), or cases where lab confirmation of protein C/S deficiency (as per inclusion criteria) has not been provided

Conditions

Hematology - Thrombosis

What will happen in the study?

We would like your permission to collect information on your diagnosis and management of NPF and enter it into this secure database along with data from other children with NPF. As part of the study, we would also like your permission to collect information from your medical chart at 6 months of age, then yearly chart reviews by our research staff for approximately 18 years. If you qualify and you decide to be in the study, the study staff will obtain the following information about you from your medical record (no visits to CCHMC will be required):

Age
Gender
Race/ethnicity
NPF symptoms
Diagnosis
Treatment and management
Other diseases
Family history of thrombosis (blood clots)
Some genetic information (Protein C and Protein S levels in child and parents, collected from testing you have already had done, we will not collect any blood or do laboratory testing for this study)

Will you/your child be paid to be in this research study?

You will not be paid to be part of this study.

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Clinical Trials / Research Studies
Clinical Trials / Research Studies