Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13. The main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant. Only data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during this study.
Study Type
OBSERVATIONAL
Enrollment
50
This is a non-interventional study.
Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
Index infusion is defined as the first Adzynma infusion date recorded in the medical record using commercially available Adzynma (excluding doses received in clinical studies or early access program).
Time frame: Up to 6 months following the index Adzynma infusion
Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions
Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported.
Time frame: Up to 6 months following the index Adzynma infusion
Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion
Number of participants with long term safety risk of hypersensitivity reactions following the index Adzynma infusion will be reported.
Time frame: Up to 6 months of follow-up time from the index Adzynma infusion
Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion
Number of participants with risk of hypersensitivity reactions after each administration of Adzynma following the index infusion will be reported.
Time frame: Up to 7 days after each administration of Adzynma
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product.
Time frame: From start of the study drug administration up to 5.5 years
Gestational age at the Time of Infant Birth
Gestational age at the time of infant birth will be reported.
Time frame: At birth
Number of Participants With Spontaneous Abortion
Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation.
Time frame: Up to 5.5 years
Number of Participants With Stillbirth
Stillbirth is defined as a fetal death after 20 weeks of gestation.
Time frame: Up to 5.5 years
Number of Participants With Induced Abortion
Number of participants with induced abortion will be reported.
Time frame: Up to 5.5 years
Number of Participants With Live Birth
Number of participants with live birth will be reported.
Time frame: Up to 5.5 years
Number of Participants With any Pregnancy Related Complications
Number of participants with any pregnancy related complications will be reported.
Time frame: Up to 5.5 years
Number of Participants Categorized According to Gestational age of Greater Than or Equal to (>=) 37 Gestational Weeks and Less Than (<) 37 Gestational Weeks at Birth
Number of participants categorized according to gestational age of greater than or equal to (\>=) 37 gestational weeks and less than (\<) 37 gestational weeks at birth will be reported.
Time frame: At birth
Number of Infants Categorized According to Year of Birth
Number of infants categorized according to year of birth will be reported.
Time frame: Up to 5.5 years
Number of Infants With Normal Birth Weight
Number of infants with normal birth weight will be reported.
Time frame: Up to 5.5 years
Number of Infants With Small for Gestational Age (SGA)
Small for gestational age is defined as birth size (weight, length or head circumference) less than the 10th centile for sex and gestational age.
Time frame: Up to 5.5 years
Number of Infants With Congenital Anomaly Detected at the Time of Birth
Number of infants with congenital anomaly detected at the time of birth will be reported.
Time frame: At birth
Number of Participants by Breastfeeding Status While Receiving Adzynma
Breastfeeding status during Adzynma treatment will be assessed and categorized based on response (Yes, No, or Unknown).
Time frame: Up to 5.5 years
Number of Infant Categorized by Growth and Development Outcomes
Number of infants categorized by growth and development outcomes (normal, abnormal \[specify\], or unknown) will be reported.
Time frame: Up to 5.5 years
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