Feb. 28, 2017
Posted by Jennifer Huron
Cleveland, Ohio and Danbury, Conn., February 28, 2017—The Platelet Disorder Support Association (PDSA) has launched the largest-ever study to research Immune Thrombocytopenia (ITP). In autoimmune diseases, the body mounts an immune attack toward one or more seemingly normal organ systems. In ITP, platelets are the target. They are marked as foreign by the immune system and eliminated in the spleen, the liver, and by other means. In addition to increased platelet destruction, some people with ITP also have impaired platelet production. ITP currently has no cure.
“The ITP Natural History Study Patient Registry will provide a complete picture of each patient’s experience with ITP,” said Caroline Kruse, Executive Director of PDSA. “We are launching this initiative to help fill the missing link researchers and medical experts need to advance research and a cure.”
To help drive awareness and participation, PDSA will promote the ITP Natural History Study Patient Registry through the organization’s various media platforms, including its website www.pdsa.org, with over 60,000 unique visitors each month, quarterly newsletter and monthly e-newsletter with a reach of 17,000 individuals, and through social media channels including Facebook, Instagram and Twitter, with a membership of 10,000+. In addition, an educational program on patient registries will be part of the ITP National Conference taking place in July 2017, and the importance of participating in a patient registry will be promoted through PDSA’s 39 local support groups.
“Our goal is to enroll as many patients, or their parents or legal guardians, as possible,” said Caroline. “The success of the registry is dependent upon community participation.”
The ITP Natural History Study Patient Registry is a natural history study that consists of electronic surveys to collect information about the patient experience and disease progression. Patients, or their caregivers or guardians, can enter information from anywhere in the world. The data is made anonymous and stored securely in an online portal called a registry. PDSA may share the data but not your personal identifying information with individuals or institutions conducting research or clinical trials, as approved by the study’s governing board that includes scientists, doctors and patient advocates.
PDSA is launching the study in collaboration with the National Organization for Rare Disorders (NORD), an independent charity that built its natural history study platform as part of its mission to help identify and treat all 7,000 rare diseases. Funding is supported by a cooperative agreement between NORD and the U.S. Food and Drug Administration (FDA). The FDA has praised NORD’s program as a helpful tool “that protects the security and privacy of personal information, while making valuable information available to a researcher or drug developer interested in creating a new therapy for a rare disease.”
NORD President and CEO Peter L. Saltonstall said, “NORD’s natural history studies platform empowers patients and families to drive research and eliminate some of the unknowns that still exist in rare diseases. We are glad to be working with our Member Organization on this project and thank the FDA for its support and ongoing commitment to help people with rare diseases.”
ITP is a rare autoimmune bleeding disorder that occurs in approximately 9.5 out of every 100,000 people. A normal platelet count is between 150,000 and 400,000/microliter of blood. If someone has a platelet count lower than 100,000/microliter of blood with no other reason for low platelets, that person is considered to have ITP. There is no accurate, definitive test to diagnose ITP. With few platelets, people with ITP often have bleeding symptoms such as spontaneous bruising, petechiae, tiny red dots on the skin, or for women, heavy menses. More severe bleeding symptoms include blood blisters on the inside of the mouth, blood in the urine or stool, or bleeding in the brain. Treatments for the disease vary depending on the platelet count, severity of symptoms, age, lifestyle, personal preferences, and any other associated diseases. Some people may choose to not treat their disease and live with low platelets.
While it may seem like ITP is a simple disease, there are nuances to the diagnosis, differences in the disease between children and adults, and variations in how the disease responds to treatments.
For more information, visit https://www.pdsa.org/join-the-community/registry.html.
About Platelet Disorder Support Association (PDSA)
The Platelet Disorder Support Association is a 501 (c)(3) organization dedicated to enhancing the lives of people with Immune Thrombocytopenia (ITP) and other platelet disorders through education, advocacy, research and support. As the leading ITP patient advocacy organization in North America, PDSA serves the worldwide ITP community of patients, practitioners, caregivers, advocates, and key disease stakeholders, promoting their needs and uniting them on a global level. We build awareness, educate the ITP community, and provide critical connections and resources that empower patients to take charge of their disease and encourage practitioners to exercise patient-centered medical care. Learn more about PDSA at www.pdsa.org.
About the National Organization for Rare Disorders (NORD)®
The National Organization for Rare Disorders (NORD)® is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD is committed to the identification, treatment and cure of the 7,000 rare diseases that affect 30 million Americans, or 1 in every 10 people. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. For more than 30 years, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research, and providing patient and family services for those who need them most. NORD represents more than 260 disease-specific member organizations and their communities and collaborates with many other organizations in specific causes of importance to the rare disease patient community. www.rarediseases.org.
For PDSA: Alexandra Kruse, Research Coordinator, email@example.com, 440-746-9003
For NORD: Jennifer Huron, firstname.lastname@example.org, 203-744-0100
 Woodcock, J. “The more we know about rare diseases, the more likely we are to find safe and effective treatments.” FDAVoice (Oct. 23, 2014)