RecruitingOBSERVATIONAL

Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center

This study, called TWI-LIGHT, is looking at health records of over 1,200 people in France who had a rare and serious blood condition called immune thrombotic thrombocytopenic purpura (iTTP). iTTP happens when the body's immune system attacks a vital enzyme, leading to dangerous blood clots. The researchers want to understand how different factors, like heart disease risks and new treatments (such as caplacizumab), affect how long people live with iTTP and their overall health. They are especially interested in groups like pregnant women, children, and older patients. The goal is to provide better, more personalised care and guidelines for everyone living with iTTP based on real-world information.

At a glance

Status

Recruiting

Sponsor

Assistance Publique - Hôpitaux de Paris

Enrolment target

1,200

Start

02 Dec 2024

Estimated completion

01 Dec 2028

Thrombotic Thrombocytopenic Purpura (TTP)Immune Thrombotic Thrombocytopenic Purpura Thrombotic Microangiopathies Microangiopathy, Thromboic Cardiovascular Diseases Autoimmune Diseases Rare Diseases Neurological Manifestations

What is this study about?

This study is called TWI-LIGHT, and it's a really important project looking into a rare but serious blood condition called immune thrombotic thrombocytopenic purpura, or iTTP. Normally, a special enzyme in your body called ADAMTS13 helps to control how your blood clots. With iTTP, your immune system mistakenly attacks this enzyme, making it less active. This leads to tiny blood clots forming throughout your body, which can cause serious problems for your organs, including your brain and heart.

Because iTTP is so serious and can be life-threatening, it's really important to understand how people live with it in the long term. This study isn't about trying a new medicine; instead, it's looking back at existing medical information from over 1,200 patients in France who were diagnosed with iTTP between 2000 and 2024. The researchers want to see how different things, like having conditions such as high blood pressure or diabetes, or receiving newer treatments like caplacizumab, affect how well people do over many years. They are also keen to learn more about how iTTP affects specific groups, like pregnant women, children, and older adults.

By carefully reviewing these records, the study hopes to find patterns and insights that can help doctors provide better care. This could mean knowing what future health problems to watch out for, how to tailor treatments more effectively, and ultimately, improving the health and quality of life for people living with iTTP. It's about using past experiences to help future patients.

Key takeaways

This study is looking back at health records of people with iTTP.
It aims to understand how iTTP affects people's long-term health and survival.
No direct patient involvement is needed; existing medical data is used.
Your privacy is protected as direct identifiers are removed.
The results could help improve future care and guidelines for iTTP patients.

Who may be eligible?

This study is looking at medical records of people who have already been diagnosed and treated for a specific type of iTTP. You won't be asked to join the study directly or change your treatment.

The researchers will be considering records from people who had a confirmed diagnosis of iTTP where their immune system was involved (called immune-mediated TTP). This diagnosis usually involves blood tests showing low platelets (cells that help blood clot), a specific type of anaemia where red blood cells are destroyed, and very low levels of the ADAMTS13 enzyme (less than 10% activity).

They won't be including records from people whose iTTP was linked to cancer, severe infections, or organ transplants. Also, if a patient or their family specifically said they didn't want their healthcare information used for research, those records won't be included. This ensures that the study uses appropriate and ethically relevant data.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Have you been diagnosed with immune Thrombotic Thrombocytopenic Purpura (iTTP)?
Was your iTTP not caused by cancer, severe infection, or an organ transplant?
Were you diagnosed between October 2000 and June 2024?
Do you have at least one year of follow-up medical records for your iTTP?
Have you *not* previously objected to your health data being used for research?

Answer every question to see your result.

What does participation involve?

As this is a retrospective study, it means the researchers are looking back at existing medical records and information that has already been collected as part of your standard healthcare. You wouldn't be asked to do anything extra. There are no study visits, no new assessments, no new medications to take, and no special follow-up appointments related to this study. Your participation simply involves the researchers using your anonymised or pseudonymised (meaning your name isn't directly attached) health information from over the years, which has been collected during your routine medical care for iTTP. The data they use will cover details from when you were first diagnosed and treatment started, to any long-term follow-up you've had.

Potential risks and benefits

There are no direct risks to individuals whose data is used in this study, as no patient contact or involvement beyond their existing medical records is required. Your personal identity will be protected by removing or scrambling identifying information, ensuring your privacy. For society, the potential benefits are significant: by understanding iTTP better, the study aims to improve long-term care plans, treatment effectiveness, and overall quality of life for future patients. Individuals also have the right to object to their data being used for research, which would mean their records are not included.

Locations (1)

Service d'Hématologie Hôpital Saint-Antoine
Verified postcode
Paris, France· Recruiting

Common questions

What is iTTP?

iTTP is a rare and serious blood disorder where your body's immune system mistakenly attacks an important enzyme, leading to dangerous small blood clots throughout the body.

Will I have to do anything for this study?

No, you don't need to do anything. This study looks at existing medical records from patients who have already had iTTP. You won't have any extra appointments or treatments.

Is my personal information safe?

Yes, your privacy is protected. The researchers use 'pseudonymised' data, which means your name and direct identifiers are removed or replaced so your records can't be linked directly back to you.

What will the study learn?

The study hopes to learn how different factors, like heart problems or certain treatments, affect the long-term health and survival of people with iTTP. This information aims to improve future care.

Is this study happening in the UK?

No, this specific study is being carried out in France, looking at records from French patients. However, the findings could be helpful for improving care for iTTP patients worldwide, including in the UK.

How to find out more

Paul COPPO, MD, PHD

aul.coppo@aphp.fr 00 33 1 49 28 34 39 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.