Research and the ITP Support Association
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by
mervyn
Research and the ITP Support Association
The ITP Support Association is a registered charity dedicated to promoting and improving the welfare of patients and families affected by Immune Thrombocytopenia (ITP). Central to our mission is the provision of clear, reliable information and meaningful support to those living with this condition. We offer written resources, online support, expert advice, mentoring, and opportunities for patients to meet and share experiences at conventions and smaller meetings.
Beyond direct patient support, the Association plays a vital role in raising awareness of ITP at regional, national, and international levels. This is achieved through close collaboration with professional societies and specialist groups, the organisation of medical seminars, and ongoing engagement with regulators, commissioners, and NHS bodies. We also work actively with other patient organisations and maintain strong links with international partners, including the Platelet Disorder Support Association (PDSA) in the United States and several European patient groups. Through these collaborations, and particularly via our links with the European Hematology Association (EHA), we have contributed to broader recognition of ITP as a bleeding disorder.
Supporting and Enabling Research
A core objective of the Association is to encourage and support high-quality research into ITP for the direct benefit of patients. We have been particularly successful in working with pharmaceutical companies to support clinical trials, helping to ensure that patient perspectives are represented and that research addresses real clinical needs. In addition, we collect and disseminate information on all available clinical trials in the UK, enabling patients to make informed decisions about participation. We also engage directly with trial sponsors to provide structured patient feedback.
While the Association is keen to fund research directly, we recognise that, as a relatively small organisation, our resources must be used strategically. We therefore avoid competing with major funding bodies where substantial external funding is already available. Instead, our approach has focused on two complementary strategies: supporting carefully selected individual research projects and funding national patient registries.
The ITP Registries
One of the Association’s most significant contributions to research has been the funding and development of the Adult and Paediatric ITP Registries, as well as more recent registries in Pregnancy and Secondary ITP. These registries have proved to be exceptionally good value for money and have generated data with a substantial impact on clinical practice.
Research arising from the Paediatric Registry contributed to both romiplostim and eltrombopag becoming available for children with ITP. Data from the Adult Registry demonstrated an increased incidence of thrombosis in ITP, highlighted the significant burden of fatigue experienced by patients, and documented changes in treatment patterns over time. Importantly, the Registry showed sustained long-term responses following cessation of thrombopoietin receptor agonists in adults.
More recent analyses involving over 300 patients treated with rituximab, using either low- or high-dose regimens, demonstrated that the lower-dose option is both effective and more economical. This finding has enabled wider use of the treatment. Registry data on spleen scanning and platelet survival have also had a major influence on clinical decision-making, leading to a marked reduction in unnecessary surgery and sparing many patients from inappropriate splenectomy.
Individual Research Projects and Pilot Studies
In addition to registry funding, the Association has provided more than £250,000 to support individual research projects over the years. All applications are rigorously assessed, and while not all projects lead to positive results, this reflects the inherent nature of research. Importantly, negative findings are often just as valuable in guiding clinical practice.
Notable projects include a study conducted at Addenbrooke’s Hospital examining the relationship between food, allergy, and ITP. This research found no association, demonstrating that while rare individual reactions may occur, diet is not a major factor in ITP. This has helped dispel common misconceptions among patients.
The Association also supported a pilot study on ITP in pregnancy led by Dr Angharad Care in Liverpool. The findings, published in the journal Platelets, led directly to the establishment of the Pregnancy Registry, which has now collected data on over 100 pregnancies. Early results have confirmed the increased risk of severe post‑delivery haemorrhage and highlighted the importance of specialist care for pregnant women with ITP.
Another important study, led by Professor Nikki Cooper at Imperial College, investigated the presence of microbleeds—tiny, clinically silent bleeds—in the brains of children with persistently low platelet counts. Although these bleeds do not cause immediate symptoms, they may have implications for long‑term neurological outcomes. This work, presented at the Association’s Convention and published in Platelets, led to a much larger NIHR‑funded PhD study, worth over £400,000, undertaken by Dr Alice Hart.
Patient‑reported outcome data from this research also identified an unmet need for education among young people with ITP. This directly informed the development of the ITP and Me book, which has since been translated into multiple languages and is widely used internationally.
More recently, Professor Cooper has led a study examining the gut microbiome in patients with ITP. Early findings have been sufficiently promising to attract further external funding from the NIHR (approximately £330,000) and will support a larger investigation into the role of the microbiome in ITP and the potential for targeted interventions.
The Trustees consider these smaller pilot studies—typically funded at levels of up to £20,000—to be an effective use of the Association’s resources. By supporting early-stage research, we help generate the preliminary data needed to attract larger grants from major funding bodies. This approach has already proven successful, including in the initial genetics study of ITP using DNA obtained through the Adult Registry, which subsequently received £75,000 from Barts Charity. In addition, the Medical Research Council has funded a PhD Fellowship to explore ITP genetics at Imperial College and Hammersmith Hospital, with partial support from the National Institute for Health and Care Research. The Association is also a non‑commercial partner of the NIHR, providing further support for research applications.
Future Directions
Looking ahead, the Association intends to continue supporting high‑quality pilot studies and is actively seeking ideas from members about priority areas for future research. We will also invite proposals from researchers working in ITP‑related fields. All grant applications will continue to be reviewed by external assessors alongside our own expert advisers, and all funded projects will be closely monitored to ensure responsible use of funds.
Acknowledgements
The Association is deeply grateful to its members for their enthusiastic and imaginative fundraising, and for the extraordinary generosity of all who support our work. We are committed to ensuring that all funds are used carefully and effectively, both to support patients and families living with ITP and to advance understanding of the disease and its treatment. In doing so, we aim to fulfil the broader objectives set out in our charitable charter.
Professor Adrian Newland CBE
Chair of Trustees
The ITP Support Association
