The UK Adult ITP Registry
In any disease it is important to collect data in order to predict the outcome of the disease, evaluate treatments etc, and until the ITP Support Association funded the UK ITP Adult Registry there was no such data collection in the UK, although a few short studies on newly diagnosed patients had been done. As many members of the ITP Support Association have suffered chronic ITP for many years (over 50 years for a few cases!) the Association felt that it was important that they too should be involved in such a study, and pressed for the UK Registry to be retrospective and all inclusive.
In 2001 The Towler family very generously funded a pilot year of the ITP Registry in memory of Carol Towler. Dr. Provan explained the importance of the registry in the December 2002 edition of The Platelet:
In 2003 we obtained approval from the London Multicentre Research Ethics Committee (MREC) to set up a registry for adult patients with ITR So what? you may ask. Let me explain the purpose of the registry.
Disorders such as ITP are uncommon, and each clinician will see relatively few affected patients, making the study of these disorders difficult. For common conditions such as heart disease, the study of large numbers of patients worldwide has significantly improved the quality of care, and we now know how to treat patients with these disorders. The reverse is true for uncommon disorders such as ITP. There have been few good.studies involving large numbers of patients and we are still struggling to decide who needs treatment, which treatment is best, how long to treat, when to stop, and so on. The UK Adult ITP Registry will allow us to collect a large amount of information about ITP patients (anonymously-patients will never be named or identifiable in any publication of results) and by analysing this information we will learn a great deal about ITP and its treatment.
There are at least 60 autoimmune diseases, of which ITP is one. Some of these diseases have been studied scientifically, using DNA from patients with these diseases. Scientists have examined the genes controlling the immune system and have come up with some interesting findings. Readers of The Platelet will know that we are currently carrying out small-scale studies of this kind in London. However, we have access to relatively small numbers of ITP patients. We can greatly increase this number by asking patients with ITP if they would be willing to take part in The Registry. If he or she agrees, a small blood sample will be collected and sent to The Royal London, where we will analyse the immune response genes to determine whether gene variations (minor changes in genetic code) can influence the outcome of the disease, or can predict the response to a treatment such as intravenous immunoglobulin or splenectomy. In the future, we may be able to use these as predictors of response, and inform patients which treatments are likely to work.
Following on from pilot registry, in 2003 Dr. Drew Provan obtained approval from the London Multicentre Research Ethics Committee (MREC) to set up an ongoing registry for adult patients with ITP at the Royal London Hospital, to be jointly funded by The ITP Support Association and a pharceutical company.
2007 Registry Report
In 2007 Ameet Sarpatwari was appointed as study co-ordinator and later reported on behalf of The Royal London Hospital: As many of you recall, we initiated an adult ITP registry for the United Kingdom nearly six years ago. Through the assemblage of a large number of patients, we hoped to uncover valuable information concerning the natural progression and overall burden of adult ITP, the effectiveness of commonly utilised treatments, and promising genetic markers of disease severity and chronicity.
The response to this study among both patients and consultant haematologists was truly overwhelming. From inception of the registry in 2001 until the conclusion of 2004, we were able to consent and enroll 609 patients, making this study one of the largest in the world. Sadly at that time, we found ourselves ill-equipped to process this wealth of information and were forced to place the study on temporary hold.
Today, we are delighted to inform you of its imminent return! Through generous support from both The ITP Support Association and GlaxoSmithKline (GSK), we have been able to secure the resources necessary to see this project through to its completion, including the full-time efforts of one PhD student from the University of Cambridge. Following receipt of ethical approval for re-initiation of the study this spring, we will commence collection of updated past participant information while enrolling new patients at The Royal London Hospital and a representative selection of haematology centres across the United Kingdom.
2014 Update from the Royal London Hospital
Under the leadership and guidance of Dr Drew Provan (Chief Investigator) and Professor Adrian Newland (Co-Investigator), the ITP Registry has been extremely busy in the last year in welcoming new local UK centres, enrolling new participants, supporting collaborating centres and undertaking current as well as planning new studies. Since starting work over a year ago, our Lead Epidemiologist, Umesh Doobaree, has been engaged in executing and delivering several studies and planning new ones for the next few years ahead while at the same time devising ways with our Data Manager, Raghava Nandigam, in ensuring data completeness and quality.
By the end of 2013, with the invaluable study coordination from Raghava, the registry had recruited 1246 participants (with 1005 data already collected) from 62 UK centres. Currently, 46 of these centres are actively engaged in enrolment and data update. However, participants receiving ITP care in Scotland, Wales and Ireland are currently underrepresented in our cohort but we continually seek to address this by encouraging more haematology teams in those parts of the UK to join this national endeavour.
The Research Ethics Committee also approved the Registry to be exempt from Site Specific Assessment (SSA) thus making collaboration with external centres straightforward and shortening the local approval process by an average of 4 weeks before local participants’ enrolment can take place. As the Registry is part of the National Institute of Health Research (NIHR) portfolio, new centres that wish to participate can contact their regional clinical research networks for more information about the local support available.
To generate robust research findings we not only require sufficient number of participants but also good quality demographic and clinical data. With this in mind we have prepared various tools and strategies to assist data collection. These include revising some data entry properties of our database, creating a data dictionary and devising a data entry manual. We are also planning to develop web-assisted live tutorials to help current and new centres. With these measures in place we hope that the data collection will be a streamlined process and assist the smooth running of the registry and the delivery of our research projects.
With regards to our research, we have completed an extensive systematic review of published studies on the risk of thromboembolism in the ITP population. An initial abstract for this paper will be available at the next British Society of Haematologist (BSH) Conference in April and its full text will be published in due time. Our Epidemiologist is also heavily engaged on describing the characteristics of the cohort, especially focusing on the natural history of the disease, platelet progression over time and prevalence and incidence of comorbid conditions over time. The next phase, should adequate support be in place, is to describe the treatment patterns in current practices and their effectiveness. One very important outcome that Drew and Umesh are working on right now is to include a patient-related outcome into some of our future studies, particularly focusing on quality of life, as this will assist us to research and understand what improves patients’ lives, alongside other biological outcomes (e.g. platelet counts improvement). At a later phase, we also plan to undertake genetic studies but we will convey more information nearer that time. At the Blizard Institute, Saleha Hassan (Bio molecular Scientist) is working with Drew and Dan Pennington (Professor in Molecular Immunology) on conducting important research on T-cell activities in ITP and their work will be initially shown at the next BSH meeting too. Our Research Nurse, Louise Taylor, continues with her valuable support and care of patients seen at the Royal London Hospital, among whom many are registered into our registry.
It is perhaps worth mentioning too that Umesh is currently engaged in developing the epidemiological research infrastructure to receive data from haematology teams based outside the UK and this should take place this year. This will undoubtedly improve our recruitment and provide more statistical power into our studies to explore treatment patterns and their effectiveness as well as the characteristics of this rare disorder.
We would like to take this opportunity to thank all our participants, the ITP Support Association and all haematology teams for their continued support and making our research possible.