ITP in Pregnancy Registry

The ITP Support Association is sole funder of the ITP Childhood Registry and part funder of the ITP Adult Registry. The data collected by these registries has increased the understanding of ITP resulting in improved management practices. Dr Sue Robinson of Guys and St Thomas's Hospital in London is about to set up a new ITP in Pregnancy Registry which the ITP Support Association has agreed to fund. It will run in conjunction with the Adult ITP Registry, and it is hoped that it will identify the women who need lots of additional care and those that just need a gentle eye. Dr Robinson explained "Pregnancy in women with ITP accounts for almost 1 in a 1000 pregnancies in the UK. Pregnancy is a precious time, health care professionals endeavour to provide patient focused care and where possible we must avoid over medicalising pregnancy in women with ITP. How doctors and midwives look after women with ITP in pregnancy has changed over time. To define best practice and determine exactly when additional treatment is really required data collection regarding expectant mothers with ITP women and their newborns is essential. The planned addition of an ITP in Pregnancy Registry to the Adult ITP registry is an opportunity not to be missed!"

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Quality Of Life

The ITP Support Association awarded a research grant to support the British part of the International ITP Quality of Life study in March 2006.

How Do You Measure Quality Of Life (QoL) In Childhood Immune Thrombocytopenic Purpura (ITP)?

by Robert J Klaassen, MD Victor S. Blanchette, FRCP, Dorothy Barnard, MD, Cindy D. Wakefield, RN, Christine Curtis, MSc and Nancy L. Young, PhD

Much of the focus by patients, doctors and other health care professionals when dealing with ITP has been on the platelet count. Fortunately, patients with even very low platelet counts may have minimal or no bleeding. Relying solely on the platelet count to decide when to treat, and what treatment is needed, does not seem to be the best way to manage this condition. Other approaches are needed.

Quality of life (QoL) has been defined by the World Health Organization as the "net consequence of life characteristics on a person's perception of their position in life, in the context of the culture and value systems in which they live, and in relation to their goals, expectations, standards, and concerns". The key to measuring QoL is that it is subjective, reflecting present lifestyle, past experience, hopes for the future, dreams and ambitions. Those of us who see patients with ITP are keenly aware of the impact it can have in all of these areas.

Back in 1997, two pediatric hematologists in Canada, Dr. Dorothy Barnard and Dr. Victor Blanchette, decided to do something about this and started the process of developing three questionnaires (also called measures) to try to measure the quality of life of children with ITP and their parents. It took a lot of work and the contribution of 140 parents and 95 children for that vision to come to fruition. Dr. David Feeney, an expert in the development of disease specific quality of life measures, provided invaluable input for the initiative which was funded by Cangene (Canada). Details of the ITP QoL instrument were published in 2003 in the Journal of Pediatric Hematology and Oncology.

But that was only the beginning. In 2004, a total of 13 families reviewed the measures in detail, allowing for further refinements to be made. The revised questionnaires were given the name Kid's ITP Tools (KIT). We then gave the KIT to 49 families with chronic ITP and 40 with acute ITP spread out over four treatment centres in Canada (Ottawa, Kingston, Toronto, and Hamilton) and two in the United States (Boston and Dallas). This North American study was led by Dr. Rob Klaassen, a pediatric hematologist in Ottawa, and Dr. Nancy Young, a Canadian expert in the field of outcome measures development. The study was a success and showed that the KIT was reliable, valid and responsive to change. This was demonstrated by the fact that the results were stable (reliable) when given repeatedly to patients whose QoL was stable, found higher QoL in children with chronic ITP when compared to children newly presenting with ITP (valid), and improved when the children's platelet count went back to normal (responsiveness).

Once we showed that the KIT worked in North America, we decided to test it in four other countries: UK (Queen's English), Germany (German), France (French) and Uruguay (Spanish). The first step in the non-English countries was to complete a rigorous process of translation. This involved translating the measures to the various languages and then back translating the questionnaires into English. We then compared the back-translated measures to the original KIT to identify any discrepancies. Three Canadian pediatric hematologists with expertise in French, German and Spanish reviewed the problem areas and corrections were made by consensus over the telephone. We had quite the phone bills!

Next we took the translated measures and asked 10-20 families in each of the four countries to review them in detail. Canadian investigators and their counterparts from the UK (Manchester), France (Nantes, Angers, Lyon), Germany (Berlin) and Uruguay (Montevideo), together with research coordinators for the study, Chris Curtis and Cindy Wakefield, met in December 2006 in Orlando, Florida to go over the comments from the families. Interestingly even after all the previous work there were many refinements and the European and South American versions were finalised.

Over the next 6 to 9 months we will be asking families in the UK, France, Germany and Uruguay to fill out the questionnaire to make sure that it works as well as it did in North America. In the UK, this will be done by Dr. John Grainger, Dr. Paula Bolton-Maggs, Dr Russell Keenan and Dr Mike Richards. If you are contacted by one of these physicians about this international ITP study please help. The questionnaires only take a few minutes to fill out and you will be contributing to the success of this study.

At this point we would like to thank the ITP Support Association for awarding us £10,000 to support the British part of the International ITP QoL study. The money will be well spent and we think the end result will be a useful tool that can be used in future ITP studies.

Our thanks are due to Cangene who have funded all parts of the development of the KIT, and to patients/families who participated in our studies and without whose help this research would not have been possible.

Study Coordinators: Cindy Wakefield, Chris Curtis, Catharine Bradley; Canada: Dr Nancy Young, Dr. Robert Klaassen, Dr. Mariana Silva, Dr. Victor Blanchette, Dr. Anthony Chan, Dr. Dorothy Barnard, Dr. Vicky Price; USA: Dr. George Buchanan, Dr. Ellis Neufeld; UK: Dr. John Grainger, Dr. Paula Bolton- Maggs, Carol Beane; France: Dr. Eliane Tarral, Dr. Isabelle Pellier, Dr. Françoise Mechinaud, Dr Anne Ponthieux, Anne Marie Favreaux; Uruguay: Dr. Gustavo Dufort, Dr. Estela Citrin; Germany: Prof. Gerhard Gaedike, Dr. Tillmann Taube, and Arne Riedlinger.

Dr. Victor S. Blanchette Dr. John Grainger

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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.

Molecular studies
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.

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Molecular analysis of treatment predictors in ITP

The ITP Support Association awarded a research grant to Dr. Drew Provan and Professor Newland at the Royal London Hospital in March 2001.

Dr. Provan said: "Professor Newland and I were delighted to hear that we have been awarded a research grant from The ITP Support Association to allow us to carry out some basic studies into ITP. Having completed many clinical studies in this area at The Royal London Hospital we have now extended our studies to include DNA-based molecular studies. Our aim is to look at some immune response genes in order to predict which patients will respond to particular therapies and also see whether we can match the DNA profiles of patients with their indium scan data. In the text that follows we have attempted to put forward our thinking in this complex area. It must be remembered that genetic analysis such as this is a new area for all diseases, not least for ITP.

We are confident, from studies carried out by other groups, that we will get some interesting answers from our patients who have kindly donated DNA samples for this study. Without volunteers such as these we would not be able to carry out any of these studies. Similarly, without the support of The ITP Support Association we would not be in a position financially to carry out these experiments. We extend our warm thanks to all who worked so hard to raise this impressive sum.

As readers of The Platelet will be well aware, ITP is a disorder affecting both adults and children. From previous studies it is obvious that childhood ITP is different to adults, inasmuch as the bruising in children may be dramatic, but they rarely require much in the way of investigations, and usually get better by themselves, the ITP resolving within a few weeks. The important point to note about childhood ITP is that it is not usually a chronic ailment, unlike the adult type, although in about 20% of cases the child will have ITP similar to that in adults. The hallmark of the adult type ITP is the long-standing relapsing and remitting low platelet count, requiring intermittent courses of steroids, intravenous immunoglobulins IVIg) and many other treatments.

We all have patients who attend our clinics who respond beautifully to steroids and have ITP which is easy to control. At the same time, we have many patients who relapse after we tail off the steroids and who require further therapy, most often with dubious benefit. Although we tend to group all of our ITP patients together, it is clear from the way patients respond to different treatments, that ITP in adults does not represent one single disorder - but rather several different types, each presumably with a different underlying cause. Unfortunately, we have no way of sub-categorising patients at present. As a result we lump all people together and label them as having 'idiopathic ITP', as if this were a single disease, expecting them to respond well to treatment equally. If only we could sub-divide patients into different groups (e.g. ITP Type 1, 2, 3, etc.), then we would be able to offer specific therapies, rather than generalised global immune suppressing drugs such as prednisolone, azathioprine and many others. In effect, we would be in a position to offer patients therapy which we knew would work.

ITP is one of many autoimmune diseases known in humans. These diseases are caused by antibodies directed against the patient's own cells or tissues. Several published studies have shown that we all produce antibodies against our own tissues at variable rates and at different times (these are the autoantibodies). In fact, we can detect T-cells and B-cells, both of which are involved in the immune response, which target 'self' tissues. Thankfully, for most of us, these self-reacting cells are removed and prevented from causing any harm, but it is likely that patients with ITP have self-reactive immune cells that somehow escape removal and cause their damage through coating cells such as platelets with antibody. Once the platelets have been coated with antibody, the spleen and other organs have no choice but to remove them since the spleen believes the platelets are 'foreign'. The platelet count then falls.

The study which we are shortly to start is a molecular study of immune response genes, in an attempt to see how they cause harm and to determine whether particular molecular 'variants' determine how a patient will respond to steroids, IVIg or splenectomy.

The immune system is an immensely complex network of interacting genes and proteins, which coordinates the destruction of invading pathogenic bacteria and viruses. The whole function of our immune system is to keep us healthy by destroying any invading micro-organism quickly and effectively. However, sometimes the immune system response overreacts by producing too much or too little of specific immune response proteins. These proteins include cytokines, and the genes for many of these have now been cloned by molecular labs and their precise DNA sequence worked out.

This provides us with sophisticated molecular methods for looking at mutations or variations (we call these polymorphisms - poly = many, morphism = form) in cytokine genes amongst patients with specific diseases, such as ITP. This has been carried out for other disorders such as juvenile chronic arthritis, which is a condition similar to adult rheumatoid arthritis, but affecting younger patients.

In 1998 a group of research workers published a detailed study showing that a single DNA base change in the interleukin-6 (IL-6) gene was strongly associated with development of juvenile chronic arthritis, and correlated with its severity. The IL-6 protein is one of the anti-inflammatory response proteins, and from studies of inflammatory versus anti-inflammatory response proteins it looks as though autoimmune diseases such as ITP and many others are caused by an imbalance between pro- and anti-inflammatory response genes. In health the body has these finely tuned with a perfect balance of pro- and anti-inflammatory proteins, but tipping the balance either way leads to a variety of disorders, of which ITP may be one example. Other factors doubtless play a role, such as virus infections and other environmental agents encountered during life.

We are also very keen to look at the effect of steroids on ITP. We know from our clinical records how patients have responded to prednisolone when this has been used as treatment for ITP. It appears that mutation within the IL-6 gene may alter responsiveness to steroids, so we are hopeful that the presence of a specific DNA sequence in patients with ITP will highlight those patients who are likely to respond to steroids, and those who are not.

A second group of genes that we wish to look at in this project is involved in the uptake of antibody-coated platelets by the spleen. There are many variants of these genes that have been described in both mice and men, and such variants are associated with differing ability of the spleen to destroy antibody-coated cells, such as platelets. It may well be that those patients who have difficult, refractory and troublesome ITP have specific variations in their antibody receptor genes, compared to other patients who have simple ITP (that responds rapidly to treatment and causes few problems). In addition, because these antibody receptors interact with IVIg the presence of specific variations may correlate with a patient's response to IVIg therapy.

The media is full of reports concerning the Human Genome Project, and you will all be aware that scientists have almost completely worked out the sequence of the entire human genome, an incredible achievement in such a short time! The increasing availability of genetic sequences for genes such as those of the immune system make it easier to design experiments to look at malfunctions of these genes, and to examine the role of such genetic variations in normal populations, and also those with diseases such as ITP.

The Royal London Hospital has been scanning patients with ITP, to determine whether the spleen is the primary site of the destruction, for some years now. These so-called indium scans are highly predictive in terms of a patient's response to splenectomy. Patients whose indium scan shows that the spleen is the main site of platelet destruction have an excellent response following removal of the spleen; however, those patients who have a pattern of platelet destruction involving the liver and spleen do much less well after splenectomy.

We have been very lucky to have the co-operation of patients who have undergone indium scanning, since they have allowed us to take an extra blood sample so that we can look at their DNA for the presence or absence of specific variations in genes, such as those described above. We intend to look at the IL-6 and antibody receptor genes in all patients undergoing indium scanning, to see if the presence of particular variations correlate with the indium scan results. We are hopeful that the presence of specific gene variants will correlate with a specific indium pattern. If we find this is the case, then we will hopefully be able to avoid indium scanning altogether and look at those specific genes, instead of having to subject patients to the indium scanning which is time-consuming and highly expensive. Looking at the genes is much simpler and cheaper - the time from the initial blood sampling to the final results will be around one day or less.

To summarise the above: we have the resources and precious analytical material in the form of ITP patient DNA for this study. We intend to look at a few critical immune response genes in order to determine whether these predict treatment to therapy and also to determine whether these correlate with the indium spleen scan data. If so, these tests will help us predict who will have easy/difficult ITP, who will respond to specific therapies and may also help us predict those who will respond to splenectomy. Like all molecular projects this is ambitious, but with your help, continuing patient support and a strong molecular genetics lab at The Royal London Hospital we are confident that we will provide some answers to this puzzling and complex disorder."

Update (2013)

We received a grant from The ITP Support Association to examine the DNA from a number of patients looking for changes within specific genes to see if there were any obvious changes which might be linked to ITP. These results showed that within the TNF-α gene there was a definite poly- morphism linked to ITP. The paper was published in the journal Hematology, volume 16, page 243 (2011).

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Study of Newly Diagnosed Children with ITP

In order to help establish a valid criterion for future ITP research it was necessary to secure medical data about the disorder and the patients being treated in the UK. An initial study collated this information from newly diagnosed ITP children.

Dr. Bolton-Maggs spearheaded the project. She told The ITP Support Association, "To help direct ITP research it would be helpful to have more information about the natural course of the illness. Often the review of information thus held leads to ideas or questions for research to answer. At present we know that some people receive treatment and others are not felt to need it; it would be useful to understand why there is this variation, and whether the outcomes are different. If we are able to keep anonymous records about all people who develop the disease, whether in childhood or in adult life, we may learn more quickly about the disorder, and where then to concentrate research effort."

The establishment of such a register was supported by the British Society for Hæmatology, with the financial assistance of the ITP Support Association and Bio Products Laboratory (BPL).

The register was kept under the supervision of Dr. Paula Bolton-Maggs at Alder Hey Children's Hospital in Liverpool, where the 1995/6 National Audit of ITP in Children was carried out.

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Can ITP be caused by something you eat?

In 2011 The ITP Support Association approached a leading specialist in gastroenterology research, Professor Hunter, to ask if he could investigate whether food intolerance might be more commonly associated with ITP than is currently recognised.

ITP and diet
It is not unusual for those with ITP to consider changing their diet in the hope that the ITP may simply be a reaction to something they eat, or that a special diet may help to raise their platelet count.

Known cases of food intolerance affecting platelet count
A few cases of food intolerance have been written up in medical journals and over the years the Association has received anecdotal reports from a few ITP sufferers who were diagnosed with coeliac disease after ITP and found that adopting a gluten free diet helped both conditions.

It was not known if these food related ITP cases are extremely rare, or whether food intolerance might be implicated with ITP to a much greater degree than is currently recognised.

How was the research done?
Professor Hunter devised a study to investigate the incidence of food intolerance in ITP patients and invited adult ITP patients from the ITP Support Association membership to take part if they met certain criteria; they should be between the ages of 18 and 65, have chronic primary thrombocytopenia with a platelet count of less than 100 for at least 12 months, and not have ITP secondary to any other condition. Patients who had undergone splenectomy were be included providing they are taking no other antibiotics than penicillin. This project, funded by the ITP Support Association, was based in Addenbrookes Hospital, Cambridge and and was run by a team including Dr Trevor Baglin (Consultant Haematologist, Addenbrookes Hospital) and experts from Cranfield University (Bedfordshire) and the Open University (Milton Keynes, Bucks).

There were no unpleasant investigations!
After an initial interview with Professor Hunter to assess suitability for the trial, patients were asked to complete a symptom questionnaire, and blood and urine samples were taken. In addition to a platelet count, the samples were examined for (a) coeliac disease, (b) food allergies to the 6 most common allergens and (c) evidence of abnormal fermentation of food residues by gut flora. Anyone proving positive for coeliac disease or food allergy was referred back to their own doctor for appropriate diet/treatment, with their platelet count regularly monitored and any improvement in their ITP noted. Patients proving positive for abnormal gut flora were invited to live on a special dietary substance for 4 weeks whilst having weekly blood tests. If their platelet count showed a marked improvement they had the opportunity to consult with a dietitian and work through an elimination diet to find which foods had been responsible for the lowered platelet count.
Flow chart for the study. Click image for a larger version, (opens in new window)

The second phase
In 2013 The Food Intolerance Study ceased recruitment of patients and controls and moved on to the second phase with certain groups of participating patients being invited to return to Addenbrookes to start the appropriate diet for their particular results.
The ITP Support Association is most grateful to all the patients who volunteered to take part in this study and also to family members who offered themselves as 'controls'.


After all the results had been carefully scrutinized,  Professor Hunter told us that the results had not proved that ITP was commonly caused by food intolerance or allergies, but there was evidence that study participants with ITP had different gut bacteria from the healthy controls. It was agreed that he and his team should move to an extension of the study, funded solely by the ITP Support Association, with gut flora being examined in more depth. Some patients who participated in the the initial study were invited to return and others who met the criteria were be recruited.

Gut Bacteria Study

The study was open to adults between the ages of 18 and 65, who had chronic primary thrombocytopenia – ie. a platelet count of less than 100 for at least 12 months, and whose ITP was not secondary to any other condition. Participants who had undergone splenectomy were eligible for inclusion provided they were taking no other antibiotics than penicillin. Participants registering on the study were invited to bring a partner or friend as a healthy volunteer so that results could be compared.

After an initial interview with the Professor Hunter to assess suitability for the trial, participants were asked to provide a sample of both blood and urine. Then they were sent a kit to collect a stool sample in the convenience and privacy of their own home which was sent directly back to the laboratory

Professor Hunter will be presenting the results of this study at the ITP Convention on 13th May 2017.

Professor Hunter

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The Childhood ITP Registry

The ITP Support Association has been sole funder of the UK childhood ITP registry since its inception in 2005.

The UK Paediatric ITP Registry by Dr. John Grainger


Children with ITP in the UK have not been systematically studied in large numbers. Many may not require any specific treatment to raise the platelet count – because they have few symptoms and usually recover spontaneously within a few weeks. However, a significant proportion (about 20%) may not spontaneously recover within six months, these have chronic ITP. Children with chronic ITP may require treatment with drugs (steroids and intravenous immunoglobulin) or surgery (splenectomy) because their risk of serious bleeding is perceived to be greater.
Because no systematic study has taken place it is not clear if there are different subgroups who may respond differently to interventional therapy, or groups who are at particular risk of bleeding complications. The research aims to establish a UK database, which is linked to the international registry initiated by the intercontinental ITP study group (ICIS). Individuals with ITP will be followed over several years enabling us to obtain a analysis of remission rate, bleeding frequency and need for therapy.
As well as giving us more information on the natural course of chronic ITP the database is also intended to identify individuals whom may teach us more about ITP. These individuals will be approached to participate in additional studies. It is expected that the information gathered by the database will be stimulate further scientific research aimed to identify those children whom would benefit from early treatment and those children whom are best served by the avoidance of therapy.

How the Registry will work

The UK Paediatric ITP Registry intends to gather information on all new cases of ITP in the UK and follow children with persistent (chronic) ITP for up to twenty years.

In contrast to other studies we are encouraging recruitment from general paediatricians in local hospitals, rather than just from specialist centres. This approach will be more difficult to organise but should give a true reflection on the severity and outcome of ITP for children in the UK.

The UK registry will work closely with the Intercontinental Childhood ITP Study Group so UK data can be compared to that from other countries. The childhood chronic ITP registry received a final favourable opinion from COREC (central ethics) on 22 March 2006 and the final round of invites to all UK Paediatricians was completed in January 2007. The online database, parent information sheets and information sheets can be accessed via our website at .

The key aims of the Childhood Registry are as follows:

  • Maintaining accurate information on the incidence and outcome of childhood ITP
  • Updating national guidelines
  • Identifying children at a higher bleeding risk who may benefit from early treatment
  • Identifying children at a lower bleeding risk who do not require treatment
  • Stimulating scientific research and trials of new agents in childhood ITP

An abstract of Dr Grainger's article based on results of this registry was published in August 2013 by the BMJ's Archives of Disease in Childhood. The full text gives an acknowledgement to the ITP Support Association for funding this work.

Dr. John Grainger

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Dr Roberto Stasi

The ITP Support Association was deeply saddened to learn of the tragic death of Dr Roberto Stasi, a leading international ITP specialist. He was always very happy to help our Association, answer medical questions from our members and see patients with problematic ITP. He gave presentations at our Conventions in 2007 and 2008, and invited a representative from our Association to speak at an international medical meeting last year, even finding the funding to make this possible. The Association is currently funding a research project Dr Stasi initiated at St George's Hospital, and before he became ill he had agreed to report on his early findings at our Convention in April. He was very highly thought of both by his patients and by his colleagues in the medical profession.

Professor Adrian Newland has written the following tribute.

Dr Roberto Stasi 24/08/1961 - 28/01/2014
Sadly, Roberto Stasi was found dead at his home in Rome on the 28th of January. Roberto was one of the leading doctors in the field of Immune Thrombocytopenia. He trained in Rome where he was one of the top students in his class and undertook a higher research degree before carrying on with his training. He worked in Rome in haematology developing research links with myself and my colleague, Drew Provan, in England, Dr Jim Bussel in New York and Professor Francesco Rodeghiero in Vicenza, Italy. He made some of the first observations on the benefits of Rituximab in ITP and did much important work on the thrombopoietin agonists. He was acknowledged as an outstanding scientist and a physician of the highest ethical stature. He was loved by his patients to whom he offered untiring and caring support. We were lucky in the UK when he transferred to St George's Hospital in 2010 and many members of the Association will have benefited from his care. He continued to develop his international standing, publishing extensively and become a leading figure in the European Hematology Association with a large network of clinical friends throughout the world. He became unwell last year and returned to Rome to recuperate where he was slowly recovering. His sudden and tragic death was a shock to the many colleagues who knew and loved him, a major loss to the patients he cared for and to the field of ITP in general. We will honour his name in a more fitting and permanent way in the future and will remember the loyal and affectionate friend we all knew and the first class doctor that he had become.
Professor Adrian Newland

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The role of bone marrow mesenchymal stromal cells in chronic ITP

In 2012 Dr Roberto Stasi, Consultant Haematologist, St George's Healthcare NHS Trust, received £18,500 from The ITP Support Association for a two-year study into the role of a particular cell in the bone marrow: mesenchymal stromal cells. Dr Stasi explained that these cells will be separated from the bone marrow of people with chronic ITP. Little is known about the function of these cells in ITP but they have been shown to be abnormal in other autoimmune disorders. The aim is to improve understanding of the causes of chronic ITP and to possibly find a new treatment i.e. healthy mesenchymal stromal cells.


November 2013 Update by Dr Ruth Pettengell and Dr Edita Hamzic.

Mesenchymal stromal cells (MSC) have been shown to be abnormal in a number of autoimmune disorders, however, little is known about these cells in ITP patients. St George’s Hospital received full ethical approval in July 2013 to compare MSC from individuals with ITP to compare directly with healthy donors. The Hammersmith Hospital in London is currently in the process of being approved as an additional research site for the study and depending on trial recruitment numbers we will seek additional research sites.

Initial work has focused on growing MSC from ITP and donor bone marrow in the laboratory and comparing the growth characteristics and stem cell properties of these cells. MSC from 2 of 3 healthy donors and 1 of 2 ITP bone marrows grew fully in the laboratory dishes showing densely packed spindle shaped cells (Figure 1). By microscopy no obvious differences in the growth ability between patient and healthy donor MSC were observed and using flow cytometry to confirm their stem cell nature there were no clear differences in the pattern seen in individuals with ITP and healthy donors.

We are now repeating these studies on a larger sample size and testing the ability of the MSC from ITP and healthy donors to support platelet production.

Figure 1. MSC growing in a laboratory dish.

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 Sadly Dr Stasi died on 28 January 2014.

Confocal bone marrow examination in ITP patients

Dr Nichola Cooper (Consultant Haematologist and Honorary Senior Lecturer at the Imperial College London/Hammersmith Hospital) has received £9,000 from The ITP Support Association to study bone marrow using modern imaging techniques. Platelets are developed from megakaryocytes, which are formed in the bone marrow and Dr Cooper is hoping to gain more understanding about bone marrow to help doctors to understand why people with ITP respond differently to currently available treatments.

This project involves staining bone marrow trephines to analyse the interactions of the immune system in the bone marrow. We have found that patients with ITP have increased T cell interactions with the megakaryocyte when compared to bone marrows from patients without ITP. We are developing novel imaging techniques to further characterize these interactions. We hope that these techniques will allow us to understand what kind of disease patients have, i.e. antibody, or T cell, or both. This should allow us to make better decisions on what treatments to use in individual patients.

Dr Nichola Cooper. December 2013

The ITP Support Association is extremely grateful to Steven Sims Cavaliers whose amazing fundraising efforts in memory of their friend Steven Sims have provided the funds enabling us to support this project.

Dr Nichola Cooper

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Identification of novel pathogenic antiplatelet antibodies in ITP

Dr James Crawley (Senior Lecturer in Haematology Imperial College London/Hammersmith Hospital) was awarded £60,000 by The ITP Support Association in 2013 for a 16 month project to investigate why antibodies bind to platelets and cause their destruction. Current techniques allowed limited research into antibody binding, and by using new techniques Dr Crawley hopes to identify antibodies binding to novel sites on the platelet. This research has the potential to improve patient monitoring and may ultimately help to guide treatment.

Dr Cooper and myself advertised for the research position associated with our ITP Support Association award. We had a promising number of applicants. We shortlisted 5 candidates for interview and offered the position to Adrienn Orosz. She is highly capable, meticulous in her approach and comes from a good lab with a strong background in haemostasis. During Adrienn's time with us she has already been fundamental to the successful development of some of the assays that we have been setting up in the lab looking at ITP patient anti-platelet antibodies. So far we have successfully achieved the following:

  • Developed an assay that can accurately and sensitively identify those ITP patients that have autoantibodies that recognise their platelets. This assay is much improved on the commercially available anti platelet antibody tests in that it is quicker, cheaper and allows detection of all anti platelet antibodies (rather than just a small subset)
  • Collected anti platelet antibodies from a new cohort of patients attending Nikki's clinic anti categorised them according to treatment, anti platelet antibody status, platelet count etc…
  • Developed an assay that will now enable us to identify the platelet surface proteins to which patient antibodies bind. We have performed this assay just once so far, but the results are very encouraging. With further optimisation, this assay will hopefully start to provide new insights in the near future into the nature of autoimmunity in ITP patients.
  • Identified a patient with anti platelet antibodies that, likely due to the antibodies, has a consequent platelet function deficiency.

We are extremely encouraged by the progress that we have made so far in getting new assays set up in a comparatively short period of time and are confident that these approaches will develop our understanding of the pathogenesis of ITP.

Dr James Crawley. May 2013

Dr James Crawley

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Severe Primary Immune Thrombocytopenia (ITP) in Pregnancy

Dr Angharad Care (Clinical Research Fellow) from Liverpool University/Liverpool Women's Hospital was awarded £16,000 by The ITP Support Association to conduct a population-based prospective analysis of all reported cases in the UK of severe primary ITP in pregnancy over 12 months using the UK Obstetric Surveillance System (UKOSS). This is essential research as ITP is more prevalent in women of reproductive age and it may affect both mother and baby. There are currently no good quality clinical trials in this area and this study should: determine the true incidence of severe ITP in pregnant women (number of severe cases in 12 months); describe how cases are managed and; report outcomes for mother and baby.

We are currently in the collection phase of our data. Since June, nationally, there have been 51 reports of ITP in pregnancy and we are continuing to collect every month. The next step is to confirm the cases by sending data collection forms to each unit reporting a case to complete. That way we ensure they meet the case definition/criteria set by this project and obtain a full set of data to analyze for the project. Of 20 data collection forms received so far by the UK Obstetric Surveillance System (UKOSS) there are 13 confirmed cases, 4 do not meet the criteria, 2 have been reported in error and for 1 the reporting unit at present do not have the case notes and therefore cannot report.

The surveillance period is currently set to run until May 2014. We will review the number of cases that we acquire in that time and if we feel that it is insufficient for analysis we would make a plan to continue recruiting for an additional period of time. Ideally a minimum of 100 cases is needed for meaningful analysis.

Dr Angharad Care. Nov 2013

Sept 2014 update by Dr Care

Up to June 2014, 90 cases of ITP in pregnancy have been reported. Information has been received for 74 cases (82%). Of these, fourteen cases were reported in error, one set of case notes was reported lost and 8 did not meet the case definition. Thus, there are currently 51 known confirmed cases in an estimated 800,000 maternities. This gives an estimated incidence of severe ITP in pregnancy in the UK of 6.4 per 100,000 maternities; however, this may be a significant underestimate as data collection is still incomplete. The data collection period has been extended to January 2015.

Dr Care presented the following results of this research in the ITP Support Association 2015 National Convention.

This national cohort study  was undertaken by 202 consultant led units between 2013 and 2015. She was  assisted by three reporting clinicians from UKOSS.
The summary of the research showed this to be the largest reported cohort of severe ITP in pregnant women. There was a high rate of postpartum haemorrhage and an extremely low maternal and neonatal morbidity level of approx. 1 in 10,000 pregnancies. There was no difference in outcomes between patients treated antenatally or during labour, but it was noted that treatment carried some risks.



Dr Angharad Care

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