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Information for adult patients
Establishment of a UK Evans’ Syndrome Registry and
molecular investigation of potential causative factors
Invitation to Participate in a Research Project
You are being invited to take part in a research study. Before you decide, it is important for you to understand
why the research is being done and what it will involve. Please take time to read the following information
carefully and discuss it with others if you wish. Ask us if there is anything that is not clear or if you would like
more information. Take time to decide whether or not you wish to take part.
What is the Evans’ Syndrome Registry?
Evans’ Syndrome is an uncommon disorder affecting your platelets and red cells. The low platelet count is
caused by an antibody that attaches to platelets and causes their removal by the spleen; the same happens to
your red cells. We know very little about the cause of Evans’ Syndrome and despite many years of treating this
disorder we are not certain which treatment is best or when treatment should be given, or even how common
the disease is. Why have we chosen to establish a registry? For uncommon diseases such as Evans’ Syndrome,
individual hospitals will only have a small number of patients with the condition. This makes it impossible to
study the disorder since meaningful results are only obtained through the study of large numbers of
individuals. Disease Registries, therefore, allow us to build up a complete picture of the disorder, since we are
able to collect information on a large number of people with Evans’ Syndrome across the UK. We can then
separate these into distinct subgroups, such as: mild Evans’ Syndrome or severe Evans’ Syndrome, people
with good response to steroids and those with poor response to steroids, and so on. We may then be able to
link this clinical information with scientific findings. For example, we may find that people with mild Evans’
Syndrome have a particular profile or pattern within their immune response genes. If we find such
associations, these may be useful in predicting the type of Evans’ Syndrome a patient is likely to have, or
which treatments might be effective. Other information that comes from disease registries includes ‘natural
history’ information, where we learn about the effects of treatment on the amount of bleeding, bruising or
anaemia suffered by Evans’ Syndrome patients over time. We can also obtain information about the long term
effects of therapies on the patients’ general well-being, and most importantly of all, it lets us know whether
our treatments are beneficial. Worldwide, there have been no trials in Evans’ Syndrome and the establishment
of the Registry will provide much of the information we are currently lacking. If you decide to register
(through your Haematologist) you will be asked to provide a few basic details about your disease e.g. age
when it was diagnosed, your blood count, what treatment you had and any associated bleeding or other
symptoms. You will be allocated a unique number so we can follow your progress over time. At the same
time, we would very much like to have a small sample of blood (10ml, ~2 teaspoonsful for adults; 5ml for
children) for the molecular study, which is described next.
All information which is collected about you will be kept strictly confidential. Any information about you
which leaves the hospital will have your name and address removed so that you cannot be recognised from it.
Molecular study of Evans’ Syndrome —What causes Evans’ Syndrome?
The ‘molecular’ study is so-called because it is a scientific study of the immune system molecules. The immune
system consists of cells and proteins, and these proteins are coded within our immune system genes. We know
that individuals have natural minor variations within the genes of the immune system and this is what we are
trying to detect in the molecular study. At present we do not know what triggers Evans’ Syndrome but it may
start following a minor viral illness after which the body makes antibodies against its own cells. Disorders
such as these are called autoimmune. We think that part of the immune system is over-reacting but we are not
sure which part of the immune system this is. Studies of other autoimmune diseases suggest one or more
immune regulation genes may be overactive.
We would very much like to see whether your immune regulation genes show the same type of variation as
Evans’ Syndrome Patient Information Sheet version 2.0, September 2004
that found in other diseases such as rheumatoid arthritis.
How might this research help?
Although this study may not have an immediate impact on your own Evans’ Syndrome, it will hopefully
benefit patients in the future and:
1. It may help us find out what causes Evans’ Syndrome and predict whether the Evans’ Syndrome will be
mild or more severe.
2. By analysing information from a large number of registered patients we should have a better understanding
of which treatments to use in the future, although it is unlikely to alter your treatment at present.
3. We may find ways of switching the overactive genes off.
What are we asking from you?
If you agree to take part in this study, it will simply involve taking an extra 10ml of blood (5ml for children)—
on one occasion only—at the same time as you have your routine blood count at the outpatient clinic. There are
no hazards in this research, and the amount of blood which we take for the research is very small and will not
make you anaemic or feel in any way unwell. All the results obtained will be coded in such a way that your
identity will be unknown to the researchers. Of course, you do not have to join the study and please be
assured that if you decide not to take part your care will not be affected in any way. Your local haematologist
will write to your GP to let him/her know you are taking part in the Registry.
What happens if you wish to withdraw from the study?
If you feel you no longer wish to take part in the study, we will remove any data relating to you from the
database. This will not affect your ongoing management or care in any way.
What will happen to the results of the research study?
The Registry information will be published periodically in the form of properly peer-reviewed papers so that
other clinicians dealing with patients who have Evans’ Syndrome are better informed about the different
treatment options available. In addition, we will publish outlines of the Registry and the scientific study on the
PISCES (Evans’ Syndrome patient association) website since this organisation is funding part of the molecular
study. We will also present information at the Annual British Society for Haematology conference to educate
other haematologists about Evans’ Syndrome. Data will be stored electronically on Dr Provan’s computer with
strict password protection. The only individuals with access to the electronic data are: Dr Provan, Professor
Newland, our Nurse Specialist and Data Manager (Registry Coordinator) and the Senior Scientist performing
the molecular tests. Since the study is a long term follow-up study, the data will be kept for 10 years. At the
end of this time the Registry data will be reviewed and if felt to be useful, will be continued. However, if all
useful information from the Registry data has been extracted the electronic files will be erased.
Who has reviewed the study
The study has been fully reviewed by The London Multicentre Research Ethics Committee. The molecular
study has been reviewed by several senior clinicians involved in the Evans’ Syndrome field, both in the US
and the UK. The funding we obtained was based on a favourable review by these reviewers.
What happens if you are worried or if you would like more information?
You will always be able to contact an investigator to discuss your concerns and/or to get help:
Name:
Address:
Telephone number:
Dr Drew Provan
The Royal London Hospital, London E1 1BB
020-7377-7178 or 07971-206092 or [email protected]
Name:
Address:
Telephone number:
Professor Adrian Newland, Head of Department of Haematology
The Royal London Hospital, London E1 1BB
020-7377-7180 or [email protected]
Name:
Address:
Telephone number:
Sister Deborah Kenny, Specialist Autoimmunity Nurse
The Royal London Hospital, London E1 1BB
020-7377-7652 or [email protected]