E.coli O157 & Haemolytic Uraemic Syndrome

Drugs trial of Eculizumab (ECUSTEC) for Haemolytic Uraemic Syndrome (HUS) to start in June this year.

16th May 2016

This trial is known as the ECUSTEC trial as a reference to the drug (ECU) combined with the condition that can cause HUS (Shingla Toxin Escherichia Coli).

The drug Eculizumab has been used for a similar condition aHUS in recent years and has proved to be successful in improving the long term outcomes for these patients. Our charity, along with others, supported the aHUS Action charity in its efforts to have this given to patients, when we were made aware of its success for them. These patients can now be prescribed this drug as a treatment.

Eculizumab has been used for some patients of HUS previously in oubreaks of E.coli abroad but, as it was not properly trialed,  there is no scientific evidence if it is an effective treatment or not. Hence the reason for this trial.

Our small national charity supports this trial bcause at the present time there is no specfic treatment for HUS despite the best efforts of Paediatric Nephrologists (Children’s Kidney Specalists) throughout the UK.

Given that approximately 1/3rd of all HUS sufferers have some long term heatlh issues, some minor and some major, we would wish to see these long term health outcomes improved.

We are one of the co-applicants for this trial, as since we were formed in 1997 we have seen no major improvements for these sufferers.

Also, given that Government has failed to legislate effectively to prevent bacterium such as E.Coli O157 which causes HUS, entering the food chain and the environment, we believe that this is at least an effort to address unnecessary suffering. Whilst we would wish to see prevention rather than cure, this has not been possible. We believe the food and farming industries, including open farms, have not addressed a problem which is essentially theirs. 

Neither Paediatric Nephrologists or our Charity know whether or not the drug will work, but it does offer a glimmer of hope, which is why we are co- applicants for this trial. 

The trial is supported by the 13 Renal Units in the UK. These units have a wealth of expertise and experience and their Paediatric Nephrologists, along with other experts in this field,  believe that this trial is important, as currently they have no specfic treatment to prevent the long term effects that some patients will suffer.

If youir child has HUS you may be asked within 24-36 hours by your treating doctor or Paediatric Nephrologist at the renal unit you are in, if you wish to take part in a drugs trial of Eculizumab for HUS.

HUS in very basic terms,  is a form of blood disorder that affects the Kidneys and may have other complications.

As a charity who represent sufferers of this condition, we realise that to make a decision in such a short time will be difficult for you, given that this diagnosis has come as a shock.

The treating doctor(s) will need to give you information both verbally and in writing. They have produced the information to enable you to make an informed decision as to whether you wish to consent to taking part in this Drugs trial. The documents have been written by the research team, along with others and our charity, for parents/guardians and children of different age ranges.

You are under no obligation to agree to take part, and you will be given the best possible medical care  should you say no. It is your right to say no, for any reason you may have without having to explain your reasoning.

The Drug

The treating doctors/ Paediatric Nephrologists believe that Eculizumab is best given within the first 36 hours after arrival at the renal unit, with a second dose being adminstered no later that 8 days after the first. (Normally at 7 Days).

The trial will invovle 50% of patients receiving the drug and 50% receiving a Placebo of Sodium chloride only. (A Placebo is a drug that has no effect)  The treating doctors/ Paediatric Nephrologists and Nurses in your renal unit will not know which patient is receiving the drug or Placebo. Only the Pharmacy will know and they will be given the list of who gets what, by a computer generated programme (This is known as a doubled blind trial). Only in special circumstances can the treating doctor(s) find out who is having Eculizumab or the Placebo.

The possible disadvantage of the drug is that there is a small risk of Meningococcal (Meningitis). To help prevent this, both the manufacturer and other experts believe that vaccination against this is essential, therefore penicillin or an alternative for those who are allergic to it, will be given. The treating doctor(s) will explain more about this requirement. They will normally consult your GP to ascertain what is required.

If your child enters the trial they may be given penicillin or an alternative twice a day for 8 weeks whether they are receiving  Eculizumab or the Placebo. This is to ensure the trial is fair and that those receiving the drug cannot be easily identified by others.

Any other known side effects of Eculizumab will be explained to you by the treating doctors. 

Eculizumab is made by a company called Alexion and is an expensive drug, however the Eculizumab used in this trial will be paid for by the National Health Service (NHS).

The research study is being funded by National Institute for Health Research, Efficacy and Mechanism Evaluation Programme (NIHR EME).

Why should I agree to let my Child take part in this trial?

The trial is subject to strict scruitiny particularly in the first year to ensure that the drug Eculizumab has no unknown side effects. Your child will be assessed daily and followed up after discharge at 14 days, 30 days, 60 days, 6 months & 12 months  This will then determine if the trial continues to the 2nd,, 3rd and 4th year. 

All the children in the trail will be monitored for side-effects and if there is any concern that these are in any way associated with the study then your child could be withdrawn from it.

Your child can also be removed from the trial at any time upon your request. 

If you have any concerns about your child at anytime during the trial, please consult the treating doctors at the renal centre your child was treated at. Also you will be kept informed of how the trial is progressing.  The information gained from this study will not identify individual patients and will be stored securely for 25 years.

If you agree to take part in the trial you will be helping other sufferers in the short and long term. The trial hopes to recruit 134 children over a four year period. The fifth and six year of the trial will consist of collating the findings from the previous four years of research. 

Should you wish to talk to someone from the charity about the trial, please contact us by either using the e-mail address or telephone number (which has an answerphone) as stated on our website.

Please note we cannot discuss the specfic medical aspects of the trial or treatment, but we can discuss any concerns you have in relation to patient information, or if you think there are any ways the trial could be improved for patients, parents or guardians.

The charity has no association with Alexion the manufacturer of  Eculizumab or any other commerical interest in the work we do, which allows us to be truly Independent.

If you are asked to consider the trial for your child, thank you for your decision, whichever way you decide.

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