Paediatrics

Principal Investigator:
Dr N Kafil Hussain

A multicentre randomised controlled trial

The purpose of this study is to perform the first randomised controlled trial to test whether refractive adaptation before patching improves the number of successfully treated children with amblyopia. Electronic monitors are used to measure how much children wear their glasses and patches each day and will determine how this relates to their improvement in vision. The study is also investigating whether different types of amblyopia respond better to different treatments.

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Principal Investigator:
Dr T Bhatti

The National Registry of Rare Kidney Diseases (RaDaR) is a research initiative by UK kidney specialists (the Renal Association and the UK Renal Registry). It is designed to gather information from patients with rare kidney diseases. This will give a much better understanding of how these illnesses affect people. It will also speed up research.

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Principal Investigator:
Dr T Bhatti

Nephrotic Syndrome is a rare disease and can be difficult for the doctors treat, as it often recurs .The Kidney Research Unit in Bristol has developed a special interest in Nephrotic syndrome and what happens to the kidney in this disease. They want to find out:

• If some patients are likely to develop the disease because of gene mutations. (If the programming of the gene is slightly altered this results in a ‘gene mutation’ which can result in a disease.)
• We know that there are some gene mutations which cause NS but we don’t know how often this occurs in the UK.
• How the disease actually affects the kidney.
• Whether NS causes a pattern of changes in the kidney cells which is the same in every patient.

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Principal Investigator:
Dr D Haddad

Peanut, tree-nut and sesame seed allergies are common and may lead to severe allergic reactions. Peanut allergy has increased in the UK and now affects around 1 in 50 children. We wish to determine how many children with a peanut, tree-nut or sesame seed allergy are allergic to other nuts and seeds, based on the gold standard for diagnosing food allergies by oral food challenge. Children will therefore find out not just which nuts or seeds they are allergic to but also which nuts and seeds they are not allergic to. Nut allergies disrupt daily activities, family and social life. We wish to assess whether regular introduction of nuts and seeds to which the child is not allergic improves quality of life. Children with nut or seed allergies may develop new nut and seed allergies over time. There is evidence which suggests that regular consumption of a food promotes tolerance to that food. Through the regular introduction of ‘safe’ nuts and seeds into the diet, the onset of new nut and seed allergies may be prevented. This study will provide safety and acceptability data for a future larger study to compare whether children with specific nut or seed allergies who avoid all nuts and seeds are more likely to develop new nut and seed allergies than children who introduce nuts and seeds into the diet to which they are not allergic.

Principal Investigator:
Dr A Thakur

The anti-TNFdrugs, Infliximab and Adalimumab, have greatly advanced the treatment of Crohns disease. However some children and adults do not respond to treatment, and others lose response to the drugs with time. In addition children and adults occasionally experience unpredictable side effects which may be severe. The purpose of this study is to identify why some patients develop these problems, whilst other patients do not. It is hoped that we might then be able to develop a test to predict which patients are likely to benefit, and least likely to suffer side effects. This information will allow these drugs to be used in a safer, more cost-effective way, tailored to the individual patient. 1200 children and adults are being invited from across the UK to participate in this study.

Principal Investigator:
Dr T Bhatti

Children with nephrotic syndrome suffer from disease relapses, where the protein in the urine returns. When these occur, treatment with high dose prednisolone is commenced and this may be associated with a number of side-effects, such as a puffy face, high blood pressure or changes in mood. For this reason, every effort is made to prevent relapses from happening. This generally involves the use of either low dose every other day prednisolone, or other drugs such as levamisole, ciclosporin or mycophenolate mofetil. It is well know that relapses may be caused by upper respiratory tract infection (URTI the common cold). Three small studies performed in the developing world (India and Sri Lanka)have shown that if a short course of daily prednisolone treatment is commenced when children develop an URTI, this reduces the risk of a relapse developing. The purpose of this study is to see whether giving a six day course of daily prednisolone when children develop an URTI prevents relapses from developing.

The study is being conducted by the kidney specialists in every children’s kidney unit in the UK

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Principal Investigator:
Dr G Baksh

The aims of the study are

1. To establish a register of patients with severe juvenile idiopathic arthritis
2. To collect and record data on children and young people prescribed etanercept for JIA including demographic data, disease type and activity, outcomes and safety data
3. To collect data on appropriate methotrexate treated controls
4. To provide for the future collection of data on longer term outcomes through flagging with the NHS Central Register.

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Principal Investigator:
Dr G Baksh

This study is documenting the use of biologic drugs in children with juvenile idiopathic arthritis (JIA) and other rheumatic diseases in order to assess their efficacy and safety during routine clinical use.

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Principal Investigator:
Dr T Bhatti

United Kingdom national randomised trial for children and young adults with acute lymphoblastic leukaemia and lymphoma 2011.

Acute Lymphoblastic Leukaemia (ALL) is a type of cancer involving the white blood cells in the blood and bone marrow. This is the most common type of cancer in children and young people. The treatment for ALL is already very successful and up to 90% of patients are cured of the disease.

However treatment could be improved further by investigating the two problems that remain:

1. There is a risk of side effects both during and after treatment, and
2. Even with current therapy we cannot cure all patients.

In this research we want to take the standard treatment that we currently use to treat patients with ALL and make a few changes to this to find out the following:

• Whether the side effects of treatment can be reduced
• Whether we can reduce the chance that the disease returns (relapse)

Children on this study come to St.Peter’s for some of their treatment and we assist with gathering data here although their main care is at Great Ormond St. or Royal Marsden Hospitals

Principal Investigator:
Dr.Bhatti

Immune thrombocytopenia (ITP)is a blood condition characterised by a low platelet count. There are many aspects of ITP that we do not fully understand, To help us answer these questions we want to collect information about children with ITP in the UK in a systematic way to create a collection of information (or registry). This will form part of an international registry (PARC, Paediatric and Adult intercontinental Registry on Chronic ITP).

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Principal Investigator:
Dr Han

The purpose is to identify children and adults newly diagnosed with type 1 who might be interested in taking part in diabetes research studies. We want to collect information and blood samples from these people to understand more about the development and progression of type 1 diabetes, and to help find suitable trials of new treatments or other studies into diabetes that they might want to take part in. When we find suitable trials or diabetes studies we will contact these people to ask if they would be interested in taking part.

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Principal Investigator:
Dr Irwin

The purpose of BIOJUME is to find the genes influencing Juvenile Mycolonic Epilepsy or “JME”. By finding the genes, we hope to develop new tests to help diagnose epilepsy and also to develop cures.

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Principal Investigator:
Dr Irwin

The purpose of REGAIN is to find the genes influencing Rolandic Epilepsy or “RE”. Differences in genes or DNA (our genetic code that is passed down from our parents) explain the colour of our hair and eyes. Differences in our genes can also explain why some people develop certain diseases, like epilepsy, and others do not. We are studying individuals with epilepsy in order to find the genes that underlie these epilepsies. By finding the genes, we hope to develop new tests to help diagnose epilepsy and also to develop cures.

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Principal Investigator:
Catherine Casewell – dietician

There is now very good evidence to indicate that a healthy gut flora (“good bacteria”) can help improve the health of infants with allergy; with the potential to reduce the number of infections they have, normalise gastro-intestinal symptoms, improve the prognosis of allergies and potentially help prevent the progression toward other allergic conditions. Infants with Cows Milk allergy (CMA) often have a poorer gut flora than other infants, so may benefit from efforts to improve their gut bacteria.

The aim of this evaluation is to find out how well infants with CMA , who require an extensively hydrolysed feed tolerate a new formula containing a synbiotic blend, consisting of the prebiotics and probiotics.