Paediatrics

RaDaR: National Registry of Rare Kidney Diseases

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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NephroS: National Study of Steroid Resistant Nephrotic Syndrome in Childhood

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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PRONUTS

Principle 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.

BSPAR: The British Society for Paediatric and Adolescent Rheumatology Etanercept Cohort Study

Principle 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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BCRD Study: The long-term safety & efficacy of biologic therapies in children with rheumatic diseases.

Principle 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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UKALL

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

United Kingdom Childhood ITP Registry

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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Address2

Principle 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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Biojume

Principle 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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Regain

Principle 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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