Treatment

My child has a Hickman line in. What should happen if he has a fever?

If your child’s temperature is 38 degrees and above they need to go to hospital straight away and be seen urgently. Do not give your child paracetamol, aspirin or ibrufen as these may mask the fever. The hospital must take blood samples from the line and start intravenous antibiotics. Sometimes children can look very well and have no other symptoms, however having a line in that goes into the bloodstream means that a child with CGD is at risk of developing an serious infection and any fever of 38 degrees and above must be treated as a line infection until the blood test confirms it is not.

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Are homeopathic/complementary/alternative therapies used to treat CGD?

There is limited scientific evidence about the benefits of homeopathic or complementary medicines. They should only be used if they support conventional medical protocols set out by your doctor or medical team.

Just because a medicine is described as “natural” or “herbal” doesn't mean it is guaranteed to be safe. Always check the contents of any homeopathic or herbal treatments with your doctor. Some treatments will not be suitable for people with CGD.

Ensure that people offering complementary therapy are properly qualified and registered with the appropriate regulatory body.

It is vital that you or your child or family member affected by CGD doesn’t stop taking your usual medicines (itraconazole and co-trimoxazole) because these prevent infections

Find out more about how to manage medication.

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Are there any special considerations for children with CGD who are offered the Fluenz vaccine to protect against flu?

Children in the UK are presently being offered a flu vaccination, which is given as a nasal spray and is a live virus. Children with CGD can have this product however careful consideration must be given to other family members if they are in close contact with someone who is about to have a bone marrow transplant or who has had one within the last two years.It is best to check with your immunology doctor first.

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Are there any special considerations for treating hay fever when you have CGD?

No. It is treated in exactly the same way as for people with healthy immune systems by taking anti-histamine tablets available from your chemist such as Piriton, Zyrtec, and Benedryl. Sometimes if your nose is really blocked you might need to visit your GP to ask for a nasal spray.


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Are viruses more of a threat to people with CGD than to people with healthy immune systems?

People with CGD are able to fight viral illness in the same way that people who are not affected by CGD.

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My son is 5 and has rarely been sick but has lost weight and has had loose stools for about a year. The doctor has found granulomas on his bowel following a colonoscopy and now wants to do a CGD test even though no other symptoms are present. Is there reason for concern and to do the test?

This is a sensible decision on behalf of your doctor.  CGD patients usually have more symptoms and these are often more severe but mild CGD cases and some restricted to gut involvement have been described. This test is worthwhile to rule out or confirm CGD so that your son can get the right treatment.

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Have there been any clinical trials of gene therapy for treating patients with the p47phox form of CGD? What were the results and what is the current status of this work?

The first ever trial was for p47phox was at the National Institutes of Health in the USA but this was with old vectors (delivery systems) and no conditioning and this was about 20 years ago. Together with the Swiss group in Zurich Professor Adrian Thrasher, at Great Ormond Street Hospital are making a p47phox vector based on the existing gp91 vector (the one that is currently being tested for X-linked CGD), hopefully to be ready for trials in 1-2 years. This work is funded partly by Action Medical Research see www.cgdsociety.org/newsroom/latestnews/genetherapyforp47phoxformofcgd

 

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Is severe diarrhoea a reaction to antibiotics?

Never assume anything where CGD is concerned, especially when problems continue. Always ask for it to be investigated. The underlying cause for diarrhoea, or vague, continued tummy pain, could be due to inflammation in the guts. Continued constipation or difficulty passing water should also be reported to doctor and investigated.

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Is there a cure for CGD?

Yes. Bone marrow transplant (BMT) can cure CGD. Although BMT is not without risk it is becoming recommended for a greater number of affected children using related and more recently, unrelated matched donors.

Read about BMT

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Is there a list that tells us exactly what my child/I should or should not do to keep safe?

There is no definitive list of things it is absolutely unsafe to do or completely safe to do. There is an element of risk in everything we do and it’s up to each individual/family to decide how they want to live their lives, balancing risk to a level they are comfortable with.

Families approach lifestyle changes in different ways. For example, some parents are not happy for their child to play rugby in case they get bruised and this leads to complications. Instead, they find a non-contact sport for them to take part in or suggest that they become a referee.

Others allow their child to swim at a clean swimming pool (swimming in lakes and rivers is not recommended in case they harbour bacteria).

Others feel that humid conditions at public pools may lead to infection. Each family needs to assess the risks and decide how to balance them with the need to lead as normal a life as possible.
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Is there any treatment?

People who are diagnosed with CGD should take daily antibiotics to prevent infection and stay healthy. This is called "prophylactic" treatment. From time to time, serious infection or illness may need to be treated with more aggressive antibiotics and a stay in hospital.

Find out more about how to manage CGD.
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I’m an X-linked carrier of CGD and one chronic health issue that I have been living with is chronic constipation and irritable bowel syndrome. Is there is any evidence that bowel issues are a symptom of CGD carrier status?

Yes although chronic constipation and irritable bowel syndrome [IBD] are problems seen in the general population there is some evidence to suggest they are linked to X-linked carrier status and these can be symptoms that carriers experience.  In a study involving 81 carriers, conducted by Dr Alex Battersby at Newcastle University, UK, 41 had bowel problems with 11 per cent having constipation and 8 of 81 having IBD.

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Is Toxoplasmosis a problem in CGD?

Toxoplasmosis is a virus that is known to be dangerous to pregnant women. People with CGD do not have any different risk factors associated with toxoplasmosis and would not be routinely tested for it unless they are being considered for bone marrow transplant.

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My child has CGD and we are considering BMT. We have heard that virus infection can be a risk before, during and after the procedure. What are these risks and what is done to minimise them?

When someone with CGD is being considered for a BMT they will be tested for specific viruses. The reason for this is because during BMT their immune system will be reduced and the new donor immune system will take many months to fully recover.During this time the viruses that the patient has already been exposed too may grow in number as there is no immune system to fight them.If untreated the viruses can cause problems however patients are routinely monitored for viruses and if detected then appropriate treatment is started.

Patients undergoing BMT should be advised of whether or not that have had these viruses and how it may affect them in transplant.


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My son has recurrent mouth ulcers that spread to his throat and cause difficulty eating. Do you have any suggestions as to what may help?

Mouth ulcers are commonly seen in those affected by CGD they can be localised to the mouth but in some cases can affect the whole of the digestive tract. Good oral hygiene is extremely important with regular dental check ups advised. Using an electric toothbrush has been recognised as helping maintain good oral hygiene. There are products that can be purchased over the counter that will help reduce pain and keep the mouth clean so the ulcers can heal. it is always advisable to discuss these options with the pharmacist.

If the ulcers occur frequently, are persistent or take a long time to heal and they reduce your son’s ability to eat and drink normally then you should seek medical advice. It would be helpful to have the ulcers swabbed to make sure that there is no underlying bacterial or viral infection that may require additional treatment. Mouth ulcers in people with CGD are generally caused by inflammation and in some cases will respond to specific drugs used in people with CGD, these would need to be prescribed or recommended by your immunology specialist. In specific circumstances a short course of oral steroids may be prescribed under medical supervision.

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Should people with CGD have the Flu jab?

Some viruses such as the Flu virus can cause serious illness in the general population so it’s really important for everyone that is offered a vaccination to have one. People with CGD will be offered a flu vaccine.

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What about immunisations?

The usual immunisations are recommended, including vaccinations against rubella, measles and mumps and polio. People with CGD should avoid having the BCG vaccine which is generally given in the teenage years. This is because people with weakened immune systems cannot be given “live” vaccines. The BCG vaccine is made from a weakened, live form of bacteria closely related to human tuberculosis. These bacteria can cause disease and complications in CGD.

People with CGD are able to fight viruses in exactly the same way as healthy people so vaccination against the polio virus can be given either ‘live’ and taken orally or ‘killed’ and injected.

Sometimes influenza can be complicated by bacterial infection so it is a good idea to have the “inactivated” flu vaccine. Inactivated means killing the disease-causing component of the vaccine with chemicals, heat, or radiation before it is used for vaccination. Please talk to our CGD specialist nurse about this. 

Find out more about how to manage CGD.

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What is a virus and what diseases can they cause?

Viruses are very small organisms that can cause an infection. Viruses need a host cell to survive, the viruses works by taking over the host cell, which will continue to reproduce allowing the virus to survive.

Viruses can by spread by simple contact such as coughing and sneezing whilst others can spread by contamination from faeces due to poor hand washing.

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What is gene therapy for CGD?

In gene therapy a healthy copy of the faulty gene is placed into the person’s own stem cells. Gene therapy is only offered to people who are very seriously ill and who have infections where the usual medicines don’t help. Although gene therapy may have the potential to be a cure for CGD at present it offers a temporary but effective treatment of life-threatening infections.

CGDS has funded pioneering gene therapy research since 1995 with 14 patients in Europe treated to date. The ultimate aim is to develop a cure that will be suitable for all people with CGD and this remains a hope for the future.

Read more about gene therapy


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What medicines should I take if I have CGD?

People with CGD have to take antibiotics and antifungal drugs everyday to fend off infection. However, is it still possible that people with CGD can get infections even with this drugs regime. In this case it is important to seek medical attention quickly.

Read about health management of CGD


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What precautions should I take to avoid infection if I have CGD?

There are several ways to avoid getting infections. Being careful with personal hygiene and avoiding environments that may have high levels of fungi are advised. This means not working with or around mulch, hay, wood chips, grass clippings, garden waste and staying out of barns, caves and other dusty or damp areas. This helps avoid inhalation of high levels of fungi.

Read more about preventing infections

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When will gene therapy become a cure?

The answer is we don’t know when gene therapy will become a permanent cure for CGD.  We do know, however, that huge advances have been made since it was first thought of and that research has led to refinements of the gene medicine now given to patients. Each time a patient is treated we learn more about what is working and what is not and this provides the necessary information for researchers to adapt their approach in developing a permanent cure.

Read more about gene therapy




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Will stem cell treatments help with CGD?

Yes - they already are. Stem cells from bone marrow and from umbilical cord blood are being used successfully to cure CGD. Treatments such as gene therapy also rely on the use of stem cells.

Read more about treatments

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