Safety first | Action Medical Research

Touching Lives - December 2004

Safety first

Preterm babies suffer with many health complications as a result of their immaturity, often from bleeding or lack of oxygen in the brain. But also around a quarter of premature babies have problems with their lungs and develop chronic lung disease.

This is because they have not sufficiently grown to be able to breathe by themselves and require prolonged oxygen in hospital and at home to survive. This chronic lung disease is associated with a large number of deaths and prolonged health problems throughout childhood and even into adult life.

Although all medicines have to go through safety procedures before they are administered to patients of all ages, it is important to do follow up studies to evaluate how well they work. One of the treatments for lung disease in premature babies is to use steroids. Steroids are drugs that mainly work by reducing inflammation and have been used for the treatment of chronic lung disease — or bronchopulmonary dysplasia — since the 1980s.

Because of their anti-inflammatory properties, steroids enabled these vulnerable babies to come off the ventilator more quickly than they would have otherwise. This is important because prolonged ventilator therapy can in itself make the chronic lung disease actually get worse. Also, ^seeing a baby on a ventilator is a frightening experience for parents who may feel unable to hold or bond with their baby^.

Steroids had a clear and immediate impact on the health of preterm babies and so entered widespread use. Doctors concluded that the short term ill effects of ventilation were greater than those of using steroids. However as with any new treatment, steroids still needed to be evaluated for their safety, effectiveness and potential side-effects in the longer term. Early studies reported that using steroids was linked with high blood pressure, raised blood sugar levels and growth impairment, and more recently concerns have been raised of an increased likelihood of a child having cerebral palsy.

Dexamethasone trials

In 1986 — at a time when steroids were just beginning to be used for treating chronic lung disease in premature babies — Action Medical Research funded an international trial into the steroid dexamethasone. The trial involved 31 centres in six countries and confirmed that babies came off ventilator therapy quicker although unfortunately there was no reduction in how many children died of lung disease. These babies were then followed up at the age of three by the National Perinatal Epidemiology Unit in Oxford to check on their development, and in this latest study 145 children were looked at again now they are between the ages of 13 and 17.

The children underwent a number of tests and assessments. The parents provided information about their respiratory symptoms, schooling, visual and hearing impairment, learning disability, cerebral palsy and epilepsy. ^A nurse visited the children and measured their growth, lung function, and vocabulary^, and the children did some non-verbal reasoning tests.

The vocabulary tests involved the child looking at a picture and then choosing one word out of four which best described the picture. Non-verbal reasoning tests involved predicting the next pattern in a series. The nurses on the project talked to the children’s teachers at school about their educational ability, and a comparison was made between the numbers of children who had cerebral palsy from the group that had the steroid treatment and those that did not.

Pleasing results

Dr Ros Jones, lead researcher on the £108,000 project, told Touching Lives, “We were really pleased that the results showed that most children with chronic lung disease in infancy have grown up to be healthy, although there were obviously a higher number with various disabilities because of their prematurity.

“We found no real differences in growth, educational development and lung function between the two groups, and this will be a comfort to the parents whose children were treated with steroids. This was a complicated trial as some of the children had the therapy at different times so there was an overlap, which makes evaluating the effectiveness of steroids more difficult. And because the babies involved were so premature, it made them very vulnerable to long term disability, and assessing what could be the potential side-effects of the steroid and what is caused by early birth is complex.

“Reports that have put all the different studies together suggest that there may be a best time to give steroids. There may also be fewer side-effects if much smaller doses are used. There is a role for the use of steroids for seriously ill babies who are on ventilators, and our research will add to the weight of other studies into the application of steroids for premature babies who have lung disease.”

Helping others

Tiffany Scannella gave birth to twins Michael and Natalie on 23rd February 1987. They were 15 weeks early, weighing just 2lb 1oz and 1lb 14oz respectively.

Mrs Scannella’s children took part in both Action Medical Research studies: “It was a long time ago but I remember the labour happening really very quickly. Then because the twins were so premature, after six weeks the doctors talked to us about participating in a trial of a treatment that could help them breathe. Of course ^we wouldn’t know which twin would get the steroids, but it was good to know that the results would help other babies^ in the long term.

As well as the problems with their breathing we were told that Natalie may need speech therapy and to wear glasses in childhood. She had pneumonia and stopped breathing at one point, and needed two blood transfusions. She was very sick — I really thought she was going to die so I was just glad to give anything a go.”

Mrs Scannella has always thought that Michael received the steroid treatment because he came off the ventilator after two and a half months, whereas Natalie was on it for three months and came home at six months, still on oxygen. Her prematurity at birth has had long-term consequences for her. “Natalie has some learning disabilities and her reading isn’t very good,” Mrs Scannella said.

“Although in every other way ^she is just a typical 17-year old^. She has a boyfriend and a part-time job, and is at college studying health and social care. Michael is doing his GNVQs and AS levels and he is hoping to join the police force. He’s six feet tall so if he did have the steroid treatment I don’t think it’s affected his growth at all!

“I like the fact that their progress has been followed up in this way by Action Medical Research. It’s great to know that someone is there and they can tell you that everything is OK and that they are within the ‘above average’ category. The kids were keen to help Dr Jones and her team with the follow up study — they understand how important it is.

“And I myself now work with children with learning difficulties. I want to help children reach their full potential and feel that I had some experience of the difficulties they face with my own children.”

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