Let's Talk Rett Syndrome Conference - Sydney, 21-22 April 2007

Moderator Bill Callaghan, President Australian Rett Association
Dr Helen Leonard, Clinical Associate Professor, University of Western Australia
Mrs Kathy Hunter, President & Founder of the International Rett Assn.

Research Recent Developments

There are 320 girls and women diagnosed with Rett Syndrome in Australia.

Kathy opened the morning by saying “History is being rewritten at the moment. Teachers believe girls with Rett Syndrome can and do understand, and are able to learn.”

Clincial trials indicate that therapy can be tailored to the actual mutation of the gene.

One of the quotes in Kathy Hunter’s revised HANDBOOK ON RETT SYNDROME is by Alavis Morrisette. “Life is not about how fast you run or how high you climb but how well you bounce.”

Dr Helen Leonard who has been Director of Rett Syndrome at the University of Western Australia since 1993 said the new research is the first time a neurological illness is proof of a theory. Fourteen years later her research is invaluable to see patterns with the variation of the mutation. The clinical data is linked to the most common mutation. She has also tested three boys with MeCP2 mutations or deletions. The irregular nature of the boys breathing alerted paediatricians that something was astray soon after their birth.

Helen said some of the more difficult behaviour patterns are associated with the milder mutations. It is as though there are two types of Rett children. The ones who have “cerebral palsy” like symptoms and show lack of motor milestones with low muscle tone but who are often happy and the “autistic type” child who is highly strung, hyperactive, not sleeping and for whom motor planning is a problem.

Behaviour

Why are these behaviour patterns happening?

Kathy said studies at the Kennedy Institute had shown that there are excessive levels of glutamate in the bodies of young girls with Rett Syndrome. This causes seizures and irritability. The levels lower with age and the girls become more peaceful. The age of 2-4 where the girls are moving between Stage 2-3 is one of the most difficult. Despite the therapies the problems often improve.

The girls often do not show a reaction to pain. They have elevated levels of beta endorphins and do not feel pain as much as normal.

The gastro reflux is very painful as food gathers and the acid burns the throat when it is regurgitated. Coughing, choking, and spluttering after eating especially with fried food and chocolate. Untreated reflux can lead to the oesophagus being damaged.

Anxiety

Dr Zogbi is working on a specially tailored drug to help with anxiety reduction in Rett Syndrome. Tooth grinding, air swallowing and hand wringing occur under stress.

Constipation is most uncomfortable and everyone has their special remedy to help bowel movements. Microlax per mouth is said to help the formation of stools. Regular time for bowel movement seemed to be important. Bowel is often full of gas in Xrays. Stools are often “stuck” high up in the bowel so that is why softeners taken per mouth are often more satisfactory than suppositories. Sometimes both are found to work well.

Drooling

Some parents used 3 drops of Atropine on the tongue, others found botox in the salivary glands was helpful. Accupressure on the points below the ear, sides of the mouth and under the nose helped one girl.

Crying & Sleeping

Kathy said she thought there were physical reasons for this behaviour. Dystonia, reflux were possibilities. Different families had different remedies but music and the child’s favourite dvds were often great solace.

GALL BLADDER problems were often associated with Rett and it is important to have this checked as passing gallstones is very painful.

Apnea and Breating Difficulties

Boys with MeCP2 mutations had been noticed because of their severe breathing problems. Helen Leonard said she thinks this will be associated with certain genotypes.

Hyperventilation

Dr Leonard said that 50% of Rett children have breathing diffiuculties

A physio said lying the patient down and massaging their torso helped relax the child. Others said if you blow onto their face the pattern will stop.

Ear Infections are common in young children and one third of 81 participants in a study had diminished hearing.

Hand Wringing

Balliol Studies show if hand movements are stopped the girls can often concentrate better. If the non dominant hand is has a splint it seems to give better control to the other hand.

Helen Leonard said the stereopathies may be related to the mutation.

Sleep

Some girls sleep well others seem to need less sleep. The crying screaming period passes although it is hard to believe at the time.

Music, music, music seems to be the great comforter.

Issues Associated with the Nervous & Sensory System

Seizures

What is a seizure, jerking are not seizures.

Different medicines seem to help different girls.

Sometimes the pain of reflux and impacted bowel motions cause seizures.

Seizures are reported to be more common with hormonal changes. Some of the girls were on birth control pills to monitor periods.

Sensory Perception

Girls seem to be more sensitive to touch, sound and smell.

Tell them when you are going to change their position etc. Some days their antennae is up and some days it is down.

Going down stairs is a problem, one physio suggested coming downstairs (with help) in a sideways position holding on to the rail. Going between carpet and wood can often stop the girls as though it is a huge step or barrier.

Issues Associated with the Digestive System

Gastrointestinal Problems

Many of the girls are malnourished inspite of their great appetites and love of food. Many of them have had “G” tubes fitted to compensate and reports are they have more energy and it is a good way to ensure even doses of liquids and medicine. This session was very helpful to those families who were contemplating G tube insertions.

Issues Associated with the Musculosceletal System

Sunday’s conference began with musculoskeletal problems relating to the feet.

The girls feet are very small, red or bluish and often swollen by circulatory problems. Contractions can become so severe that it pays to deal with the problem quickly.

Some families had had serial casting which forced the feet down into a neutral position where shoes could be worn again. Circulation issues make casting more of a problem for some girls. There have been some unfortunate incidents with the girls inability to feel pain.

Helen Leonard said it would be good to have some research as to the success of heel cord surgery. Some girls walk on their toes like ballet dancers. Hammer toes do not seem to cause too many problems. Kathy said they result from the girls clamping their toes down to keep their balance.

HL said the girls have a short fourth toe which may be genetic in origin.

Botox Injections

Some families reported that botox injections had helped the girls for periods of 3-6 months. In some states they are funded for certain parts of the body but not others. Physiotherapists said it is important to keep up stretching exercises after the injections to maximise their benefit.

Botox injections to alleviate muscular tightness in the neck region are funded by some states and not by others. Cerebral Palsy children have access to funding because of research saying the children have been helped by this procedure.

KH said not to overlook dystonia pain with the girls and her book has a section on this.

Hips

A lot of discussion ensured on the partial dislocation of the hips from long periods of sitting and with the problems related to curvature of the spine.

A Melbourne Professor has a paper being published on problems relating to the hips in Rett girls and women. A physio said keeping knees apart in the wheel chair was important and stretching exercises would help hips from getting worse. Positioning in the wheelchair was vital and a wedge was important to keep the back straight.

The sleeping position of the girls should also be studies as pillows and cushions cut out for the hips so the girls could sleep in a neutral position was helpful.

Tight hamstring muscles can be uncomfortable in the wheel chair, once again remember a wedge can help.

Scoliosis

Helen Leonard said her study showed that by the age of thirteen years most girls have scoliosis. The median age was 9.8 years which is lower than expected.

She said an English surgeon at the Belgium conference had thought earlier surgical intervention brought better results. There is a new study and Guide lines for the Management of Scoliosis will be published as a new study. There are expandable rods for younger children.

Issues such as bracing were discussed and Kathy said many expensive braces wiled their time away in cupboards because the girls found them to be too uncomfortable. You need to wear them 23 hours a day.

In Australia 50-60 girls out of 320 had had surgery. The majority of the girls were not mobile before surgery. One Mother said she had extreme physical therapy for her daughter at the time of puberty when her back curved at an alarming rate and she is still walking many years later.

Aspects of Communication

Bill Callaghan introduced this session emphasising the need to allow time for the girls to process information. It was important to start with their favourite activities such as food, water, siblings etc.

Repetition was important however, the girls got bored with too much repetition.

The on /off buttons of the tape recorder gave the girls choice.

PC programmes with YES/NO and MORE/FINISHED where enjoyed by the girls.

Some parents reported excellent results with facilitated communication

Comprehension, concentration, memory response time varied with moods and interest level. Music was great for concentration and singing instructions worked in many cases.

Everyone had their favourite DVD’s and interesting stories were told about girls screaming with frustration when carers had put on the wrong DVD.

DVD’s obviously are like favourite stories which give stimulation and comfort.

Kathy said when they introduce Stacey to a new DVD they put it on in 15min segments over a few days.

Kathy said “Not being able to talk is not the same as having nothing to say.”

Girls love to be given a choice however, we need to wait for the response.

Music Therapy

Kathy went back to the first Rett conference in 1984 when 39 Parents met in Baltimore and Dr Rett said Music seems to be in part of the brain which is untouched by the condition. Because the girls cannot copy speech they often miss out on speech therapy where you need to be ready to copy the sounds.

Music can bridge the gap with communication. All parents agreed music was the most soothing and most enjoyed of the therapies.

Horse riding was good for balance.

Hydro therapy was excellent for relaxation and stretching muscles.

Massage was great to relieve tension.

Physical therapy with up to date stretches was important to keep muscles strong.

If using splints stretching needed to be consistent.

A standing frame in front of the computer gave some exercise to the back muscles.

The organisation MOVE had interesting ideas to get people to stand through movement.

Step Out was a walker that was strapped on which had helped several children learn to walk.

The kind of wheel chair one chooses should have good back support. A Renault car can be converted to take.

Wheelchairs but still seem like a car instead of a van.

Dolphin therapy was a special event for Allanah.

Education

IEP – An individual programme was necessary for each child to reach potential.

A Note book with the child’s medication and favourite activities was a must when leaving a child with carers or visits to hospital. Ideas such as putting tablets in yoghurt to disguise the taste would be helpful to nurses and teachers.

Schooling varied from family to family and State to state.

Post school – with more girls living longer it is important to work on some of these issues. Also once you are past the age of the children’s hospital it is more difficult to get help from the Adult hospital.

Care of the Care Giver

Kathy gave an impassioned speech about finding time to devote to oneself.

Sometimes taking a break can restore one’s sense of balance. Men & Women cope with the grief of the diagnosis in different ways. Men are fixers and so like projects to help with the child’s development. Women seek solace with their family and friends while they work out their own emotions.

“Your daughter will teach you the most important lessons in life. The most important things in life are your health and having people to love. Stacey gave us courage, inspiration and was a facilitator to reach out to others because of what she cannot do rather than what she can do. The struggle to survive has made her triumphant.”

Siblings have been shown to be more tolerant and accepting of difference and more willing to help others. They have appreciated their own life of health and developed their gifts.