The following are summaries of the talks from the 1998 Cri du Chat conference,
held in Warrington, England. The information comes from notes I made at
the conference and I apologise if I have misunderstood anything and got
it wrong!
Joan Overhauser's talk about Cri du Chat syndrome
When given a diagnosis based on genetic testing that involves the deletion
of part of a chromosome we are given a string of numbers e.g. 46,XY,del(5)(p15.1).
Joan explained what this means:
46 = the total number of chromosomes
XY = the sex of the person ( in this case male, XX = female)
del = deletion ( a piece missing)
(5) = which chromosome is affected( in this case the fifth largest)
(p15.1) = where the breakpoint is i.e.:
p = the short arm (q would be the long arm)
15.1 = the location. On chromosome 5 the specific bands are 11 (being the
biggest deletion), 12, 13, 14, 15.1, 15.2,
15.3 (being just the tip)
Why do breaks occur?
Most importantly, there is no reason! It isn't the fault of either
parent!
10 -15% of all eggs and sperm have some kind of chromosomal abnormality,
and all chromosomes can get deletions, but those with deletions on chromosome
5 seem to survive better than those with deletions elsewhere.
50% of all pregnancies result in a miscarriage, often due to a chromosomal
abnormality or a more basic unknown reason. The body seems to know when
a foetus is not developing properly and aborts it. It is infact amazing
how many of us were born normal!
What does this loss mean?
Loss of genetic material on the short arm of chromosome 5 shows these
typical features - babies and children tend to be small, tend to grow slowly,
and have some kind of learning disability. They also have a strange high-
pitched cry as babies, which often sounds like a cat - hence the name (Cat
Cry). Cri du chat children may also have other disorders (though not all
do) e.g. heart defects, seizures, reflux. If there are no major defects
or immunity problems, that person's life span is expected to be normal.
Does the size of the deletion relate to the severity of the condition?
There are conflicting reports about this, but according to Joan's research
the answer is no. Don't forget there are another 45 chromosomes also involved
with brain development! However, if the person only has the very tip missing
they seem to be completely normal, and if they only have a deletion at
15.3 their symptoms are pretty mild. The crucial building block for Cri
du Chat syndrome seems to be somewhere in 15.2
Why is it important to do research?
Research helps us to get a more accurate description of the features
of Cri du Chat syndrome, so that babies can be diagnosed as early a possible
(so that early intervention problems can be started quickly). A more accurate
prognosis may be given to the parents. Growth and development charts can
be drawn up specifically for Cri du Chat children so that they can be compared
with like. Specific genes and proteins can be identified and their function
determined, which will hopefully help to develop therapies some time in
the future.
Kim Cornish's talk about self-injurous behaviour
Kim has studied children from 66 families so far and is planning to undertake
more research in the future (subject to funding).
She looked at self-injurous behaviour, aggressive/destructive behaviour
against people or objects and stereotypic behaviour which was not goal
directed.
She found the following behaviours occurred:
Behaviour
%
Self biting
47
Pulling own hair
34.8
Scratching self
40.9
Vomiting/regurgitating
57.6
Hitting head or body
54
Hitting others
65.2
Pulling other's hair
65.2
Scratching others
36.4
Pinching others
45.5
Destructive behaviour
40.9
Rocking
71.2
Twirling/twisting and object
40.9
Repetitive vocalisations
39.4
This is only part of the list, and Kim was keen to find out if behaviours
increased with age, decreased with age or remained the same. She found
that none increased with age though some remained pretty constant. However
the following ones decreased with age: hitting own head, biting others,
pulling other's hair, scratching others, vomiting.
A new collaborative study is to be done in the future which will correlate
the genetics, behaviour, cognitive development (intelligence) and language
acquisition.
Margaret Collins's talk about TNI (targeted nutritional intervention)
Margaret has been looking at TNI in Cri du chat Syndrome, Downs
syndrome and the autistic spectrum. Her own son has Downs syndrome. The
aim of TNI is to correct or ameliorate metabolic anomalies due to the over
expression of genes or abnormalities in gene products. Before intervention
can begin though, there is a need for assessment:
Anthropometric assessment (weighing and measuring)
Dietary assessment
Behavioural assessment
Behavioural assessment
Laboratory assessment
In the 1970's and 1980's it was hoped that big doses of vitamins and
minerals would boost intelligence in Downs syndrome, but studies showed
this not to be true, the control (placebo) group improved too! Also there
were concerns about side effects, just because a little of something is
good for you, it doesn't mean that alot of it will be also. TNI is widely
used amongst Downs families, as many parents report improvements, but there
is no scientific evidence and further research is needed, so clinicians
do not recommend it's use. even less research has been done on Cri du Chat
children. Research is therefor being proposed which will link genetic,
psychological and nutritional information, which may provide a rationale
for nutritional or other treatment interventions. the purpose of Targeted
Nutritional Intervention is to prevent damage and maintain health in an
individual.