Patient care in children with white matter disorders
The first and most important question is whether there is a causal treatment for a particular disease. The answer depends on the cause of the disease, but chances for cure are usually not very good.
Early in the course of the disease, one may consider bone marrow
transplantation in some lysosomal disorders and in the cerebral form of
X-linked adrenoleukodystrophy. There is a chance of a beneficial influence
on the course of the disease or even partial or complete reversal of
clinical signs. The procedure itself carries considerable risks in terms of
morbidity and mortality. These risks are lower in case of bone marrow
donation by an HLA-identical sibling. Newer techniques, including stem cell
transplantation are promising.
In mitochondrial disease, many drugs have been advocated with anecdotal beneficial effects, which could not be confirmed in systematic studies.
In generalized peroxisomal disorders, there is evidence of a beneficial effect of a drug called DHA on some functions.
In amino acidopathies and organic acidurias, dietary treatment together
with specific supplementation is often successful in preventing illness and
guaranteeing good life quality. Similar successful treatment is available
for galactosemia and Wilson's disease.
In infectious white matter disease, specific antimicrobial treatment may be installed with variable success. Congenital CMV which is still active at birth may benefit from antiviral treatment, but in most cases CMV infection is of earlier date and treatment will not prevent the brain damage.
In inflammatory white matter disease, the acute episodes can be treated with high doses of intravenous corticosteroids. For multiple sclerosis, many other drug are available which modify the course of the disease.
Preventive measures are most important in hypoxic-ischemic encephalopathies. Results of neuroprotective measure after the incident/incidents has/have occurred, are disappointing.
Supportive care is very important in white matter disorders. First of all, physical therapy and special education are almost always necessary. Often, more intensive rehabilitative treatment is required. Psychological support is essential.