Hi,
Thank you for writing to HCM,
Brief Answer -
Curative treatment , Symptomatic treatment , A multidisciplinary approach
Explained Answer -
I would like to suggest you below things -
1. Curative treatment -
Over the last decades many therapies have been tried in X-ALD.
Treatment with Lorenzo’s oil, ß-interferon, intravenous
immunoglobulins,
immunosuppression with cytostatic drugs, and plasma exchange so far have not been able to arrest or delay the disease progression once neurological symptoms are present.
So far, bone marrow or stem cell
transplantation in boys with mild brain involvement is the only treatment that has been shown to be beneficial.
In men and women with AMN like symptoms however, bone marrow or stem cell transplantation should not be performed, as this aggressive treatment is directed against the
brain inflammation, and not at the slowly progressive loss of function of the nerve cells in the spinal cord and peripheral nerves, which occurs in AMN and female carriers.
2. Symptomatic treatment -
Spasticity can be treated with drugs that reduce the muscle tone, such as baclofen (Kemstro ®), tizanidine (Zanaflex ®) and dantrolene (Dantrium ®). Alternatives for short-term use are benzodiazepines, such as diazepam (Valium ®) or clonazepam (Klonopin ®).
Many carriers experience lower back pain, or pain in the ankles, knees and hips, caused by the increased muscle tone in the legs and consequent abnormal walking.
Sometimes surgery may be considered. Knees and hips can be replaced, bladder surgery may help in coping with incontinence. It should be clear though that the underlying disease is not treated with these procedures, and that things may not get better.
3. A multidisciplinary approach -
Female carriers with neurological symptoms may benefit from a multidisciplinary approach.
The
neurologist can treat some of the symptoms, but a symptomatic carrier should also consult a physical therapist, rehabilitation physician and urologist.
A specialized nurse who knows the ins and outs of X-ALD is invaluable.
If necessary, a
psychologist or
psychiatrist may be consulted, in order to cope with the disease itself, or the feeling of guilt that may overwhelm a carrier once it has been established that one of her children is affected.
Last but not least, the clinical geneticist should not be forgotten, as the mode of inheritance of X-ALD, the availability of prenatal testing, and the need for family screening must be discussed.
Its long enough i know but i have to write this for your define your query in detail.
Hope it will helpful to you.
Thanks & Ragards,
Dr. Gaurav Prajapati