Treatments available for patients with paramyloidosis
  • Tratamentos
Patients with paramyloidosis should be followed by a multidisciplinary team involving a neurologist, a cardiologist, a nephrologist, an ophthalmologist or other specialists according to the involvement of other organs. If the disease has already manifested, it is normal to have a consultation every 4 to 6 months to evaluate the disease.
Liver Transplantation
Since the liver is the primary source of mutated TTR, it has become a well-established therapy to stop the progression of the disease and increase the quality of life if performed early in the evolution of the disease. In December 2010, 1917 transplants related to paramyloidosis had been performed worldwide4-5. In fact, this procedure may delay the course of the symptoms, although recovery of the damage to the body is not to be expected.
That is why transplantation is generally considered primarily for people at the onset of the disease. The results are better in people with the genetic mutation Val30Met and in younger people, and even in those in whom the symptoms are less advanced and that they possess good general physical condition.
Thus, transplantation may not be possible in some people due to the advanced severity of the disease and its general poor physical condition. Although with recognized results, liver transplantation presents limitations because it is an invasive therapy associated with non-negligible perioperative mortality, adverse effects associated with prolonged immunosuppression (anti-rejection drugs), as well as the shortage of donors. Liver transplantation is a relevant surgical intervention that must be performed with the coordination of a specialized center experienced in patients with paramyloidosis. In the case of a liver transplant, the team performing the intervention keeps a close follow-up of the patient. It is also possible to provide psychological support, social services, physiotherapy and advice on daily life.
New PAF-TTR treatments
Other treatments have been developed, based on the different mechanisms of the disease. The European Medicines Agency approved in 2011 a specific drug for the treatment of paramyloidosis in adult patients with stage I (non-supported walking) paramyloidosis in Europe. New treatments for paramyloidosis are currently under development.~
Supportive care
The signs and symptoms of paramyloidosis are varied, and people may need multiple treatments to maintain their daily activities. As the disease progresses, people will need more supportive care to help them manage their symptoms and maximize their autonomy and quality of life. ´
Supportive care may minimize the symptoms associated with polyneuropathy.
Available Treatments
Treatments available for patients with paramyloidosis
Liver Transplantation
Salvador was an always cheerful child ...
My liver transplant