(RxWiki News) Familial amyloid polyneuropathy is a very rare condition. New research shows that a relatively common medication may slow progression of the disease.
These researchers found that patients taking the medication experienced slowed progression of this nervous system disease.
The authors of this study suggested that the medication may provide a lower cost treatment option for people with the rare condition.
"Discuss treatment options for nervous system disorders with a neurologist."
John Berk, MD, of the Amyloidosis Center in Boston, led this study.
Familial amyloid polyneuropathy (FAP) is a rare disease in which the body's nerves malfunction, causing disability that can be fatal.
Although a liver transplant is the standard treatment for FAP, the availability of organs is limited and the authors of this study argued that alternative treatments are necessary.
To see if diflunisal, a non-steroidal anti-inflammatory drug (NSAID), could treat FAP, these researchers recruited 130 FAP patients in a two-year trial.
NSAIDs are typically used to reduce inflammation and fever. This class of medications are commonly prescribed to treat arthritis, headaches and muscle stiffness.
The patients in this study were assigned to one of two groups. The first group received a daily dose of diflunisal, and the second group received a placebo, or fake medication.
The patients visited their physicians after one, three and 18 months from the beginning of the study. The doctors evaluated their disease progression.
At the end of the study period, the researchers measured disease progression using nerve tests and the Neuropathy Impairment Score.
The researchers also evaluated the patients' quality of life using a questionnaire.
These researchers found that the group taking diflunisal experienced significantly slower disease progression.
The placebo group's change in Neuropathy Impairment Score and nerve tests was three times greater than the group taking diflunisal.
Additionally, the participants taking diflunisal had steady quality of life scores, while participants on a placebo reported decreasing scores.
Although some participants reported adverse events, there was no significant difference in serious side effects between the placebo and diflunisal groups.
The researchers concluded that, for patients with familial amyloid polyneuropathy, the use of diflunisal significantly slowed disease progression and improved quality of life.
The authors of this study suggested that diflunisal could provide a low-cost alternative treatment for patients with this rare disease.
"The results of this (albeit small) study offer a promising and lower-cost treatment option for FAP," said E. Lee Carter, RPh, Clinical Pharmacy Specialist at the Department of Veterans Affairs in Prestonsburg, Kentucky.
"Up to now, liver transplantation and immunotherapy (both expensive options) have been the primary treatment modalities for this disease. Diflunisal is one of the older NSAIDs, is available generically, and the medical community has several decades of experience with this medication, and is generally well-tolerated," Carter told dailyRx News.
"Precautions for diflunisal include possible dosing adjustments in the face of kidney disease, and taking this medication with food to reduce the occurrence of possible gastrointestinal effects of this drug. A few of the more common side-effects of diflunisal include abdominal pain, indigestion, nausea, headache, and dizziness," he said.
This study was published in JAMA on December 24.
The research was funded by grants from the National Institute of Neurological Diseases and Stroke, the US Food and Drug Administration, the Young Family Amyloid Research Fund and the National Center for Advancing Translational Sciences.
Some of the authors have received honoraria or financial support from pharmaceutical companies.