Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Amyloidosis Center, |
RCV000663430 | SCV000778367 | pathogenic | Amyloidosis, hereditary systemic 1 | 2018-03-16 | no assertion criteria provided | clinical testing | A 37 year old African American man presented with sensorimotor polyneuropathy, and myopathy in his lower extremities. Deposits of variant TTR were detected in the right shin skin using tandem mass spectrometry. A fat aspirate was Congo red positive for amyloid deposits, the patient's brother was also diagnosed with amyloidosis by sural nerve biopsy. Following nine months of diflunisal treatment (250mg/daily), the patient demonstrated progression of sensorimotor polyneuropathy, and also autonomic neuropathy. Diflunisal treatment was stopped due to lack of efficacy. The patient was found to have a six base pair duplication in TTR exon 3 (c.212_217dupAGTCTG) by DNA sequencing of the TTR gene. The patient died from recurrent aspiration pneumonia, debilitating peripheral and autonomic neuropathy. |