Hostname: page-component-cd9895bd7-jn8rn Total loading time: 0 Render date: 2024-12-23T19:37:32.785Z Has data issue: false hasContentIssue false

A.05 An epidemiologic study of SLC52A2-related Riboflavin Transport Deficiency

Published online by Cambridge University Press:  02 June 2017

JK Mah
Affiliation:
(Calgary)
M Menezes
Affiliation:
(Sydney)
K Massey
Affiliation:
(Calgary)
Rights & Permissions [Opens in a new window]

Abstract

Core share and HTML view are not available for this content. However, as you have access to this content, a full PDF is available via the ‘Save PDF’ action button.

Background: Riboflavin transporter deficiency (RTD), formerly known as Brown-Vialetto-van Laere syndrome, is an early-onset neurodegenerative disorder with distinctive phenotypes. RTD is caused by mutations in either the SLC52A2 or SLC52A3 genes that encode riboflavin transporters RFVT-2 and RFVT-3, respectively. Methods: This was a 3-year retrospective case review from the Cure RTD International Registry. Results: 73 individuals (~60% female, 14 deceased) from 56 families had genetically confirmed RTD Type 2, including 30 novel SLC52A2 mutations (24 missense, 2 nonsense, 4 deletion). The mean ages at symptom onset and at diagnosis were 2.4 years (SD 1.5, range 0.25–8, n=63) and 12.0 years (SD 10.2, range 0.75–52, n=56) respectively. Most common presenting symptoms were sensory ataxia (n=43), sensorineural hearing loss (n=22), nystagmus/visual loss secondary to optic atrophy (n=14), upper limb weakness (n=11), and respiratory insufficiency (n=9). Treatment included high dose riboflavin, other supplements, and supportive care; 7 individuals required transfusions for anemia pre-riboflavin treatment and 17 (25%) received a cochlear implant. The minimum prevalence of RTD was estimated to be 1 per million, with >100 new cases each year. Conclusions: This is the largest case series of RTD to date. Early recognition and prompt riboflavin treatment is essential for survival and optimal outcome.

Type
Platform Presentations
Copyright
Copyright © The Canadian Journal of Neurological Sciences Inc. 2017