Randomized trial of l-serine in patients with hereditary sensory and autonomic neuropathy type 1
dc.contributor.author | Fridman, Vera | |
dc.contributor.author | Suriyanarayanan, Saranya | |
dc.contributor.author | Novak, Peter | |
dc.contributor.author | David, William | |
dc.contributor.author | Macklin, Eric A. | |
dc.contributor.author | McKenna-Yasek, Diane | |
dc.contributor.author | Walsh, Kailey | |
dc.contributor.author | Aziz-Bose, Razina | |
dc.contributor.author | Oaklander, Anne Louise | |
dc.contributor.author | Brown, Robert H. Jr. | |
dc.contributor.author | Hornemann, Thorsten | |
dc.contributor.author | Eichler, Florian | |
dc.date | 2022-08-11T08:09:52.000 | |
dc.date.accessioned | 2022-08-23T16:46:36Z | |
dc.date.available | 2022-08-23T16:46:36Z | |
dc.date.issued | 2019-01-22 | |
dc.date.submitted | 2019-02-28 | |
dc.identifier.citation | <p>Neurology. 2019 Jan 22;92(4):e359-e370. doi: 10.1212/WNL.0000000000006811. Epub 2019 Jan 9. <a href="https://doi.org/10.1212/WNL.0000000000006811">Link to article on publisher's site</a></p> | |
dc.identifier.issn | 0028-3878 (Linking) | |
dc.identifier.doi | 10.1212/WNL.0000000000006811 | |
dc.identifier.pmid | 30626650 | |
dc.identifier.uri | http://hdl.handle.net/20.500.14038/40914 | |
dc.description.abstract | OBJECTIVE: To evaluate the safety and efficacy of l-serine in humans with hereditary sensory autonomic neuropathy type I (HSAN1). METHODS: In this randomized, placebo-controlled, parallel-group trial with open-label extension, patients aged 18-70 years with symptomatic HSAN1 were randomized to l-serine (400 mg/kg/day) or placebo for 1 year. All participants received l-serine during the second year. The primary outcome measure was the Charcot-Marie-Tooth Neuropathy Score version 2 (CMTNS). Secondary outcomes included plasma sphingolipid levels, epidermal nerve fiber density, electrophysiologic measurements, patient-reported measures, and adverse events. RESULTS: Between August 2013 and April 2014, we enrolled and randomized 18 participants, 16 of whom completed the study. After 1 year, the l-serine group experienced improvement in CMTNS relative to the placebo group (-1.5 units, 95% CI -2.8 to -0.1, p = 0.03), with evidence of continued improvement in the second year of treatment (-0.77, 95% CI -1.67 to 0.13, p = 0.09). Concomitantly, deoxysphinganine levels dropped in l-serine-treated but not placebo-treated participants (59% decrease vs 11% increase; p < 0.001). There were no serious adverse effects related to l-serine. CONCLUSION: High-dose oral l-serine supplementation appears safe in patients with HSAN1 and is potentially effective at slowing disease progression. CLINICALTRIALSGOV IDENTIFIER: NCT01733407. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that high-dose oral l-serine supplementation significantly slows disease progression in patients with HSAN1. | |
dc.language.iso | en_US | |
dc.relation | <p><a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=30626650&dopt=Abstract">Link to Article in PubMed</a></p> | |
dc.rights | Copyright © 2019 The Author(s). This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal. | |
dc.rights.uri | http://creativecommons.org/licenses/by-nc-nd/4.0/ | |
dc.subject | L-serine | |
dc.subject | hereditary sensory autonomic neuropathy type I | |
dc.subject | HSAN1 | |
dc.subject | Amino Acids, Peptides, and Proteins | |
dc.subject | Congenital, Hereditary, and Neonatal Diseases and Abnormalities | |
dc.subject | Nervous System Diseases | |
dc.subject | Neurology | |
dc.subject | Therapeutics | |
dc.title | Randomized trial of l-serine in patients with hereditary sensory and autonomic neuropathy type 1 | |
dc.type | Journal Article | |
dc.source.journaltitle | Neurology | |
dc.source.volume | 92 | |
dc.source.issue | 4 | |
dc.identifier.legacyfulltext | https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=4726&context=oapubs&unstamped=1 | |
dc.identifier.legacycoverpage | https://escholarship.umassmed.edu/oapubs/3714 | |
dc.identifier.contextkey | 13921316 | |
refterms.dateFOA | 2022-08-23T16:46:36Z | |
html.description.abstract | <p>OBJECTIVE: To evaluate the safety and efficacy of l-serine in humans with hereditary sensory autonomic neuropathy type I (HSAN1).</p> <p>METHODS: In this randomized, placebo-controlled, parallel-group trial with open-label extension, patients aged 18-70 years with symptomatic HSAN1 were randomized to l-serine (400 mg/kg/day) or placebo for 1 year. All participants received l-serine during the second year. The primary outcome measure was the Charcot-Marie-Tooth Neuropathy Score version 2 (CMTNS). Secondary outcomes included plasma sphingolipid levels, epidermal nerve fiber density, electrophysiologic measurements, patient-reported measures, and adverse events.</p> <p>RESULTS: Between August 2013 and April 2014, we enrolled and randomized 18 participants, 16 of whom completed the study. After 1 year, the l-serine group experienced improvement in CMTNS relative to the placebo group (-1.5 units, 95% CI -2.8 to -0.1, p = 0.03), with evidence of continued improvement in the second year of treatment (-0.77, 95% CI -1.67 to 0.13, p = 0.09). Concomitantly, deoxysphinganine levels dropped in l-serine-treated but not placebo-treated participants (59% decrease vs 11% increase; p < 0.001). There were no serious adverse effects related to l-serine.</p> <p>CONCLUSION: High-dose oral l-serine supplementation appears safe in patients with HSAN1 and is potentially effective at slowing disease progression.</p> <p>CLINICALTRIALSGOV IDENTIFIER: NCT01733407.</p> <p>CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that high-dose oral l-serine supplementation significantly slows disease progression in patients with HSAN1.</p> | |
dc.identifier.submissionpath | oapubs/3714 | |
dc.contributor.department | Department of Neurology | |
dc.source.pages | e359-e370 |