Academic Journal
Randomized trial of l-serine in patients with hereditary sensory and autonomic neuropathy type 1
| العنوان: | Randomized trial of l-serine in patients with hereditary sensory and autonomic neuropathy type 1 |
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| المؤلفون: | Fridman, Vera, Suriyanarayanan, Saranya, Novak, Peter, David, William, Macklin, Eric A., McKenna-Yasek, Diane, Walsh, Kailey, Aziz-Bose, Razina, Oaklander, Anne Louise, Brown, Robert H. Jr., Hornemann, Thorsten, Eichler, Florian |
| المساهمون: | Department of Neurology |
| المصدر: | Neurology ; 92 ; 4 ; e359-e370 |
| سنة النشر: | 2022 |
| المجموعة: | University of Massachusetts, Medical School: eScholarship@UMMS |
| مصطلحات موضوعية: | L-serine, hereditary sensory autonomic neuropathy type I, HSAN1, Amino Acids, Peptides, and Proteins, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nervous System Diseases, Neurology, Therapeutics |
| الوصف: | 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. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| Relation: | Link to Article in PubMed; Neurology. 2019 Jan 22;92(4):e359-e370. doi:10.1212/WNL.0000000000006811. Epub 2019 Jan 9. Link to article on publisher's site; 0028-3878 (Linking); http://hdl.handle.net/20.500.14038/40914; https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=4726&context=oapubs&unstamped=1; https://escholarship.umassmed.edu/oapubs/3714; oapubs/3714 |
| DOI: | 10.1212/WNL.0000000000006811 |
| الاتاحة: | https://doi.org/10.1212/WNL.0000000000006811 https://hdl.handle.net/20.500.14038/40914 https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=4726&context=oapubs&unstamped=1 https://escholarship.umassmed.edu/oapubs/3714 |
| 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. ; http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| رقم الانضمام: | edsbas.72E2AE68 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1212/WNL.0000000000006811 |
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