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      Stability of serial platelet and urine protein measurements in patients receiving nusinersen for spinal muscular atrophy

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          Abstract

          Introduction/Aims

          Patients undergoing nusinersen treatment for spinal muscular atrophy are subject to measurements of platelet count and urine protein before each injection due to concern for platelet depletion and renal dysfunction according to the prescribing information. These tests may be uncomfortable or inconvenient and may cause delays in treatment. However, it is still unclear whether these values have been significantly affected by nusinersen treatment. Our aim in this study was to determine whether these measurements ever reached critical values that necessitated withholding treatment at our center.

          Methods

          Records from 57 patients treated with nusinersen at our institution between 2017 and 2020 were retrospectively analyzed. Laboratory values for platelet count, random urine protein, and total urine protein:creatinine ratio were collected from all patients before each procedure.

          Results

          Mean patient age was 28.9 years (range, 2‐76 years). Mean platelet count was 307 × 10 9/L (range, 96‐755 × 10 9/L; normal lab limits, 150‐450 × 10 9/L), mean random urine protein was 0.164 g/L (range, <0.05‐0.73 g/L), and mean total urine protein:creatinine ratio was 0.885 g per gram creatinine (range, 0.12‐9.71 g per gram creatinine). No laboratory values precluded continuing treatment for any patient.

          Discussion

          Although further study on a larger cohort is warranted for more definitive conclusions, it may not be necessary to measure platelet count and urine protein before each nusinersen treatment, particularly in the maintenance phase.

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          Most cited references10

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          Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

          New England Journal of Medicine, 377(18), 1723-1732
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            Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

            Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 ( SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA).
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              Safety of antisense oligonucleotide and siRNA-based therapeutics.

              Oligonucleotide-based therapy is an active area of drug development designed to treat a variety of gene-specific diseases. Two of the more promising platforms are the antisense oligonucleotides (ASOs) and short interfering RNAs (siRNAs), both of which are often directed against similar targets. In light of recent reports on clinical trials of severe thrombocytopenia with two different ASO drugs and increased peripheral neuropathy with an siRNA drug, we compared and contrasted the specific safety characteristics of these two classes of oligonucleotide therapeutic. The objectives were to assess factors that could contribute to the specific toxicities observed with these two classes of promising drugs, and get a better understanding of the potential mechanism(s) responsible for these rare, but serious, adverse events.
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                Author and article information

                Contributors
                nagar048@umn.edu
                Journal
                Muscle Nerve
                Muscle Nerve
                10.1002/(ISSN)1097-4598
                MUS
                Muscle & Nerve
                John Wiley & Sons, Inc. (Hoboken, USA )
                0148-639X
                1097-4598
                11 May 2022
                July 2022
                : 66
                : 1 ( doiID: 10.1002/mus.v66.1 )
                : 76-79
                Affiliations
                [ 1 ] Department of Radiology University of Minnesota Minneapolis Minnesota
                [ 2 ] Biostatistics Core, Masonic Cancer Center University of Minnesota Minneapolis Minnesota
                Author notes
                [*] [* ] Correspondence

                Eric K. Nagarajan, Department of Radiology, University of Minnesota, MMC 292, 420 Delaware Street SE, Minneapolis, MN 55455.

                Email: nagar048@ 123456umn.edu

                Author information
                https://orcid.org/0000-0002-4135-9875
                Article
                MUS27564
                10.1002/mus.27564
                9321971
                35466424
                f8971a36-8ded-4667-9c64-27b29d487aea
                © 2022 The Authors. Muscle & Nerve published by Wiley Periodicals LLC.

                This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

                History
                : 17 April 2022
                : 13 September 2021
                : 19 April 2022
                Page count
                Figures: 1, Tables: 1, Pages: 4, Words: 2422
                Categories
                Clinical Research Short Report
                Clinical Research Short Reports
                Custom metadata
                2.0
                July 2022
                Converter:WILEY_ML3GV2_TO_JATSPMC version:6.1.7 mode:remove_FC converted:26.07.2022

                Neurosciences
                creatinine,nusinersen,platelet,spinal muscular atrophy,urine protein
                Neurosciences
                creatinine, nusinersen, platelet, spinal muscular atrophy, urine protein

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