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Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen.
Front Neurol 2019; 10:1179FN

Abstract

Background:

Nusinersen is an antisense-oligonucleotide (ASO) approved for treatment of 5q-spinal muscular atrophy (SMA). Since the drug cannot cross the blood-brain barrier (BBB), it must be administered into the cerebrospinal fluid (CSF) space repeatedly by lumbar puncture. However, little is known whether ASOs have an impact on CSF routine parameters that may yield information on CSF flow and/or intrathecal inflammation. The objective of this study was to examine CSF routine parameters in SMA patients treated with nusinersen.

Methods:

Routine CSF parameters [white cell count, total protein, CSF/serum quotients of albumin (Qalb), lactate, and oligoclonal IgG bands (OCB)] of 60 SMA patients (type 1, 2, and 3, aged 7-60 years) were retrospectively analyzed.

Results:

White cells ranged from 0 to 4/μL in CSF; a singular case of pleocytosis (8/μL) was observed in a patient in parallel with a systemic infection. Total protein and Qalb showed a mild increase from baseline to the following lumbar punctures (except for total protein in CSF at the fourth injection of nusinersen). Lactate levels revealed a stable course. In one patient, positive OCB in CSF were transiently observed. The slight change in total CSF protein and Qalb may be caused by repeated lumbar puncture and/or intrathecal administration of the drug.

Conclusion:

Our data suggest that a regular examination of routine CSF parameters in patients in which intrathecal ASOs are administered is important to obtain information on possible side effects and to gain further insights into intrathecal processes.

Authors+Show Affiliations

Department of Neurology, Ulm University, Ulm, Germany.Department of Neurology, University Medicine Göttingen, Göttingen, Germany.Department of Neurology, Klinikum Rechts der Isar der Technischen Universität München, Munich, Germany.Institute of Epidemiology and Medical Biometry, Ulm University, Ulm, Germany.Department of Neurology, Ulm University, Ulm, Germany.Department of Neurology, Ulm University, Ulm, Germany.Department of Neurology, Ulm University, Ulm, Germany.Department of Pediatrics, Ulm University, Ulm, Germany.Department of Orthopedic Surgery, RKU - University and Rehabilitation Clinics, Ulm University, Ulm, Germany.Department of Neuroradiology, RKU - University and Rehabilitation Clinics, Ulm University, Ulm, Germany.Department for Neurodegenerative Disorders and Gerontopsychiatry, Bonn University, Bonn, Germany.Department of Anesthesiology, RKU - University and Rehabilitation Clinics, Ulm University, Ulm, Germany.Department of Neurology, Ulm University, Ulm, Germany. German Center for Neurodegenerative Diseases (DZNE) Ulm, Ulm, Germany.Department of Neurology, Klinikum Rechts der Isar der Technischen Universität München, Munich, Germany.Department of Neurology, Klinikum Rechts der Isar der Technischen Universität München, Munich, Germany.Department of Neurology, Ulm University, Ulm, Germany. Specialty Hospital of Neurology Dietenbronn, Schwendi, Germany.Translational Neurodegeneration Section "Albrecht-Kossel", Department of Neurology, University Medical Center Rostock, University of Rostock, Rostock, Germany. German Center for Neurodegenerative Diseases (DZNE) Rostock, Rostock, Germany.Department of Neurology, Technische Universität Dresden, Dresden, Germany. German Center for Neurodegenerative Diseases (DZNE) Dresden, Dresden, Germany.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31787927

Citation

Wurster, Claudia D., et al. "Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen." Frontiers in Neurology, vol. 10, 2019, p. 1179.
Wurster CD, Koch JC, Cordts I, et al. Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen. Front Neurol. 2019;10:1179.
Wurster, C. D., Koch, J. C., Cordts, I., Dreyhaupt, J., Otto, M., Uzelac, Z., ... Günther, R. (2019). Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen. Frontiers in Neurology, 10, p. 1179. doi:10.3389/fneur.2019.01179.
Wurster CD, et al. Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen. Front Neurol. 2019;10:1179. PubMed PMID: 31787927.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen. AU - Wurster,Claudia D, AU - Koch,Jan C, AU - Cordts,Isabell, AU - Dreyhaupt,Jens, AU - Otto,Markus, AU - Uzelac,Zeljko, AU - Witzel,Simon, AU - Winter,Benedikt, AU - Kocak,Tugrul, AU - Schocke,Michael, AU - Weydt,Patrick, AU - Wollinsky,Kurt, AU - Ludolph,Albert C, AU - Deschauer,Marcus, AU - Lingor,Paul, AU - Tumani,Hayrettin, AU - Hermann,Andreas, AU - Günther,René, Y1 - 2019/11/07/ PY - 2019/07/02/received PY - 2019/10/22/accepted PY - 2019/12/3/entrez PY - 2019/12/4/pubmed PY - 2019/12/4/medline KW - antisense-oligonucleotide KW - lumbar puncture KW - motor neuron disease KW - routine CSF parameters KW - spinal muscular atrophy SP - 1179 EP - 1179 JF - Frontiers in neurology JO - Front Neurol VL - 10 N2 - Background: Nusinersen is an antisense-oligonucleotide (ASO) approved for treatment of 5q-spinal muscular atrophy (SMA). Since the drug cannot cross the blood-brain barrier (BBB), it must be administered into the cerebrospinal fluid (CSF) space repeatedly by lumbar puncture. However, little is known whether ASOs have an impact on CSF routine parameters that may yield information on CSF flow and/or intrathecal inflammation. The objective of this study was to examine CSF routine parameters in SMA patients treated with nusinersen. Methods: Routine CSF parameters [white cell count, total protein, CSF/serum quotients of albumin (Qalb), lactate, and oligoclonal IgG bands (OCB)] of 60 SMA patients (type 1, 2, and 3, aged 7-60 years) were retrospectively analyzed. Results: White cells ranged from 0 to 4/μL in CSF; a singular case of pleocytosis (8/μL) was observed in a patient in parallel with a systemic infection. Total protein and Qalb showed a mild increase from baseline to the following lumbar punctures (except for total protein in CSF at the fourth injection of nusinersen). Lactate levels revealed a stable course. In one patient, positive OCB in CSF were transiently observed. The slight change in total CSF protein and Qalb may be caused by repeated lumbar puncture and/or intrathecal administration of the drug. Conclusion: Our data suggest that a regular examination of routine CSF parameters in patients in which intrathecal ASOs are administered is important to obtain information on possible side effects and to gain further insights into intrathecal processes. SN - 1664-2295 UR - https://www.unboundmedicine.com/medline/citation/31787927/Routine_Cerebrospinal_Fluid__CSF__Parameters_in_Patients_With_Spinal_Muscular_Atrophy__SMA__Treated_With_Nusinersen_ L2 - https://doi.org/10.3389/fneur.2019.01179 DB - PRIME DP - Unbound Medicine ER -