Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen

被引：24

作者：

Wurster, Claudia D. ^{[1
]}

Koch, Jan C. ^{[2
]}

Cordts, Isabell ^{[3
]}

Dreyhaupt, Jens ^{[4
]}

Otto, Markus ^{[1
]}

Uzelac, Zeljko ^{[1
]}

Witzel, Simon ^{[1
]}

Winter, Benedikt ^{[5
]}

Kocak, Tugrul ^{[6
]}

Schocke, Michael ^{[7
]}

Weydt, Patrick ^{[8
]}

Wollinsky, Kurt ^{[9
]}

Ludolph, Albert C. ^{[1
,10
]}

Deschauer, Marcus ^{[3
]}

Lingor, Paul ^{[3
]}

Tumani, Hayrettin ^{[1
,11
]}

Hermann, Andreas ^{[12
,13
]}

Guenther, Rene ^{[14
,15
]}

机构：

[1] Ulm Univ, Dept Neurol, Ulm, Germany

[2] Univ Med Gottingen, Dept Neurol, Gottingen, Germany

[3] Tech Univ Munich, Dept Neurol, Klinikum Rechts Isar, Munich, Germany

[4] Ulm Univ, Inst Epidemiol & Med Biometry, Ulm, Germany

[5] Ulm Univ, Dept Pediat, Ulm, Germany

[6] Ulm Univ, Dept Orthoped Surg, RKU Univ & Rehabil Clin, Ulm, Germany

[7] Ulm Univ, Dept Neuroradiol, RKU Univ & Rehabil Clin, Ulm, Germany

[8] Univ Bonn, Dept Neurodegenerat Disorders & Gerontopsychiat, Bonn, Germany

[9] Ulm Univ, Dept Anesthesiol, RKU Univ & Rehabil Clin, Ulm, Germany

[10] German Ctr Neurodegenerat Dis DZNE Ulm, Ulm, Germany

[11] Specialty Hosp Neurol Dietenbronn, Schwendi, Germany

[12] Univ Rostock, Translat Neurodegenerat Sect Albrecht Kossel, Dept Neurol, Univ Med Ctr Rostock, Rostock, Germany

[13] German Ctr Neurodegenerat Dis DZNE Rostock, Rostock, Germany

[14] Tech Univ Dresden, Dept Neurol, Dresden, Germany

[15] German Ctr Neurodegenerat Dis DZNE Dresden, Dresden, Germany

来源：

FRONTIERS IN NEUROLOGY | 2019年 / 10卷

关键词：

spinal muscular atrophy; motor neuron disease; antisense-oligonucleotide; routine CSF parameters; lumbar puncture; BARRIER DYSFUNCTION; MULTIPLE-SCLEROSIS; PROTEIN; DIAGNOSIS; PHASE-1;

D O I：

10.3389/fneur.2019.01179

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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/mu L in CSF; a singular case of pleocytosis (8/mu L) 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.

引用

页数：10

共 42 条

[1] CEREBROSPINAL-FLUID IN THE DIAGNOSIS OF MULTIPLE-SCLEROSIS - A CONSENSUS REPORT [J].