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Neurochemical markers in CSF of adolescent and adult SMA patients undergoing nusinersen treatment.
Ther Adv Neurol Disord 2019; 12:1756286419846058TA

Abstract

Background

There is limited information on neurochemical markers being used to support and monitor the affection of motoneurons in patients with spinal muscular atrophy (SMA). The objective of this study was to examine neurochemical markers in cerebrospinal fluid (CSF) under treatment with the antisense-oligonucleotide (ASO), nusinersen.

Methods

We measured markers of axonal degeneration [neurofilament light chain (NfL) and phosphorylated neurofilament heavy chain (pNfH)] along with basic CSF parameters in 25 adolescent and adult SMA type 2 and 3 patients at baseline and after four intrathecal injections of nusinersen. Neurochemical markers were compared with controls. In addition, neurochemical markers in SMA patients were related to the Hammersmith Functional Rating Scale Expanded (HFMSE).

Results

No significant difference in neurofilament (Nf) values was observed between SMA and control group, neither at baseline nor after four injections of nusinersen. NfL, protein and quotients of albumin (Qalb) increased slightly in SMA patients after the fourth injection. The slight increase of NfL could be related to the development of mild CSF flow change. No relations were observed between changes in Nf and HFMSE.

Conclusion

We assume that Nf levels in CSF in these patients may result from slow disease progression in this stage of disease, pre-existing loss of motoneurons due to long disease duration besides affection of the LMN only. Therefore, we conclude that Nf levels in CSF do not seem useful as diagnostic and monitoring markers in adolescent and adult SMA type 2 and 3 patients.

Authors+Show Affiliations

Department of Neurology, University of Ulm, Oberer Eselsberg 45, Ulm 89081, Germany.Department of Neurology, Technische Universität Dresden, Dresden, Germany German Center for Neurodegenerative Diseases (DZNE) Dresden, Dresden, Germany.Department of Neurology, Ulm University, Ulm, Germany.Institute of Epidemiology and Medical Biometry, Ulm University, Ulm, Germany.Department of Anesthesiology, Ulm University, Ulm, Germany.Department of Neurology, Ulm University, Ulm, Germany.Department of Neurology, Ulm University, Ulm, Germany.Department of Orthopedic Surgery, Ulm University, Ulm, Germany.Department of Pediatrics, Ulm University, Ulm, Germany.Department of Neurology, University Medicine Göttingen, Göttingen, Germany.Department of Neurology, University Medicine Göttingen, Göttingen, Germany.Department of Neurology, Hannover Medical School, Hannover, Germany.Department of Neurology, Ulm University, Ulm, Germany.Department of Neurology, Technische Universität Dresden, Dresden, Germany.Department of Neurology, Ulm University, Ulm, Germany.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31205491

Citation

Wurster, Claudia D., et al. "Neurochemical Markers in CSF of Adolescent and Adult SMA Patients Undergoing Nusinersen Treatment." Therapeutic Advances in Neurological Disorders, vol. 12, 2019, p. 1756286419846058.
Wurster CD, Günther R, Steinacker P, et al. Neurochemical markers in CSF of adolescent and adult SMA patients undergoing nusinersen treatment. Ther Adv Neurol Disord. 2019;12:1756286419846058.
Wurster, C. D., Günther, R., Steinacker, P., Dreyhaupt, J., Wollinsky, K., Uzelac, Z., ... Otto, M. (2019). Neurochemical markers in CSF of adolescent and adult SMA patients undergoing nusinersen treatment. Therapeutic Advances in Neurological Disorders, 12, p. 1756286419846058. doi:10.1177/1756286419846058.
Wurster CD, et al. Neurochemical Markers in CSF of Adolescent and Adult SMA Patients Undergoing Nusinersen Treatment. Ther Adv Neurol Disord. 2019;12:1756286419846058. PubMed PMID: 31205491.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Neurochemical markers in CSF of adolescent and adult SMA patients undergoing nusinersen treatment. AU - Wurster,Claudia D, AU - Günther,René, AU - Steinacker,Petra, AU - Dreyhaupt,Jens, AU - Wollinsky,Kurt, AU - Uzelac,Zeljko, AU - Witzel,Simon, AU - Kocak,Tugrul, AU - Winter,Benedikt, AU - Koch,Jan C, AU - Lingor,Paul, AU - Petri,Susanne, AU - Ludolph,Albert C, AU - Hermann,Andreas, AU - Otto,Markus, Y1 - 2019/05/10/ PY - 2018/11/28/received PY - 2019/04/01/accepted PY - 2019/6/18/entrez PY - 2019/6/18/pubmed PY - 2019/6/18/medline KW - SMA KW - antisense-oligonucleotide KW - neurofilaments KW - nusinersen SP - 1756286419846058 EP - 1756286419846058 JF - Therapeutic advances in neurological disorders JO - Ther Adv Neurol Disord VL - 12 N2 - Background: There is limited information on neurochemical markers being used to support and monitor the affection of motoneurons in patients with spinal muscular atrophy (SMA). The objective of this study was to examine neurochemical markers in cerebrospinal fluid (CSF) under treatment with the antisense-oligonucleotide (ASO), nusinersen. Methods: We measured markers of axonal degeneration [neurofilament light chain (NfL) and phosphorylated neurofilament heavy chain (pNfH)] along with basic CSF parameters in 25 adolescent and adult SMA type 2 and 3 patients at baseline and after four intrathecal injections of nusinersen. Neurochemical markers were compared with controls. In addition, neurochemical markers in SMA patients were related to the Hammersmith Functional Rating Scale Expanded (HFMSE). Results: No significant difference in neurofilament (Nf) values was observed between SMA and control group, neither at baseline nor after four injections of nusinersen. NfL, protein and quotients of albumin (Qalb) increased slightly in SMA patients after the fourth injection. The slight increase of NfL could be related to the development of mild CSF flow change. No relations were observed between changes in Nf and HFMSE. Conclusion: We assume that Nf levels in CSF in these patients may result from slow disease progression in this stage of disease, pre-existing loss of motoneurons due to long disease duration besides affection of the LMN only. Therefore, we conclude that Nf levels in CSF do not seem useful as diagnostic and monitoring markers in adolescent and adult SMA type 2 and 3 patients. SN - 1756-2856 UR - https://www.unboundmedicine.com/medline/citation/31205491/Neurochemical_markers_in_CSF_of_adolescent_and_adult_SMA_patients_undergoing_nusinersen_treatment_ L2 - http://journals.sagepub.com/doi/full/10.1177/1756286419846058?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub=pubmed DB - PRIME DP - Unbound Medicine ER -