Email this article The efficacy and safety of nusinersen within the expanded access program in Russia
- Authors: Artemieva S.B.1, Kuzenkova L.M.2, Ilyina E.S.3, Kursakova Y.A.3, Kolpakchi L.M.3, Sapego E.Y.4, Golenko A.A.5, Popovich S.G.2, Parshin D.V.5, Shidlovskaya O.A.1, Papina Y.O.1, Monakhova A.V.1, Vlodavets D.V.1
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Affiliations:
- Veltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical University, the Ministry of Health of Russia
- National Medical Research Center for Children’s Health, the Ministry of Health of Russia
- Pirogov Russian National Research Medical University, the Ministry of Health of Russia
- Regional Children’s Clinical Hospital No. 1, the Ministry of Health of Russia
- Altai Regional Clinical Center for Maternity and Childhood Protection, the Ministry of Health of Russia
- Issue: Vol 10, No 3 (2020)
- Pages: 35-41
- Section: ORIGINAL REPORTS
- Published: 06.12.2020
- URL: https://nmb.abvpress.ru/jour/article/view/396
- DOI: https://doi.org/10.17650/2222-8721-2020-10-3-35-41
- ID: 396
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Abstract
Introduction. Spinal muscular atrophy is a severe neuromuscular disease characterized by rapid progression of muscle weakness and early death. Pathogenetic therapy with nusinersen can significantly change the course of the disease and enable the patient to acquire new skills. The study of the efficacy and safety of nusinersen therapy in patients with type 1 spinal muscular atrophy should be continued.
The aim of the study was to assess the safety and efficacy of nusinersen therapy in patients with type 1 spinal muscular atrophy for 6 months as part of an expanded access program at federal and regional healthcare facilities.
Materials and methods. Patients with type 1 spinal muscular atrophy received nusinersen therapy under the expanded access program (NCT02865109). Patients were evaluated before starting treatment and 6 months after starting treatment. Overall motor development and motor function was assessed using the Hammersmith Infant Neurological Status Scale Part 2 and the Philadelphia Pediatric Hospital Neonatal Motor Scale.
Results. 41 children aged 6 to 38 months received nusinersen therapy between July 2019 and March 2020. At the time of analysis, all patients were alive and continued treatment. The average improvement on the Hammersmith Hospital Neurological Status Scale was 3.7 points after 6 months of therapy compared to baseline (n = 39, p <0.001). The mean improvement on the Children’s Hospital of Philadelphia Scale for the Diagnosis of Motor Function in Newborns was 9.8 after 6 months of therapy from baseline (n = 30, p <0.001).
Conclusion. The efficacy and safety of nusinersen have been demonstrated in real clinical practice in Russia in the treatment of early-onset spinal muscular atrophy.
About the authors
S. B. Artemieva
Veltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Author for correspondence.
Email: artemievasb@gmail.com
ORCID iD: 0000-0002-8876-7462
2 Taldomskaya St., Moscow 125412 Russian Federation
L. M. Kuzenkova
National Medical Research Center for Children’s Health, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0002-9562-3774
62 Build. 2, Lomonosovsky prospect, Moscow 119296
Russian FederationE. S. Ilyina
Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0002-5496-605X
1 Ostrovityanova St., Moscow 117997 Russian Federation
Yu. A. Kursakova
Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0002-8900-9508
1 Ostrovityanova St., Moscow 117997 Russian Federation
L. M. Kolpakchi
Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0003-4244-2379
1 Ostrovityanova St., Moscow 117997 Russian Federation
E. Yu. Sapego
Regional Children’s Clinical Hospital No. 1, the Ministry of Health of Russia
Email: fake@neicon.ru
32 Seraphim Deryabina St., Yekaterinburg 620149 Russian Federation
A. A. Golenko
Altai Regional Clinical Center for Maternity and Childhood Protection, the Ministry of Health of Russia
Email: fake@neicon.ru
179 Gushchina St., Barnaul 656019 Russian Federation
S. G. Popovich
National Medical Research Center for Children’s Health, the Ministry of Health of Russia
Email: fake@neicon.ru
62 Build. 2, Lomonosovsky prospect, Moscow 119296 Russian Federation
D. V. Parshin
Altai Regional Clinical Center for Maternity and Childhood Protection, the Ministry of Health of Russia
Email: fake@neicon.ru
179 Gushchina St., Barnaul 656019 Russian Federation
O. A. Shidlovskaya
Veltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0003-2017-1651
2 Taldomskaya St., Moscow 125412 Russian Federation
Yu. O. Papina
Veltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0003-3794-6855
2 Taldomskaya St., Moscow 125412 Russian Federation
A. V. Monakhova
Veltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0001-9828-9348
2 Taldomskaya St., Moscow 125412 Russian Federation
D. V. Vlodavets
Veltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical University, the Ministry of Health of Russia
Email: fake@neicon.ru
ORCID iD: 0000-0003-2635-2752
2 Taldomskaya St., Moscow 125412 Russian Federation
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