Nusinersen for adults with spinal muscular atrophy

ARSLAN D., İNAN B., KILINÇ M., Bekircan-Kurt C. E., ERDEM ÖZDAMAR S., Tan E.

Neurological Sciences, cilt.44, sa.7, ss.2393-2400, 2023 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 44 Sayı: 7
  • Basım Tarihi: 2023
  • Doi Numarası: 10.1007/s10072-023-06698-9
  • Dergi Adı: Neurological Sciences
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, BIOSIS, CINAHL, EMBASE, Index Islamicus, MEDLINE, Psycinfo
  • Sayfa Sayıları: ss.2393-2400
  • Anahtar Kelimeler: Nusinersen, Adults, Spinal muscular atrophy, Hammersmith Functional Motor Scale Expanded (HFMSE)
  • Hacettepe Üniversitesi Adresli: Evet

Özet

Introduction: Nusinersen was effective in improvingmotor function and survival in infantile and childhood-onset spinalmuscular atrophy (SMA), and the value ofreal-world experiences in adult SMA patients increase gradually.Here, we present our clinical experience in adult SMA patientstreated with nusinersen according to CHERISH study. Material and methods: Thirty-two SMA patients treated withnusinersen were included in the study. Results: Median age atnusinersen initiation was 33.5 (20.0–60.0) years and 23 of SMApatients were male. Six (18.8%) patients had SMA type 2, and 26(81.2%) had SMA type 3. Median follow-up period of patients undernusinersen treatment was 17 months (9–21). Twenty-three patientsimproved by at least 3 HammersmithFunctional Motor Scale Expanded (HFMSE)points after loading doses. There was significant HFMSE scoreincrease in type 3 patients at each time point, whereas type 2patients seem to benefit from nusinersen loading doses, subsequentlystayed stable. Motor improvement was positively correlated withbaseline HFMSE scores in patients whose baseline HFMSE scores were≤47. There was a correlation between the changes in AmyotrophicLateral Sclerosis Functional Rating Scale Revised(ALSFRS-R) score and HFMSE scores. Ambulatory patients who could notshow clinically meaningful increase in HFMSE scores improved at least30 m by 6-min walktest (6MWT). Conclusion: Overall, 78% of patients have respondedto treatment according to HFMSE or 6MWT. ALSFRS-R and 6MWT may bealternative tools to monitor nusinersen effect.