Page 85 - 《中国药房》2024年15期

[31] Scottish Medicines Consortium. Onasemnogene abepar- safety results from the phase 2 NURTURE study[J]. Neu‐
vovec for the treatment of patients with 5q SMA with a romuscul Disord，2019，29（11）：842-856.
biallelic mutation in the SMN1 gene and a clinical diagno‐ [41] CASTRO D，FINKEL R S，FARRAR M A，et al. Nu-
sis of SMA Type 1，or patients with 5q SMA with a bial‐ sinersen in infantile-onset spinal muscular atrophy：results
lelic mutation in the SMN1 gene and up to 3 copies of the from longer-term treatment from the open-label shine ex‐
SMN2 gene[EB/OL]. （2021-03-08）[2023-09-27]. https：// tension study：1640[EB/OL]. （2021-04-05）[2023-09-27].
database.inahta.org/article/19582. https：//clinicaltrials.gov/study/NCT02865109.
[32] CADTH. Reimbursement review. Risdiplam （Evrysdi ） [42] DAY J W，FINKEL R S，CHIRIBOGA C A，et al.
®
[EB/OL]. （2021-08-26） [2023-09-27]. https：//www.cadth. Onasemnogene abeparvovec gene therapy for sympto-
ca/risdiplam. matic infantile-onset spinal muscular atrophy in patients
[33] National Centre for Pharmacoeconomics. Risdiplam with two copies of SMN2 （STR1VE）：an open-label，
（Evrysdi ）[EB/OL].（2022-03-15）[2023-09-27]. https：// single-arm，multicentre，phase Ⅲ trial[J]. Lancet Neurol，
®
www.ncpe.ie/risdiplam-evrysdi-hta-id-21006/. 2021，20（4）：284-293.
[34] HU J H，SUTHERLAND C S，RIBERO V A，et al. Cost- [43] MERCURI E，MUNTONI F，BARANELLO G，et al.
effectiveness of risdiplam versus nusinersen for treating Onasemnogene abeparvovec gene therapy for sympto-
patients with spinal muscular atrophy type 1 in China matic infantile-onset spinal muscular atrophy type 1
[EB/OL]. （2022-12-03）[2023-09-27]. https：//www.ispor. （STR1VE-EU）：an open-label，single-arm，multicentre，
org/docs/default-source/euro2021/113922jiahaohuhandout- phase 3 trial[J]. Lancet Neurol，2021，20（10）：832-841.
pdf.pdf？sfvrsn=54432135_0. [44] MENDELL J R，AL-ZAIDY S A，LEHMAN K J，et al.
[35] KOLBIN A S，KURYLEV A A，MISHINOVA S A，et al. Five-year extension results of the phase 1 START trial of
The health-economic evaluation of risdiplam in patients onasemnogene abeparvovec in spinal muscular atrophy[J].
with spinal muscular atrophy[EB/OL]. （2022-10-23） JAMA Neurol，2021，78（7）：834-841.
[2023-09-27]. https：//www. ispor. org/docs/default-source/ [45] MASSON R， MAZURKIEWICZ-BEŁDZIŃSKA M，
euro2021/hearisdiplam-pdf.pdf？sfvrsn=c8ecea7c_0. ROSE K，et al. Safety and efficacy of risdiplam in patients
[36] CADTH. Reimbursement review. Nusinersen （Spinraza ） with type 1 spinal muscular atrophy （FIREFISH part 2）：
®
[EB/OL]. （2022-08-11） [2023-09-27]. https：//www.cadth. secondary analyses from an open-label trial[J]. Lancet
ca/nusinersen-1. Neurol，2022，21（12）：1110-1119.
[37] FINKEL R S，MERCURI E，DARRAS B T，et al. Nusi- [46] MERCURI E，DECONINCK N，MAZZONE E S，et al.
nersen versus sham control in infantile-onset spinal mus‐ Safety and efficacy of once-daily risdiplam in type 2 and
cular atrophy[J]. N Engl J Med，2017，377（18）：1723- non-ambulant type 3 spinal muscular atrophy （SUNFISH
1732. part 2）：a phase 3，double-blind，randomised，placebo-
[38] MERCURI E，DARRAS B T，CHIRIBOGA C A，et al. controlled trial[J]. Lancet Neurol，2022，21（1）：42-52.
Nusinersen versus sham control in later-onset spinal [47] CHIRIBOGA C A，BRUNO C，DUONG T，et al. Ris‐
muscular atrophy[J]. N Engl J Med，2018，378（7）： diplam in patients previously treated with other therapies
625-635. for spinal muscular atrophy：an interim analysis from the
[39] DARRAS B T，CHIRIBOGA C A，IANNACCONE S T， JEWELFISH study[J]. Neurol Ther，2023，12 （2） ：
et al. Nusinersen in later-onset spinal muscular atrophy： 543-557.
long-term results from the phase 1/2 studies[J]. Neuro- [48] KHAN K A，PETROU S，RIVERO-ARIAS O，et al. Map‐
logy，2019，92（21）：e2492-e2506. ping EQ-5D utility scores from the PedsQL generic core
TM
[40] DE VIVO D C，BERTINI E，SWOBODA K J，et al. scales[J]. Pharmacoeconomics，2014，32（7）：693-706.
Nusinersen initiated in infants during the presymptomatic （收稿日期：2023-12-12 修回日期：2024-06-05）
stage of spinal muscular atrophy：interim efficacy and （编辑：胡晓霖）

中国药房 2024年第35卷第15期 China Pharmacy 2024 Vol. 35 No. 15 · 1875 ·