Ashrafi Mahmoud Reza, Babaee Marzieh, Hashemi Nazari Seyed Saeed, Barzegar Mohammad, Ghazavi Mohammadreza, Beiraghi Toosi Mehran, Nafissi Shahriar, Inaloo Soroor, Zamani Ghaletaki Gholamreza, Fatehi Farzad, Heshmat Ramin, Ghahvechi Akbari Masood, Abdi Alireza, Bakhtiary Hassan, Montazerlotfelahi Hadi, Abbaskhanian Ali, Hosseini Seyed Ahmad, Farshadmoghadam Hossein, Hosseiny Seyyed Mohammad Mahdi, Shariatmadari Fakhreddin, Ziaadini Bentolhoda, Babaei Meisam, Tavasoli Azita, Nikbakht Sedighe, Momen Aliakbar, Khajeh Ali, Aminzadeh Vahid, Mollamohammadi Mohsen, Taghdiri Mohammad Mehdi, Nasehi Mohammad Mehdi, Memarian Sara, Badv Reza Shervin, Heidari Morteza, Jafari Narjes
Department of Pediatrics, Division of Pediatric Neurology, Children's Medical Center, Pediatrics Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran.
Pediatric Cell and Gene Therapy Research Center, Gene, Cell & Tissue Research Institute, Tehran University of Medical Sciences, Tehran, Iran.
J Neuromuscul Dis. 2024 Nov;11(6):1190-1199. doi: 10.1177/22143602241288087. Epub 2024 Dec 20.
Three medications have been approved for spinal muscular atrophy (SMA) treatment. No head-to-head clinical trials have directly compared the efficacy of nusinersen and risdiplam. We compare the efficacy of them in Type 2 and 3 SMA patients, with 6 months of follow-up.
A multicenter cohort study was conducted. Demographic, genetic and clinical findings containing Hammersmith Functional Motor Scale Expanded (HFMSE) and revised upper limb module (RULM) scales were gathered. An increase of at least 3 points in HFMSE and RULM scores was considered a positive response.
73 (58.4%) children received risdiplam, and 52 (41.6%) received nusinersen non-randomly, based on clinical decision. The difference in HFMSE and RULM scores compared to the baseline was significant in both groups (P-value <0.001). However, there was no significant difference between mean difference changes in HFMSE and RULM scores between the two groups. 80.4% of patients in the risdiplam group and 72% in the nusinersen group achieved the 3-point cutoff after 6 months, and there is no significant difference between the two groups (P-Value:0.33).
This study showed that both medications significantly changed the HFSME and RULM after 3 and 6 months of follow-up. However, there was no significant difference between the two drugs according to the HFSME.
三种药物已被批准用于治疗脊髓性肌萎缩症（SMA）。尚无直接比较诺西那生钠和利司扑兰疗效的头对头临床试验。我们比较了它们在2型和3型SMA患者中的疗效，并进行了6个月的随访。
进行了一项多中心队列研究。收集了人口统计学、遗传学和临床数据，包括哈默史密斯功能运动量表扩展版（HFMSE）和修订的上肢模块（RULM）量表。HFMSE和RULM评分至少增加3分被视为阳性反应。
根据临床决策，73名（58.4%）儿童非随机接受了利司扑兰，52名（41.6%）接受了诺西那生钠。两组与基线相比，HFMSE和RULM评分差异均有统计学意义（P值<0.001）。然而，两组间HFMSE和RULM评分的平均差异变化无显著差异。利司扑兰组80.4%的患者和诺西那生钠组72%的患者在6个月后达到了3分的临界值，两组间无显著差异（P值：0.33）。
本研究表明，两种药物在随访3个月和6个月后均能显著改变HFSME和RULM。然而，根据HFSME，两种药物之间无显著差异。
