Reply to Aljabali et al. Comment on “Abbas et al. The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Medicina 2022, 58, 213”
- (1)
- The efficacy of nusinersen in some SMA subgroups such as respiratory insufficiency, bulbar manifestations, gastric feeding tube, severe contractures or severe scoliosis, or medical disability. The exclusion criteria mentioned by Aljabali et al. were also mentioned in Mercuri et al. [5]. On the other hand, Finkel et al. [4] included patients with and without gastrostomy, as mentioned in the Supplementary Materials, and Acsadi et al. [3] reported exploratory efficacy endpoints that involved a change from baseline in ventilator use (including bilevel positive airway pressure, tracheostomy, and endotracheal tube), which means that he included patients with respiratory insufficiency. These examples go against Aljabali et al.’s comment that the clinical trial excluded those patients. Due to the lack of data and the few RCTs published so far, more data are still needed before delivering conclusion about those patients subgroup. We also mentioned that in the Limitations section “A further limitation of the present meta-analysis is the small number of available RCTs with outcomes that were suitable for pooling through meta-analysis; thus, more RCTs are needed before making clinical recommendations based on these studies”.
- (2)
- Adult SMA patients. As indicated in our manuscript title, we only included infantile patients, and we did not comment on adult patients throughout our manuscript.
- (3)
- The long-term efficacy and safety of nusinersen. We also reported that in the Limitations section, and we conducted our meta-analysis based on available RCTs.
Author Contributions
Funding
Data Availability Statement
Conflicts of Interest
References
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Abdelazeem, B.; Abbas, K.S.; Brašić, J.R. Reply to Aljabali et al. Comment on “Abbas et al. The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Medicina 2022, 58, 213”. Medicina 2022, 58, 793. https://doi.org/10.3390/medicina58060793
Abdelazeem B, Abbas KS, Brašić JR. Reply to Aljabali et al. Comment on “Abbas et al. The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Medicina 2022, 58, 213”. Medicina. 2022; 58(6):793. https://doi.org/10.3390/medicina58060793
Chicago/Turabian StyleAbdelazeem, Basel, Kirellos Said Abbas, and James Robert Brašić. 2022. "Reply to Aljabali et al. Comment on “Abbas et al. The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Medicina 2022, 58, 213”" Medicina 58, no. 6: 793. https://doi.org/10.3390/medicina58060793