Agosto Caterina, Salamon Eleonora, Giacomelli Luca, Papa Simonetta, Benedetti Francesca, Benini Franca
Pediatric Pain and Palliative Care Service, Department of Women's and Children's Health, Padua University Hospital, Padua, Italy.
Polistudium SRL, Milan, Italy.
Front Neurol. 2021 Sep 21;12:704928. doi: 10.3389/fneur.2021.704928. eCollection 2021.
Nusinersen is the first oligonucleotide-based drug that is approved for the treatment of spinal muscular atrophy. In January 2020, the WHO declared COVID-19 a pandemic and nusinersen-provider centers had to postpone planned infusions for some children along with other related interventions. Considering the important contribution that the intrathecal infusions and other support activities could have on the quality of life of spinal muscular atrophy patients and their families, this emergency could have a relevant impact on the course of the pathology. The present work aims to assess the clinical and social issues that arise for spinal muscular atrophy children in care at the referral pediatric palliative care Centre of Padua (Veneto) from a delay in nusinersen infusions, resulting from the contingent COVID-19 restrictions. This evaluation has been carried out in both the short and long term after the first lockdown period and can be considered as a "proxy" of a situation of a possible delay in administration or management of infusions, due to other different causes.
诺西那生钠是首个获批用于治疗脊髓性肌萎缩症的寡核苷酸类药物。2020年1月,世界卫生组织宣布新冠疫情为大流行,提供诺西那生钠的中心不得不推迟一些儿童的计划输注以及其他相关干预措施。考虑到鞘内输注和其他支持活动对脊髓性肌萎缩症患者及其家庭生活质量可能产生的重要贡献,这一紧急情况可能会对疾病进程产生重大影响。本研究旨在评估在帕多瓦(威尼托)转诊儿科姑息治疗中心接受治疗的脊髓性肌萎缩症儿童因新冠疫情临时限制导致诺西那生钠输注延迟而出现的临床和社会问题。该评估在首次封锁期后的短期和长期内均已进行,并且可被视为由于其他不同原因导致输注给药或管理可能延迟情况的一个“代表”。