Amico Silvia, Scoscina Dalila, Milazzo Ivan, Martiniani Monia, Meco Leonard, Siliquini Sabrina, Specchia Nicola, Gigante Antonio P
Department of Clinical and Molecular Sciences, Polytechnic University of Marche, Ancona, Italy -
Department of Clinical and Molecular Sciences, Polytechnic University of Marche, Ancona, Italy.
Minerva Pediatr (Torino). 2023 May 8. doi: 10.23736/S2724-5276.23.07198-7.
Nusinersen, the recently approved medical therapy in the treatment of spinal muscular atrophy (SMA), has revolutionized the natural history of this disease. Until now, surgical treatment of scoliosis in SMA patients was an exclusion criterion for drug therapy. In fact, the bone graft positioned posteriorly during surgery, in order to obtain a solid fusion, prevented the lumbar puncture necessary for the intrathecal administration of the drug. The aim is to describe a surgical technique that allows for safe and easy intrathecal administration of nusinersen.
We present a single-center, single-surgeon case series descriptive study. From 2019 to 2021 seven consecutive patients affected by genetically confirmed SMA suitable for treatment with nusinersen and suffered from neuromuscular scoliosis needing posterior spinal fusion surgery were included in the present study. During posterior spinal fusion surgery a L3-L4 or L2-L3 laminectomy was performed to provide safer access to intrathecal injection. The drainage scar was used as a skin landmark so as to facilitate future procedures.
The median of operative time was 250 min (range: 200-370 min). The median correction rate was 57% (range: 43.5-68). The median of intraoperative blood loss was 650 mL (range 320-940 mL). The median value of the correction loss at the last follow-up was 10% (range: 4.5-15%).
The surgical procedure allowed all patients to receive nusinersen therapy without complications. The procedure described is simple and effective in providing safe intrathecal access to make these patients suitable for undertaking or continuing the protocol of treatment with nusinersen.
最近获批用于治疗脊髓性肌萎缩症（SMA）的药物诺西那生钠彻底改变了这种疾病的自然病程。到目前为止，SMA患者脊柱侧弯的手术治疗是药物治疗的排除标准。事实上，手术过程中为实现牢固融合而在后方放置的骨移植材料阻碍了鞘内注射药物所需的腰椎穿刺。目的是描述一种能实现诺西那生钠安全、简便鞘内给药的手术技术。
我们呈现一项单中心、单术者的病例系列描述性研究。2019年至2021年，本研究纳入了7例经基因确诊、适合用诺西那生钠治疗且患有神经肌肉型脊柱侧弯需要后路脊柱融合手术的连续患者。在后路脊柱融合手术期间，进行L3 - L4或L2 - L3椎板切除术以提供更安全的鞘内注射途径。引流瘢痕用作皮肤标志以便于后续操作。
手术时间中位数为250分钟（范围：200 - 370分钟）。矫正率中位数为57%（范围：43.5 - 68）。术中失血量中位数为650毫升（范围320 - 940毫升）。最后一次随访时矫正丢失的中位数为10%（范围：4.5 - 15%）。
该手术方法使所有患者能够接受诺西那生钠治疗且无并发症。所描述的手术操作简单有效，能提供安全的鞘内给药途径，使这些患者适合接受或继续诺西那生钠治疗方案。
