Supplementary Material for: Patient and Caregiver Reported Outcome Measures after Single-Level Selective Dorsal Rhizotomy in Pediatric and Young Adult Patients with Spastic Cerebral Palsy

Introduction: The aim of this cohort study is to assess the outcome of single-level selective dorsal rhizotomy (SDR) in children and young adults with spastic cerebral palsy (CP) treated at our institution, focusing on patient-reported outcome measures (PROMs) and quality of life (QoL) of patients and their caregivers. Methods: We included consecutive patients undergoing SDR from 2018 to 2020 at our institution. Subjective outcome was measured through PROMs, while functional outcome was measured through baseline characteristics, operative outcome, as well as short- and long-term follow-up. Furthermore, the effect of age at the time of surgery on patient/caregiver satisfaction was analyzed. Results: Seven patients (3 female, 43%) with a median age at surgery of 11.9 years (IQR 8.7-15.5) were included. All patients had a GMFCS score of at least IV before surgery. Five surgeries were palliative and two non-palliative. Based on PROMs, SDR showed very good QoL, and health-related outcome measures for both palliative and non-palliative patients. Patients/caregivers satisfaction was higher for the early subgroup (age ≤ 11) than the late subgroup (age > 11). Functional outcome showed reduced spasticity in both groups. Blood transfusions were never needed, while no CSF leak, infection, or permanent morbidity was seen. Conclusion: Based on PROMs, SDR leads to high satisfaction and improved QoL, especially if done at an early age. Further studies with larger cohorts are necessary to underline and confirm our observations.

引言：本队列研究旨在评估本医疗机构收治的痉挛性脑性瘫痪（spastic cerebral palsy，CP）儿童及青年患者接受单节段选择性脊神经后根切断术（single-level selective dorsal rhizotomy，SDR）后的预后情况，重点关注患者及其照护者的患者报告结局指标（patient-reported outcome measures，PROMs）与生活质量（quality of life，QoL）。
研究方法：纳入本机构2018年至2020年期间接受SDR治疗的连续性入组患者。主观结局通过PROMs进行评估，功能结局则通过基线特征、手术结局以及短期与长期随访结果进行评价。此外，本研究还分析了手术时的年龄对患者/照护者满意度的影响。
研究结果：本研究共纳入7例患者（其中女性3例，占比43%），患者手术时的中位年龄为11.9岁（四分位数间距8.7~15.5岁）。所有患者术前粗大运动功能分级系统（Gross Motor Function Classification System，GMFCS）评分均不低于IV级。其中5例手术为姑息性手术，2例为非姑息性手术。基于PROMs评估结果，无论姑息性还是非姑息性患者，SDR术后均获得了良好的QoL及健康相关结局指标。早期亚组（年龄≤11岁）的患者/照护者满意度高于晚期亚组（年龄>11岁）。两组患者的功能结局均显示痉挛症状得到缓解。本研究未出现输血需求，亦未观察到脑脊液漏（CSF leak）、感染或永久性并发症发生。
结论：基于PROMs评估结果，SDR可提升患者满意度并改善QoL，尤其在患者年龄较小时实施该手术效果更佳。后续需开展更大样本量的队列研究以验证并巩固本研究结果。