1Division of Pediatric Neurosurgery, Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois.
2Department of Neurosurgery, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
J Neurosurg Pediatr. 2023 Dec 8;33(2):179-184. doi: 10.3171/2023.11.PEDS23473. Print 2024 Feb 1.
Intrathecal baclofen (ITB) is an effective treatment for refractory hypertonia in children. ITB has long been effective for the treatment of spasticity, and indications have naturally evolved to include dystonia and mixed pediatric movement disorders (PMDs). The established uses for ITB trials are insurance prerequisite, mixed tone, and family request. Despite agreement for ITB therapy by a multidisciplinary group of subspecialists in a complex PMD program, insurance companies often require an ITB trial be performed. A longitudinal cohort was identified to determine the safety and efficacy of ITB trials and to determine the utility of test dosing in this population.
Retrospective data analysis was performed for patients with hypertonia who underwent ITB bolus trials at the authors' institution between 2021 and 2023. Nonmodifiable risk factors and clinical variables were collected.
Thirty-one patients (11 female) underwent 32 ITB trials. Of these patients, 67.7% had a diagnosis of mixed hypertonia, 32.3% pure spasticity, and 9.1% secondary dystonia. The mean age at test dose was 12.8 years, and 58.1% of patients were born premature. The mode Gross Motor Function Classification System score was 5. The mean difference in Barry-Albright Dystonia Scale (BADS) scores was -7.33 points (p = 0.01) at 2.5 hours postoperatively. The mean difference in upper-extremity modified Ashworth Scale (mAS) scores was -5.36 points (p = 0.003), and that for lower-extremity mAS scores was -6.61 (p < 0.001). In total, 21.9% of patients developed a post-dural puncture headache. Conversion to a permanent baclofen pump was performed in 22/32 (68.8%) patients. Of those who did not pursue pump placement, 1 patient had high surgical risk, 1 had an ineffective response, 1 had a bad reaction to the test dose and cited both regression and increased discomfort, and 2 declined despite an effective trial owing to family preferences.
ITB trials require hospitalization in some form and carry risks of procedural complications. The decision to pursue a trial should be made on a case-by-case basis by clinicians and should not be determined by insurance companies. The complication rate of ITB trials is high, and a test dose is unnecessary in this fragile population.
鞘内注射巴氯芬（ITB）是治疗儿童难治性张力亢进的有效方法。ITB 长期以来一直是治疗痉挛的有效方法，适应证自然已扩展至包括肌张力障碍和混合性儿科运动障碍（PMD）。ITB 试验的既定用途是保险先决条件、混合音调和家庭要求。尽管在复杂的 PMD 计划中，由一组多学科亚专科医生组成的小组同意进行 ITB 治疗，但保险公司通常仍要求进行 ITB 试验。本研究确定了一个纵向队列，以确定 ITB 试验的安全性和有效性，并确定在该人群中进行测试剂量的效用。
对 2021 年至 2023 年在作者所在机构接受 ITB 推注试验的张力亢进患者进行回顾性数据分析。收集不可变风险因素和临床变量。
31 名患者（11 名女性）接受了 32 次 ITB 试验。这些患者中，67.7%的患者患有混合性张力亢进，32.3%的患者患有单纯性痉挛，9.1%的患者患有继发性肌张力障碍。测试剂量时的平均年龄为 12.8 岁，58.1%的患者早产。模式粗大运动功能分类系统评分均为 5 分。术后 2.5 小时巴瑞-阿尔布赖特肌张力障碍量表（BADS）评分平均差值为-7.33 分（p=0.01）。上肢改良 Ashworth 量表（mAS）评分平均差值为-5.36 分（p=0.003），下肢 mAS 评分平均差值为-6.61 分（p<0.001）。总的来说，21.9%的患者出现了硬膜穿刺后头痛。22/32（68.8%）患者转为永久性巴氯芬泵。在未进行泵植入的患者中，1 例患者手术风险高，1 例患者反应无效，1 例患者对测试剂量反应不佳，同时出现退行和不适增加，2 例患者尽管试验有效，但因家庭偏好而拒绝。
ITB 试验需要以某种形式住院，并且存在程序并发症的风险。是否进行试验应由临床医生根据具体情况决定，而不应由保险公司决定。ITB 试验的并发症发生率较高，在这个脆弱的人群中，测试剂量是不必要的。
