Bascou Nicholas A, Marcos Maria C, Beltran Quintero Maria L, Roosen-Marcos Mercedes C, Cladis Franklyn P, Poe Michele D, Escolar Maria L
Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
Department of Anesthesia, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
Paediatr Anaesth. 2019 Oct;29(10):1053-1059. doi: 10.1111/pan.13714. Epub 2019 Sep 4.
Krabbe disease and metachromatic leukodystrophy are leukodystrophies characterized by neurologic degeneration and early death. Patients often require general anesthesia for diagnostic and therapeutic interventions.
A retrospective review of medical records was conducted for patients with Krabbe disease and metachromatic leukodystrophy receiving general anesthesia at a large children's hospital between 2012 and 2017. Patient complications and American Society of Anesthesiologists Physical Status were recorded for all procedures. The Neurodevelopment in Rare Disorders classification system was created to categorize the severity of the patient's disease progression based on clinical markers. Descriptive and inferential statistics were used to compare: (a) complication rate of affected patients vs the general hospital population; (b) the accuracy of the novel Neurodevelopment in Rare Disorders classification system vs American Society of Anesthesiologists Physical Status regarding the assessment of complication risk; (c) complication rate in patients with hematopoietic stem cell transplantation vs those without transplantation; (d) complication rate in immunosuppressed patients vs nonimmunosuppressed patients; and (e) complication rate of the three most commonly performed procedures.
A total of 96 patients underwent 287 procedures. Of these, 11 cases had complications, yielding a rate of 3.8%. This is significantly higher than the overall complication rate at our institution of 0.246%. Statistical analysis showed better correlation between the Neurodevelopment in Rare Disorders classification system and complication rate than American Society of Anesthesiologists Physical Status and complication rate. The system also showed better accuracy in differentiating low-risk and high-risk patients. No statistically significant difference in complication rate was found for patients with transplantation vs those without transplantation or for immunosuppressed vs nonimmunosuppressed patients. Of the three most common procedures, central catheter placement/removal exhibited the highest complication rate.
Although the complication rate for patients with Krabbe disease and metachromatic leukodystrophy is higher than the general population, most complications were mild and self-limiting. These results suggest that, in experienced hands, general anesthesia is well tolerated in most children. Findings show that the Neurodevelopment in Rare Disorders classification system is a better indicator for assessing complication risk in patients with Krabbe and metachromatic leukodystrophy than American Society of Anesthesiologists Physical Status.
克拉伯病和异染性脑白质营养不良是两种以神经退行性变和早亡为特征的脑白质营养不良症。患者在进行诊断和治疗干预时通常需要全身麻醉。
对2012年至2017年期间在一家大型儿童医院接受全身麻醉的克拉伯病和异染性脑白质营养不良患者的病历进行回顾性研究。记录所有手术的患者并发症及美国麻醉医师协会身体状况分级。创建罕见病神经发育分类系统，根据临床指标对患者疾病进展的严重程度进行分类。采用描述性和推断性统计进行比较：(a) 患病患者与医院普通人群的并发症发生率；(b) 新型罕见病神经发育分类系统与美国麻醉医师协会身体状况分级在评估并发症风险方面的准确性；(c) 接受造血干细胞移植患者与未接受移植患者的并发症发生率；(d) 免疫抑制患者与非免疫抑制患者的并发症发生率；(e) 三种最常进行的手术的并发症发生率。
共有96例患者接受了287次手术。其中，11例出现并发症，发生率为3.8%。这显著高于我们机构0.246%的总体并发症发生率。统计分析表明，罕见病神经发育分类系统与并发症发生率之间的相关性优于美国麻醉医师协会身体状况分级与并发症发生率之间的相关性。该系统在区分低风险和高风险患者方面也具有更高的准确性。接受移植患者与未接受移植患者或免疫抑制患者与非免疫抑制患者的并发症发生率未发现统计学上的显著差异。在三种最常见的手术中，中心静脉导管置入/拔除的并发症发生率最高。
尽管克拉伯病和异染性脑白质营养不良患者的并发症发生率高于普通人群，但大多数并发症较轻且为自限性。这些结果表明，在经验丰富的医生手中，大多数儿童对全身麻醉耐受性良好。研究结果表明，罕见病神经发育分类系统在评估克拉伯病和异染性脑白质营养不良患者的并发症风险方面比美国麻醉医师协会身体状况分级更好。
