左旋多巴-卡比多巴肠凝胶治疗晚期帕金森病：LRRK2 和 GBA1 突变的影响。
Levodopa-carbidopa intestinal gel for advanced Parkinson's disease: Impact of LRRK2 and GBA1 mutations.
机构信息
Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel; Movement Disorders Unit, Neurological Institute, Tel-Aviv Medical Center, Tel-Aviv, Israel; Laboratory of Early Markers of Neurodegeneration, Neurological Institute, Tel-Aviv Medical Center, Tel-Aviv, Israel; Sagol School of Neuroscience, Tel-Aviv University, Tel-Aviv, Israel.
Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel; Department of Neurology, Sheba Medical Center, Tel-Hashomer, Israel; The Movement Disorders Institute, Sheba Medical Center, Tel-Hashomer, Israel.
出版信息
Parkinsonism Relat Disord. 2024 Oct;127:107115. doi: 10.1016/j.parkreldis.2024.107115. Epub 2024 Aug 28.
BACKGROUND
Advanced Parkinson's disease (PD) can be treated with Levodopa-Carbidopa Intestinal Gel (LCIG).
OBJECTIVE
To compare descriptive data of LCIG treatment in GBA1-PD and LRRK2-PD.
METHODS
This multicenter retrospective study compared clinical data obtained from electronic medical records of PD patients treated with LCIG. Patients were grouped based on their genetic status.
RESULTS
Fifty-two iPD, 15 LRRK2-PD and 23 GBA1-PD were included in this study. No difference in daily dose of LCIG or levodopa equivalent daily dose were detected. GBA1-PD had significantly shorter disease duration at LCIG initiation (p = 0.01) and experienced more hallucinations (p = 0.03) compared with LRRK2-PD and iPD. LRRK2-PD and iPD had significantly longer duration of LCIG treatment compared with GBA1-PD (p < 0.01).
CONCLUSION
Overall, LCIG treatment was well tolerated in LRRK2-PD and GBA1-PD. GBA1-PD required LCIG earlier in their course of their disease and had higher frequencies of hallucinations during treatment, attesting to a more severe disease course.
背景
晚期帕金森病（PD）可以用左旋多巴-卡比多巴肠凝胶（LCIG）治疗。
目的
比较 GBA1-PD 和 LRRK2-PD 患者 LCIG 治疗的描述性数据。
方法
这项多中心回顾性研究比较了接受 LCIG 治疗的 PD 患者电子病历中的临床数据。根据患者的遗传状况对其进行分组。
结果
本研究纳入了 52 名特发性帕金森病患者、15 名 LRRK2-PD 患者和 23 名 GBA1-PD 患者。LCIG 的每日剂量或左旋多巴等效日剂量无差异。与 LRRK2-PD 和特发性帕金森病相比，GBA1-PD 患者在开始 LCIG 治疗时的疾病持续时间明显更短（p=0.01），且出现幻觉的频率更高（p=0.03）。与 GBA1-PD 相比，LRRK2-PD 和特发性帕金森病的 LCIG 治疗持续时间明显更长（p<0.01）。
结论
总体而言，LRRK2-PD 和 GBA1-PD 患者对 LCIG 治疗的耐受性良好。GBA1-PD 在疾病早期就需要 LCIG 治疗，并且在治疗过程中出现幻觉的频率更高，这表明疾病进程更为严重。
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