{"title":"左旋多巴-卡比多巴肠道凝胶治疗晚期帕金森病：LRRK2和GBA1突变的影响","authors":"Avner Thaler , Saar Anis , Penina Ponger , Tsviya Fay-Karmon , Vered Livneh , Achinoam Faust-Socher , Lior Greenbaum , Johnathan Reiner , Ariela Hilel , Hertzel Shabtai , Roy N. Alcalay , Ruth Djaldetti , Sharon Hassin-Baer , Adi Ezra , Anat Mirelman , Nir Giladi , Tanya Gurevich","doi":"10.1016/j.parkreldis.2024.107115","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><p>Advanced Parkinson's disease (PD) can be treated with Levodopa-Carbidopa Intestinal Gel (LCIG).</p></div><div><h3>Objective</h3><p>To compare descriptive data of LCIG treatment in <em>GBA1-</em>PD and <em>LRRK2-</em>PD.</p></div><div><h3>Methods</h3><p>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.</p></div><div><h3>Results</h3><p>Fifty-two iPD, 15 <em>LRRK2</em>-PD and 23 <em>GBA1</em>-PD were included in this study. No difference in daily dose of LCIG or levodopa equivalent daily dose were detected. <em>GBA1</em>-PD had significantly shorter disease duration at LCIG initiation (p = 0.01) and experienced more hallucinations (p = 0.03) compared with <em>LRRK2</em>-PD and iPD. <em>LRRK2</em>-PD and iPD had significantly longer duration of LCIG treatment compared with <em>GBA1</em>-PD (p < 0.01).</p></div><div><h3>Conclusion</h3><p>Overall, LCIG treatment was well tolerated in <em>LRRK2</em>-PD and <em>GBA1</em>-PD. <em>GBA1</em>-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.</p></div>","PeriodicalId":19970,"journal":{"name":"Parkinsonism & related disorders","volume":"127 ","pages":"Article 107115"},"PeriodicalIF":3.1000,"publicationDate":"2024-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Levodopa-carbidopa intestinal gel for advanced Parkinson's disease: Impact of LRRK2 and GBA1 mutations\",\"authors\":\"Avner Thaler , Saar Anis , Penina Ponger , Tsviya Fay-Karmon , Vered Livneh , Achinoam Faust-Socher , Lior Greenbaum , Johnathan Reiner , Ariela Hilel , Hertzel Shabtai , Roy N. Alcalay , Ruth Djaldetti , Sharon Hassin-Baer , Adi Ezra , Anat Mirelman , Nir Giladi , Tanya Gurevich\",\"doi\":\"10.1016/j.parkreldis.2024.107115\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Background</h3><p>Advanced Parkinson's disease (PD) can be treated with Levodopa-Carbidopa Intestinal Gel (LCIG).</p></div><div><h3>Objective</h3><p>To compare descriptive data of LCIG treatment in <em>GBA1-</em>PD and <em>LRRK2-</em>PD.</p></div><div><h3>Methods</h3><p>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.</p></div><div><h3>Results</h3><p>Fifty-two iPD, 15 <em>LRRK2</em>-PD and 23 <em>GBA1</em>-PD were included in this study. No difference in daily dose of LCIG or levodopa equivalent daily dose were detected. <em>GBA1</em>-PD had significantly shorter disease duration at LCIG initiation (p = 0.01) and experienced more hallucinations (p = 0.03) compared with <em>LRRK2</em>-PD and iPD. <em>LRRK2</em>-PD and iPD had significantly longer duration of LCIG treatment compared with <em>GBA1</em>-PD (p < 0.01).</p></div><div><h3>Conclusion</h3><p>Overall, LCIG treatment was well tolerated in <em>LRRK2</em>-PD and <em>GBA1</em>-PD. <em>GBA1</em>-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.</p></div>\",\"PeriodicalId\":19970,\"journal\":{\"name\":\"Parkinsonism & related disorders\",\"volume\":\"127 \",\"pages\":\"Article 107115\"},\"PeriodicalIF\":3.1000,\"publicationDate\":\"2024-08-28\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Parkinsonism & related disorders\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1353802024011271\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Parkinsonism & related disorders","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1353802024011271","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Levodopa-carbidopa intestinal gel for advanced Parkinson's disease: Impact of LRRK2 and GBA1 mutations
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.
期刊介绍：
Parkinsonism & Related Disorders publishes the results of basic and clinical research contributing to the understanding, diagnosis and treatment of all neurodegenerative syndromes in which Parkinsonism, Essential Tremor or related movement disorders may be a feature. Regular features will include: Review Articles, Point of View articles, Full-length Articles, Short Communications, Case Reports and Letter to the Editor.
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