背景:控制持续性运动波动的复杂多重用药方案会导致晚期帕金森病(APD)患者严重的药丸负担。这项研究评估了卡比多巴/左旋多巴肠内混悬液(CLES)和深部脑刺激(DBS)减轻APD患者药丸负担的有效性。
方法:我们使用了2014年至2018年与CLES患者支持计划(PSP)数据相关的100%Medicare服务费用索赔。CLES发起者(CLES-I)与未开始治疗(CLES-NI)(N=188)或接受DBS的PSP患者的倾向匹配为1:1,接受DBS的患者为1:3(N=204,N=612)。平均每日药丸负荷和左旋多巴等效日剂量(LEDD)在基线测量,随访0-6个月和7-12个月。
结果:CLES-I和CLES-NI在基线时的服药负荷高于DBS患者。然而,在治疗后6个月,CLES-I的药丸/天明显少于CLES-NI(4.7对11.4,p<0.05)和DBS(4.8对7.4,p<0.05)。CLES-I(p<0.001)在0-6个月(46.3%)和7-12个月(68.3%)随访时观察到药丸负担显着降低,而CLES-NI(10.5%,p<0.05和+8.2%,p>0.05),星展银行的降幅不显著(-3.9%和-6.1%,p>0.05)。与CLES-NI相比,CLES-I中的平均调整药丸负担在0-6个月时减少了57.3%,在7-12个月时减少了74.1%。与DBS相比减少了49.6%和70.1%。与基线(935至237mg)以及CLES-NI(237mg对1112mg)和DBS患者(236mg对594mg)相比,CLES-I在7-12个月时显示LEDD降低。
结论:与CLES-NI和DBS患者相比,CLES导致药丸负担和口服LEDD显著降低。减轻药丸负担可以被认为是APD患者的治疗目标,这些患者受到复杂的多药物治疗方案的挑战,这些方案会干扰日常生活活动和生活质量。
晚期帕金森病患者不可控运动的管理依赖于口服左旋多巴治疗。非运动症状如抑郁和焦虑可通过其他口服药物治疗。随着时间的推移,需要更高和更频繁的口服药物剂量,导致影响生活质量和依从性的复杂药物治疗方案。2014年至2018年期间,一项针对10,752名帕金森病患者的现实世界研究评估了两种设备辅助疗法减轻药丸负担的有效性。卡比多巴/左旋多巴肠内悬浮和深部脑刺激。卡比多巴/左旋多巴混悬液治疗涉及通过附接到便携式泵的手术端口将左旋多巴连续递送至肠。大脑刺激涉及将金属线连接到大脑以通过植入的刺激器发送电脉冲的手术。由于帕金森病主要影响老年人,我们将接受卡比多巴/左旋多巴混悬液的Medicare患者与未接受混悬液的配对对照组和接受脑刺激的患者进行了比较.在接受装置辅助治疗之前(基线)和治疗后0-6个月和7-12个月(随访)测量平均药丸负荷/天。上面的图表显示,治疗后6个月,使用卡比多巴/左旋多巴混悬液的患者需要的药丸比没有混悬液的患者少(4.7对11.4),12个月时进一步减药(3.5对11.1)。底部的图表显示,到6个月,使用卡比多巴/左旋多巴混悬液的患者比使用脑刺激治疗的患者需要更少的药丸(4.8对7.4),在12个月时进一步减少(3.6对7.0)。口服丸剂负荷的减少表明卡比多巴/左旋多巴悬浮液可能提供简化治疗方案的机会。
BACKGROUND: Complex polypharmacy regimens to manage persistent motor fluctuations result in significant pill burden for patients with advanced Parkinson\'s disease (APD). This study evaluated the effectiveness of carbidopa/levodopa enteral suspension (CLES) and deep brain stimulation (DBS) on reducing pill burden in APD patients.
METHODS: We utilized 100% Medicare fee-for-service claims from 2014 to 2018 linked to CLES Patient Support Program (PSP) data. CLES initiators (CLES-I) were propensity matched 1:1 with patients enrolled in PSP who did not initiate treatment (CLES-NI) (N = 188) or undergo DBS, and 1:3 with patients who received DBS (N = 204, N = 612). Average daily pill burden and levodopa equivalent daily dosage (LEDD) were measured at baseline, 0-6 months and 7-12 months follow-up.
RESULTS: CLES-I and CLES-NI had higher pill burden than DBS patients at baseline. However, at 6 months post-treatment, CLES-I had significantly fewer pills/day than CLES-NI (4.7 versus 11.4, p < 0.05) and DBS (4.8 versus 7.4, p < 0.05). A significant reduction in pill burden was observed at 0-6 months (46.3%) and 7-12 months (68.3%) follow-up for CLES-I (p < 0.001) versus increased burden for CLES-NI (+10.5%, p < 0.05 and +8.2%, p > 0.05) and insignificant reductions for DBS (-3.9% and -6.1%, p > 0.05). Mean adjusted pill burden showed 57.3% fewer pills at 0-6 months and 74.1% at 7-12 months among CLES-I compared with CLES-NI, and 49.6% and 70.1% reduction compared with DBS. CLES-I showed a decrease in LEDD at 7-12 months compared with baseline (935 to 237 mg) and to CLES-NI (237 mg versus 1112 mg) and DBS patients (236 mg versus 594 mg).
CONCLUSIONS: CLES led to a significant reduction in pill burden and oral LEDD compared with CLES-NI and DBS patients. Pill burden reduction could be considered a treatment goal for patients with APD challenged by complex polypharmacy regimens that interfere with activities of daily living and quality of life.
Management of uncontrollable motor movements in patients with advanced Parkinson’s disease rely on oral levodopa-based treatments. Non-motor symptoms such as depression and anxiety are managed with additional oral medications. Over time, higher and more frequent dosing of oral medications is required, resulting in complex medication regimens that impact quality of life and adherence.A real-world study of 10,752 Parkinson’s disease patients between 2014 and 2018 evaluated the effectiveness of two device-aided therapies to reduce pill burden, carbidopa/levodopa enteral suspension and deep brain stimulation. Carbidopa/levodopa suspension treatment involves continuous delivery of levodopa to the intestines through a surgical port attached to a portable pump. Brain stimulation involves surgery to attach metal wires to the brain to send electrical pulses via an implanted stimulator.As Parkinson’s disease predominately affects the elderly, we compared Medicare patients on carbidopa/levodopa suspension to a matched control group receiving no suspension and to those receiving brain stimulation. Average pill burden/day was measured prior to receiving a device-aided treatment (baseline) and at 0–6 months and 7–12 months post-treatment (follow-up).The top graph shows that by 6-months post-treatment, patients on carbidopa/levodopa suspension required fewer pills than those without suspension (4.7 versus 11.4), with further pill reduction at 12 months (3.5 versus 11.1). The bottom graph shows that by 6 months, patients on carbidopa/levodopa suspension required fewer pills than patients treated with brain stimulation (4.8 versus 7.4), with further reduction at 12 months (3.6 versus 7.0). The reduction in oral pill burden suggests that the carbidopa/levodopa suspension may present an opportunity to simplify treatment regimens.