Gómez-Arango Catalina, Gorostiza Inigo, Úcar Eduardo, García-Vivar Maria Luz, Pérez Clara Eugenia, De Dios Juan Ramon, Alvarez Belen, Ruibal-Escribano Ana, Stoye Claudia, Vasques Margarida, Belzunegui Joaquin, Escobar Antonio, Trancho Ziortza, Ruiz Del Agua Ainhoa, Del Rio Lorena, Jorquera Cristina, Diez Eli, Martínez Antonio, Nagore Daniel
Department of Rheumatology, Osakidetza Basque Health Service, Basurto University Hospital, Bilbao, Spain.
Research Unit, Osakidetza Basque Health Service, Basurto University Hospital, Bilbao, Spain.
Rheumatol Ther. 2021 Sep;8(3):1323-1339. doi: 10.1007/s40744-021-00345-5. Epub 2021 Jul 18.
To assess the clinical and cost-effectiveness of therapeutic drug monitoring (TDM) based on serum adalimumab levels compared to standard of care in patients with rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis.
This was a non-inferiority, multicentric, non-randomized, pragmatic trial including adult patients diagnosed with moderate-to-severe, clinically stable rheumatic diseases treated with adalimumab. Consecutive patients were assigned 1:2 to the control (CG) or the intervention group (IG), based on the site of inclusion, and followed up for 18 months. Adalimumab serum levels were measured at each study visit and released to the IG only to modify dosing strategy. Data on disease activity, healthcare resource utilization and health-related quality of life (HRQoL) measured through the EQ-5D-5L were collected. Number of persistent and overall flares, time to first flare, days experiencing high disease activity, total direct costs, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratio (ICER) were calculated.
Of the 169 recruited patients, 150 were included in the analysis (52 and 98 patients in the CG and IG, respectively). The primary endpoint was not met as persistent flares were not significantly lower in the IG, although mean (SD) number of flares was numerically lower in the IG (0.67 [0.70] versus 0.90 [0.82], P = 0.073), respectively. Based on EQ-5D-5L utilities, HRQoL was significantly higher in the IG at 3 (P = 0.001) and 6 months (P = 0.035), which overall translated into 0.075 QALYs gained per patient for the IG at month 18. Overall, direct costs were significantly lower for the IG patients (€15,311.59 [4,870.04] versus €17,378.46 [6,556.51], P = 0.030), resulting in the intervention being dominant, leading to increased QALY at a lower overall cost CONCLUSION: Adalimumab dose tapering based on TDM for rheumatic patients led to an increased quality of life and QALY gain and entailed lower costs, being a more cost-effective alternative than clinically guided management.
评估与类风湿关节炎、银屑病关节炎和强直性脊柱炎患者的标准治疗相比，基于血清阿达木单抗水平的治疗药物监测（TDM）的临床效果和成本效益。
这是一项非劣效性、多中心、非随机、实用性试验，纳入了使用阿达木单抗治疗的中度至重度、临床稳定的成年风湿性疾病患者。根据纳入地点，将连续入组的患者按1:2分配至对照组（CG）或干预组（IG），并随访18个月。在每次研究访视时测量阿达木单抗血清水平，仅向IG提供该结果以调整给药策略。收集通过EQ-5D-5L测量的疾病活动、医疗资源利用和健康相关生活质量（HRQoL）数据。计算持续发作和总体发作的次数、首次发作时间、疾病高活动期天数、总直接成本、质量调整生命年（QALY）和增量成本效益比（ICER）。
在169名招募的患者中，150名纳入分析（CG组和IG组分别为52名和98名患者）。主要终点未达到，因为IG组的持续发作次数没有显著降低，尽管IG组的平均（标准差）发作次数在数值上较低（分别为0.67[0.70]和0.90[0.82]，P = 0.073）。基于EQ-5D-5L效用，IG组在3个月（P = 0.001）和6个月（P = 0.035）时的HRQoL显著更高，总体而言，在第18个月时，IG组每位患者获得0.075个QALY。总体而言，IG组患者的直接成本显著更低（15311.59欧元[4870.04]对17378.46欧元[6556.51]，P = 0.030），导致干预措施具有优势，即在总体成本更低的情况下提高了QALY。结论：基于TDM对风湿性疾病患者进行阿达木单抗剂量滴定可提高生活质量和QALY，并降低成本，是一种比临床指导管理更具成本效益的替代方案。
