Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment.
Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment.
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DOI：
10.1186/s13023-020-01667-3
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发表时间：
2021-01-20
影响因子：
3.7
通讯作者：
Shi L
中科院分区：
文献类型：
--
作者：
Monnette A;Chen E;Hong D;Bazzano A;Dixon S;Arnold WD;Shi L
检查患者/护理者对脊髓性肌萎缩症（SMA）治疗的关键属性的偏好。在快速发展的SMA治疗领域，了解潜在治疗的属性如何影响患者/护理人员的选择是至关重要的。在定性访谈的基础上，开展了离散选择实验调查。SMA患者（≥18岁）和患者的护理人员通过美国患者组织招募。受访者在12组假设的治疗方案中分别做出选择。比较五个治疗特征的相对重要性（通过条件logit模型的回归系数[RC]测量）：(1)运动功能的改善或稳定，(2)呼吸功能的改善或稳定，(3)适用于所有年龄或仅适用于儿科患者，(4)给药途径[反复注射（IT），一次性静脉（IV）输注，每日口服]和(5)潜在危害（轻度，中度，严重/危及生命）。患者年龄从小于1岁到67岁不等（n = 101, 65例自我报告，36例护理人员报告），其中64例为女性。总SMA亚型包括：1型（n = 21）， 2型（n = 48）， 3型（n = 29），其他（n = 3）。47例患者报告既往脊柱手术。Nusinersen和onasemnogene abparvovexii分别在59例和10例患者中使用。运动和呼吸功能的改善得到高度评价[RC: 0.65, 95%可信区间（CI）： 0.47-0.83和RC: 0.79, 95% CI: 0.60-0.98]。口服药物和一次性输注比反复注射更受欢迎（RC: 0.80, 95% CI: 0.60-0.98, RC: 0.51, 95% CI: 0.30-0.73）。患者最不喜欢年龄限制标签/批准使用（≤2岁）（RC: - 1.28, 95% CI: - 1.47至- 1.09）。交叉属性权衡决策表明，尽管有额外的疗效增加，但高风险治疗的意愿较低。对于一些患者来说，可能会愿意牺牲额外的疗效来改变给药途径，从反复鞘内给药到口服给药。运动/呼吸功能的改善，适应症广泛，口服或一次性输注，以及最小的风险是首选的治疗属性。治疗决定应在临床背景下做出，并根据患者的需要量身定制。
To examine patient/caregiver preference for key attributes of treatments for spinal muscular atrophy (SMA). In the rapidly evolving SMA treatment landscape, it is critically important to understand how attributes of potential treatments may impact patient/caregiver choices. A discrete choice experiment survey was developed based on qualitative interviews. Patients with SMA (≥ 18 years) and caregivers of patients were recruited through a U.S. patient organization. Respondents made choices in each of 12 sets of hypothetical treatments. The relative importance of five treatment characteristics was compared (measured by regression coefficients [RC] of conditional logit models): (1) improvement or stabilization of motor function, (2) improvement or stabilization of breathing function, (3) indication for all ages or pediatric patients only, (4) route of administration [repeated intrathecal (IT) injections, one-time intravenous (IV) infusion, daily oral delivery] and (5) potential harm (mild, moderate, serious/life threatening). Patient ages ranged from less than 1 to 67 years (n = 101, 65 self-reported and 36 caregiver-reported) and 64 were female. Total SMA subtypes included: type 1 (n = 21), type 2 (n = 48), type 3 (n = 29), other (n = 3). Prior spinal surgery was reported in 47 patients. Nusinersen and onasemnogene abeparvovec-xioi use were reported in 59 and 10 patients, respectively. Improvement in motor and breathing function was highly valued [RC: 0.65, 95% confidence interval (CI): 0.47–0.83 and RC: 0.79, 95% CI: 0.60–0.98, respectively]. Oral medication and one-time infusion were strongly preferred over repeated IT injections (RC: 0.80, 95% CI: 0.60–0.98 and RC: 0.51, 95% CI: 0.30–0.73, respectively). Patients least preferred an age-restricted label/approved use (≤ 2 years of age) (RC: − 1.28, 95% CI: − 1.47 to − 1.09). Cross-attributes trade-off decision suggested a lower willingness for a high-risk therapy despite additional efficacy gain. For some patients, there may be willingness to trade off additional gains in efficacy for a change in route of administration from repeated intrathecal administration to oral medication. Improvements in motor/breathing function, broad indication, oral or one-time infusion, and minimal risk were preferred treatment attributes. Treatment decisions should be made in clinical context and be tailored to patient needs.
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影响因子：
64.5
作者：
LEFEBVRE, S;BURGLEN, L;MELKI, J
通讯作者：
MELKI, J
影响因子：
11.5
作者：
Dhillon, Sohita
通讯作者：
Dhillon, Sohita
影响因子：
2.5
作者：
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通讯作者：
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影响因子：
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作者：
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通讯作者：
Bridges, John F. P.
影响因子：
2.2
作者：
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通讯作者：
Young, Guy