目的:尿素循环障碍(UCDs)可引起氨蓄积和中枢神经系统毒性。氮结合药物可能是有效的,但某些属性可能会对依从性产生负面影响。这项研究旨在量化影响总体处方选择和患者依从性的管理相关属性。方法:基于网络,包括离散选择实验(DCE)方法在内的定量调查收集了医疗服务提供者对UCD患者的反应.一系列假设的治疗概况集具有诸如给药途径等属性,味道/气味,准备说明,包装,剂量测量,并提出了体重使用限制。从16套3个假设的产品概况中,受访者评估了处方选择或患者依从性最优选的属性。如果相对重要性(RI)得分>16.67%(如果所有属性具有同等重要性,则该值),则属性假定更高的总体偏好。评估偏好体重评分。一个九点李克特量表评估了受访者的态度,比如满意。结果:共有51名受访者完成了调查。受访者表示对目前的治疗不满意(平均值[SD]=5.4[1.7])。对于处方选择,实现的四个属性RI>16.67%:味道/气味(24%),体重限制(21%),准备说明(18%),和给药途径(17%)。为了坚持,与给药相关的三个属性达到RI>16.67%:味道/气味(28%),准备说明(21%),和给药途径(17%)。对于处方选择(平均值[SD];1.52[1.10]vs-1.52[1.10])和治疗依从性(73.8[55.2]vs-73.8[55.2]),“掩味/掩味”的权重高于“未掩味”。局限性:这项研究包含相对较小的样本量。调查受访者的选择,使用假设的产品概况,排除非药物治疗方案可能导致潜在的偏见.结论:在测试的属性中,味道/气味是影响处方和患者依从性总体偏好的最重要属性,与味道/气味掩蔽首选。通过掩蔽味道/气味来优化氮结合药物可以支持UCD中改善的患者依从性和结果。
UNASSIGNED: Urea cycle disorders (UCDs) can cause ammonia accumulation and central nervous system toxicity. Nitrogen-binding medications can be efficacious, but certain attributes may negatively impact adherence. This study sought to quantify the administration-related attributes influencing overall prescription selection and patient adherence.
UNASSIGNED: A web-based, quantitative survey including discrete choice experiment (DCE) methodology captured responses from health care providers for patients with UCDs. A series of hypothetical treatment profile sets with attributes such as route of administration, taste/odor, preparation instructions, packaging, dose measurement, and weight use restrictions were presented. From 16 sets of 3 hypothetical product profiles, respondents evaluated attributes most preferred for prescription selection or patient adherence. Attributes assumed a higher overall preference if relative importance (RI) scores were >16.67% (the value if all attributes were of equal importance). Preference weight scores were assessed. A nine-point Likert scale assessed respondent attitudes, such as satisfaction.
UNASSIGNED: A total of 51 respondents completed the survey. Respondents reported dissatisfaction with current treatments (mean [SD] = 5.4 [1.7]). For prescription selection, four attributes achieved RI >16.67%: taste/odor (24%), weight restrictions (21%), preparation instructions (18%), and route of administration (17%). For adherence, three attributes related to administration achieved RI >16.67%: taste/odor (28%), preparation instructions (21%), and route of administration (17%). Preference weights for \"taste/odor masked\" were higher than \"not taste/odor masked\" for prescription selection (mean [SD]; 1.52 [1.10] vs -1.52 [1.10]) and treatment adherence (73.8 [55.2] vs -73.8 [55.2]).
UNASSIGNED: This study contained a relatively small sample size. Survey respondent selection, the use of hypothetical product profiles, and exclusion of non-pharmacologic treatment options could have contributed to potential biases.
UNASSIGNED: Among attributes tested, taste/odor was the most important attribute influencing overall preference for both prescribing and patient adherence, with taste/odor masking preferred. Optimizing nitrogen-binding medications through masking taste/odor may support improved patient adherence and outcomes in UCDs.