Adherence with metreleptin therapy and health self-perception in patients with lipodystrophic syndromes
Adherence with metreleptin therapy and health self-perception in patients with lipodystrophic syndromes
复制标题
脂肪营养不良综合征患者美曲普汀治疗的依从性和健康自我认知
DOI：
10.1186/s13023-019-1141-2
复制
发表时间：
2019
影响因子：
3.7
通讯作者：
C. Vigouroux
中科院分区：
文献类型：
--
作者：
C. Vatier;D. Kalbasi;M. Vantyghem;O. Lascols;I. Jéru;A. Daguenel;J. Gautier;M. Buyse;C. Vigouroux
背景尽管美曲瘦素替代疗法被证明可以改善脂肪营养不良综合征的代谢改变，但患者对治疗的依从性和满意度从未被评估过。20例脂肪营养不良综合征患者参加了法国的metreleptin治疗的同情项目，填写了一份自我问卷，包括依从性评估测试，药物治疗满意度问卷（TSQM®-vII），以及有关身体外观的项目。结果15例患者为女性，中位年龄为32.5岁（IQT 25-75（16.2;49.5）），18例患有糖尿病。与metreleptin（每日一次皮下注射）的依从性差，25%，25%的优秀和50%的患者可接受。在0-100量表上，患者对metreleptin治疗的有效性满意度评分为66.7（52.1;81.2），易用性/舒适度为55.6（44.4;66.7），总体满意度为83.3（52.1;83.3）。自我报告的副作用为频繁的注射部位反应100（79.2;100）。部分（n = 10）或全身性（n = 10）脂肪营养不良综合征患者的满意度评分没有差异，与代谢改善无关，但依从性患者的满意度评分显著较高，副作用较少。据报道，在17 patients.ConclusionsMetreleptin治疗的13改善形态学外观，增加健康的自我认知，减少形态相关的污名化，在大多数患者脂肪营养不良综合征，但舒适度差的使用和局部副作用削弱坚持。
BackgroundAlthough metreleptin replacement therapy was shown to improve metabolic alterations in lipodystrophic syndromes, patients’ adherence and satisfaction with treatment have never been evaluated. The 20 patients with lipodystrophic syndromes participating in the French compassionate program of metreleptin therapy filled in a self-questionnaire including an Adherence Evaluation Test, the Treatment Satisfaction Questionnaire for Medication (TSQM®-vII), and items about physical appearance.Results15 patients were women, median age was 32.5 years (IQT 25–75 (16.2;49.5), 18 had diabetes. Adherence with metreleptin (one daily subcutaneous injection) was poor in 25%, excellent in 25% and acceptable in 50% of patients. On a 0-to-100 scale, patients’ satisfaction scores reached 66.7 (52.1;81.2) for effectiveness, 55.6 (44.4;66.7) for ease/comfort of use, and 83.3 (52.1;83.3) for global satisfaction with metreleptin therapy. Self-reported side effects were frequent injection site reactions 100 (79.2;100). Satisfaction scores did not differ in patients with partial (n = 10) or generalized (n = 10) lipodystrophic syndromes, did not correlate with metabolic improvement, but were significantly higher in compliant patients with fewer side effects. Morphological appearance was reported improved under metreleptin therapy in 13 among 17 patients.ConclusionsMetreleptin increases health self-perception and decreases morphotype-associated stigmatization in most patients with lipodystrophic syndromes, but poor comfort of use and local side effects weaken adherence.