Improvement in quality of life after Asfotase Alfa treatment in adults with Pediatric-onset Hypophosphatasia: data from 5 patient-reported outcome measures

IF 4.3 3区材料科学 Q1 ENGINEERING, ELECTRICAL & ELECTRONIC

ACS Applied Electronic Materials Pub Date : 2024-05-07 DOI:10.1093/jbmrpl/ziae062

K. Dahir, Steven W Ing, Chad Deal, Andrew Messali, Toby Bates, E. Rush

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引用次数: 0

Abstract

Hypophosphatasia (HPP) is a rare inherited metabolic disorder caused by deficient tissue-nonspecific alkaline phosphatase activity. This study assessed the impact of treatment with asfotase alfa on patient-reported outcomes (PROs) in adults with pediatric-onset HPP. A longitudinal telephone-based survey was administered to eligible individuals enrolled in a patient support program. Interviews were conducted at study entry (prior to asfotase alfa initiation) and after 3, 6, and 12 months. PROs—Patient Health Questionnaire-9 [PHQ-9], Work Productivity and Activity Impairment–Specific Health Problem [WPAI:SHP], Patient-Reported Outcomes Measurement Information System 29 [PROMIS-29], and Routine Assessment of Patient Index Data 3 [RAPID3]—were assessed at each time point. Appropriate statistical tests were performed to assess score changes. Among 50 enrolled patients (mean age: 46 years [SD: 15.4]; 80% female; 94% white), 49 were evaluable at 3 months, 44 at 6 months, and 29 at 12 months. By Month 3, statistically significant improvements from baseline were detected in PHQ-9 scores (10.6 vs. 5.8 [p < 0.0001]), PROMIS-29 domain scores (overall physical function: 38.0 vs. 43.0 [p = 0.001]; anxiety: 57.5 vs. 51.5 [p = 0.0011]; fatigue: 63.3 vs. 55.3 [p < 0.0001]; sleep disturbances: 58.8 vs. 54.3 [p = 0.0099]; ability to participate in social roles and activities: 42.6 vs. 47.7 [p = 0.0012]; and pain interference: 63.8 vs. 58.4 [p = 0.001]) and RAPID3 domain scores (functional status: 2.7 vs. 1.1 [p < 0.0001]; pain tolerance: 6.0 vs. 3.2 [p < 0.0001]; and global health estimate: 5.1 vs. 2.7 [p < 0.0001]). Improvements persisted at Month 12. Patients also showed improvements in WPAI:SHP domain scores at Month 6 (presenteeism: 39.6% vs. 14.1% [p < 0.0001] and work productivity loss: 41.9% vs. 14.1% [p < 0.0001]). Treatment with asfotase alfa was associated with improved quality of life across several domains.

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阿斯福通α治疗成人儿童型低磷酸盐血症后生活质量的改善：5 项患者报告的结果测量数据

低磷酸盐血症（HPP）是一种罕见的遗传性代谢性疾病，由组织非特异性碱性磷酸酶活性不足引起。本研究评估了使用阿斯福通α治疗对儿童型HPP成人患者报告结果（PROs）的影响。研究人员通过电话对参加患者支持计划的合格患者进行了纵向调查。访谈在研究开始时（开始使用阿斯福太酶α之前）以及 3、6 和 12 个月后进行。在每个时间点都对PROs--患者健康问卷-9 [PHQ-9]、工作效率和活动障碍--特定健康问题[WPAI:SHP]、患者报告结果测量信息系统29 [PROMIS-29]和患者指数数据常规评估3 [RAPID3]进行了评估。对评分变化进行了适当的统计检验。在 50 名入组患者中（平均年龄：46 岁 [标码：15.4]；80% 为女性；94% 为白人），49 人在 3 个月时接受了评估，44 人在 6 个月时接受了评估，29 人在 12 个月时接受了评估。到第 3 个月时，PHQ-9 评分（10.6 vs. 5.8 [p < 0.0001]）、PROMIS-29 领域评分（总体身体功能：38.0 vs. 43.0 [p<0.0001]）与基线相比均有统计学意义上的显著改善：63.3 vs. 55.3 [p < 0.0001]；睡眠障碍：58.8 vs. 54.3 [p = 0.0099]；参与社会角色和活动的能力：42.6 vs. 47.7 [p = 0.0012]；疼痛干扰：63.8 vs. 58.4 [p = 0.001]）和 RAPID3 领域得分（功能状态：2.7 vs. 1.1 [p < 0.0001]；疼痛耐受性：6.0 vs. 3.2 [p < 0.0001]；总体健康估计：5.1 vs. 2.7 [p < 0.0001]）。这些改善在第 12 个月仍在持续。第 6 个月时，患者的 WPAI:SHP 领域得分也有所改善（缺勤率：39.6% vs. 14.0% [p < 0.0001]）：39.6% vs. 14.1% [p < 0.0001]）和工作效率损失（41.9% vs. 14.1% [p < 0.0001]）：41.9% vs. 14.1% [p < 0.0001]）。阿斯福通α治疗与多个领域的生活质量改善相关。

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来源期刊

ACS Applied Electronic Materials Multiple-

CiteScore

7.20

自引率

4.30%

发文量

567

期刊介绍： ACS Applied Electronic Materials is an interdisciplinary journal publishing original research covering all aspects of electronic materials. The journal is devoted to reports of new and original experimental and theoretical research of an applied nature that integrate knowledge in the areas of materials science, engineering, optics, physics, and chemistry into important applications of electronic materials. Sample research topics that span the journal's scope are inorganic, organic, ionic and polymeric materials with properties that include conducting, semiconducting, superconducting, insulating, dielectric, magnetic, optoelectronic, piezoelectric, ferroelectric and thermoelectric. Indexed/Abstracted： Web of Science SCIE Scopus CAS INSPEC Portico