K. Dahir, Steven W Ing, Chad Deal, Andrew Messali, Toby Bates, E. Rush
{"title":"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","authors":"K. Dahir, Steven W Ing, Chad Deal, Andrew Messali, Toby Bates, E. Rush","doi":"10.1093/jbmrpl/ziae062","DOIUrl":null,"url":null,"abstract":"\n 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.","PeriodicalId":14611,"journal":{"name":"JBMR Plus","volume":null,"pages":null},"PeriodicalIF":3.4000,"publicationDate":"2024-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Improvement in quality of life after Asfotase Alfa treatment in adults with Pediatric-onset Hypophosphatasia: data from 5 patient-reported outcome measures\",\"authors\":\"K. Dahir, Steven W Ing, Chad Deal, Andrew Messali, Toby Bates, E. Rush\",\"doi\":\"10.1093/jbmrpl/ziae062\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\\n 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]). 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引用次数: 0
摘要
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。
Improvement in quality of life after Asfotase Alfa treatment in adults with Pediatric-onset Hypophosphatasia: data from 5 patient-reported outcome measures
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.