Weitzman Elissa R, Wisk Lauren E, Salimian Parissa K, Magane Kara M, Dedeoglu Fatma, Hersh Aimee O, Kimura Yukiko, Mandl Kenneth D, Ringold Sarah, Natter Marc
Division of Adolescent/Young Adult Medicine, Boston Children's Hospital, 300 Longwood Ave, Boston, MA 02115, USA.
Department of Pediatrics, Harvard Medical School, Boston 02115, USA.
J Patient Rep Outcomes. 2018;2. doi: 10.1186/s41687-017-0025-2. Epub 2018 Jan 4.
Children with Juvenile Idiopathic Arthritis (JIA) often have poor health-related quality of life (HRQOL) despite advances in treatment. Patient-centered research may shed light on how patient experiences of treatment and disease contribute to HRQOL, pinpointing directions for improving care and enhancing outcomes.
Parent proxies of youth enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry shared patient-reported outcomes about their child's HRQOL and experiences of disease and treatment burden (pain interference, morning stiffness, history of medication side effects and methotrexate intolerance). Contributions of these measures to HRQOL were estimated using generalized estimating equations accounting for site and patient demographics.
Patients ( = 180) were 81.1% white non-Hispanic and 76.7% female. Mean age was 11.8 (SD = 3.6) years, mean disease duration was 7.7 years (SD = 3.5). Mean Total Pediatric Quality of Life was 76.7 (SD = 18.2). Mean pain interference score was 50.1 (SD = 11.1). Nearly one-in-five (17.8%) youth experienced >15 min of morning stiffness on a typical day, more than one quarter (26.7%) reported ≥1 serious medication side effect and among 90 methotrexate users, 42.2% met criteria for methotrexate intolerance. Measures of disease and treatment burden were independently negatively associated with HRQOL (all -values <0.01). Negative associations among measures of treatment burden and HRQOL were attenuated after controlling for disease burden and clinical characteristics but remained significant.
For youth with JIA, HRQOL is multidimensional, reflecting disease as well as treatment factors. Adverse treatment experiences undermine HRQOL even after accounting for disease symptoms and disease activity and should be assessed routinely to improve wellbeing.
尽管治疗取得了进展，但幼年特发性关节炎（JIA）患儿的健康相关生活质量（HRQOL）往往较差。以患者为中心的研究可能会揭示患者的治疗体验和疾病如何影响HRQOL，从而为改善护理和提高治疗效果指明方向。
参与儿童关节炎和风湿病研究联盟（CARRA）注册研究的青少年的家长代理人分享了患者报告的有关其子女HRQOL以及疾病和治疗负担（疼痛干扰、晨僵、药物副作用史和甲氨蝶呤不耐受）的结果。使用考虑到研究地点和患者人口统计学特征的广义估计方程来估计这些指标对HRQOL的影响。
患者（n = 180）中81.1%为非西班牙裔白人，76.7%为女性。平均年龄为11.8岁（标准差 = 3.6），平均病程为7.7年（标准差 = 3.5）。儿童生活质量总平均分是76.7（标准差 = 18.2）。平均疼痛干扰评分为50.1（标准差 = 11.1）。近五分之一（17.8%）的青少年在典型的一天中晨僵时间超过15分钟，超过四分之一（26.7%）的青少年报告有≥1种严重药物副作用，在90名使用甲氨蝶呤的患者中，42.2%符合甲氨蝶呤不耐受标准。疾病和治疗负担指标与HRQOL独立呈负相关（所有P值<0.01）。在控制疾病负担和临床特征后，治疗负担指标与HRQOL之间的负相关有所减弱，但仍具有显著性。
对于JIA青少年，HRQOL是多维度的，反映了疾病以及治疗因素。即使在考虑了疾病症状和疾病活动后，不良的治疗体验仍会损害HRQOL，应定期进行评估以改善健康状况。
