METHODS: Adult patients and caregivers of paediatric patients self-enrolled in an online survey (March-April 2021). Outcomes included EQ-5D-5L, PROMIS® GH and INF1-QOL (adult patients only), and EQ-5D-5L, CarerQol and WPAI (caregivers only). Utilities were estimated from EQ-5D-5L responses using the UK crosswalk value set. Linear regression models explored univariable associations between adult patient characteristics and HRQoL.
RESULTS: Mean (± standard deviation) EQ-5D utility in adult patients with NF1 PN was 0.65 (± 0.29; n = 35; age-/sex-matched norm: 0.89 [± 0.04]). Moderate-extreme pain/discomfort and anxiety/depression were reported by 14/35 (40.0%) and 18/35 (51.4%) patients, respectively. Mean PROMIS® GH physical and mental health scores were 43.6 (± 9.19) and 41.7 (± 11.5; n = 35; matched norm: 50.0 [± 10.0]). Mean INF1-QOL score was 11.03 (± 6.02; n = 33). Chronic itching, at least one symptom, at least one comorbidity, PN location at extremities (arms/legs) and pain were associated with worse HRQoL scores. Mean caregiver EQ-5D utility was 0.72 (± 0.24; n = 8; age-/sex-matched norm: 0.88 [± 0.03]). Moderate pain/discomfort and moderate-severe anxiety/depression were reported by 4/8 (50.0%) and 2/8 (25.0%) caregivers, respectively. Mean CarerQol score was 69.3 (± 13.9; n = 8). Mean WPAI regular activity productivity loss was 36.3% (± 31.6%; n = 8).
CONCLUSIONS: NF1 PN worsens adult patient and caregiver HRQoL compared to the general population, notably affecting pain and discomfort, anxiety and depression and caregiver productivity.
方法:成人患者和儿科患者的照顾者自行参加在线调查(2021年3月至4月)。结果包括EQ-5D-5L,PROMIS®GH和INF1-QOL(仅限成人患者),和EQ-5D-5L,CarerQol和WPAI(仅限护理人员)。公用事业是使用英国人行横道值集从EQ-5D-5L响应中估算的。线性回归模型探讨了成人患者特征与HRQoL之间的单变量关联。
结果:NF1PN成年患者的EQ-5D效用平均值(±标准差)为0.65(±0.29;n=35;年龄/性别匹配的标准:0.89[±0.04])。14/35(40.0%)和18/35(51.4%)患者报告了中度极度疼痛/不适和焦虑/抑郁,分别。平均PROMIS®GH身体和心理健康评分分别为43.6(±9.19)和41.7(±11.5;n=35;匹配标准:50.0[±10.0])。平均INF1-QOL评分为11.03(±6.02;n=33)。慢性瘙痒,至少有一个症状,至少有一种合并症,四肢(胳膊/腿)的PN位置和疼痛与HRQoL评分较差相关。平均护理人员EQ-5D效用为0.72(±0.24;n=8;年龄/性别匹配标准:0.88[±0.03])。4/8(50.0%)和2/8(25.0%)护理人员报告了中度疼痛/不适和中重度焦虑/抑郁,分别。平均CarerQol评分为69.3(±13.9;n=8)。平均WPAI常规活动生产率损失为36.3%(±31.6%;n=8)。
结论:与普通人群相比,NF1PN使成年患者和照顾者的HRQoL恶化,特别是影响疼痛和不适,焦虑、抑郁和护理人员的生产力。