Milligan A, Hughes D, Goodwin S, Richfield L, Mehta A
Lysosomal Storage Disorders Unit, Royal Free Hospital, London, UK.
Br J Nurs. 2006;15(6):330-3. doi: 10.12968/bjon.2006.15.6.20681.
This article evaluates satisfaction with enzyme replacement therapy (ERT) at home and at hospital in adult patients with Fabry and Gaucher diseases. A questionnaire was developed and sent to 34 patients with Fabry disease who were receiving ERT with agalsidase alfa (Replagal) and to 49 patients with type I Gaucher disease who were receiving ERT with glucocerebrosidase (Cerezyme). Of the 45 returned questionnaires, 20 were from patients with Fabry disease and 25 from patients with Gaucher disease. Hospital treatment visits were reported as stressful by 18 patients (40%), whereas only 4 (9%) patients reported that home therapy was stressful. Both groups of patients adjusted well to receiving home-based therapy. Nearly all of the patients with Fabry disease (19 patients, 95%) and Gaucher disease (21 patients, 84%) preferred home-based therapy. Treatment in the home was reported as more comfortable, less stressful, more effective and had less impact on family life. Only 4 (9%) patients chose to continue receiving infusions in hospital. The majority of patients with Fabry disease and Gaucher disease found home-based therapy to be more convenient and less stressful than hospital-based therapy.
本文评估了法布里病和戈谢病成年患者在家中和医院接受酶替代疗法（ERT）的满意度。设计了一份问卷，并发送给34名正在接受阿加糖酶α（瑞普佳）ERT治疗的法布里病患者以及49名正在接受葡萄糖脑苷脂酶（思而赞）ERT治疗的I型戈谢病患者。在回收的45份问卷中，20份来自法布里病患者，25份来自戈谢病患者。18名患者（40%）报告称医院治疗就诊有压力，而只有4名（9%）患者表示家庭治疗有压力。两组患者都很好地适应了家庭治疗。几乎所有法布里病患者（19名患者，95%）和戈谢病患者（21名患者，84%）都更喜欢家庭治疗。据报告，在家中治疗更舒适、压力更小、更有效，对家庭生活的影响也更小。只有4名（9%）患者选择继续在医院接受输液。大多数法布里病和戈谢病患者发现家庭治疗比医院治疗更方便、压力更小。
