Gardulf Ann, Nicolay Uwe, Math Dipl, Asensio Oscar, Bernatowska Ewa, Böck Andreas, Costa-Carvalho Beatriz T, Granert Carl, Haag Stefan, Hernández Dolores, Kiessling Peter, Kus Jan, Matamoros Nuria, Niehues Tim, Schmidt Sigune, Schulze Ilka, Borte Michael
Swedish Centre for Immunodeficiencies, Division of Clinical Immunology at the Department of Laboratory Medicine, Karolinska Institutet at Karolinska University Hospital, Stockholm, Sweden.
J Allergy Clin Immunol. 2004 Oct;114(4):936-42. doi: 10.1016/j.jaci.2004.06.053.
A large number of children and adults with primary antibody deficiencies need lifelong IgG replacement therapy. It is mostly unknown what effect the choice of replacement therapy has on the patients' health-related quality of life (HRQOL) and treatment satisfaction (TS).
To investigate whether a switch from hospital-based intravenous IgG (IVIG) to home-based subcutaneous IgG (SCIG) therapy would improve the HRQOL and TS.
Fifteen children (<14 years; hospital-based IVIG therapy at enrollment) and 32 adults (> or =14 years; 22 on hospital-based IVIG and 10 on home-based SCIG therapy at enrollment) were included. Questionnaires were completed at baseline and at 6 and 10 months: the Child Health Questionnaire-Parental Form 50 (children) or Short Form 36 (adults), the Life Quality Index, and questions regarding therapy preferences.
The SCIG home therapy was reported to give better health (P=.001) and improved school/social functioning (P=.02) for the children, reduced emotional distress (P=.02) and limitations on personal time for the parents (P=.004), and fewer limitations on family activities (P=.002). Adults switching therapy reported improved vitality (P=.04), mental health ( P=.05), and social functioning ( P=.01). Adults already on SCIG home therapy at enrollment retained high HRQOL and TS scores. The SCIG home therapy improved TS because it led to greater independence and better therapy convenience ( P <.05). The patients preferred the SCIG administration route and having the treatment at home.
Home-based SCIG therapy improves several important aspects of HRQOL and provides the patients with primary antibody deficiencies and their families with greater independence and better control of the therapy situation and daily life. SCIG home therapy is an appreciated therapeutic alternative for adults and children in need of lifelong IgG replacement therapy.
大量原发性抗体缺陷的儿童和成人需要终身进行免疫球蛋白G（IgG）替代治疗。目前大多不清楚替代治疗的选择对患者健康相关生活质量（HRQOL）和治疗满意度（TS）有何影响。
研究从医院静脉注射IgG（IVIG）治疗转换为家庭皮下注射IgG（SCIG）治疗是否会改善HRQOL和TS。
纳入15名儿童（<14岁；入组时接受医院IVIG治疗）和32名成人（≥14岁；入组时22人接受医院IVIG治疗，10人接受家庭SCIG治疗）。在基线以及6个月和10个月时完成问卷调查：儿童健康问卷家长版50项（儿童）或简明健康调查问卷36项（成人）、生活质量指数以及有关治疗偏好的问题。
据报告，SCIG家庭治疗使儿童健康状况更佳（P = 0.001），学校/社交功能得到改善（P = 0.02），减少了情绪困扰（P = 0.02）以及家长个人时间受限情况（P = 0.004），家庭活动受限情况也减少（P = 0.002）。转换治疗的成人报告活力（P = 0.04）、心理健康（P = 0.05）和社交功能（P = 0.01）有所改善。入组时已接受SCIG家庭治疗的成人保持了较高的HRQOL和TS评分。SCIG家庭治疗提高了TS，因为它带来了更大的独立性和更好的治疗便利性（P < 0.05）。患者更喜欢SCIG给药途径以及在家中接受治疗。
家庭SCIG治疗改善了HRQOL几个重要方面，为原发性抗体缺陷患者及其家庭提供了更大的独立性，以及对治疗情况和日常生活更好的掌控。SCIG家庭治疗是需要终身IgG替代治疗的成人和儿童值得赞赏的治疗选择。
