Serramontmany Eugènia, Muñoz Marina, Fernández-Polo Aurora, Morillo María, Gómez-Ganda Laura, Cañete-Ramírez Carme, Ariceta Gema
Pharmacy Department, Vall d'Hebron University Hospital, Barcelona, Spain.
Pediatric Nephrology Department, Vall d'Hebron University Hospital, Barcelona, Spain.
Front Pediatr. 2021 Jan 14;8:614535. doi: 10.3389/fped.2020.614535. eCollection 2020.
Congenital nephrotic syndrome of the Finnish type (CNF) is a rare, severe glomerular disease caused by mutations in the NPHS1 gene, which codes for nephrin. It is characterised by massive proteinuria and severe edoema. Progression to end-stage kidney failure occurs during early childhood and the only curative treatment is kidney transplantation. Nowadays, patients need aggressive medical treatment, which includes daily albumin infusions (for months) until they get clinical stability to receive transplant. In our paediatric hospital, we implemented a multidisciplinary program for the home infusion of albumin with outpatient follow-up. The aim of the study was to assess the safety and efficacy of this program for the first four years of its implementation. Retrospective observational study of CNF paediatric patients treated with home albumin infusion therapy from March 2014 to July 2018 at a tertiary care paediatric hospital. Information on albumin administration was obtained from the electronic prescription assistance program and details on clinical and care-related variables from the hospital's electronic information systems. Four patients with CNF received albumin infusions for 18, 21, 22 months, and 3 years. The treatment was safe, and the complication rates were to be expected considering the severity of disease. Patients required a median of two hospital admissions a year (19 in total); 47% due to catheter-related complications, but there were just three catheter infections. In our experience, home albumin infusion therapy is safe and effective and helps to improve children health and quality of life.
芬兰型先天性肾病综合征(CNF)是一种罕见的严重肾小球疾病,由编码nephrin的NPHS1基因突变引起。其特征为大量蛋白尿和严重水肿。在儿童早期会进展为终末期肾衰竭,唯一的治愈性治疗方法是肾脏移植。如今,患者需要积极的医学治疗,包括每日输注白蛋白(持续数月),直至临床稳定以接受移植。在我们的儿科医院,我们实施了一项多学科计划,进行白蛋白家庭输注并门诊随访。本研究的目的是评估该计划实施头四年的安全性和有效性。对2014年3月至2018年7月在一家三级儿科医院接受白蛋白家庭输注治疗的CNF儿科患者进行回顾性观察研究。白蛋白给药信息来自电子处方辅助程序,临床和护理相关变量的详细信息来自医院的电子信息系统。4例CNF患者接受白蛋白输注的时间分别为18个月、21个月、22个月和3年。治疗是安全的,考虑到疾病的严重程度,并发症发生率在意料之中。患者每年平均住院两次(共19次);47%是由于导管相关并发症,但只有3例导管感染。根据我们的经验,白蛋白家庭输注治疗是安全有效的,有助于改善儿童健康和生活质量。