Zotta Federica, Vivarelli Marina, Emma Francesco
Department of Pediatric Subspecialties, Division of Nephrology, Bambino Gesù Children's Hospital - IRCCS, Piazza S. Onofrio 4, 00165, Rome, Italy.
Pediatr Nephrol. 2022 Feb;37(2):303-314. doi: 10.1007/s00467-021-04983-3. Epub 2021 Mar 5.
Steroid-sensitive nephrotic syndrome (SSNS) is a rare condition that develops primarily in preadolescent children after the age of 1 year. Since the 1950s, oral corticosteroids have been the mainstay of treatment of all children presenting with nephrotic syndrome, with most patients responding within 4 weeks to an oral course of prednisone (PDN). However, corticosteroids have important side effects and 60-80 % of patients relapse, developing frequently relapsing or steroid-dependent forms. For these reasons, many patients require second-line steroid-sparing immunosuppressive medications that have considerably improved relapse-free survival, while avoiding many PDN-related toxicities. Since most patients will eventually heal from their disease with a normal kidney function, the morbidity of SSNS is primarily related to side effects of drugs that are used to maintain prolonged remission. Therefore, treatment is essentially based on balancing the use of different drugs to achieve permanent remission with the lowest cumulative number of side effects. Treatment choice is based on the severity of SSNS, on patient age, and on drug tolerability. This review provides an update of currently available therapeutic strategies for SSNS.
类固醇敏感性肾病综合征（SSNS）是一种罕见疾病，主要发生在1岁以后的青春期前儿童。自20世纪50年代以来，口服糖皮质激素一直是所有肾病综合征患儿的主要治疗方法，大多数患者在接受泼尼松（PDN）口服疗程后4周内有反应。然而，糖皮质激素有重要的副作用，60%-80%的患者会复发，常发展为频繁复发或类固醇依赖型。由于这些原因，许多患者需要二线类固醇节省免疫抑制药物，这些药物显著提高了无复发生存率，同时避免了许多与PDN相关的毒性。由于大多数患者最终会康复且肾功能正常，SSNS的发病率主要与用于维持长期缓解的药物的副作用有关。因此，治疗基本上基于平衡使用不同药物，以实现永久缓解并使副作用的累积数量降至最低。治疗选择基于SSNS的严重程度、患者年龄和药物耐受性。本综述提供了SSNS当前可用治疗策略的最新信息。
