Clinical and Experimental Haematology Unit, Department of Haematology/Oncology, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Br J Haematol. 2016 Feb;172(4):524-34. doi: 10.1111/bjh.13866. Epub 2015 Dec 2.
The management of Evans Syndrome in children is challenging due to the lack of evidence-based data on treatment. Steroids, the first-choice therapy, are successful in about 80% of cases. For children who are resistant, relapse or become steroid-dependent, rituximab is considered a valid second-line treatment, with the exception of those with an underlying diagnosis of autoimmune lymphoproliferative syndrome who may benefit from other options such as mycophenolate mofetil and sirolimus. Better knowledge of the immunological mechanisms underlying cytopenias and the availability of new immunosuppressive drugs can be helpful in the choice of more targeted therapies that would enable the reduction of the use of long-term steroid administration or other more aggressive options, such as splenectomy or stem cell transplantation. This manuscript provides an overview of the pathogenic background of the disease, and suggests a clinical approach to diagnosis and treatment with a particular focus on the management of relapsing/resistant disease.
儿童 Evans 综合征的治疗具有挑战性，因为缺乏针对治疗的循证数据。皮质类固醇是首选治疗方法，约 80%的病例有效。对于耐药、复发或依赖皮质类固醇的儿童，利妥昔单抗被认为是一种有效的二线治疗方法，但对于那些潜在诊断为自身免疫性淋巴组织增生综合征的儿童，可能需要其他选择，如霉酚酸酯和西罗莫司。更好地了解血细胞减少的免疫机制和新型免疫抑制药物的可用性，有助于选择更具针对性的治疗方法，从而减少长期皮质类固醇治疗的使用或其他更具侵袭性的治疗方法，如脾切除术或干细胞移植。本文综述了该病的发病机制背景，并提出了一种诊断和治疗的临床方法，特别关注复发/耐药疾病的管理。
