Department of Hematology/Oncology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, 00165 Rome, Italy.
Department of Neurology, Bambino Gesù Children's Hospital, 00165 Rome, Italy.
Int J Mol Sci. 2020 Jul 26;21(15):5304. doi: 10.3390/ijms21155304.
Neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorders (NMOSD) are a group of autoimmune inflammatory disorders of the central nervous system (CNS). Understanding of the molecular basis of these diseases in the last decades has led to an important improvement in the treatment of this disease, in particular, to the use of immunotherapeutic approaches, such as monoclonal antibodies and Hematopoietic Stem Cell Transplantation (HSCT). The aim of this review is to summarize the pathogenesis, biological basis and new treatment options of these disorders, with a particular focus on HSCT applications. Different HSCT strategies are being explored in NMOSD, both autologous and allogeneic HSCT, with the new emergence of therapeutic effects such as an induction of tolerance to auto-antigens and graft versus autoimmunity effects that can be exploited to hopefully treat a disease that still has prognosis.
视神经脊髓炎(NMO)和视神经脊髓炎谱系疾病(NMOSD)是一组中枢神经系统(CNS)自身免疫性炎症性疾病。在过去几十年中,对这些疾病的分子基础的了解导致了该病治疗的重要改进,特别是免疫治疗方法的应用,如单克隆抗体和造血干细胞移植(HSCT)。本文综述的目的是总结这些疾病的发病机制、生物学基础和新的治疗选择,特别关注 HSCT 的应用。不同的 HSCT 策略正在 NMOSD 中进行探索,包括自体和异体 HSCT,新出现的治疗效果,如对自身抗原的诱导耐受和移植物抗自身免疫效应,这些效果可以被利用来治疗预后仍然不佳的疾病。