HSCT for stiff person syndrome and myasthenia gravis.

Q2 Medicine

Handbook of clinical neurology Pub Date : 2024-01-01 DOI:10.1016/B978-0-323-90242-7.00020-1

V D Boccia, Giacomo Boffa, Matilde Inglese

引用次数: 0

Abstract

Recent advances in neuroimmunology have shed light on the pathogenic mechanisms underlying rare neuroimmunologic conditions such as myasthenia gravis (MG) and stiff person syndrome (SPS). Despite the rarity of these conditions, their complex manifestations and potential for irreversible disability necessitate effective therapeutic strategies. This chapter reviews the current understanding of the safety and efficacy of hematopoietic stem cell transplantation (HSCT) in MG and SPS. Several case reports and retrospective studies have demonstrated promising outcomes following HSCT in refractory MG and SPS, with significant clinical improvement and even discontinuation of chronic immunomodulatory therapy in some cases. Furthermore, HSCT may offer insights into the underlying pathophysiologic mechanisms of these conditions, particularly the role of cellular immunity. Although more research is needed to fully understand the impact of HSCT on disease pathology and outcomes, current evidence suggests that HSCT could be a valuable therapeutic option for patients with refractory MG and SPS.

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造血干细胞移植治疗僵人综合征和重症肌无力。

神经免疫学的最新进展揭示了罕见神经免疫疾病（如重症肌无力（MG）和僵人综合征（SPS））的致病机制。尽管这些病症十分罕见，但其复杂的表现形式和可能造成的不可逆转的残疾却需要有效的治疗策略。本章回顾了目前对造血干细胞移植（HSCT）治疗MG和SPS的安全性和有效性的认识。一些病例报告和回顾性研究显示，造血干细胞移植治疗难治性MG和SPS取得了良好的疗效，临床症状明显改善，有些病例甚至停止了慢性免疫调节治疗。此外，造血干细胞移植可能有助于深入了解这些疾病的潜在病理生理机制，尤其是细胞免疫的作用。尽管还需要更多的研究才能充分了解造血干细胞移植对疾病病理和预后的影响，但目前的证据表明，造血干细胞移植可能是难治性 MG 和 SPS 患者的一种重要治疗选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Handbook of clinical neurology Medicine-Neurology (clinical)

CiteScore

4.10

自引率

0.00%

发文量

302

期刊介绍： The Handbook of Clinical Neurology (HCN) was originally conceived and edited by Pierre Vinken and George Bruyn as a prestigious, multivolume reference work that would cover all the disorders encountered by clinicians and researchers engaged in neurology and allied fields. The first series of the Handbook (Volumes 1-44) was published between 1968 and 1982 and was followed by a second series (Volumes 45-78), guided by the same editors, which concluded in 2002. By that time, the Handbook had come to represent one of the largest scientific works ever published. In 2002, Professors Michael J. Aminoff, François Boller, and Dick F. Swaab took on the responsibility of supervising the third (current) series, the first volumes of which published in 2003. They have designed this series to encompass both clinical neurology and also the basic and clinical neurosciences that are its underpinning. Given the enormity and complexity of the accumulating literature, it is almost impossible to keep abreast of developments in the field, thus providing the raison d''être for the series. The series will thus appeal to clinicians and investigators alike, providing to each an added dimension. Now, more than 140 volumes after it began, the Handbook of Clinical Neurology series has an unparalleled reputation for providing the latest information on fundamental research on the operation of the nervous system in health and disease, comprehensive clinical information on neurological and related disorders, and up-to-date treatment protocols.