Part 5: Allogeneic HSCT in refractory SJIA with lung disease; recent cases from centers in North America & Europe

IF 2.8 3区医学 Q1 PEDIATRICS

Pediatric Rheumatology Pub Date : 2024-01-05 DOI:10.1186/s12969-023-00868-x

Alexei A. Grom, Scott W. Canna, Rolla F. Abu-Arja, Rashmi Sinha, Luciana Peixoto, Elvira Cannizzaro, Shanmuganathan Chandrakasan, Kyla Driest, Rebecca Marsh, Bénédicte Neven, Karen Onel, Sampath Prahalad, Susan Prockop, Pierre Quartier, Johannes Roth, Grant Schulert, Juliana M.F. Silva, Donna Wall, Ulrike Zeilhofer

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引用次数: 0

Abstract

It has been increasingly recognized that there is a subset of patients with refractory systemic JIA, who have failed all available medications and may benefit from HSCT. The increasing experience with HSCT in SJIA, suggests that despite the complicated post-HSCT course, short-term, the transplanted patients either achieved SJIA remission or reduced burden of disease. Longer follow-up, however, is needed to better define the long-term outcomes. The discussion at the NextGen 2022 conference was focused on the optimal timing for the procedure, the need for a good control of inflammatory SJIA activity prior to HSCT, and the role of the reduced intensity conditioning regimens as there was a remote concern that such regimens might increase the risk of SJIA relapse after the transplantation. There was unanimous agreement about the importance of long-term registries to address these questions.

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第 5 部分：异基因造血干细胞移植治疗难治性 SJIA 合并肺部疾病；来自北美和欧洲中心的最新病例

越来越多的人认识到，有一部分难治性系统性 JIA 患者在所有药物治疗无效后，可能会从造血干细胞移植中获益。越来越多的造血干细胞移植治疗 SJIA 的经验表明，尽管造血干细胞移植后病程复杂，但短期内，接受移植的患者要么 SJIA 得到缓解，要么疾病负担减轻。然而，要更好地确定长期疗效，还需要更长时间的随访。在 NextGen 2022 会议上，与会者重点讨论了手术的最佳时机、造血干细胞移植前对 SJIA 炎症活动进行良好控制的必要性，以及降低调理强度方案的作用，因为有人担心这种方案可能会增加移植后 SJIA 复发的风险。与会者一致认为，长期登记对于解决这些问题非常重要。

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

Pediatric Rheumatology PEDIATRICS-RHEUMATOLOGY

CiteScore

4.10

自引率

8.00%

发文量

审稿时长

>12 weeks

期刊介绍： Pediatric Rheumatology is an open access, peer-reviewed, online journal encompassing all aspects of clinical and basic research related to pediatric rheumatology and allied subjects. The journal’s scope of diseases and syndromes include musculoskeletal pain syndromes, rheumatic fever and post-streptococcal syndromes, juvenile idiopathic arthritis, systemic lupus erythematosus, juvenile dermatomyositis, local and systemic scleroderma, Kawasaki disease, Henoch-Schonlein purpura and other vasculitides, sarcoidosis, inherited musculoskeletal syndromes, autoinflammatory syndromes, and others.