Autologous hematopoietic stem cell transplantation in Clarkson disease.
Autologous hematopoietic stem cell transplantation in Clarkson disease.
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自体造血干细胞移植治疗克拉克森病。
DOI：
10.1016/j.jaip.2022.10.023
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发表时间：
2023
期刊：
影响因子：
--
通讯作者：
Cunningham-Rundles,Charlotte
中科院分区：
文献类型：
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作者：
Druey,KirkM;Eisch,ARobin;Cunningham-Rundles,Charlotte
临床意义512例Clarkson病(单克隆性伽玛病相关性特发性全身性毛细血管渗漏综合征[ISCLS])患者在自体干细胞移植(ASCT)治疗骨髓瘤后症状缓解或治疗要求减少53例，其中54例提示单克隆性伽马病在ISCLS发病机制中的作用。5556ISCLS耀斑通常在感染之前，特别是呼吸道病毒感染57，包括新冠肺炎(1，2)。血流动力学崩溃发生在发作开始时，通常为58例，随后是静脉输液引起的大量外周水肿。红斑可能合并多器官功能障碍综合征(MODS)、60血浓缩导致的血管血栓形成和间隔室综合征(1)。使用高剂量静脉注射免疫球蛋白(IVIG)每月无限期预防可导致疾病缓解，但费用昂贵，给药不便。停用IVIG与频繁复发和增加63例死亡率有关(3)。即使接受静脉注射丙种球蛋白治疗的患者也能出现突破性发作，特别是在新冠肺炎的背景下的(4)。这些发现为探索65种根治疗法提供了理论基础。66
Clinical implications 512 patients with Clarkson disease (monoclonal gammopathy-associated idiopathic systemic 52 capillary leak syndrome [ISCLS]) had either remission of symptoms or reduced treatment 53 requirements following autologous stem cell transplantation (ASCT) for myeloma, 54 suggesting a role for the monoclonal gammopathy in ISCLS pathogenesis. 5556 ISCLS flares are frequently preceded by infections, particularly respiratory virus infections 57 including COVID-19 (1, 2). Hemodynamic collapse occurs at the onset of episodes, typically 58 followed by massive peripheral edema resulting from IV fluid administration. Flares may be 59 complicated by multi-organ dysfunction syndrome (MODS), vascular thrombosis due to 60 hemoconcentration, and compartment syndrome (1). Indefinite monthly prophylaxis with high 61 dose intravenous immunoglobulins (IVIG) induces disease remission but is expensive and 62 inconvenient to administer. IVIG withdrawal is associated with frequent relapse and increased 63 mortality (3). Breakthrough episodes can occur even in patients treated with IVIG, particularly in 64 the setting of COVID-19 (4). These findings provide a rationale for exploration of curative 65 therapies. 66