A retrospective analysis of 20 adult patients with histopathological and clinical diagnoses of ECD was conducted at a single institution over a twenty-year period (2002–2022). Clinical responses were compared on the basis of treatments rendered, which included chemotherapy, immunotherapy, systemic corticosteroids, surgery and radiation, or targeted agents, referring to any small molecular inhibitors. Treatment response evaluation varied by the anatomic site(s) of disease, the extent of disease at diagnosis, and the imaging modality employed. In this analysis, patients were treated with a combination of targeted agents, myelosuppressive therapies, and radiation at various points in their disease courses. Of these, the most common treatment modality rendered was targeted therapy, employed in 11 of 20 patients. Partial responses or better were observed in 15 of 20 patients. Rates of stable disease trended towards being more frequent with targeted therapy versus conventional therapy but did not reach significance (p= 0.2967). Complete response rates trended towards being more common with conventional therapy than molecular (p= 0.5) but were equivocal overall. Trends of peripheral blood absolute monocytes with relation to disease activity were reviewed as recent literature implied that monocyte levels surrounding disease progression were of potential prognostic significance in histiocytic diseases. Amongst the patients who progressed at any point during their treatment course, absolute monocyte count (in K/µL) was identified at the closest available timepoint prior to or following disease progression and at the lowest value (nadir) following re-institution of therapy prior to any additional agent(s) being employed. There was no statistically significant difference in either of these monocyte values nor in disease outcomes with respect to treatments rendered within our cohort. However, our cohort consists of a heterogenous population of patients with ECD with data that highlights several trends over a longitudinal period, spanning the advent of targeted therapy. Significant differences are anticipated in ongoing analyses.
在一家机构对20名经组织病理学和临床诊断为埃尔德海姆-切斯特病（ECD）的成年患者进行了为期二十年（2002–2022年）的回顾性分析。根据所接受的治疗方案（包括化疗、免疫治疗、全身性皮质类固醇、手术和放疗，或靶向药物，此处指任何小分子抑制剂）对临床反应进行比较。治疗反应的评估因疾病解剖部位、诊断时疾病范围及所用影像学模式而异。本分析中，患者在病程的不同阶段接受了靶向药物、骨髓抑制疗法和放疗的联合治疗。其中，最常见的治疗模式是靶向治疗，20名患者中有11名接受了该治疗。20名患者中有15名观察到部分缓解或更好的疗效。与常规治疗相比，靶向治疗的疾病稳定率有更频繁的趋势，但未达到统计学显著性（p=0.2967）。常规治疗的完全缓解率较分子靶向治疗有更常见的趋势（p=0.5），但总体结果尚不明确。鉴于近期文献提示，疾病进展前后的单核细胞水平在组织细胞疾病中可能具有预后意义，本研究回顾了外周血绝对单核细胞计数与疾病活动性的关联趋势。在治疗过程中任何时间点出现疾病进展的患者中，我们获取了疾病进展前后最近时间点、以及重启治疗后（在启用任何其他药物前）最低值（谷值）的绝对单核细胞计数（单位：K/µL）。在本队列中，这些单核细胞数值以及不同治疗方案间的疾病结局均无统计学显著差异。然而，本队列包含异质性ECD患者群体，其数据揭示了靶向治疗问世以来纵向观察期的若干趋势。预期在持续分析中将发现显著差异。
肿瘤(癌症)患者之家