背景:播散性黄色瘤(XD)是一种罕见的非朗格汉斯组织细胞增生症,广泛累及皮肤。缺乏基于证据的治疗决策建议。以前的病例报告已经确定了嘌呤类似物,尤其是克拉屈滨,作为一种充满希望的一线治疗选择,但缺乏对临床和病理反应的表征。
目的:根据XD患者的系列检查,描述克拉屈滨单药治疗的临床和病理反应。
方法:我们回顾性研究了临床,在我们医院接受静脉注射克拉屈滨单药治疗和连续检查的5例XD患者的病理和实验室数据。与基线特征相比,确定并分析了临床特征和病理模式的变化。我们还对XD患者中克拉屈滨治疗的报道病例进行了文献综述。
结果:本研究涉及4名男性和1名女性患者。所有患者在5至10个周期后对克拉屈滨单药治疗均表现出满意的临床反应。我们观察到在治疗期间从经典黄色肉芽肿到过渡纤维组织细胞浸润的病理转变,病理反应预示着持续的临床改善。除了嗜血中性粒细胞减少症,未发现突出的不良事件.在随访期间,所有五名患者均实现了可持续的病灶清除。从19到66个月不等。
结论:克拉屈滨单药治疗XD患者是一种有效且耐受性良好的治疗选择。病理转化是临床反应的标志,可能揭示黄色肉芽肿家族疾病的潜在组织细胞生物学。
BACKGROUND: Xanthoma disseminatum (XD) is a rare form of non-Langerhans histiocytosis with extensive cutaneous involvement. There is a paucity of evidence-based recommendations for treatment decision-making. Previous
case reports have established purine analogues, especially cladribine, as a hopeful first-line treatment option, but characterization of the clinical and pathological responses is lacking.
OBJECTIVE: To characterize the clinical and pathological responses to cladribine monotherapy based on serial examinations in XD patients.
METHODS: We retrospectively studied the clinical, pathological and laboratory data in a cohort of five XD patients who received intravenous cladribine monotherapy with serial examinations in our hospital. Compared with baseline characteristics, changes in clinical features and pathological patterns were identified and analysed. We also conducted a literature review of reported cases of cladribine treatment in XD patients.
RESULTS: Four male and one female patient were involved in the study. All patients demonstrated satisfactory clinical responses to cladribine monotherapy after 5 to 10 cycles. We observed a pathological shift in pattern from classic xanthogranuloma to transitional fibrohistiocytic infiltration during the treatment, and pathological responses heralded persistent clinical improvement. Other than afebrile neutropenia, no prominent adverse events were identified. Sustainable lesion clearance was achieved in all five patients during the follow-up period, ranging from 19 to 66 months.
CONCLUSIONS: Cladribine monotherapy is an effective and well-tolerated therapeutic option for XD patients. Pathological transformation is a signature of the clinical response and possibly unveils the underlying histiocyte biology of diseases in the xanthogranuloma family.