Department of Dermatology, Beijing Tsinghua Changgung Hospital, School of Clinical Medicine, Tsinghua University, Beijing, China.
Department of Hematology and Oncology, Beijing Tsinghua Changgung Hospital, School of Clinical Medicine, Tsinghua University, Beijing, China.
Eur J Dermatol. 2023 Jun 1;33(3):270-279. doi: 10.1684/ejd.2023.4502.
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.
To characterize the clinical and pathological responses to cladribine monotherapy based on serial examinations in XD patients.
MATERIALS & 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.
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.
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.
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。目前针对治疗决策，缺乏循证推荐。既往病例报告已经证实嘌呤类似物，特别是克拉屈滨，是一种有希望的一线治疗选择，但对其临床和病理反应的特征描述尚缺乏。
根据 XD 患者的连续检查，描述克拉屈滨单药治疗的临床和病理反应。
我们回顾性研究了在我院接受静脉注射克拉屈滨单药治疗的 5 例 XD 患者的临床、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。我们还对报道的 XD 患者克拉屈滨治疗病例进行了文献复习。
研究纳入 4 名男性和 1 名女性患者。所有患者在接受 5 至 10 个周期的克拉屈滨单药治疗后均获得满意的临床反应。我们观察到在治疗过程中，从经典黄色瘤到过渡性纤维组织细胞浸润的病理模式发生转变，且病理反应预示着持续的临床改善。除了无发热性中性粒细胞减少症外，未发现明显的不良事件。在随访期间，所有 5 例患者均实现了持续的皮损清除，时间范围为 19 至 66 个月。
克拉屈滨单药治疗是 XD 患者的一种有效且耐受良好的治疗选择。病理转化是临床反应的特征，可能揭示了黄色瘤家族疾病中组织细胞生物学的潜在机制。
