There is no therapeutic agent approved in cutaneous mastocytosis and mast cell activation syndrome. We report the efficacy of hydroxychloroquine in four patients with cutaneous mastocytosis (n = 2) and mast cell activation syndrome (n = 2).

We show that this molecule reduces the long-term survival of primary human mast cells, interferes with lysosome function and leads to the accumulation of non-functional tryptase in the mast cell granules. Furthermore, hydroxychloroquine decreases the production of pro-inflammatory mediators.

中文翻译：

---

羟氯喹作为肥大细胞活化疾病的新型治疗方法

在皮肤肥大细胞增多症和肥大细胞活化综合症中尚无治疗药物被批准。我们报告了羟基氯喹对四名皮肤肥大细胞增多症（n  = 2）和肥大细胞活化综合征（n = 2）患者的疗效。

我们显示该分子降低了主要人类肥大细胞的长期存活，干扰了溶酶体功能，并导致肥大细胞颗粒中无功能类胰蛋白酶的积累。此外，羟氯喹减少促炎性介质的产生。