Dermatology Department, Center for Clinical Studies, Houston, Texas.
Department of Internal Medicine, Memorial Sloan Kettering Cancer Center, New York, New York.
Dermatol Ther. 2019 May;32(3):e12848. doi: 10.1111/dth.12848. Epub 2019 Feb 20.
Mastocytosis describes a heterogeneous group of disorders arising from a clonal proliferation of mast cells. Given the lack of curative treatments for the cutaneous form, there is a significant need for superior therapies. Omalizumab is a recombinant DNA-derived humanized IgG monoclonal antibody that selectively binds to human immunoglobulin E (IgE). It represents a potential treatment for the management of cutaneous mastocytosis, which currently has no standard treatment.
Two patients were treated with subcutaneous omalizumab 300 mg every 4 weeks.
Patient 1 experienced 50% reduction in cutaneous infiltration and moderate improvement in pruritus. Patient 2 underwent 90% complete clearance of cutaneous lesions and reported full resolution of pruritus. The median duration of treatment was 24 weeks and time to response was 8 weeks. No significant changes in tryptase levels were observed. Both patients experienced injection site reactions.
We provide evidence from two cases supporting the efficacy of IgE-mediated therapy in the treatment of cutaneous mastocytosis. Even at a higher-than-standard dose (300 mg vs. 150 mg), the drug was well-tolerated. As we await the results of pivotal clinical trials, omalizumab appears to be a promising treatment option in patients with cutaneous mastocytosis unresponsive to traditional therapies.
肥大细胞增多症描述了一组源于肥大细胞克隆性增殖的异质性疾病。鉴于皮肤形式缺乏治愈性治疗方法，因此非常需要更好的治疗方法。奥马珠单抗是一种重组 DNA 衍生的人源化 IgG 单克隆抗体，可选择性地与人类免疫球蛋白 E（IgE）结合。它代表了治疗皮肤肥大细胞增多症的一种潜在疗法，目前尚无标准治疗方法。
两名患者接受皮下奥马珠单抗 300mg，每 4 周一次。
患者 1 的皮肤浸润减少了 50%，瘙痒中度改善。患者 2 的皮肤病变完全消退了 90%，并报告瘙痒完全缓解。治疗的中位持续时间为 24 周，反应时间为 8 周。未观察到类胰蛋白酶水平的显著变化。两名患者均出现注射部位反应。
我们提供了两例病例的证据，支持 IgE 介导的治疗在治疗皮肤肥大细胞增多症中的疗效。即使在高于标准剂量（300mg 比 150mg）的情况下，该药物也具有良好的耐受性。在我们等待关键临床试验结果的同时，奥马珠单抗似乎是对传统治疗方法无反应的皮肤肥大细胞增多症患者的一种有前途的治疗选择。
