Abstract：
Mastocytosis patients often experience a number of symptoms, including mastocytosis-associated pain that is difficult to manage due to resistance to usual antalgic treatments and/or the patient\'s poor tolerance. Mastocytosis patients display significantly higher levels of indoleamine-2,3-dioxygenase-1 (IDO1) activity, leading to hyperactivation of the N-methyl-D-aspartate receptor. As cannabidiol (CBD) is known to inhibit IDO1\'s enzymatic activity, we hypothesized that pharmaceutical-grade CBD is an effective treatment for mastocytosis-associated pain. Patients with non-advanced mastocytosis and refractory pain were eligible for inclusion in this observational pilot study. CBD was initiated at 50 mg/day and increased to a maximum of 900 mg/day. Pain was scored on a 0-to-10 numerical rating scale (NRS). A total of 44 patients were included over a 2-year period. The median dose of CBD prescribed was 300 mg/day (range: 50-900 mg/day). Elevated liver enzymes were observed in one patient. The mean ± standard deviation NRS pain score decreased significantly from 7.27 ± 1.35 before treatment to 3.78 ± 1.99 after 3 months of treatment (p < 0.0001). Fifteen patients (34%) were able to discontinue all their previous antalgic medications. CBD treatment might be a safe, effective treatment for mastocytosis-associated pain and its use requires confirmation in a randomized, controlled trial.
摘要：
肥大细胞增多症患者通常会出现许多症状，包括与肥大细胞增多症相关的疼痛，由于对常规抗药治疗的抵抗和/或患者的耐受性差而难以治疗。肥大细胞增多症患者的吲哚胺-2,3-双加氧酶-1（IDO1）活性水平显着升高，导致N-甲基-D-天冬氨酸受体过度激活。由于已知大麻二酚（CBD）抑制IDO1的酶活性，我们假设药物级CBD是肥大细胞增多相关疼痛的有效治疗方法.非晚期肥大细胞增多症和难治性疼痛的患者有资格纳入这项观察性试点研究。CBD以50mg/天开始并增加至最高900mg/天。在0-10数字评定量表（NRS）上对疼痛进行评分。在2年的时间里，共纳入了44例患者。规定的CBD的中位剂量为300mg/天（范围：50-900mg/天）。在一名患者中观察到肝酶升高。平均±标准差NRS疼痛评分从治疗前的7.27±1.35显著下降至治疗3个月后的3.78±1.99(p<0.0001)。15名患者（34％）能够停止所有以前的抗药药物。CBD治疗可能是安全的，肥大细胞增多症相关疼痛的有效治疗及其使用需要在一个随机的确认，对照试验。
