Abstract：
Determine efficacy and adverse events (AEs) of hydroxyurea (HU) in mast cell activation syndrome (MCAS) patients who were refractory to standard medical therapy. An electronic chart review was performed to find MCAS patients who received HU in a MCAS medical practice. Diagnosis of MCAS was established on the basis of mast cell (MC) activation symptoms in ≥ 5 systems plus ≥ 1 abnormal MC mediators and/or ≥ 20 MC/high power field on duodenal biopsies. Medicines not providing significant clinical improvement prior to HU were tabulated. The following symptoms were evaluated by patients on a 0-10 scale prior to and at the study conclusion: bone pain, abdominal pain, diarrhea, bloating, and nausea. Safety labs were obtained on a regular basis. Twenty out of three hundred ten (8.4%) MCAS patients received HU. Patients included 22 females, average age 42.4 years. Dysautonomia was present in 60%. An average of 10.6 (SD 1.7, range 8-13) medications were used prior to adding HU to various concomitant medications. Average dose of HU was 634 mg. In 20 patients who continued therapy for ≥ 2 months, there was statistically significant reduction of bone pain, abdominal pain, diarrhea, bloating, and nausea. Fourteen patients noted prolonged success with therapy. Six patients stopped HU within 6 weeks owing to AEs. Four patients treated ≥ 2 months had AEs and 2 led to HU cessation. All AEs were reversible. Refractory MCAS patients showed clear significant improvement in bone pain and gastrointestinal symptoms on HU. Systematic monitoring was effective in preventing the occurrence of severe HU-induced adverse events.
摘要：
确定羟基脲（HU）在标准药物治疗难治性肥大细胞活化综合征（MCAS）患者中的疗效和不良事件（AE）。进行了电子图表审查，以寻找在MCAS医疗实践中接受HU的MCAS患者。MCAS的诊断是根据≥5个系统中的肥大细胞（MC）激活症状加上十二指肠活检上的≥1个异常MC介质和/或≥20个MC/高倍视野而建立的。列出了在HU之前未提供显着临床改善的药物。在研究结论之前和研究结论时，患者以0-10量表评估了以下症状：骨痛，腹痛,腹泻,腹胀,和恶心。定期获得安全实验室。三百十分之二十（8.4％）MCAS患者接受了HU。患者包括22名女性，平均年龄42.4岁。60%存在自主神经失调。在将HU添加到各种伴随药物中之前,使用平均10.6种(SD1.7,范围8-13)药物。HU的平均剂量为634mg。在20名持续治疗≥2个月的患者中，有统计学意义的骨痛减轻，腹痛,腹泻,腹胀,和恶心。14名患者注意到治疗的长期成功。6例患者因AEs在6周内停止HU。4例治疗≥2个月的患者出现AE，2例导致HU停止。所有AE都是可逆的。难治性MCAS患者在HU上显示骨痛和胃肠道症状的明显改善。系统监测可有效预防HU引起的严重不良事件的发生。
