Risk factors for stomatitis in patients with lymphangioleiomyomatosis during treatment with sirolimus: A multicenter investigator-initiated prospective study.
Risk factors for stomatitis in patients with lymphangioleiomyomatosis during treatment with sirolimus: A multicenter investigator-initiated prospective study.
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西罗莫司治疗期间淋巴管平滑肌瘤病患者口腔炎的危险因素：一项多中心研究者发起的前瞻性研究。
DOI：
10.1002/pds.4259
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发表时间：
2017
期刊：
影响因子：
--
通讯作者：
Nakata K.
中科院分区：
文献类型：
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作者：
Kitamura N;Seyama K;Inoue Y;Nagai K;Suzuki M;Moriyama H;Takada T;Tazawa R;Hirai T;Mishima M;Hayashida M;Hirose M;Arai T;Sugimoto C;Hattori N;Watanabe K;Tamada T;Akazawa K;Tanaka T;Nakata K.
淋巴管肌瘤病是一种罕见的肺部疾病，由异常的平滑肌样细胞增殖引起，多见于绝经前妇女。西罗莫司现在是治疗淋巴管肌瘤病的一线药物。西罗莫司所致口腔炎是治疗过程中最常见的不良事件。为了确定危险因素，我们利用多中心长期西罗莫司试验的数据，调查了口腔炎发病率与患者背景数据和治疗参数的关系。方法受试者接受西罗莫司治疗2年，剂量调整为将低谷血药浓度维持在5至15 ng/mL。口腔炎的发生率与基线人口学、临床特征和纵向数据的变化相关。结果最常见的不良事件是口腔炎，9个月时累积发生率达88.9%，高于肾移植后报告的发生率。口腔炎事件的重复性、持续时间和严重程度因患者而异。我们发现，低Hb(14.5g/dL)患者的发病率明显高于高Hb患者(≥为14.5g/dL，P<；0.01)。口腔炎的累积发生率与平均红细胞体积的减少显著相关，而Hb水平不变；因此，患者的红细胞计数在研究期间增加。结论治疗期间基线Hb水平和平均红细胞体积的减少与口腔炎的发生相关。
PurposeLymphangioleiomyomatosis is a rare lung disease caused by proliferation of abnormal smooth muscle‐like cells and typically occurs in premenopausal women. Sirolimus is now the first‐line drug for the treatment of lymphangioleiomyomatosis. Sirolimus‐induced stomatitis is the most frequent adverse event experienced during treatment. To identify risk factors, we investigated the association of stomatitis incidence with patient background data and treatment parameters, using data from the multicenter long‐term sirolimus trial.MethodsSubjects received sirolimus for 2 years at doses adjusted to maintain a trough blood level of 5 to 15 ng/mL. The incidence of stomatitis was correlated with baseline demographics, clinical characteristics, and changes in the longitudinal data. Risk factors at baseline were assessed by using univariate and multivariate analyses.ResultsThe most frequent adverse event was stomatitis, with the cumulative rate reaching 88.9% by 9 months, higher than that reported in postrenal transplant patients. The repetition, the duration, and the severity of stomatitis events were variable among patients. We found that patients with low hemoglobin (Hb) (<14.5 g/dL) showed significantly higher incidence than those with high Hb (≥14.5 g/dL,P< .01). The cumulative rate for stomatitis incidence was significantly associated with a decrease in the mean corpuscular volume, while the Hb level was constant; thus, red blood cell count in patients increased during the study.ConclusionsBaseline Hb levels and a decrease in mean corpuscular volume during treatment were correlated with the incidence of stomatitis.