A multicenter phase II prospective clinical trial of glucocorticoid for patients with untreated IgG4-related disease
A multicenter phase II prospective clinical trial of glucocorticoid for patients with untreated IgG4-related disease
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DOI：
10.1080/14397595.2016.1259602
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发表时间：
2017-01-01
影响因子：
2.2
通讯作者：
Sumida, Takayuki
中科院分区：
文献类型：
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作者：
Masaki, Yasufumi;Matsui, Shoko;Sumida, Takayuki
目的：虽然糖皮质激素对 IgG4 相关疾病患者有效，但治疗尚未标准化。因此，应制定治疗策略。 患者及方法：符合明确IgG4相关疾病综合诊断标准的患者开始使用泼尼松龙（0.6mg/kg体重），每两周减量。随后的维持剂量和泼尼松龙的需要是针对个体患者确定的。主要终点是一年时的完全缓解（CR）率。次要终点包括总体缓解率 (ORR)、维持剂量、复发率和不良事件。 结果：本研究纳入了 61 名患者。经过临床病理学审查，排除了 3 名患者，分别有 1 名、13 名和 44 名患者被诊断为可能、可能和明确的 IgG4 相关疾病。 44 例确诊 IgG4-RD 的患者中，29 例（65.9%）达到 CR，ORR 为 93.2%。没有患者对初级治疗有抵抗力。最常见的不良事件是葡萄糖不耐受。 6例患者复发。结论：糖皮质激素治疗通常对IgG4-RD患者有效，当患者糖皮质激素难治时，应检查是否有其他疾病的可能性。有些患者被误诊，使得诊断的中央临床病理学审查在进行临床研究中非常重要。
Objective: Although glucocorticoids are effective for patients with IgG4-related disease, the treatment has not yet been standardized. Therefore, the treatment strategy should be established.Patients and methods: Patients who fulfilled the comprehensive diagnostic criteria for definite IgG4-related disease were started on prednisolone (0.6mg/ kg body weight) with the dose reduced every two weeks. The subsequent maintenance dose and need for prednisolone were determined for individual patients. The primary endpoint was the complete remission (CR) rate at one year. Secondary end-points included overall response rate (ORR), the maintenance dose, the relapse rate, and adverse events.Results: This study enrolled 61 patients. After clinicopathological review, three patients were excluded, and one, 13, and 44 patients were diagnosed with probable, possible, and definite IgG4-related disease, respectively. Of the 44 patients with definite IgG4-RD, 29 (65.9%) achieved CR, and the ORR was 93.2%. No patient was refractory to primary treatment. The most frequent adverse events were glucose intolerance. Six patients relapsed.Conclusions: Glucocorticoid treatment is usually effective for patients with IgG4-RD, and we should examine the possibility of other disorders when a patient is glucocorticoid refractory. Some patients are misdiagnosed, making central clinicopathological review of diagnosis very important in conducting clinical studies.