医学
沙利度胺
朗格汉斯细胞组织细胞增多症
环磷酰胺
养生
内科学
胃肠病学
地塞米松
外科
中性粒细胞减少症
耐火材料(行星科学)
临床终点
挽救疗法
化疗
临床试验
多发性骨髓瘤
疾病
物理
天体生物学
作者
Jinuo Wang,Ting Liu,Ailin Zhao,Boju Pan,Jian Sun,Jian Li,Daobin Zhou,Xinxin Cao,Minghui Duan
出处
期刊:Leukemia
[Springer Nature]
日期:2022-03-31
卷期号:36 (6): 1619-1624
被引量:2
标识
DOI:10.1038/s41375-022-01555-8
摘要
Langerhans cell histiocytosis (LCH) is a clonal histiocytic neoplasm with various clinical manifestations and heterogeneous prognoses. No standard therapy is available for recurrent/refractory LCH patients. This single-center, single-arm, phase 2 study enrolled 32 patients diagnosed with recurrent/refractory LCH. The TCD regimen (thalidomide 100 mg daily, cyclophosphamide 300 mg/m2 Day 1, 8, 15, and dexamethasone 40 mg Day 1, 8, 15, 22 every 4 weeks) was administered for 12 cycles and thalidomide alone as maintenance for 12 months. The primary endpoint was event-free survival (EFS). Events were defined as progression during or after TCD therapy or death from any cause. After a median follow-up of 22 months (range 5–24 months), no patient died of all causes. The overall response rate was 87.5%, including 18 patients (56.3%) achieving complete remission and 10 patients (31.3%) as partial remission. The estimated 24-month EFS was 64.0%. Patients with risk organ involvement had similar EFS compared to patients without risk organ involvement (P = 0.38). The common toxicities of TCD regimen include grade 1–2 neutropenia (18.8%), grade 1–2 constipation (12.5%), grade 1–2 tiredness (9.4%) and grade 2 peripheral neuropathy (12.5%). Oral thalidomide, cyclophosphamide and dexamethasone are effective and safe regimen for recurrent/refractory LCH patients, particularly for patients with risk organ involvement.
科研通智能强力驱动
Strongly Powered by AbleSci AI