Treatment Optimization for Children with De Novo and Relapsed Langerhans cell Histiocytosis (LCH) as Part of the International Collaborative Study LCH IV

作为国际合作研究 LCH IV 的一部分，针对新发和复发朗格汉斯细胞组织细胞增多症 (LCH) 儿童的治疗优化

• 批准号: 280500934
• 负责人: Professor Dr. Thomas Lehrnbecher
• 金额: --
• 依托单位: Schwerpunkt Pädiatrische Onkologie, Hämatologie und Hämostaseologie
• 依托单位国家: 德国
• 项目类别: Clinical Trials
• 财政年份: 2016
• 资助国家: 德国
• 起止时间: 2015-12-31 至 2020-12-31
• 项目状态: 已结题
• 来源: https://gepris.dfg.de/gepris/projekt/280500934?language=en
• 关键词: Treatment; Optimization; Children; De; Novo

Langerhans cell Histiocytosis (LCH) is a malignant disorder affecting 3-5/1 million children. Previous trials decreased mortality by intensifying induction chemotherapy, but up to 40% of patients with LCH still reactivate, and permanent sequelae occur in up to 50% of patients. The proposed phase III open-label multicenter randomized trial will evaluate strategies of maintenance therapy [prolongation (12 vs 24 months)±intensification with 6-mercaptopurine] in all patients with de novo LCH (Stratum I) and will evaluate a new compound (indomethacin) in patients with relapsed LCH (Stratum II) to decrease relapse rate and permanent morbidity. Primary endpoint is reactivation-free survival, secondary endpoints include toxicity and risk of permanent consequences. Although many patients suffer from significant morbidity, LCH is a rare disease. Germany alone does not have enough patients for a sufficiently powered study, which has therefore to be done in an international setting. A collaborative trial (LCH-IV) has already started in several countries, and the participation of Germany in the frame of the proposed study is necessary, as Germany is expected to enroll >20% of all patients. This trial may ultimately decrease the risk of relapse and permanent sequelae, thus improving quality of life and reducing socio-economic burden in patients with LCH.

朗格汉斯细胞组织细胞增生症（LCH）是一种恶性疾病，影响3-5/100万儿童。以前的试验通过加强诱导化疗降低了死亡率，但高达40%的LCH患者仍然复发，高达50%的患者发生永久性后遗症。拟定的III期开放标签多中心随机试验将在所有新发LCH（I层）患者中评价维持治疗策略[延长（12 vs 24个月）± 6-巯基嘌呤强化]，并将在复发性LCH（II层）患者中评价一种新化合物（吲哚美辛）以降低复发率和永久性发病率。主要终点是无再激活生存期，次要终点包括毒性和永久性后果的风险。虽然许多患者患有显着的发病率，LCH是一种罕见的疾病。仅德国没有足够的患者进行充分把握度的研究，因此必须在国际环境中进行。一项合作试验（LCH-IV）已经在几个国家开始，德国参与拟议的研究是必要的，因为预计德国将招募超过20%的患者。该试验可能最终降低复发和永久性后遗症的风险，从而改善LCH患者的生活质量并减轻社会经济负担。