Hybrid ImmunoTherapy (ATG/Dexamethasone/Etoposide) for Hemophagocytic Lymphohisti

描述（由申请人提供）： 噬血细胞性淋巴组织细胞增生症（HLH）是一种异常和严重破坏性的免疫激活疾病。尽管最近的医学进步，大量的儿童与HLH仍然在治疗的初始阶段死亡。值得注意的是，治疗HLH的两种不同方法， 有类似的结果。我们建议进行一项名为“HLH混合免疫疗法”（HIT-HLH）试验的临床试验，该试验测试了结合两种方法（ATG和依托泊苷）的治疗方案可能会为HLH患者带来更好的结果的想法。通过进行这项多中心试点试验，HIT-HLH联盟希望在使用这种创新方案治疗后以显着的精确度确定应答率和生存率。我们还希望将这些患者的结果与研究参与者收集的大量临床数据相关联。这些目标很重要，因为推进对这种毁灭性疾病的治疗需要进行大规模试验，将标准治疗与这种（或其他）新的创新疗法进行比较。如果不首先测试新的"混合"方法并了解其可行性和有效性，就不能进行这些试验。这些研究也很重要，因为没有已知的临床特征可以预测HLH患者的复发或死亡，这些知识对于根据患者需求定制未来治疗至关重要。此外，进行HIT-HLH试验将汇集一个独特的合格合作研究者网络，这些研究者有兴趣并有动力测试HLH患者的新疗法。该网络的影响和努力将远远超出目前的审判。通过这一努力，我们打算提高HLH儿童的长期生存率，并对HLH和人体免疫功能产生重要的新见解。

DESCRIPTION (provided by applicant): Hemophagocytic lymphohistiocytosis (HLH) is a disorder of abnormal and severely damaging immune activation. Despite recent medical progress, significant numbers of children with HLH still die during the initial phases of therapy. Notably, two different approaches for treating HLH, with similar outcomes, have been reported. We are proposing to conduct a clinical trial, called the 'Hybrid Immunotherapy for HLH' (HIT-HLH) trial, which tests the idea that a treatment regimen combining aspects of both approaches (ATG and etoposide) may result in better outcomes for patients with HLH. By conducting this multicenter pilot trial, the HIT-HLH consortium hopes to define response and survival rates with significant precision after treatment with this innovative regimen. We also hope to correlate outcomes of these patients with extensive clinical data collected on study participants. These goals are important because advancing therapy for this devastating disorder will require large trials comparing standard treatment with this (or other) new innovative therapies. These trials cannot be performed without first testing the new 'hybrid' approach and understanding how feasible and effective it may be. These studies are also important because there are no known clinical features which can predict relapse or death in patients with HLH, and such knowledge will be essential for tailoring future therapies to patients needs. Furthermore, conducting the HIT-HLH trial will gather together a uniquely qualified network of collaborative investigators who are interested and motivated to test novel therapies for patients with HLH. The impact and efforts of this network will extend well beyond the current trial. Through this effort we intend to improve the long term survival of children with HLH and develop significant new insights into HLH and human immune function.