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

噬血细胞性淋巴组织炎的混合免疫疗法（ATG/地塞米松/依托泊苷）

基本信息

批准号：
8444429
负责人：
Michael Jordan
金额：
$ 21.85万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
依托单位国家：
美国
项目类别：
财政年份：
2012
资助国家：
美国
起止时间：
2012-03-15 至 2015-02-28
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8444429
关键词：
Adrenal Cortex Hormones Affect Aftercare Animal Model Animals Antithymoglobulin Bone Marrow Brain Cells Cessation of life Child Clinical Clinical Data Clinical Trials Clinical Trials Network Consensus Data Defect Dexamethasone Diagnosis Disease Dose Etoposide Functional disorder Future Genetic Goals Hemophagocytic Lymphohistiocytoses Human Hybrids Immune Immune response Immune system Immunotherapy In complete remission Infant Inflammation Innovative Therapy International Knowledge Laboratories Laboratory Finding Lead Life Liver Mediating Medical Mus Neoadjuvant Therapy Organ failure Outcome Participant Patients Pattern Phase Qualifying Randomized Regimen Relapse Reporting Research Personnel Resources Sampling Serotherapies Specimen Stress Survival Rate T memory cell T-Lymphocyte T-Lymphocyte Subsets Testing Therapeutic Transplantation Treatment Protocols abstracting base chemotherapy cohort cytotoxic hematopoietic cell transplantation immune activation immune function improved innovation insight interest killings novel pilot trial pre-clinical prospective randomized trial response screening standard care standard of care trial comparing

项目摘要

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.

描述（由申请人提供）：噬血细胞性淋巴组织细胞增多症 (HLH) 是一种异常且严重破坏性免疫激活的疾病。尽管最近医学取得了进展，但仍有大量 HLH 儿童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，已报道过类似的结果。我们提议进行一项名为“HLH 混合免疫疗法”(HIT-HLH) 试验的临床试验，该试验测试了以下观点：结合两种方法（ATG 和依托泊苷）的治疗方案可能会为患有以下疾病的患者带来更好的结果： HLH。通过开展这项多中心试点试验，HIT-HLH 联盟希望能够非常精确地定义采用这种创新疗法治疗后的缓解率和生存率。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联系起来。这些目标很重要，因为推进这种破坏性疾病的治疗需要进行大型试验，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，就无法进行这些试验。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，而这些知识对于根据患者需求定制未来的治疗方法至关重要。此外，进行 HIT-HLH 试验将聚集一个独特合格的合作研究人员网络，他们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当前的试验。通过这项努力，我们打算提高 HLH 儿童的长期生存率，并对 HLH 和人类免疫功能产生重要的新见解。