INTO-HLH- Insight Into the Natural History and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis (HLH): A Disease Registry for Patients With HLH
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 2
- 主要终点
- Number of patients with infections (e.g., EBV, CMV, HHV6, HIV, fungal, bacterial) at the time of diagnosis.
研究概览
简要总结
The purpose of this observational study is to collect data on the natural history of disease of patients with Hemophagocytic Lymphohistiocytosis (HLH) including diagnosis, treatments, responses, and outcomes.
详细描述
Hemophagocytic Lymphohistiocytosis (HLH) is a complex, hyperinflammatory syndrome resulting from the interplay of genetic predisposition and various environmental factors. Despite available treatment options for HLH, approximately 30% of patients do not respond to therapy. Moreover, the standard therapy is constrained by its toxicities, and safer treatments are pursued.
There is an unmet need for a deeper understanding of the natural history, clinical/etiologic diversity, complications, and treatment outcomes of patients with HLH, specifically from North America. The proposed study, a collaboration between Cincinnati Children's Hospital Medical Center (CCHMC), Texas Children's Hospital, and Sobi Inc. aims to establish a robust registry that will enable investigators to better define the natural history of HLH.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with clinically suspected or confirmed HLH, including those meeting the HLH-2004 diagnostic criteria (primary or secondary forms, including malignancy) and other forms of HLH (macrophage activation syndrome [MAS], cytokine release syndrome [CRS], etc.)
- •Signed and dated informed consent and assent (adolescents)
排除标准
- 未提供
研究组 & 干预措施
Patients with clinically suspected or confirmed Hemophagocytic Lymphohistiocytosis
Multi-institutional cohort registry of patients with clinically suspected or confirmed Hemophagocytic Lymphohistiocytosis
结局指标
主要结局
Number of patients with infections (e.g., EBV, CMV, HHV6, HIV, fungal, bacterial) at the time of diagnosis.
时间窗: Up to 1 month from HLH diagnosis.
The presence of infections at HLH diagnosis (serology and polymerase chain reaction).
Number of patients with organ failure.
时间窗: Up to 1 year from HLH diagnosis.
Data will be gathered on organ failure related to HLH (e.g., kidney, lung, CNS).
Time to HLH diagnosis from the initial presentation
时间窗: Interval between date of presentation, as defined as the day of appearance of initial HLH symptom, and the date of full HLH diagnosis, as defined by fulfilling the HLH diagnostic criteria, will be measured. Timeframes up to 6 months will be assessed.
Date of initial presentation and the date of HLH diagnosis as defined by HLH diagnostic criteria (HLH-2004/MAS classification criteria)
Number of patients with an autoimmune disease at the time of HLH diagnosis
时间窗: Up to 1 month from HLH diagnosis
Presence of an autoimmune disease at the time of diagnosis (e.g., Systemic juvenile idiopathic arthritis, lupus)
Number of patients with malignancy at the time of HLH diagnosis
时间窗: Up to 1 month from HLH diagnosis.
Presence of hematologic and solid malignancies at the time of HLH diagnosis.
Number of patients treated with immune-activating agents before HLH diagnosis
时间窗: Up to 1 month before HLH diagnosis.
The number of patients treated with immune-activating agents before initial diagnosis (checkpoint inhibitors, CAR-T constructs)
Number of patients with long-term disease-related complications.
时间窗: Up to 5 years from HLH diagnosis.
Data on long-term complications (e.g., impaired growth, impaired cognitive development) will be gathered.
Number of patients with central nervous system (CNS) involvement during the HLH disease course.
时间窗: Up to 1 month from HLH diagnosis.
CNS involvement as defined by elevated neopterin, white blood cells, or protein at a cerebrospinal fluid or changes in MRI
Frequency of a genetic diagnosis underlying the HLH.
时间窗: Up to 1 month from HLH diagnosis.
Data on genetic testing will be gathered and investigators will summarize the number to calculate the frequency of a genetic diagnosis.
次要结局
- Treatment response rate to HLH-related treatments.(Week two from the start of treatment.)
- Frequency of hematopoietic stem cell transplantation (HSCT) related complications(From HSCT up to 5 years post HSCT.)
- Time to response to HLH-related therapy for patients in the registry.(Assessed up to 12 weeks from start of treatment.)
- The survival probability of patients in the registry(From HLH diagnosis to last follow-up or death, whichever comes first, assessed up to 5 years post-HLH diagnosis.)
- Number of patients who received hematopoietic stem cell transplantation (HSCT)(From HLH diagnoses up to 5 years post-HLH diagnosis.)
- Number of participants with treatment-related adverse events >/= 3 as assessed by CTCAE 5.0(From initiation of HLH related treatment up to 30 days following discontinuation of treatment.)