Retrospective and Prospective Longitudinal Observational Study on Invasive Fungal Disease After Allogeneic Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 6,000
- 试验地点
- 1
- 主要终点
- Overall survival after allo-HSCT, measured as time from transplantation to death from any cause
研究概览
简要总结
Invasive fungal disease (IFD) is one of the serious complications after hematopoietic stem cell transplantation (HSCT), characterized by high incidence and high mortality. According to the data from a multi-center study in China (CAESAR 2.0), even with the extensive use of antifungal active drugs for prevention, the cumulative incidence of IFD one year after HSCT still reached 6.3%, and the IFD-related mortality rate was 48.28%. In recent years, with the improvement of transplantation techniques, the application of new antifungal drugs and the optimization of diagnostic methods, the pathogen spectrum and clinical characteristics of IFD have undergone significant changes. Compared with ten years ago, the proportion of non-Aspergillus pathogens (such as Candida and Mucophora) has significantly increased, while the proportion of Aspergillus has relatively decreased. In addition, different types of invasive mycosis (such as invasive aspergillosis and invasive fusarium) show significant differences in clinical manifestations, onset time and prognosis. However, at present, large-scale prospective cohort studies on IFD after HSCT in China are still relatively scarce, and the diagnosis and treatment norms and prevention strategies in clinical practice still need to be further optimized. This study intends to conduct a multi-center retrospective and prospective combined longitudinal cohort study to comprehensively register the basic information, diagnosis, treatment and prognosis of IFD patients after HSCT, providing evidence-based medical basis for establishing new clinical diagnosis and treatment technologies and improving the long-term survival rate of patients.
详细描述
Invasive fungal disease refers to a severe infection caused by fungi invading human tissues, blood or body fluids, mainly affecting people with weakened immune systems. Under the background of HSCT, due to the transplantation recipients undergoing high-dose chemotherapy pretreatment, graft-versus-host disease (GVHD), and the use of immunosuppressants, the immune function of the body is severely impaired, and the risk of IFD occurrence significantly increases. The clinical manifestations of IFD after HSCT are diverse, which can involve multiple organs and systems such as the lungs, blood, central nervous system, and skin. It is difficult to diagnose, challenging to treat, and has a poor prognosis, seriously affecting the long-term survival and quality of life of transplant patients.
In recent years, the epidemiological characteristics of IFD after HSCT have undergone significant changes. According to the CAESAR 2.0 study, among 2015 Chinese patients who received allo-HSCT, the cumulative one-year incidence of IFD (proven + probable) was 6.3%. It is worth noting that compared with the CAESAR study ten years ago, the pathogen spectrum has undergone a significant transformation, which is closely related to the evolution of antifungal prevention strategies - ten years ago, fluconazole was mainly used for prevention, while currently about three quarters of patients use antifungal active drugs such as voliconazole or posaconazole. Although this shift in preventive strategies has reduced the occurrence of aspergillosis, it may increase the risk of infection from drug-resistant pathogens such as Mucor.
IFD after HSCT remains a major clinical challenge affecting the prognosis of patients. The current research has the following deficiencies: (1) There is still a lack of multi-center, large-sample prospective cohort studies in China; (2) The dynamic changes in the pathogen spectrum and clinical characteristics of IFD require continuous monitoring. (3) The diagnosis and treatment strategies and prognostic factors of different types of IFD need in-depth research. Based on the above background, this study intends to establish a longitudinal observational cohort that combines retrospective and prospective approaches to systematically evaluate the epidemiological characteristics, treatment strategies, and short-term and long-term outcomes of IFD patients after HSCT.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Since January 1, 2014, patients who underwent allo-HSCT at Peking University People's Hospital and other assistance centers.
排除标准
- •For any reason, such as the occurrence of severe mental disorders, the follow-up information may be unavailable;
- •Patients deemed unsuitable for the study by the researchers.
研究组 & 干预措施
Prospective cohort
Patients whose first visit to our institution occur after the opening of this study will contribute to the prospective cohort.
Retrospective cohort
Patients whose first visit to our institution and the termination of follow-up both occurred before the opening of this study will contribute to the retrospective cohort.
Retrospective/Prospective cohort
Patients whose first visit to our institution occurred before the opening of this study and whose follow-up will terminate after the opening of this study will contribute to the ambispective cohort.
结局指标
主要结局
Overall survival after allo-HSCT, measured as time from transplantation to death from any cause
时间窗: From date of allo-HSCT to death, last contact, or 5 years after allo-HSCT, whichever occurs first
Overall survival is defined as the time from the date of allogeneic hematopoietic stem cell transplantation to death from any cause. Participants without documented death will be censored at the date of last contact or at 5 years after transplantation, whichever occurs first. Overall survival probabilities at 1, 3, and 5 years after transplantation will be estimated using the Kaplan-Meier method.
次要结局
- Proportion of participants with invasive fungal disease who have overall response at 1 year(1 year after diagnosis of invasive fungal disease)
- Cumulative incidence of proven or probable invasive fungal disease within 1 year after allo-HSCT(From date of allo-HSCT to first diagnosis of invasive fungal disease, death, last contact, or 1 year after allo-HSCT, whichever occurs first)
- Number and percentage of participants with post-transplant complications within 5 years after allo-HSCT(From date of allo-HSCT to death, last contact, or 5 years after allo-HSCT, whichever occurs first)
- Number and percentage of participants with treatment-related adverse events as assessed by CTCAE v5.0(From initiation of systemic antifungal therapy to 30 days after the last dose, death, or last contact, whichever occurs first)
- Total length of hospital stay within 5 years after allo-HSCT, measured in inpatient days(From date of allo-HSCT to death, last contact, or 5 years after allo-HSCT, whichever occurs first.)
研究者
Xiao Hui Zhang
Vice President of Peking University Institute of Hematology Affiliation: Peking University People's Hospital
Peking University People's Hospital