Invasive Fungal DiseaseNCT07569055
Longitudinal Cohort Study on Invasive Fungal Disease After Allogeneic Hematopoietic Stem Cell Transplantation
An observational study of Invasive Fungal Disease and Allogeneic Hematopoietic Stem Cell Transplantation (HSCT), run by Peking University People's Hospital at 1 site in China.
Trial summary
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Invasive Fungal Disease, Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- How many people
- 6,000 participants
- Where
- 1 siteChina
- Study dates
- 15 Mar 2025to 14 Mar 2031
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Study population: Patients who underwent allo-HSCT at Peking University People's Hospital and other assistance centers since January 1, 2014. Outcome Assessment: Patients were followed up for the development of invasive fungal disease (IFD) after transplantation.
Who may be able to join
- Since January 1, 2014, patients who underwent allo-HSCT at Peking University People's Hospital and other assistance centers.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University People's Hospital
Beijing, Beijing Municipality, 100044
Xiaohui Zhang, MD, 01088326666, wujin1996@126.com
About this study
Official title: Retrospective and Prospective Longitudinal Observational Study on Invasive Fungal Disease After Allogeneic Hematopoietic Stem Cell Transplantation
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.
Read the detailed description
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.
- Keywords
- Allogeneic Hematopoietic Stem Cell Transplantation, Invasive Fungal Disease, Prediction, Prognosis
- Medical subject headings
- Invasive Fungal Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. | |
| Prospective cohort | Patients whose first visit to our institution occur after the opening of this study will contribute to the prospective 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival after allo-HSCT, measured as time from transplantation to death from any cause
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.
Measured: From date of allo-HSCT to death, last contact, or 5 years after allo-HSCT, whichever occurs first
Secondary outcomes (5)
Proportion of participants with invasive fungal disease who have overall response at 1 year
Overall response of invasive fungal disease is defined as complete response or partial response at 1 year after diagnosis of invasive fungal disease. Response will be assessed according to the Mycoses Study Group and European Organization for Research and Treatment of Cancer consensus criteria for treatment response in invasive fungal diseases. Participants who die before the 1-year assessment or do not meet complete or partial response criteria will be classified as not having overall response. The outcome will be summarized as the number and percentage of participants with complete or partial response.
Measured: 1 year after diagnosis of invasive fungal disease
Cumulative incidence of proven or probable invasive fungal disease within 1 year after allo-HSCT
Invasive fungal disease is defined as the first episode of proven or probable invasive fungal disease after allo-HSCT according to protocol-defined diagnostic criteria based on EORTC/MSGERC definitions. Death before invasive fungal disease will be treated as a competing event. Participants who do not develop invasive fungal disease and do not die will be censored at the date of last contact or at 1 year after allo-HSCT, whichever occurs first. The cumulative incidence function will be used to estimate the incidence of invasive fungal disease.
Measured: 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
Post-transplant complications include acute graft-versus-host disease, chronic graft-versus-host disease, cytomegalovirus infection or reactivation, Epstein-Barr virus infection or reactivation, bacterial bloodstream infection, organ dysfunction, relapse of the underlying hematologic disease, graft failure, intensive care unit admission. The outcome will be summarized as the number and percentage of participants with at least one post-transplant complication and by complication category.
Measured: 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
Treatment-related adverse events are adverse events considered related to systemic antifungal therapy. Adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. The outcome will be summarized as the number and percentage of participants with at least one treatment-related adverse event. Grade 3 or higher adverse events and serious adverse events will be summarized separately.
Measured: 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
Total length of hospital stay is defined as the cumulative number of inpatient days recorded for each participant after allogeneic hematopoietic stem cell transplantation and before death, last contact, or 5 years after transplantation, whichever occurs first. The outcome will be summarized as inpatient days per participant using descriptive statistics.
Measured: From date of allo-HSCT to death, last contact, or 5 years after allo-HSCT, whichever occurs first.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2026
- Study start15 Mar 2025
- Primary completion14 Mar 2031 (estimated)
- Study completion14 Mar 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Invasive Fungal DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 6,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- First Affiliated Hospital of Guangxi Medical University, First Affiliated Hospital of Harbin Medical University, The First Hospital of Jilin University, Jining Medical University, Qianfoshan Hospital, Shandong Provincial Hospital, Shanxi Province Cancer Hospital, Shengjing Hospital, First Affiliated Hospital of Xinjiang Medical University, First Affiliated Hospital of Zhejiang University, The General Hospital of Northern Theater Command
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
- Investigators
- Xiao-Hui Zhang, MD, Principal investigator, Peking University Institute of Hematology, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07569055
- Sponsor's protocol ID
- HSCT-IFD2026
- Status last verified
- Mar 2026
- First submitted
- 18 Apr 2026
- Last updated
- 6 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No