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NCT Number: NCT07569055

Longitudinal Cohort 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.

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Key information

Conditions

Invasive Fungal Disease Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) Bacterial Infections and Mycoses Infections Invasive Fungal Infections Mycoses

Sex eligibility

All sexes

Study type

Observational

Primary location

Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

Location status: Recruiting

Location contact

Xiaohui Zhang, MD

CONTACT

[email protected]

01088326666

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Since January 1, 2014, patients who underwent allo-HSCT at Peking University People's Hospital and other assistance centers.

Exclusion criteria

  • 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.

Treatment and study plan

Primary outcomes

  1. Overall survival after allo-HSCT, measured as time from transplantation to death from any cause

    Time frame: 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.

Secondary outcomes

  1. Proportion of participants with invasive fungal disease who have overall response at 1 year

    Time frame: 1 year after diagnosis of invasive fungal disease

    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.

  2. Cumulative incidence of proven or probable invasive fungal disease within 1 year after allo-HSCT

    Time frame: From date of allo-HSCT to first diagnosis of invasive fungal disease, death, last contact, or 1 year after allo-HSCT, whichever occurs first

    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.

  3. Number and percentage of participants with post-transplant complications within 5 years after allo-HSCT

    Time frame: From date of allo-HSCT to death, last contact, or 5 years after allo-HSCT, whichever occurs first

    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.

  4. Number and percentage of participants with treatment-related adverse events as assessed by CTCAE v5.0

    Time frame: From initiation of systemic antifungal therapy to 30 days after the last dose, death, or last contact, whichever occurs first

    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.

  5. Total length of hospital stay within 5 years after allo-HSCT, measured in inpatient days

    Time frame: From date of allo-HSCT to death, last contact, or 5 years after allo-HSCT, whichever occurs first.

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Fanhao Nie

CONTACT

[email protected]

086-18887323790

Jin Wu, MD

CONTACT

[email protected]

086-01088326002

Sponsors and collaborators

Lead sponsor

Peking University People's Hospital

Other

Collaborators

  • First Affiliated Hospital of Guangxi Medical University
  • First Affiliated Hospital of Harbin Medical University
  • First Affiliated Hospital of Xinjiang Medical University
  • Jining Medical University
  • Qianfoshan Hospital
  • Shandong Provincial Hospital
  • Shanxi Province Cancer Hospital
  • Shengjing Hospital
  • The First Hospital of Jilin University
  • The General Hospital of Northern Theater Command
  • Zhejiang University

Registry information

Official study title

Retrospective and Prospective Longitudinal Observational Study on Invasive Fungal Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Important dates

Study start
2025
Primary completion
2031
Study completion
2031
First posted
May 6, 2026
Registry last updated
May 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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