Prospective Validation of Engraftment Syndrome Phenotypes and Outcomes After Hematopoietic Stem Cell Transplantation

NCT07577375 | Not Yet Recruiting DMC

• 1 other identifier: ES-HSCT-001
• Study Type: observational
• Target: 250
• Locations: 0 countries
• Sites: N/A

Brief Summary

Engraftment syndrome (ES) is an early inflammatory complication after hematopoietic stem cell transplantation (HSCT) and has been associated with subsequent transplant-related complications and adverse clinical outcomes. However, ES is clinically heterogeneous, and its relationship with acute graft-versus-host disease (aGVHD), survival, and other post-transplant outcomes remains incompletely defined. This prospective observational cohort study aims to validate previously identified ES-associated risk factors, severity-oriented ES phenotypes, and their associations with grade II-IV aGVHD and clinical outcomes after HSCT. Patients undergoing HSCT will be prospectively followed for the development of ES, grade II-IV aGVHD, and clinical outcomes including overall survival, disease-free survival, relapse, and non-relapse mortality. The study will evaluate whether ES phenotypes and ES-related clinical characteristics can stratify patients according to subsequent aGVHD risk and post-transplant prognosis.

Conditions

Engraftment Syndrome; Allo-HSCT

Keywords

Engraftment Syndrome; allo-HSCT; acute GVHD

Outcome Measures

Primary Outcomes (1)

• Time to Grade II-IV Acute Graft-Versus-Host Disease

  Time from hematopoietic stem cell transplantation to the first diagnosis of grade II-IV acute graft-versus-host disease among patients with engraftment syndrome. Acute graft-versus-host disease will be graded according to institutional standard criteria.

  From hematopoietic stem cell transplantation to the first diagnosis of grade II-IV acute graft-versus-host disease, assessed up to 180 days after transplantation.

Secondary Outcomes (6)

• Incidence of Grade II-IV Acute Graft-Versus-Host Disease

  Up to 180 days after transplantation.

• Incidence of Chronic Graft-Versus-Host Disease

  Up to 2 years after transplantation.

• Overall Survival

  From transplantation to death from any cause, assessed up to 2 years after transplantation.

• Disease-Free Survival

  From transplantation to relapse, disease progression, or death from any cause, assessed up to 2 years after transplantation.

• Cumulative Incidence of Relapse

  Up to 2 years after transplantation.

Other Outcomes (5)

• Severity-Oriented Engraftment Syndrome Phenotype

  From transplantation to 100 days after transplantation.

• Association Between Engraftment Syndrome Phenotype and Grade II-IV Acute Graft-Versus-Host Disease

  Engraftment syndrome phenotype assessed up to 100 days after transplantation; grade II-IV acute graft-versus-host disease assessed up to 180 days after transplantation.

• Association Between Engraftment Syndrome Phenotype and Overall Survival

  Engraftment syndrome phenotype assessed up to 100 days after transplantation; overall survival assessed up to 2 years after transplantation.

Study Arms (1)

Patients With Engraftment Syndrome

Patients who develop engraftment syndrome after hematopoietic stem cell transplantation will be enrolled and prospectively followed. Engraftment syndrome will be diagnosed according to predefined clinical criteria during the early post-transplant period. Clinical features, laboratory parameters, transplant-related characteristics, ES phenotypes, subsequent grade II-IV acute graft-versus-host disease, and clinical outcomes will be recorded and analyzed.

Other: No Intervention: Observational Cohort

Interventions

No Intervention: Observational Cohort OTHER

No study-specific intervention will be administered. Participants will receive standard clinical care after hematopoietic stem cell transplantation according to institutional practice and treating physician discretion. The study will prospectively collect observational data on engraftment syndrome characteristics, laboratory parameters, subsequent grade II-IV acute graft-versus-host disease, and clinical outcomes.

Patients With Engraftment Syndrome

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will include patients who develop engraftment syndrome after allogeneic hematopoietic stem cell transplantation at the participating center. Patients will be prospectively enrolled after the diagnosis of engraftment syndrome and followed for subsequent grade II-IV acute graft-versus-host disease and post-transplant outcomes, including overall survival, disease-free survival, relapse, and non-relapse mortality. Clinical features, laboratory parameters, engraftment kinetics, and organ-injury markers related to engraftment syndrome will be collected to validate ES phenotypes and their prognostic significance.

You may qualify if:

• Patients undergoing allogeneic hematopoietic stem cell transplantation at the participating center.
• Development of engraftment syndrome after hematopoietic stem cell transplantation, diagnosed according to predefined institutional or published clinical criteria.
• Availability of key clinical and laboratory data required for engraftment syndrome phenotyping, including engraftment kinetics, clinical manifestations, inflammatory markers, and organ-injury parameters.
• Ability to undergo prospective follow-up for post-transplant outcomes, including acute graft-versus-host disease and survival outcomes.
• Written informed consent provided by the patient or legally authorized representative, when required by the institutional review board or ethics committee.

You may not qualify if:

• Patients who do not develop engraftment syndrome after hematopoietic stem cell transplantation.
• Patients with insufficient clinical or laboratory data to confirm the diagnosis of engraftment syndrome.
• Patients with missing essential follow-up information for assessment of primary outcome measures.
• Patients who withdraw consent or decline participation in prospective follow-up.
• Patients enrolled in another study that, in the opinion of the investigators, may interfere with the observational assessment of engraftment syndrome phenotypes or post-transplant outcomes.

Sponsors & Collaborators

• Xiao Hui Zhang: lead
• Guangdong Provincial Hospital of Chinese Medicine, Zhuhai: collaborator
• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: collaborator
• The First Affiliated Hospital of Zhengzhou University: collaborator
• The Third Xiangya Hospital of Central South University: collaborator
• The First Hospital of Jilin University: collaborator
• Guangzhou First People's Hospital: collaborator

Central Study Contacts

Yejun Wu

CONTACT

+8618800181620; wyejun1999@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 3, 2026
• First Posted: May 11, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2029
• Last Updated: May 11, 2026

Record last verified: 2026-05