Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients (ImmBridge)

Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients: A Multicenter, Randomized Controlled, Open-Label, Phase 2 Clinical Trial

This study aims to evaluate whether immediate allogeneic hematopoietic stem cell transplantation (HSCT) is non-inferior to HSCT following bridging therapy in patients with higher-risk myelodysplastic syndrome (HR-MDS).

Study Overview

• Status: Recruiting
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Other: Immediate HSCT Group; Other: Bridging Therapy Group

Detailed Description

A total of 236 patients will be randomized in a 1:1 ratio into the immediate transplantation group (n=118) and the disease control group (n=118). The study will continue until at least 124 events occur.

• Study Type: Interventional
• Enrollment (Estimated): 236
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Jiang Erlie, doctor; Phone Number: +86-15122538106; Email: jiangerlie@ihcams.ac.cn
• Study Contact Backup: Name: Xiao Zhijian, doctor; Phone Number: +86-13821085716; Email: xiaozhijian@ihcams.ac.cn

Study Locations

• China
  • Shijia Zhuang, China
    • Recruiting
    • The Second Hospital of Hebei Medical University
    • Contact: Fuxu Wang; Phone Number: +86-13931100360; Email: wfxhebmu@163.com
  • Beijing Municipality
    • Beijin, Beijing Municipality, China, 100044
      • Recruiting
      • Peking University People's Hospital
      • Contact: Hao Jiang
  • Henan
    • Zhengzhou, Henan, China, 450000
      • Recruiting
      • Zhengzhou university first affiliated hospital
      • Contact: weijie cao; Phone Number: +86-15093360671; Email: 945509679@qq.com
  • Shandong
    • Jinan, Shandong, China
      • Recruiting
      • The 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation Army
      • Contact: FANG ZHOU; Phone Number: +86-13969179221
  • Sichuan
    • Chengdu, Sichuan, China, 610083
      • Recruiting
      • People's Liberation Army The General Hospital of Western Theater Command
      • Contact: hai yi; Phone Number: +86-17760428229; Email: yihaimail@163.com
      • Contact: yan deng; Phone Number: +86-15828495714; Email: 285924840@qq.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥18 years

2. High relapse risk MDS, defined by:

   • IPSS-R score ≥3.5.
   • IPSS-M stratification as intermediate-high, high, or very high risk.
3. Eligible for allogeneic HSCT (including matched or mismatched related/unrelated donor transplantations).
4. Karnofsky Performance Status (KPS) ≥60.
5. Signed informed consent.

Exclusion Criteria:

1. Severe organ dysfunction:

   • Left ventricular ejection fraction <50%.
   • Oxygen supplementation requirement.
   • Serum bilirubin >1.5x upper limit of normal (unless due to Gilbert syndrome) or AST/ALT >5x upper limit of normal.
   • Estimated glomerular filtration rate (eGFR) <50 mL/min.
2. History of prior allogeneic HSCT.
3. Any condition deemed unsuitable by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Immediate HSCT Group	Other: Immediate HSCT Group; Patients undergo direct allogeneic HSCT.
Active Comparator: Bridging Therapy Group	Other: Bridging Therapy Group; Patients receive one to two cycles of bridging therapy before undergoing allogeneic HSCT. o Bridging Therapy Regimen: Hypomethylating agents (HMA) alone or HMA-based combination chemotherapy, e.g., azacitidine (AZA) 100 mg/day + venetoclax (VEN) 400 mg/day for 7 days. Targeted therapies (e.g., IDH1 inhibitors) will be used for eligible patients.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
2-year Disease-Free Survival (DFS) post-HSCT	Defined as the time from transplantation to two years post-HSCT, with primary events including death or failure to achieve CR or CR equivalent at the time of assessment	2-year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative Incidence of Allogeneic HSCT	The proportion of patients who undergo HSCT at 4, 8, 16, and 24 weeks post-randomization.	The proportion of patients who undergo HSCT at 4, 8, 16, and 24 weeks post-randomization.
Complete Remission (CR) or CR Equivalent Rate from Randomization	The percentage of patients achieving CR or CR equivalent, defined as the first documented occurrence.	2-year
2-year Overall Survival (OS) post-HSCT	Defined as the time from HSCT to death from any cause within two years.	2-year
2-year Leukemia-Free Survival (LFS) from Randomization	The time from randomization to the occurrence of disease progression, relapse, or death from any cause within two years.	2-year
2-year Quality of Life (QoL) Assessment: 2-year Quality of Life (QoL) Assessment from Randomization	Defined as the assessment of patient-reported QoL starting from randomization over a 2-year period, evaluated using the EORTC QLQ-C30 questionnaire.	2-year
Molecular Clearance Rate	Molecular clearance was defined by two consecutive blood samples obtained at least 4 weeks apart after that were negative for driver mutations in a patient who had been positive before transplantation.	2-year

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Actual): July 24, 2025
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2028

Study Registration Dates

• First Submitted: March 11, 2025
• First Submitted That Met QC Criteria: April 2, 2025
• First Posted (Actual): April 9, 2025

Study Record Updates

• Last Update Posted (Estimated): September 12, 2025
• Last Update Submitted That Met QC Criteria: September 5, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes
• Other Study ID Numbers: IIT2025016

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No