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

Phase 2

Not yet recruiting

• Conditions: Myelodysplastic Syndromes

• Registration Number: NCT06918834
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Brief Summary

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).

Detailed Description

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).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 Timeline

• Study First Submitted: 2025-03-11
• Status Verified: 2025-04
• Study Start: 2025-04-01
• Study First Submitted QC: 2025-04-02
• Last Update Submitted: 2025-04-02
• Study First Posted: 2025-04-09
• Last Update Posted: 2025-04-09
• Primary Completion: 2028-04-01
• Study Completion: 2028-04-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 236

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
2-year Disease-Free Survival (DFS) post-HSCT	2-year	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

Secondary Outcome Measures

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