Immediate versus bridging allogeneic stem cell transplantation for higher-risk myelodysplastic syndrome

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 trial tries to see if getting an allogeneic stem cell transplant right away or after short bridging therapy works better for adults with higher-risk myelodysplastic syndrome.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 236 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (other)
• Locations: 5 sites (Beijin, Beijing Municipality and 4 other locations)
• Trial ID: NCT06918834 on ClinicalTrials.gov

What this trial studies

This Phase 2 randomized study will enroll 236 adults with higher-risk MDS who are eligible for allogeneic hematopoietic stem cell transplantation. Participants are randomized 1:1 to either immediate transplantation or to receive bridging therapy followed by transplantation. The primary design is a non-inferiority comparison and the study will continue until at least 124 events have occurred. Key outcomes include relapse, overall survival, and transplant-related complications to determine the impact of timing and pretransplant disease control.

Who should consider this trial

Good fit: Adults (age ≥18) with higher-risk MDS by IPSS-R (≥3.5) or IPSS-M intermediate-high to very high risk who are eligible for allogeneic HSCT and have Karnofsky ≥60.

Not a fit: Patients with severe organ dysfunction (reduced LVEF, need for oxygen, marked liver or kidney impairment), prior allogeneic HSCT, or otherwise judged unsuitable by investigators are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, the study could show that delaying transplant with bridging therapy provides similar long-term outcomes while allowing disease control or improved patient readiness for transplant.

How similar studies have performed: Prior studies have explored pretransplant therapies and timing in MDS and related conditions with mixed results, and randomized non-inferiority comparisons of immediate versus bridged HSCT remain limited.

Eligibility criteria

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.

Where this trial is running

Beijin, Beijing Municipality and 4 other locations

• Peking University People's Hospital — Beijin, Beijing Municipality, China (RECRUITING)
• Zhengzhou University First Affiliated Hospital — Zhengzhou, Henan, China (RECRUITING)
• The 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation Army — Jinan, Shandong, China (RECRUITING)
• People's Liberation Army The General Hospital of Western Theater Command — Chengdu, Sichuan, China (RECRUITING)
• The Second Hospital of Hebei Medical University — Shijia Zhuang, China (RECRUITING)

Study contacts

• Study coordinator: Jiang Erlie, doctor

Conditions: Myelodysplastic Syndromes