Preventing relapse in high-risk MDS patients after transplantation

Prophylactic Intervention for Relapse Prevention Post-Allogeneic Transplantation in Very High-Risk MDS Patients Based on IPSS-M Stratification: A Single-Arm, Prospective, Single-Center Clinical Study

This study is testing a new way to help very high-risk patients with myelodysplastic syndromes avoid relapse after a stem cell transplant.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine (Academic / other)
• Locations: 1 site (Shanghai)
• Trial ID: NCT06612944 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the effectiveness of a prophylactic intervention designed to prevent relapse in patients with very high-risk myelodysplastic syndromes (MDS) following allogeneic transplantation. The study will collect interventional clinical data to evaluate survival and relapse conditions, comparing results with historical data. The primary endpoints include measuring the 1-year and 2-year relapse-free survival rates post-transplantation, focusing on the time until disease progression or death. The trial targets patients classified as very high risk based on the IPSS-M stratification system.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 to 70 diagnosed with very high-risk MDS and having a suitable hematopoietic stem cell donor.

Not a fit: Patients with low-risk MDS or those without a suitable donor may not benefit from this study.

Why it matters

Potential benefit: If successful, this intervention could significantly improve post-transplant survival rates for patients with very high-risk MDS.

How similar studies have performed: While similar approaches have been explored, this specific intervention for very high-risk MDS patients post-transplantation is novel.

Eligibility criteria

Inclusion Criteria:

1. Age between 18 and 70 years, inclusive, both male and female. Diagnosed with MDS according to WHO criteria and classified as very high-risk by IPSS-M scoring. The patient must have a suitable hematopoietic stem cell donor for allogeneic transplantation: Related donors must be at least 5/10 matched for HLA-A, -B, -C, -DQB1, and -DRB1
2. Unrelated donors must be at least 8/10 matched for HLA-A, -B, -C, -DQB1, and -DRB1. Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score of ≤ 2. ECOG performance status of 0-2. Adequate liver, kidney, cardiac, and pulmonary functions as follows: Serum creatinine ≤ 1.5× upper limit of normal (ULN)
3. Cardiac function: Ejection fraction ≥ 50%
4. Baseline oxygen saturation \> 92%
5. Total bilirubin ≤ 1.5× ULN
6. ALT and AST ≤ 2.0× ULN
7. Pulmonary function: DLCO (corrected for hemoglobin) ≥ 40% and FEV1 ≥ 50%. Patients must be capable of understanding and willing to participate in the study, and must sign an informed consent form.

Exclusion Criteria:

1. Failure to proceed with stem cell reinfusion after unsuccessful pre-transplant conditioning. History of previous hematopoietic stem cell transplantation (HSCT). ECOG performance status \> 2. Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score ≥ 3. Any unstable systemic disease including, but not limited to: unstable angina, cerebrovascular accident or transient ischemic attack within the past 3 months, myocardial infarction within the past 3 months, congestive heart failure (New York Heart Association \[NYHA\] class ≥ III), post-pacemaker implantation requiring medication for severe arrhythmias, severe liver, kidney, or metabolic diseases
2. patients with pulmonary arterial hypertension. Active, uncontrolled infection: hemodynamic instability related to infection, new symptoms or signs of worsening infection, radiological evidence of new infectious foci, persistent fever without signs or symptoms that cannot exclude infection. Need for treatment for Grade ≥2 epilepsy, paralysis, aphasia, new cerebral infarction, severe brain trauma, dementia, Parkinson\'s disease, schizophrenia. HIV infection. Active hepatitis B (HBV) or hepatitis C (HCV) requiring antiviral treatment
3. patients at risk of HBV reactivation, indicated by positive hepatitis B surface antigen or core antibody without antiviral therapy for hepatitis B. Pregnant or breastfeeding women. Men and women of childbearing potential unwilling to use contraception during the treatment and for 12 months post-treatment. Allergic to intervention drugs such as azacitidine, decitabine, or venetoclax.

Where this trial is running

Shanghai

• Shanghai General Hospital — Shanghai, China (Recruiting)

Study contacts

• Study coordinator: Xianmin Song, MD

Conditions Myelodysplastic Syndromes, Adult