This study was designed as a prospective, multicenter, open-label, randomized controlled trial. Eligible participants were patients aged 15-65 years with high risk or relapsed/refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic neoplasms (MDS), diagnosed based on bone marrow morphology, immunophenotyping, genetic testing, and treatment response assessment. The experimental group received SHR2554 combined with azacitidine as an overlapped sequential combination with the mBuCy conditioning regimen, whereas the control group received the mBuCy conditioning regimen, both followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint is 1-year event-free survival (EFS). Secondary endpoints include 2-year overall survival, 2-year cumulative incidence of relapse, transplant-related mortality, incidence of acute/chronic GVHD, and safety profiles.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
180
SHR2554 350 mg BID and azacitidine 75 mg/m² daily on days -9 to -3, overlapping with mBUCY conditioning:semustine 250 mg/m² on day -8; cytarabine 2 g/m² q12h on day -7; busulfan 0.8 mg/kg q6h on days -6,-5, -4 (total 3.2 mg/kg/day); cyclophosphamide 1.8 g/ m²/day on days -3 and -2.
semustine 250 mg/m² on day -8; cytarabine 2 g/m² q12h on day -7; busulfan 0.8 mg/kg q6h on days -6,-5, -4 (total 3.2 mg/kg/day); cyclophosphamide 1.8 g/ m²/day on days -3 and -2
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
Event-Free Survival (EFS)
It is measured from the time of entry into this trial to the date of first event (relapse, death from any cause); patients not known to have experienced any event at last follow-up are censored on the date they were last known to be event-free.
Time frame: 1 years
Overall survival（OS）
It is measured from the date of entry into this trial to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive.
Time frame: 2 years
Cumulative incidence of relapse（CIR）
It is measured the date from complete remission after transplantation to hematological relapse was recorded. Patients who had no relapse at the last follow-up were considered as censored data, and non-relapse death was regarded as a competing risk event.
Time frame: 2 years
event-free survival (EFS)
It is measured from the time of entry into this trial to the date of first event (relapse, death from any cause, or grade III-IV acute GVHD); patients not known to have experienced any event at last follow-up are censored on the date they were last known to be event-free.
Time frame: 2 years
transplant related mortality (TRM)
cumulative incidence of transplant related mortality
Time frame: 2 years
graft-versus-host disease (GvHD)
incidence and severity of acute (aGvHD) and chronic graft-versus-host disease (cGvHD) (aGvHD refer to Glucksberg Criteria and cGvHD refer to the National Institutes of Health Consensus)
Time frame: 2 years
Time period for hematopoietic reconstruction
Granulogenetic hematopoietic reconstitution: The absolute neutrophil count in peripheral blood needs to reach or exceed 0.5×10\^9 cells/L for 3 consecutive days. Megakaryotic hematopoietic reconstitution: platelet count needs to be more than 20×10\^9/L and does not rely on platelet transfusion for 7 consecutive days.
Time frame: 24 weeks
Regimen related toxicity
Number of participants with regimen related toxicity as assessed by CTCAE v5.0
Time frame: 2 years
veno-occlusive disease (VOD)
incidence of veno-occlusive disease (VOD) events (refer to modified Seattle Criteria of VOD)
Time frame: 2 years
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