Age-Stratified Conditioning Regimen Efficacy for MDS Haplo-HSCT
ConditionMyelodysplastic Syndromes
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorPeking University People's Hospital
About this trial
This study plan aims to enroll adult patients diagnosed with myelodysplastic syndrome (MDS) who are scheduled to receive T-cell-replete haploidentical hematopoietic stem cell transplantation. After obtaining written informed consent, participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged \<55 years) followed by unified post-transplant immunosuppression and supportive care. The objective is to prospectively characterize the 1-year transplant-related mortality and comprehensively evaluate hematopoietic engraftment, graft-versus-host disease, infection, relapse, survival outcomes and conditioning-related organ toxicity among all enrolled patients undergoing haploidentical transplantation.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
Disqualifiers
Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure
Trial design
Design model
Single group
Treatments tested in this trial
RTC conditioning regimen and Modified Bu/Cy conditioning regimen
DrugRTC preconditioning regimen consisted of cytarabine (2g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m2/day, days -6 to -2), semustine (250 mg/m2, day-3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France). Bu/Cy preconditioning regimen consisted of tandard institutional myeloablative busulfan + cyclophosphamide + rabbit ATG regimen per department routine dosing guidelines.
Treatment groups
120 Participants
are divided into 1 treatment groupGroup A: Age-Stratified Conditioning RegimenOther 1 intervention
Trial outcomes
Primary outcomes
1
transplant-related mortality (TRM)
Death without disease progression or relapse
Time frame
Participants will be followed for an expected average of 1 yearsSecondary outcomes
1
Hematopoietic engraftment
Neutrophil engraftment is defined as the first day of sustained absolute neutrophil count (ANC) ≥0.5×10⁹/L for 3 consecutive days; platelet engraftment is defined as the first day of sustained platelet count (PLT) ≥20×10⁹/L without transfusion support for 7 consecutive days. Cumulative incidence of neutrophil engraftment at Day 30 and platelet engraftment at Day 90 will be calculated.
Time frame
Day 30 and Day 90 post-transplantation2
Cumulative incidence of graft-versus-host disease (GVHD)
Cumulative incidence of Grade II-IV and Grade III-IV acute GVHD at Day 100, as well as chronic GVHD and moderate-severe chronic GVHD within 3 years after transplantation, graded per Seattle criteria.
Time frame
Day 100 and 3 years after transplantation3
Cumulative incidence of CMV and EBV reactivation
Defined as any detectable CMV or EBV viral load elevation requiring clinical intervention within 1 year after transplantation.
Time frame
1 year after transplantation4
Cumulative incidence of hematologic relapse
Defined as bone marrow blast recurrence or extramedullary disease relapse within 1 year after transplantation.
Time frame
1 year after transplantationSponsors and contacts
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Source ClinicalTrials.gov