Myelodysplastic Syndromes
Conditions
Brief summary
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.
Interventions
DRUGRTC conditioning regimen and Modified Bu/Cy conditioning regimen
RTC 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.
Sponsors
Peking University People's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
Exclusion criteria
* Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| transplant-related mortality (TRM) | Participants will be followed for an expected average of 1 years | Death without disease progression or relapse |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hematopoietic engraftment | Day 30 and Day 90 post-transplantation | 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. |
| Cumulative incidence of graft-versus-host disease (GVHD) | Day 100 and 3 years after transplantation | 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. |
| Cumulative incidence of CMV and EBV reactivation | 1 year after transplantation | Defined as any detectable CMV or EBV viral load elevation requiring clinical intervention within 1 year after transplantation. |
| Cumulative incidence of hematologic relapse | 1 year after transplantation | Defined as bone marrow blast recurrence or extramedullary disease relapse within 1 year after transplantation. |
| Disease-free survival (DFS) | 1 year after transplantation | Defined as the time from transplantation until hematologic relapse or death from any cause. |
| Overall survival（OS） | 1 year after transplantation | Defined as the time from treatment until death from any cause or the last follow-up. |
| Conditioning-related organ toxicities | From conditioning start to Day 30 post-transplantation | Incidence of grade 1-4 cardiac, renal, hepatic, gastrointestinal and oral mucosal toxicities from conditioning initiation to Day +30 post-transplantation, with separate analysis of Grade 3-4 severe organ injury, hepatic veno-occlusive disease (VOD), and conditioning-related death, graded per WHO organ toxicity scale. |
Countries
China
Contacts
CONTACTYu Wang
PRINCIPAL_INVESTIGATORXiao-Jun Huang
Peking University Institute of Hematology
Outcome results
None listed