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Decitabine Plus mBU/CY for High Risk Acute Leukemia With MRD Pre-HSCT

Phase 2
Recruiting
Conditions
Leukemia Relapse
Stem Cell Transplant Complications
Leukemia, Myeloid, Acute
Interventions
Drug: mBU/CY
Drug: mBU/CY and ATG
Registration Number
NCT03793517
Lead Sponsor
Peking University People's Hospital
Brief Summary

Allogeneic haematopoietic stem cell transplantation (allo-HSCT) remains one of the currently available curative therapies for acute leukemia (AL). Leukemia relapse is one of the mainly causes of transplant failure. We reported previously that patients with high-risk molecular biomarkers who still have detectable minimal residual disease(MRD) pre-HSCT were at very high risk of relapse, with cumulative relapse rate of 50-80%. Decitabine has been demonstrated efficacy in the treatment of patients with recurrent or refractory leukemia and myelodysplastiv syndrome. It was reported that the combination of decitabine, with busufan and cyclophosphamide as a preparative regimen for allo-HSCT using HLA-matching donors was safe and effective. In this prospective, single-arm clinical trial, we aimed to examine the efficacy of combining decitabine with modified busulfan and cyclophosphamide (mBU/CY) as a preparative regimen for allo-HSCT in patients with very high-risk AL and detectable MRD pre-HSCT.

Detailed Description

Patients enrolled in this study would receive decitabine 200mg·m-2·d-1 on day -12 and -11 pre-HSCT. The conditioning therapy for human leukocyte antigen (HLA)-mismatched HSCT patients was modified BU/CY plus ATG (thymoglobulin; Sang Stat, France) consisting of cytarabine (Ara-C 4 g·m-2·d-1) intravenously on days -10 to -9, busulfan (BU 3.2 mg·kg-1·d-1) intravenously on days -8 to -6, cyclophosphamide (CY 1.8 g·m-2·d-1), intravenously on days -5 to -4, semustine (Me-CCNU, 250 mg·m-2), orally once on day -3, and ATG (2.5 mg·kg-1·d-1) intravenously on days -5 to -2. In matched sibling transplantations, patients received hydroxycarbamide (80 mg·kg-1) orally on day -10 and a lower dose of Ara-C (2 g·m-2·d-1) on day -9, but otherwise an identical regimen to the HLA-mismatched patients without ATG.

BM samples from patients were obtained to assess leukemia status after HSCT. The time points that we monitored BM samples included at time of allo-HSCT; 1 month, 2 months, 3 months, 4.5 months, 6 months, 9 months, and 12 months after allo-HSCT; and every 6 months thereafter to the defined endpoints or for at least until 5 years after transplantation.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
55
Inclusion Criteria
  • acute leukemia patients with MLL-r,TLS-ERG,or SIL-TAL1,whose minimal residual disease were detectable pre-HSCT
Exclusion Criteria
  • pregnancy women
  • uncontrolled severe infection

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Decitabine plus mBU/CY for matched sibling transplantmBU/CYDecitabine plus mBU/CY as precondition regimen for High Risk Acute Leukemia With MRD at the time of matched sibling transplant. Details: In matched sibling transplantations, patients received decitabine 100mg·m-2·d-1 q12h on days-12 and -11,hydroxycarbamide (80 mg/kg) orally on day -10 and a lower dose of Ara-C (2 g·m-2·d-1) on day -9, busulfan (BU 3.2 mg·kg-1·d-1) intravenously on days -8 to -6, cyclophosphamide (CY 1.8 g·m-2·d-1), intravenously on days -5 to -4, simustine (Me-CCNU, 250 mg/m2), orally once on day -3.
Decitabine plus mBU/CY for HLA-mismatched HSCTmBU/CY and ATGDecitabine plus mBU/CY as precondition regimen for High Risk Acute Leukemia With MRD at the time of HLA-mismatched HSCT. Details: The conditioning therapy for human eukocyte antigen (HLA)-mismatched HSCT patients was decitabine plus modified BU/CY and ATG,consisting of decitabine 100mg·m-2·d-1 q12h on days-12 and -11,cytarabine (Ara-C 4 g·m-2·d-1) intravenously on days -10 to -9, busulfan (BU 3.2 mg·kg-1·d-1) intravenously on days -8 to -6, cyclophosphamide (CY 1.8 g·m-2·d-1), intravenously on days -5 to -4, simustine (Me-CCNU, 250 mg/m2), orally once on day -3, and ATG (2.5 mg·kg-1·d-1) intravenously on days -5 to -2.
Decitabine plus mBU/CY for HLA-mismatched HSCTDecitabineDecitabine plus mBU/CY as precondition regimen for High Risk Acute Leukemia With MRD at the time of HLA-mismatched HSCT. Details: The conditioning therapy for human eukocyte antigen (HLA)-mismatched HSCT patients was decitabine plus modified BU/CY and ATG,consisting of decitabine 100mg·m-2·d-1 q12h on days-12 and -11,cytarabine (Ara-C 4 g·m-2·d-1) intravenously on days -10 to -9, busulfan (BU 3.2 mg·kg-1·d-1) intravenously on days -8 to -6, cyclophosphamide (CY 1.8 g·m-2·d-1), intravenously on days -5 to -4, simustine (Me-CCNU, 250 mg/m2), orally once on day -3, and ATG (2.5 mg·kg-1·d-1) intravenously on days -5 to -2.
Decitabine plus mBU/CY for matched sibling transplantDecitabineDecitabine plus mBU/CY as precondition regimen for High Risk Acute Leukemia With MRD at the time of matched sibling transplant. Details: In matched sibling transplantations, patients received decitabine 100mg·m-2·d-1 q12h on days-12 and -11,hydroxycarbamide (80 mg/kg) orally on day -10 and a lower dose of Ara-C (2 g·m-2·d-1) on day -9, busulfan (BU 3.2 mg·kg-1·d-1) intravenously on days -8 to -6, cyclophosphamide (CY 1.8 g·m-2·d-1), intravenously on days -5 to -4, simustine (Me-CCNU, 250 mg/m2), orally once on day -3.
Primary Outcome Measures
NameTimeMethod
1 year cumulative incidence of relapse1 year post allo-HSCT

The cumulative incidence of relapse at 1 year post allo-HSCT

2 year cumulative incidence of relapse2 years post allo-HSCT

The cumulative incidence of relapse at 2 years post allo-HSCT

Secondary Outcome Measures
NameTimeMethod
5 years overall survival5 years post allo-HSCT

The overall survival at 5 years post allo-HSCT

1 year leukemia free survival1 year post allo-HSCT

The leukemia free survival at 1 years post allo-HSCT

Non-relapse mortality1 year post allo-HSCT

The cumulative incidence of non-relapse mortality at 1 year post allo-HSCT

1 year overall survival1 year post allo-HSCT

The overall survival at 1 year post allo-HSCT

5 years leukemia free survival5 years post allo-HSCT

The leukemia free survival at 5 years post allo-HSCT

engraftment100 days post allo-HSCT

The total neutrophil and platelet engraftment rate

Acute graft versus host disease100 days post allo-HSCT

The cumulative incidence of grade II-IV acute graft versus host disease

Chronic graft versus host disease1 years post allo-HSCT

The cumulative incidence of intermediate to severe chronic graft versus host disease

Trial Locations

Locations (1)

Peking University Institute of Hematology,Beijing

🇨🇳

Beijing, Beijing, China

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