This study is a multicenter, single-arm, prospective phase II clinical trial that evaluates the efficacy and safety of an intensive conditioning regimen with thiotepa combined with busulfan, fludarabine, and cytarabine for allogeneic hematopoietic stem cell transplantation in the treatment of myeloid malignancies with extramedullary involvement. The conditioning regimen includes thiotepa at a dose of 5mg/kg/d from d -9 to d -8 (2 days), fludarabine at 30mg/m2/d from d -7 to d -3 (5 days), cytarabine at 1-1.5g/m2/d from d -7 to d -3 (5 days), and busulfan at 3.2mg/kg/d from d -5 to d -3 (3 days). Conditioning begins on day -9, and donor hematopoietic stem cell infusion is performed on day 0. All patients will undergo bone marrow examination on day 14 and day 28 post-transplant, followed by bone marrow examinations every 30 days within the first year after transplantation, and every 60 days within the second year after transplantation. If disease relapse is suspected during the follow-up period, bone marrow or extramedullary relapse site examinations will be conducted at any time. The primary study endpoints are the 1-year and 2-year progression-free survival (PFS) rates post-transplant. Secondary study endpoints include the incidence of acute graft-versus-host disease (GVHD) within 180 days post-transplant, cumulative relapse rates at 1 year and 2 years post-transplant, 1-year and 2-year overall survival (OS), graft-versus-host disease-free, relapse-free survival (GRFS), non-relapse mortality (NRM), cumulative incidence of chronic GVHD, and the incidence of Cytomegalovirus (CMV)and Epstein-Barr virus(EBV)reactivation within 1 year.
Study Type
OBSERVATIONAL
Enrollment
50
Shanghai General Hospital
Shanghai, Shanghai Municipality, China
RECRUITING1y and 2y-PFS
1-year and 2-year progression-free survival (PFS) rates post-transplant
Time frame: 2023-2027
aGVHD
acute graft-versus-host disease (GVHD) within 180 days post-transplant
Time frame: 2023-2025
1y and 2y-CIR
cumulative relapse rates (CIR) at 1 year and 2 years post-transplant
Time frame: 2023-2027
1y and 2y-OS
overall survival (OS) at 1 year and 2 years post-transplant
Time frame: 2023-2027
GRFS
graft-versus-host disease-free, relapse-free survival (GRFS) at 2 years post-transplant
Time frame: 2023-2027
NRM
non-relapse mortality (NRM) at 2 years post-transplant
Time frame: 2023-2027
cGVHD
cumulative incidence of chronic GVHD at 2 years post-transplant
Time frame: 2023-2027
CMV and EBV reactivation
the incidence of CMV and EBV reactivation within 1 year
Time frame: 2023-2026
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.