Prospective Single-Arm Clinical Study of Thiotepa-Containing Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in Chronic Myelomonocytic Leukemia
Key claim: A prospective single-arm trial is evaluating whether a thiotepa/busulfan/fludarabine (± ATG) conditioning regimen provides effective and safe allo-HSCT outcomes in intermediate-2 or high-risk CMML patients.
Abstract
This is a prospective, single-arm clinical study. It aims to evaluate the effectiveness and safety of a conditioning regimen containing thiotepa (in combination with busulfan and fludarabine, with or without ATG) of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with chronic myelomonocytic leukemia (CMML) who have an intermediate-2 or high-risk prognosis.
The main goal is to see 1 year RFS and OS. Other goals include assessing engraftment, overall survival, transplant-related complications, and side effects. A total of 31 participants will be enrolled.
Why this matters
Conditioning evidence specific to CMML is thin, so once a higher-risk CMML patient clears the decision-layer threshold for transplant, regimen selection is largely extrapolated from AML/MDS data. A prospective trial of a thiotepa-intensified FluBu backbone gives the execution layer disease-specific data, and — if effective — could lower the perceived toxicity ceiling that currently pushes some CMML referrals later than risk scores would suggest.