Prospective Single-Arm Clinical Study of Thiotepa-Containing Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in Chronic Myelomonocytic Leukemia
Sun Yuqian
31 participants
Feb 13, 2026
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria17
- Age ≥ 18 years, any sex/gender.
- Confirmed diagnosis of chronic myelomonocytic leukemia (CMML) according to the 2022 WHO classification.
- Intermediate-2 or high-risk CMML based on CPSS or CPSS-mol score, and planned to receive allo-HSCT.
- Has a suitable hematopoietic stem cell donor:
- For haploidentical donor: at least 5/10 HLA match at HLA-A, -B, -C, -DQB1, and -DRB1.
- For unrelated donor: at least 9/10 HLA match at the same five loci.
- For matched sibling donor: 10/10 HLA match at the same five loci.
- Hematopoietic cell transplantation comorbidity index (HCT-CI) ≤ 2, with generally good health and no significant organ abnormalities or major comorbidities.
- Adequate organ function as defined below:
- Left ventricular ejection fraction (LVEF) ≥ 50%, and no uncontrolled tachycardia or bradycardia-tachycardia syndrome.
- Total bilirubin ≤ 1.5 × upper limit of normal (ULN); ALT ≤ 2 × ULN; AST ≤ 2 × ULN.
- Serum creatinine ≤ 1.5 × ULN.
- Baseline oxygen saturation > 92%.
- Pulmonary function: DLCO (corrected for hemoglobin) ≥ 40%, FEV1 ≥ 50%.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
- Agrees not to participate in any other interventional study during the treatment period.
- Willing and able to provide written informed consent, understand the nature, purpose, and procedures of the study, and voluntarily comply with study requirements.
Exclusion Criteria8
- Previous allogeneic HSCT for CMML that later relapsed.
- Unwilling or unable to receive the study treatment regimen.
- Active hepatitis B or C, or chronic active hepatitis; known human immunodeficiency virus (HIV) infection.
- Active uncontrolled infection, including: hemodynamic instability related to infection, new or worsening signs/symptoms of infection, new infection lesions on imaging, or persistent fever without explanation despite no symptoms/signs.
- History of stroke or intracranial hemorrhage within 6 months before enrollment.
- Known pregnancy (positive urine pregnancy test), or currently breastfeeding.
- Diagnosis of another malignancy within the past 2 years, except for localized skin cancer, superficial bladder cancer, carcinoma in situ of the cervix, breast cancer, or localized prostate cancer (Gleason score ≤ 6) that has been treated with curative intent.
- Any other condition that, in the investigator's judgment, makes the patient unsuitable for study participation.
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Interventions
Intravenous thiotepa 5 mg/kg/d on days -11,-10; busulfan 3.2 mg/kg/d on days -8,-7,-6; fludarabine 30 mg/m²/d on days -6 to -2; ATG per donor type (haplo/unrelated: 2.5 mg/kg/d days -5 to -2; MSD: 1.125 mg/kg/d days -5 to -2). Allogeneic stem cells infused on day 0. GVHD prophylaxis: CsA, MMF, MTX per protocol.
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT07588594