The Optimization of Haploidentical Hematopoietic Stem Cell Transplantation
- Conditions
- Hematological Malignancy
- Interventions
- Registration Number
- NCT05629260
- Lead Sponsor
- Peking University People's Hospital
- Brief Summary
The goal of this observational study is to compare the incidence of relapse in G-CSF/ATG based and PT-Cy based haploidentical transplantation\] in \[patients aged 18 to 55 years with a diagnosis of hematological malignancies who unmanipulated haplo-HSCT with myeloablative conditioning\]. The main question it aims to answer are:
Primary objective: To compare the incidence of relapse in G-CSF/ATG based and PT-Cy based haploidentical transplantation and illustrate the possible immune mechanism.
Secondary objectives: To compare CMV infection, GVHD and survival outcomes, and to observe the dynamic immune reconstitution of G-CSF/ATG based or PT-Cy based model.
Exploratory objectives: To compare the long-term quality of life among recipients who receive G-CSF/ATG based or PT-Cy based protocol.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 300
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Subjects diagnosed as acute leukemia with transplant indications in ≤ CR2;
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Lack of available, HLA-identical, related sibling or unrelated donor;
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Female or male, age: 18-55 years old;
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ECOG performance status 0-2;
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Adequate organ function as defined by the following criteria:
Serum aspartate transaminase (AST) and serum alanine transaminase (ALT) ≤2.5× upper limit of normal (ULN), or AST and ALT ≤5× ULN if liver function abnormalities are due to underlying malignancy Total serum bilirubin≤1.5× ULN Serum creatinine≤2.5× ULN
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Signed and dated informed consent document indicating that the patient (or legally acceptable representative) has been informed of all the pertinent aspects of the trial prior to enrollment;
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Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures.
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Uncontrollable active infection;
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Severe organic impairment: hepatic and renal impairment;
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Any of the following within 6 months prior to starting study treatment: myocardial infarction, severe/unstable angina, congestive heart failure, or cerebrovascular accident including transient ischemic attack;
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Pregnancy or breastfeeding;
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Psychiatric disorders;
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Don't sign the informed consent;
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Prior/concurrent clinical study experience;
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Other conditions:
- Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
- Participants are employees of the clinical study site or other individuals directly involved in the conduct of the study, or immediate family members of such individuals (in conjunction with section 1.61 of the ICH-GCP Ordinance E6)
- Any specific situation during study implementation/course that may rise ethics considerations
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description PT-Cy group Post-transplantation cyclophosphamide Patients in PT-Cy group received a Bu/Flu/Cy-based conditioning regimen as follows: Flu (40 mg/m2 or 1 mg/kg IV) on days -8 to -4; Bu (3.2 mg kg/ d-1 IV) on days -7 to -4; Cy (14.5-40 mg/kg IV) on days -3 to -2;±cytarabine (3 g/m2/d IV) on days -9 to -8; and ± IDA (15 mg/m2/d IV) on days -9 to -8. The addition of IDA and/or cytarabine depended on the performance status of the patients and whether the patients were in relapse status. High-dose PTCy (50 mg/kg/d IV) was administered on days +3 and +4. G-CSF/ATG group ATG Patients in the G-CSF/ATG group received a modified Bu/Cy plus ATG conditioning regimen as follows: cytarabine (4/g m2 per day IV.) on days -10 to -9; Bu (3.2 mg/kg per day IV.) on days -8 to -6; Cy (1.8 g/m2 per day IV.) on days -5 to -4; methyl chloride hexamethylene urea nitrate (Me-CCNU) (250 mg/m2 per day orally) once on day -3; and ATG (2.5 mg/kg per day IV; rabbit, Sang Stat, Lyon, France) on days -5 to -2.
- Primary Outcome Measures
Name Time Method Percent of participants with disease relapse 1 year The cumulative incidence of relapse of the primary disease.
- Secondary Outcome Measures
Name Time Method Transplantation-related mortality 1 year Death due to causes unrelated to the underlying disease
Incidence of CMV disease 6 months The cumulative incidences of CMV disease in participants after transplantation
Cumulative incidences of aGVHD 100 days The diagnosis and grading of aGVHD are based on the modified Glucksberg grading standard.
Cumulative incidences of cGVHD 1 year Chronic GVHD can be classified as "limited" or "extensive" according to the Seattle criteria, and also be classified as "mild" or "moderate" or "severe" according to the National Institutes of Health (NIH) criteria.
Percent of participants with overall survival 1 year Overall survival (OS) is defined as the time from randomization to death resulting from any cause.
Dynamic immune reconstitution 1 year The main immune cell subsets include: T cell, B cell, NK cell, and Monocytes
Neutrophil engraftment 1 month Neutrophil engraftment is defined as the first of 3 consecutive days with an absolute neutrophil count \> 0.5 × 10\^9/L.
Platelet engraftment 1 month Platelet engraftment is defined as the first of 7 consecutive days with an absolute platelet count \> 20 × 10\^9/L independent from transfusion
Trial Locations
- Locations (1)
People's Hospital of Peking University
🇨🇳Beijing, Beijing, China