Safety and Efficacy of Unrelated Umbilical Cord Blood Microtransplantation in Patients With Higher-risk MDS
Study Details
Study Description
Brief Summary
This study aimed to evaluate the safety and efficacy of unrelated umbilical cord blood microtransplantation in the treatment of above-mentioned MDS patients by observing the factors related to the efficacy and adverse reactions.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: chemotherapy+Unrelated Umbilical Cord Blood microtransplantation Intermittent infusion of HLA mismatched or incompletely matched granulocyte colony-stimulating factor (G-CSF) mobilized peripheral blood stem cells (G-PBSC) during routine chemotherapy without the use of immunosuppressants, abbreviated as "microtransplantation" |
Biological: Unrelated Umbilical Cord Blood
The requirements of unrelated umbilical cord blood: recipient-donor HLA match is 0-3/10, same blood type.
Drug: Venetoclax
Patients will be treated with Venetoclax (100 mg on day 1, 200 mg on day 2 and 400 mg on days 3 to 21)
Drug: Decetabine
Patients will be treated with Decetabine (20mg/m^2/d on days 1 to 5) or Azacitidine (75mg/m^2/d on days 1 to 7)
Drug: Azacitidine
Patients will be treated with Decetabine (20mg/m^2/d on days 1 to 5) or Azacitidine (75mg/m^2/d on days 1 to 7)
|
Outcome Measures
Primary Outcome Measures
- Complete response (CR) rate [28±7 days]
CR rate is defined as the percentage of patients who met the following conditions: Myelogram: naïve cells less than 5% (at least 200 nucleated cells); Hemogram: absolute neutrophil count > 1.0×10^9/L, platelet count > 100×10^9/L; Clinical diagnosis: no symptoms and signs caused by leukemia infiltration, and patients do not rely on blood transfusion.
- Hematopoietic recovery time [28±7 days]
The time of absolute neutrophil count>0.5×10^9/L and platelet count >30×10^9/L for 3 consecutive days.
Secondary Outcome Measures
- Time to Progression（TTP） [1 year]
Time from enrollment to objective progression of disease
- Disease Free Survival（DFS） [1 year]
From CR to recurrence or death or to the date of last follow-up
- Overall Survival（OS） [1 year]
From the beginning of treatment to death or to the date of last follow-up
- Early mortality rate [3 months]
Death within the first 3 months of induction therapy
Other Outcome Measures
- Changes in lymphocyte subsets (NK cells, T cells) before and after treatment [At the time of enrollment and at 1 month]
The concentration of lymphocyte subsets in peripheral blood can be detected by flow cytometry
- Correlation between human leukocyte antigen (HLA) matching and the concentration of lymphocyte subsets [HLA typing and matching will be assessed at the time of enrollment, and the concentration of lymphocyte subsets in peripheral blood will be detected at the time of enrollment and at 1 month]
HLA typing and matching can be assessed by sequence based genotyping (PCR-SBT), and the concentration of lymphocyte subsets in peripheral blood can be detected by flow cytometry
Eligibility Criteria
Criteria
Inclusion Criteria:
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Patients diagnosed with MDS with WHO criteria through bone marrow morphology, histochemistry, immunophenotyping, pathological testing, etc. Patients can be newly diagnosed, recurrent or unresponsive, with International prognostic scoring system (IPSS-R) score> 3.5
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Patients aged 14-80, gender and race are not limited;
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Karnofsky score ≥ 60%, Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2;
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Expected survival time ≥ 3 months;
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The examination results meet the following requirements:
ALT and AST ≤ 3 × Upper limit of normal value (ULN); Total bilirubin ≤ 3 × ULN; Creatinine ≤ 2 × ULN or creatinine clearance rate ≥ 40mL/min; The left ventricular ejection fraction (LVEF) measured by echocardiography or multigated acquisition (MUGA) scanning is within the normal range (>50%);
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The recipient and selected donor should be 0-3/10 HLA-matched with same blood type;
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Patients who voluntarily participate in this clinical study and have signed an informed consent.
Exclusion Criteria:
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Patients who have suffered from malignant tumors;
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Patients have suffered from hematopoietic failure after chemotherapy, and have undergone ineffective blood transfusion with unknown cause;
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Patients who have undergone Class II or above surgery within 4 weeks prior to enrollment;
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Suffering from life-threatening diseases other than MDS;
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Allergic to the drugs in the research;
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Patient with severe cardiac insufficiency, including uncontrolled or symptomatic arrhythmia, congestive heart failure, myocardial infarction within 6 months, or any grade 3 (moderate) or grade 4 (severe) heart disease;
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Patients with test positive for HIV, HCV or HBV;
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Stroke or intracranial hemorrhage occurred within 6 months prior to enrollment;
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Warfarin or vitamin K antagonists (such as phenprocoumarin) is necessary for anticoagulation;
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Patients with mental illnesses or cognitive impairments;
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Patients have participated within the month prior to enrollment or patients are currently participating in other clinical trials;
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There are other conditions that the investigators consider inappropriate for inclusion.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | The First Affiliated Hospital of University of Science and Technology of China | Hefei | Anhui | China | 230001 |
Sponsors and Collaborators
- Anhui Provincial Hospital
Investigators
- Principal Investigator: Xiaoyu Zhu, MD, The First Affiliated Hospital of University of Science and Technology of China
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- UCB-MST&MDS