Hematologic Diseases
Conditions
Keywords
Hematopoietic cell transplantation, HLA mismatch related, Hematologic diseases
Brief summary
This study is a single center, prospective, single arm exploratory clinical trial that includes patients with hematological malignancies who are indicated for allogeneic hematopoietic stem cell transplantation (allo HSCT) but lack suitable donors. This project plans to use human leukocyte antigen (HLA) mismatched donors. Ultimately, a HLA mismatched allo HSCT transplantation plan will be established to improve the disease prognosis of these patients and truly enter the era of everyone has a donor.
Detailed description
This study is a single center, prospective, single arm exploratory clinical trial that includes patients with hematological malignancies who are indicated for allogeneic hematopoietic stem cell transplantation (allo HSCT) but lack suitable donors. This project plans to use human leukocyte antigen (HLA) mismatched donors. Ultimately, a HLA mismatched allo HSCT transplantation plan will be established to improve the disease prognosis of these patients and truly enter the era of everyone has a donor.
Interventions
For myeloablative conditioning: Busulfan (Bu，3.2 mg/kg/d IV -8d \ -6d) For reduced intensity conditioning: (Bu，3.2 mg/kg/d IV -7d\ -5d)
Cyclophosphamide is only used in myeloablative conditioning (Cy，1.8g/m2, -5d, -4d)
For myeloablative conditioning: Fludarabine (Flu，30 mg/m2/d IV -6d \ -2d) For reduced intensity conditioning: Fludarabine（Flu，30mg/m2 /d IV -10d\ -5d）
For both myeloablative and reduced intensity conditioning: Semustine (MECCNU，250 mg/m2 orally-3d).
Sponsors
He Huang
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* Adult patients (18-60 years old) with hematological malignancies and indications for hematopoietic stem cell transplantation with no available related human leukocyte antigen (HLA) identical sibling donors; * No available donor with HLA high-resolution typing ≥ 9/10 or those who have difficulty finding donors due to urgent medical conditions; * No suitable HLA matching haploidentical donor available; * There is a suitable donor with mismatched HLA typing; * The subjects or their legal representatives shall sign an informed consent form before the start of the clinical study.
Exclusion criteria
* Patients with severe liver and kidney function (alanine aminotransferase\>2.5 times the upper limit of normal, blood creatinine\>1.5 times the upper limit of normal) and cardiopulmonary dysfunction (New York Heart Association (NYHA) III/IV heart function, ejection fraction\<50%, severe obstructive or restrictive ventilation dysfunction); * Merge active infections; * Eastern Cooperative Oncology Group (ECOG) score ≥ 2 points; * Secondary tumors with merged activity; * Severe central nervous system or mental illness leading to the inability to autonomously choose to enter or exit clinical trials; * Combine other allo HSCT contraindications.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall survival | 1-year | Overall survival after allogeneic cell transplantation refers to the length of time a patient remains alive from the date of transplantation, regardless of disease status or complications. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Platelet engraftment rate | 28 days | Platelet engraftment rate is assessed, and platelet engraftment is defined as achievement of a platelet count of ≥ 20 × 10⁹/L (or ≥ 50 × 10⁹/L in some definitions) without transfusion support for at least seven consecutive days. |
| Relapse | 1-year | Cumulative incidence of relapse |
| Neutrophil engraftment rate | 28 days | The neutrophil engraftment rate is assessed, and neutrophil engraftment is defined as achievement of an absolute neutrophil count (ANC) of ≥ 0.5 × 10⁹/L for three consecutive days |
| Progression free survival | 1-year | Progression-free survival after allogeneic cell transplantation refers to the length of time during which a patient remains alive without any signs of disease relapse or progression. |
| GRFS | 1-year | GRFS (Graft-versus-host disease-free, relapse-free survival) after allogeneic cell transplantation is the length of time a patient remains alive without experiencing significant graft-versus-host disease, disease relapse, or death. |
| Cumulative incidence of relapse | 1-year | Progression-free survival after allogeneic cell transplantation refers to the length of time during which a patient remains alive without any signs of disease relapse or progression. |
Countries
China
Contacts
Primary ContactYanmin Zhao, MD., PhD
Backup ContactYishan Ye, MD., PhD
Outcome results
None listed