Hematopoietic Stem Cell Transplantation
Conditions
Keywords
HSCT, busulfan, PPK, EFS
Brief summary
Objectives To evaluate the correlation between BU exposure and post-transplant clinical results (efficacy and safety) to establish the optimal BU treatment window for myeloablative conditioning in Chinese pediatrics, provide theoretical basis and the new strategy for BU individualized dosage, further optimize transplant treatment and reduce drug-related toxicity. Population 500 participants of any sex between the age of 0.1 and 18 years. Patients receiving the BU-based myeloablative conditioning before transplantation. Endpoint primary To establish BU pop-PK model and analyze the association between BU AUC and event-free survival (EFS)or overall survival (OS) after transplantation in Chinese pediatrics. Secondary The investigators are also interested in transplantation-related mortality (TRM), acute toxicity and chronic GvHD.
Detailed description
Objectives To evaluate the influence factors of BU metabolism in children and the correlation between BU exposure and post-transplant clinical results (efficacy and safety) to establish the optimal BU treatment window for myeloablative conditioning in Chinese pediatrics, provide theoretical basis and the new strategy for BU individualized dosage, further optimize transplant treatment and reduce drug-related toxicity. Endpoint primary The main study endpoints are event-free survival (EFS) and overall survival (OS). EFS is calculated from the time of transplant until death, relapse of disease, or graft failure (defined as non-engraftment or rejection), whichever occurred first. OS is the time between transplantation and death of any cause. All surviving patients are censored at day of last contact. Duration of follow-up is the time from transplant to the last assessment for surviving patients or death. Secondary The investigators are also interested in transplantation-related mortality (TRM), acute toxicity and chronic GvHD. TRM is defined as death unrelated to underlying disease. Acute toxicities are defined as VOD (diagnosed according to modified Seattle criteria), acute GvHD grade II-IV (diagnosed and graded according to Mount Sinai Acute GvHD International Consortium (MAGIC) criteria, oral mucositis (grade II-IV) and hemorrhagic cystitis (grade II-IV). Two of the most commonly utilised scales for oral mucositis are the WHO and NCI-CTCAE scales. Chronic GvHD should be graded as mild, moderate or severe according to the National Institutes of Health (NIH) consensus criteria.
Interventions
DRUGBusulfan
The BU administration regimen was four times a day (Q6h), continuous intravenous infusion for 2 hours, 3 or 4 days in a row.
Sponsors
Children's Hospital of Soochow University
Children's Hospital of Fudan University
Fujian Medical University Union Hospital
Guangzhou Women and Children's Medical Center
West China Second University Hospital
Beijing Children's Hospital
Shenzhen Children's Hospital
The First Affiliated Hospital of Zhengzhou University
The First Affiliated Hospital of Soochow University
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
0 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. The subjects or their legal guardians voluntarily sign the informed consent, and can complete the study in accordance with the requirements of the program; 2. No age or gender restriction; 3. Patients with blood disease confirmed by histopathology or cytology; 4. BU-based regimen will be adopted. The BU administration regimen was four times a day (Q6h), continuous intravenous infusion for 2 hours, 3 or 4 days in a row.
Exclusion criteria
1. Patients with difficulty in vein blood sampling (if there is a needle, blood history); 2. BU TDM was not performed during the treatment; 3. Subjects who are considered unfit to participate in the trail by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| event-free survival (EFS) | 2 years after administration of busulfan | EFS is calculated from the time of transplant until death, relapse of disease, or graft failure (defined as non-engraftment or rejection), whichever occurred first. |
| overall survival (OS) | 2 years after administration of busulfan | OS is the time between transplantation and death of any cause. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| transplantation-related mortality (TRM) | 2 years after administration of busulfan | TRM is defined as death unrelated to underlying disease. |
| acute toxicity | 2 years after administration of busulfan | Acute toxicities are defined as VOD (diagnosed according to modified Seattle criteria),, acute GvHD grade II-IV (diagnosed and graded according to Mount Sinai Acute GvHD International Consortium (MAGIC) criteria, oral mucositis (grade II-IV) and hemorrhagic cystitis (grade II-IV). Two of the most commonly utilised scales for oral mucositis are the WHO and NCI-CTCAE scales. |
| chronic GvHD | 2 years after administration of busulfan | Chronic GvHD should be graded as mild, moderate or severe according to the National Institutes of Health (NIH) consensus criteria |
Countries
China
Contacts
Primary ContactChenrong Huang, PhD
Backup ContactXiaohuan Du, MS
Outcome results
None listed