Autologous Hematopoietic Stem Cell Transplantation, Delayed Platelet Engraftment, Neuroblastoma
Conditions
Keywords
Hetrombopag, delayed platelet engraftment, neuroblastoma, pediatrics, autologous hematopoietic stem cell transplantation
Brief summary
The goal of this clinical trial is to learn if hetrombopag promotes platelet engraftment in neuroblastoma children undergoing autologous hematopoietic stem cell transplantation. The main question it aims to answer is: · Does drug hetrombopag promote platelet engraftment? Participants will take hetrombopag every day for 6 weeks or until platelet > 100\*10\^9/L.
Interventions
DRUGHetrombopag
Patients enrolled were administered Hetrombopag starting from +4d after transplantation on routine medication of recombinant human thrombopoietin injection (rhTPO). The starting dose of Hetrombopag is 2.5mg or 5mg, oral, once daily (starting at 5mg/d for body weight\>20kg; starting from 2.5mg/d for body weight ≤ 20kg). Adjust the dosage based on platelet count every two weeks, with a maximum dose of 7.5mg per day. Stop the medication after 6 weeks of continuous use or when PLT reaches 100 × 10\^9/L.
Sponsors
Beijing Children's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
Inclusion criteria
* Age\<18 years old; * Diagnosed as neuroblastoma; * ECOG ≤ 2; * First time receiving autologous hematopoietic stem cell transplantation; * The subjects or their legal guardians agree to participate and sign the informed consent form.
Exclusion criteria
* ALT/AST \> 3 ULN, or TBIL \> 1.5 ULN; * Have experienced congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring medication treatment within one year prior to enrollment, or have experienced myocardial infarction or cerebral infarction within three months prior to enrollment; * Suffering from thromboembolic diseases; * Other situations that are not suitable for inclusion in the study determined by researchers.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Accumulated platelet engraftment rate | From enrollment to 60 days post-transplant | Platelet engraftment is defined as a continuous 7-day PLT ≥ 20 × 10\^9/L without platelet infusion. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of platelet transfusions | From enrollment to 60 days post-transplant | — |
| Time to platelet engraftment | From enrollment to 60 days post-transplant | — |
| progression-free survival (PFS) | From enrollment to 60 days post-transplant | The time from enrollment to the first occurrence of disease progression or death from any cause |
| Adverse events | From medication to 30 days after discontinuation | Any adverse events within 30 days of discontinuation |
Countries
China
Contacts
Primary ContactDr. Yang
Outcome results
None listed