Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma
Status:
Not yet recruiting
Trial end date:
2027-03-01
Target enrollment:
Participant gender:
Summary
Neuroblastoma (NB) is the most common extracranial solid tumor of embryonal origin in
children. According to the International Neuroblastoma Risk Group (INRG) staging criteria and
the International Neuroblastoma Staging System (INSS) ，NB preoperative staging is divided
into L1, L2, M and Ms stages, the postoperative staging is divided into 1 to 4 stages and 4s
stage. Among them, 4/M stage is of the highest degree of malignancy and the worst prognosis.
Despite the aggressive combination therapy, the 5-year survival rate (OS) is still less than
15%, and the 2-year relapse rate is 80%. Currently, no effective treatment is accessible for
refractory/relapsed stage 4/M NB after completing conventional therapy.
In hematopoietic stem cell transplantation (HSCT) , conditioning regimen with high-dose
radiotherapy and chemotherapy is implemented to eradicate tumor cells and abnormal clonal
cells in the patient, block the pathogenesis, and restore the patient's hematopoietic and
immune systems by transplanting normal hematopoietic stem cells. According to the source of
hematopoietic stem cells, HSCT can be divided into two types: autologous hematopoietic stem
cell transplantation (auto-HSCT) and allogeneic hematopoietic stem cell transplantation
(allo-HSCT). It has been confirmed that benefiting from the graft versus tumor(GVT) effect,
allo-HSCT can clear residual lesions in refractory/relapsed NB patients post-auto-HSCT，and
prolong the survival time of patients. Our center has explored the conditioning regimen,
treatment of residual tumor lesions before transplantation, and strategies to reduce
transplantation-related death (TRM) and enhance the GVT effect. However, the sample size is
small, and multicenter and larger sample size research are needed. This study will further
observe the clinical efficacy and safety of allo-HSCT in the treatment of 4/M stage NB, and
provide a new treatment method and option for 4/M stage NB.
Phase:
Phase 2
Details
Lead Sponsor:
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University