The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.
ObjectiveTo evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen in aplastic anemia. Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. We designed this study to improve the conditioning regimen and optimize the GHVD prevention measures to improve the transplant success rate and prolong patient survival, while minimizing the occurrence of GVHD and reducing the recurrence rate of the disease.30 patients with aplastic anemia were planned to be enrolled.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Conditioning regimen: fludarabine 30mg/m2×5\~6d,cyclophosphamide 50mg/kg×2d, melphalan 100mg/m2×1d aGVHD prophylaxis: PTCY 25mg/kg×2d, ATG 4.5mg/kg total, MMF 15mg/kg +5d\~+35d, Ruxolitinib 5mg bid -1d\~+50d and 2.5mg bid +51d\~+110d, CSA
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, China
RECRUITINGoverall survival (OS)
2-year overall survival (OS) and graft-versus-host disease and relapse-free survival (GRFS) after transplantation
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Acute graft-versus-host disease incidence
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Transplantation-related motality
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Chronic graft-versus-host disease incidence
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
incidence of Intensive fungal disease
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
EB virus reactivation rate
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
CMV reactivation rate
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
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