Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia

ConditionSevere Aplastic Anemia

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age3-65

SponsorHematology department of the 920th hospital

About this trial

The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Patients with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, aplastic anemia, etc. diagnosed by NCCN guidelines, and requiring allogeneic hematopoietic stem cell transplantation as determined by the researchers;

Age 3-65 years old;

Weight 10Kg-100Kg;

Eastern Cooperative Oncology Group (ECOG) score ≤3;

Disqualifiers

patients with nonhematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;

patients with an expected survival of less than 1 month;

patients with previous autologous or allogeneic hematopoietic stem cell transplantation;

pregnant patients;

Trial design

Design model

Single group

Treatments tested in this trial

Modified transplantation system
Drug
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

Treatment groups

30 Participants

are divided into 1 treatment group

Group A: Experimental groupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

overall survival (OS)

2-year overall survival (OS) and graft-versus-host disease and relapse-free survival (GRFS) after transplantation

Time frame