Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Modified transplantation system.
- Who it may be relevant to
- Registry conditions: Severe Aplastic Anemia. Basic parameters: 3 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Prospective, Multi-center, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
Overview
The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.
Detailed description
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.
Interventions
- Drug Modified transplantation system
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
Primary outcome measures
- overall survival (OS) [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]]
Secondary outcome measures (5)
- 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]]
Eligibility criteria
Inclusion criteria
- 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;
- No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
- No severe infection;
- Subjects voluntarily participated in this clinical trial and signed the informed consent.
Exclusion criteria
- 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;
- patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
- other conditions that the investigator determines to be inappropriate for enrollment.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China — Kunming
Identifiers
NCT: NCT06378060 · KM04