Clinical trial NCT06069180: The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA
Official title: To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Severe Aplastic Anemia
Keywords: HLA matched donor transplantation
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2023-10-05
Enrollment: 160
Sex: ALL
Minimum age (years): 1
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previous studies have shown that Fludarabine (Flu)/ Cyclophosphamide (Cy)/ antithymocyte globulin (antithymocyte globulin), ATG) and Cy/ATG conditioning regimens had higher rates of mixed chimerism and poorer failure-free survival. A small cohort study has suggested that adding busulfan to Flu/Cy/ATG or Cy/ATG can reduce the incidence of mixed chimerism and improve failure-free survival. This study was a prospective, multicenter, randomized controlled trial to compare the efficacy and safety of different conditioning regimens in the treatment of severe aplastic anemia (SAA) after hematopoietic stem cell transplantation (HSCT) from HLA-identical sibling or unrelated donor.


Eligibility criteria: Inclusion Criteria:

1. Diagnosed as SAA/vSAA
2. Indication for hematopoietic stem cell transplantation
3. Available HLA matched sibling or unrelated donor
4. No active infection
5. No serious organ damage: liver and kidney function (ALT and AST \< 2.5 times normal value, normal renal function, no cardiac insufficiency)
6. Signed informed consent
7. High risk factors of mixed chimerism, at least one of the following

   1. Age \< 18 years old
   2. Ferritin level ≥2500ng/ml before transplantation

Exclusion Criteria:

1. Age \> 50 years old
2. ECOG≥3
3. Active infections that were difficult to control
4. Severe liver and kidney dysfunction
5. Mental illness
6. Not signing the informed consent
7. pregnant or lactating women
8. Any condition considered by the investigators to be unsuitable for enrollment

Primary outcomes:
measure: Failure free survival
timeFrame: 1 year post HSCT
Secondary outcomes:
measure: The incidence of mixed chimerism
timeFrame: 1 year post HSCT
measure: Regimen related toxicity
timeFrame: 100 days post HSCT
measure: Myeloid and platelet engraftment
timeFrame: 100 days post HSCT
measure: The incidence of graft versus host disease
timeFrame: 100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD
measure: The incidence of CMV and EBV reactivation
timeFrame: 100 days post HSCT
measure: The incidence of Transplantation related mortality
timeFrame: 1 year post HSCT
measure: The probability of Overall survival
timeFrame: 1 year post HSCT
Other outcomes:
Locations:
city: Beijing
country: China
facility: Deparment of Hematology, Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University People's Hospital
name: Xiao-Jun Huang
role: PRINCIPAL_INVESTIGATOR
References: