Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

Study Overview
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Brief Summary
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The haplotype HSCT system including Bu(0.8mg/kg Q6hx2d)CTX(50mg/kgx4d)rATG(2.5mg/kgx4d) , established in Institute of Hematology of Peking University ,has been evaluated to be effective for acquired SAA.But some patients with high risk factors may not tolerate CTX 200mg/kg,alternative conditioning regimen including Bu/Fludarabine/dercreased CTX was studied in this trial.

Detailed Description
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Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d. BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that investigators monitor BM or blood samples at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.

Official Title
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Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

Conditions
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Aplastic Anemia, Stem Cell Transplant Complications, Engraft Failure

Intervention / Treatment
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* Drug: Fludarabine


Participation Criteria
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Eligibility Criteria
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A：inclusion criteria Patients diagnosed as acquired severe aplastic anemia(SAA) /very vSAA patients with age 3-55 years patients have no matched sibling donor Patients have no matched unrelated donor patients have no severe infection Patients have no severe organ dysfunction patients have risk factors of potential intolerance to previous condition regimen including BuCy(200mg/kg)and ATG Consent form signed B. Exclusion criteria ： patients with congenital SAA/vSAA patients with age< 3years or >55 years patients with matched sibling donor patients with matched URD patients with severe infection patients with severe organ dysfunction pregnancy women no Consent form signed

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d. BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BFCA regimen<br>Detail: Patients enrolled in this study would receive Busulfan(B) (IV)0.8mg/kg Q6hx2d,Fludarabine(F) 30mg/m2x5d ,cyclophosphamide(C) 25mg/kg/dx4d,thymoglobulin (A :rATG ,Sang Stat,France) 2.5mg/kg/dx4d. BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT. | Drug: Fludarabine<br>* Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX (cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.<br>* Other names: Thymoglobulin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 1 year cumulative incidence overall survival | Tne cumulative incidence of overall survival at 1 year post HSCT | 1 year post HSCT | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| one month Transplantation related mortality | Tne cumulative incidence of transplantation related motality at 1 month post HSCT | 1 month post HSCT | 
| Engraftment | Tne cumulative incidence of engraftment at 1 moths post HSCT | 1 month post HSCT | 
| 0ne month regimen-related toxicity | Tne cumulative incidence of regimen related toxicity at 1 month post HSCT | 1 month post HSCT | 
| aGVHD | Tne cumulative incidence of acute GVHD at Day 100 post HSCT | 100 days post HSCT | 
| failure-free survival | The cumulative incidence of failure-free survival at 1year post HSCT | 1 year post HSCT | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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haploidentical transplantation, aplastic anemia