Aplastic Anemia, Engraft Failure, Stem Cell Transplant Complications
Conditions
Keywords
haploidentical transplantation, aplastic anemia
Brief summary
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
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.
Interventions
DRUGFludarabine
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.
Sponsors
Peking University People's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention 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.
Eligibility
Sex/Gender
ALL
Age
3 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
A：inclusion criteria 1. Patients diagnosed as acquired severe aplastic anemia(SAA) /very vSAA 2. patients with age 3-55 years 3. patients have no matched sibling donor 4. Patients have no matched unrelated donor 5. patients have no severe infection 6. Patients have no severe organ dysfunction 7. patients have risk factors of potential intolerance to previous condition regimen including BuCy(200mg/kg)and ATG 8. Consent form signed B.
Exclusion criteria
： 1. patients with congenital SAA/vSAA 2. patients with age\< 3years or \>55 years 3. patients with matched sibling donor 4. patients with matched URD 5. patients with severe infection 6. patients with severe organ dysfunction 7. pregnancy women 8. no Consent form signed
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 1 year cumulative incidence overall survival | 1 year post HSCT | Tne cumulative incidence of overall survival at 1 year post HSCT |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 0ne month regimen-related toxicity | 1 month post HSCT | Tne cumulative incidence of regimen related toxicity at 1 month post HSCT |
| one month Transplantation related mortality | 1 month post HSCT | Tne cumulative incidence of transplantation related motality at 1 month post HSCT |
| Engraftment | 1 month post HSCT | Tne cumulative incidence of engraftment at 1 moths post HSCT |
| aGVHD | 100 days post HSCT | Tne cumulative incidence of acute GVHD at Day 100 post HSCT |
| failure-free survival | 1 year post HSCT | The cumulative incidence of failure-free survival at 1year post HSCT |
Countries
China
Contacts
Primary ContactXiaojun Huang, Prof.
Backup ContactLanping Xu
Outcome results
None listed