Aplastic Anaemia, Hematopoietic Cell Transplant, Severe Aplastic Anemia
Conditions
Brief summary
The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.
Interventions
DRUGBFCA conditiong regimen
busulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2).
Sponsors
Peking University People's Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
40 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* Severe aplastic anemia; * Aged 40-60 years old; * Weight 45Kg-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
* With other hematologic diseases; * Expected survival of less than 1 month; * 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. * Current or recent (\<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection * A history of symptomatic herpes zoster infection within 12 weeks prior to screening * Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) * Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB * Exposure to a live vaccine within 12 weeks prior to enrollment or expected to receive a live vaccine during the study * Clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled * Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure * A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data * Any of the following specific abnormalities on screening laboratory tests: 1. ALT or AST \>2 x ULN, or total bilirubin ≥1.5 x ULN 2. hemoglobin \<9 g/dL, or total white blood cell (WBC) count \<2,500/µL, or neutropenia (absolute neutrophil count \<1,200/µL), or lymphopenia (lymphocyte count \<750/µL) 3. eGFR \<50 mL/min/1.73 m2
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Faliure free survival (FFS) | From enrollment to 2 years post-HSCT | survival with a response to therapy after HSCT. Death, graft faliue and relapse were considered as treatment failure. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The probability of Overall survival | From enrollment to 2 years post-HSCT | Overall survival was defined as the time from transplantation to death from any cause or to the last follow-up |
| Myeloid and platelet engraftment | 100 days post HSCT | Myeloid and platelet engraftment were defined as international criteria. |
| The incidence of mixed chimerism | 1 year post HSCT | The mixed chimerism was defined as the presence of 5%-95% donor haematopoietic cells. |
| The incidence of graft versus host disease(GVHD) | 100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD | The severity of acute and chronic GVHD was evaluated according to standard criteria. |
| Regimen related toxicity | 100 days post HSCT | The regimen related toxicity (RTT) was measured according to the Seattle Toxicity Criteria (Bearman et al, 1988). |
| The incidence of Cytomegalovirus(CMV) and Epstein-Barr virus(EBV) reactivation | 180 days post HSCT | The incidence of CMV and EBV reactivation was defined as CMV and EBV viremia. |
| The incidence of Transplantation related mortality | 2 years post HSCT | Transplantation related mortality was defined as death without disease progression. |
Countries
China
Contacts
CONTACTTingting Han
Outcome results
None listed