Haploidentical Transplantation in Severe Aplastic Anemia
- Conditions
- Severe Aplastic Anemia
- Interventions
- Other: MSD-HSCTOther: HFD-HSCT
- Registration Number
- NCT03246178
- Lead Sponsor
- Wu Xiaoxiong
- Brief Summary
This is a prospective case-control study on SAA patients treated with HSCT, order to further discuss and assess the safety, feasibility and effectiveness of HFD-HSCT which performed with reduced-intensity fludarabine-based conditioning regimen.Our findings would indicate that SAA patients who lack MSD benefited most if HFD-HSCT was performed with reduced-intensity fludarabine-based conditioning regimen, and our improved outcomes with HFD-HSCT may lead to a salvaged therapy and an expanded direct role for SAA in the future.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 60
(i) Diagnosis of SAA, very SAA or SAA and paroxysmal nocturnal hemoglobinuria (PNH) according to the International Aplastic Anemia Study Group; (ii) SAA patients no response to previous IST; (iii) adequate performance status [Eastern Cooperative Oncology Group (ECOG) score 0-2].
(i) Congenital forms of aplastic anemia; (ii)Patients with any severe pulmonary, cardiac, liver, or renal diseases or active infection.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description MSD-HSCT MSD-HSCT This group received treatment of matched sibling donor - hematopoietic stem cell transplantation (MSD-HSCT). HFD-HSCT HFD-HSCT This group received treatment of haploid family donor - hematopoietic stem cell transplantation (HFD-HSCT).
- Primary Outcome Measures
Name Time Method Engraftment In the first months after infusion Neutrophil engraftment was defined as the first of three consecutive days in which the neutrophil counts (ANC) exceeded 0.50 × 109/L, and platelet engraftment was defined as the first of five consecutive days in which the platelet count exceeded 20 × 109/L without transfusion. GF was classified as follows: (1) primary non-engraftment (failure to reach a neutrophil count of 0.5×109/L after transplant); (2) rejection (decrease in blood counts to \< 0.5×109/L neutrophils, after achieving a neutrophil count of 0.5×109/L); (3) late graft failure (decrease of blood counts after day 100 to \< 1.0×109/L neutrophils and \< 30×109/L platelets).
Toxicity grading TRT was defined as toxic effects occurring within 40 days after HSCT The transplantation-related toxicity (TRT) was graded using the National Cancer Institute Common Toxicity Criteria for Adverse Events version 4.0. Organ damage due to GVHD or infectious complications were excluded.
Chimerism analyses +30 Days +30 after HSCT Chimerism would be evaluated in recipient BM cells usually on days +30 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
Chimerism analyses +100 Days +100 after HSCT Chimerism would be evaluated in recipient BM cells usually on days +180 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
Chimerism analyses +180 Days +180 after HSCT Chimerism would be evaluated in recipient BM cells usually on days +100 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
Chimerism analyses +365 Days +365 after HSCT Chimerism would be evaluated in recipient BM cells usually on days +365 after HSCT using cytogenetic G-banding or fluorescence in situ hybridization. Sex-matched donor-recipient chimerism was assessed using PCR-based analyses of polymorphic minisatellite or microsatellite regions. HLA typing was performed for patients with HLA-haploidentical donors.
OS 1-year 1-year after HSCT OS was defined as the time from transplantation to death from any cause or the last follow-up.
OS 2-year 2-year after HSCT OS was defined as the time from transplantation to death from any cause or the last follow-up.
OS 5-year 5-year after HSCT OS was defined as the time from transplantation to death from any cause or the last follow-up.
EFS 1-year 1-year after HSCT EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure.
EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure.EFS 2-year 2-year after HSCT EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure.
EFS 5-year 5-year after HSCT EFS was defined as survival with a response to therapy. Death, GF and relapse were considered as treatment failure.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Department of Hematology, 304th Clinical Division, Chinese PLA General Hospital
🇨🇳Beijing, China