HAPLO-IDENTICAL TRANSPLANTATION FOR ACQUIRED SEVERE APLASTIC ANEMIA IN A MULTI-CENTER PROSPECTIVE STUDY
(Abstract release date: 05/19/16)
EHA Library. Xu Z. 06/12/16; 135325; S831
Dr. Zhengli Xu
Contributions
Contributions
Abstract
Abstract: S831
Type: Oral Presentation
Presentation during EHA21: On Sunday, June 12, 2016 from 08:45 - 09:00
Location: Room H5
Background
Severe aplastic anemia (SAA) is a life-threatening disorder for which allogeneic hematopoietic stem cell transplantation (HSCT) is the available curative approach. Haplo-identical donor (HID) as an alternative source, should be considered in the absence of matched related or unrelated donor after failing to respond to immunosuppressive therapy (IST). Obvious superiority arises from the immediate availability of a suitable haplo-identical donor for most patients within appropriate time-frame. However, initial outcomes of HID for SAA were far from satisfactory owing to poor engraftment, refractory graft-versus-host disease (GVHD) and delayed immune reconstitution.Several small-sample studies using various approaches of haplo-identical transplantation revealed the mixed success, with short-term survival rates reaching 67%>100% and graft failure rates ranging from 0% to 25%. However, the recent reports of haplo-identical transplantation for SAA are rather heterogeneous and almost all series are limited by small numbers of patients.
Aims
To further prove the therapeutic effect of haplo-identical SCT in the treatment of SAA, we conducted a prospective multi-center study using uniform protocol, and compared outcomes of haploidentical SCT with those of all contemporaneous transplantation from MRD.
Methods
This was a disease-specific, multicenter, prospective study. All eligible patients treated at 11 participating institutions were included. Patients were administered with BU/CY+ATG conditioning regimen, G-BM combined with peripheral blood stem cells without in vitro T-cell depletion and a combination of CsA, MTX and MMF as graft-versus-host disease (GVHD) prophylaxis. The primary endpoint was engraftment. Various outcomes of HID were compared with those of contemporaneous transplantation from matched related donors (MRD).
Results
For patients receiving HID-SCT, all cases surviving for more than 28 days achieved donor myeloid engraftment. The median time for myeloid engraftment was 12 (range, 9-25) and for platelet was 15 (range, 7-101) days with a cumulative platelet engraftment incidence of 94.1±0.1%. With a median follow up of 18.3 (3.0-43.6) months and when compared with MRD, HID recipients had more cumulative incidence of grade II-IV acute GVHD (aGVHD; 33.7% vs. 4.2%, P＜0.001), more chronic GVHD (cGVHD; 22.4% vs. 6.6%, P=0.014) at 1 year, but similar grade III-IV aGVHD (7.9% vs. 2.1%, P=0.157), 3-year estimated overall survival (OS, 89.0% vs. 91.0%, P=0.555) and 3-year estimated failure free survival (FFS, 86.8% vs. 80.3%, P=0.659). Multivariate analysis showed no significant difference in primary engraftment and survival outcomes between two cohorts.
Conclusion
Haplo-identical transplantation using our protocol was proved to be an effective and safe choice in SAA patients who failed previous IST. It could be recommended for those who lack a MRD as higher priority.
Session topic: Non-malignant hematopoietic disorders
Keyword(s): Aplastic anemia, Haploidentical stem cell transplantation
Type: Oral Presentation
Presentation during EHA21: On Sunday, June 12, 2016 from 08:45 - 09:00
Location: Room H5
Background
Severe aplastic anemia (SAA) is a life-threatening disorder for which allogeneic hematopoietic stem cell transplantation (HSCT) is the available curative approach. Haplo-identical donor (HID) as an alternative source, should be considered in the absence of matched related or unrelated donor after failing to respond to immunosuppressive therapy (IST). Obvious superiority arises from the immediate availability of a suitable haplo-identical donor for most patients within appropriate time-frame. However, initial outcomes of HID for SAA were far from satisfactory owing to poor engraftment, refractory graft-versus-host disease (GVHD) and delayed immune reconstitution.Several small-sample studies using various approaches of haplo-identical transplantation revealed the mixed success, with short-term survival rates reaching 67%>100% and graft failure rates ranging from 0% to 25%. However, the recent reports of haplo-identical transplantation for SAA are rather heterogeneous and almost all series are limited by small numbers of patients.
Aims
To further prove the therapeutic effect of haplo-identical SCT in the treatment of SAA, we conducted a prospective multi-center study using uniform protocol, and compared outcomes of haploidentical SCT with those of all contemporaneous transplantation from MRD.
Methods
This was a disease-specific, multicenter, prospective study. All eligible patients treated at 11 participating institutions were included. Patients were administered with BU/CY+ATG conditioning regimen, G-BM combined with peripheral blood stem cells without in vitro T-cell depletion and a combination of CsA, MTX and MMF as graft-versus-host disease (GVHD) prophylaxis. The primary endpoint was engraftment. Various outcomes of HID were compared with those of contemporaneous transplantation from matched related donors (MRD).
Results
For patients receiving HID-SCT, all cases surviving for more than 28 days achieved donor myeloid engraftment. The median time for myeloid engraftment was 12 (range, 9-25) and for platelet was 15 (range, 7-101) days with a cumulative platelet engraftment incidence of 94.1±0.1%. With a median follow up of 18.3 (3.0-43.6) months and when compared with MRD, HID recipients had more cumulative incidence of grade II-IV acute GVHD (aGVHD; 33.7% vs. 4.2%, P＜0.001), more chronic GVHD (cGVHD; 22.4% vs. 6.6%, P=0.014) at 1 year, but similar grade III-IV aGVHD (7.9% vs. 2.1%, P=0.157), 3-year estimated overall survival (OS, 89.0% vs. 91.0%, P=0.555) and 3-year estimated failure free survival (FFS, 86.8% vs. 80.3%, P=0.659). Multivariate analysis showed no significant difference in primary engraftment and survival outcomes between two cohorts.
Conclusion
Haplo-identical transplantation using our protocol was proved to be an effective and safe choice in SAA patients who failed previous IST. It could be recommended for those who lack a MRD as higher priority.
Session topic: Non-malignant hematopoietic disorders
Keyword(s): Aplastic anemia, Haploidentical stem cell transplantation
Abstract: S831
Type: Oral Presentation
Presentation during EHA21: On Sunday, June 12, 2016 from 08:45 - 09:00
Location: Room H5
Background
Severe aplastic anemia (SAA) is a life-threatening disorder for which allogeneic hematopoietic stem cell transplantation (HSCT) is the available curative approach. Haplo-identical donor (HID) as an alternative source, should be considered in the absence of matched related or unrelated donor after failing to respond to immunosuppressive therapy (IST). Obvious superiority arises from the immediate availability of a suitable haplo-identical donor for most patients within appropriate time-frame. However, initial outcomes of HID for SAA were far from satisfactory owing to poor engraftment, refractory graft-versus-host disease (GVHD) and delayed immune reconstitution.Several small-sample studies using various approaches of haplo-identical transplantation revealed the mixed success, with short-term survival rates reaching 67%>100% and graft failure rates ranging from 0% to 25%. However, the recent reports of haplo-identical transplantation for SAA are rather heterogeneous and almost all series are limited by small numbers of patients.
Aims
To further prove the therapeutic effect of haplo-identical SCT in the treatment of SAA, we conducted a prospective multi-center study using uniform protocol, and compared outcomes of haploidentical SCT with those of all contemporaneous transplantation from MRD.
Methods
This was a disease-specific, multicenter, prospective study. All eligible patients treated at 11 participating institutions were included. Patients were administered with BU/CY+ATG conditioning regimen, G-BM combined with peripheral blood stem cells without in vitro T-cell depletion and a combination of CsA, MTX and MMF as graft-versus-host disease (GVHD) prophylaxis. The primary endpoint was engraftment. Various outcomes of HID were compared with those of contemporaneous transplantation from matched related donors (MRD).
Results
For patients receiving HID-SCT, all cases surviving for more than 28 days achieved donor myeloid engraftment. The median time for myeloid engraftment was 12 (range, 9-25) and for platelet was 15 (range, 7-101) days with a cumulative platelet engraftment incidence of 94.1±0.1%. With a median follow up of 18.3 (3.0-43.6) months and when compared with MRD, HID recipients had more cumulative incidence of grade II-IV acute GVHD (aGVHD; 33.7% vs. 4.2%, P＜0.001), more chronic GVHD (cGVHD; 22.4% vs. 6.6%, P=0.014) at 1 year, but similar grade III-IV aGVHD (7.9% vs. 2.1%, P=0.157), 3-year estimated overall survival (OS, 89.0% vs. 91.0%, P=0.555) and 3-year estimated failure free survival (FFS, 86.8% vs. 80.3%, P=0.659). Multivariate analysis showed no significant difference in primary engraftment and survival outcomes between two cohorts.
Conclusion
Haplo-identical transplantation using our protocol was proved to be an effective and safe choice in SAA patients who failed previous IST. It could be recommended for those who lack a MRD as higher priority.
Session topic: Non-malignant hematopoietic disorders
Keyword(s): Aplastic anemia, Haploidentical stem cell transplantation
Type: Oral Presentation
Presentation during EHA21: On Sunday, June 12, 2016 from 08:45 - 09:00
Location: Room H5
Background
Severe aplastic anemia (SAA) is a life-threatening disorder for which allogeneic hematopoietic stem cell transplantation (HSCT) is the available curative approach. Haplo-identical donor (HID) as an alternative source, should be considered in the absence of matched related or unrelated donor after failing to respond to immunosuppressive therapy (IST). Obvious superiority arises from the immediate availability of a suitable haplo-identical donor for most patients within appropriate time-frame. However, initial outcomes of HID for SAA were far from satisfactory owing to poor engraftment, refractory graft-versus-host disease (GVHD) and delayed immune reconstitution.Several small-sample studies using various approaches of haplo-identical transplantation revealed the mixed success, with short-term survival rates reaching 67%>100% and graft failure rates ranging from 0% to 25%. However, the recent reports of haplo-identical transplantation for SAA are rather heterogeneous and almost all series are limited by small numbers of patients.
Aims
To further prove the therapeutic effect of haplo-identical SCT in the treatment of SAA, we conducted a prospective multi-center study using uniform protocol, and compared outcomes of haploidentical SCT with those of all contemporaneous transplantation from MRD.
Methods
This was a disease-specific, multicenter, prospective study. All eligible patients treated at 11 participating institutions were included. Patients were administered with BU/CY+ATG conditioning regimen, G-BM combined with peripheral blood stem cells without in vitro T-cell depletion and a combination of CsA, MTX and MMF as graft-versus-host disease (GVHD) prophylaxis. The primary endpoint was engraftment. Various outcomes of HID were compared with those of contemporaneous transplantation from matched related donors (MRD).
Results
For patients receiving HID-SCT, all cases surviving for more than 28 days achieved donor myeloid engraftment. The median time for myeloid engraftment was 12 (range, 9-25) and for platelet was 15 (range, 7-101) days with a cumulative platelet engraftment incidence of 94.1±0.1%. With a median follow up of 18.3 (3.0-43.6) months and when compared with MRD, HID recipients had more cumulative incidence of grade II-IV acute GVHD (aGVHD; 33.7% vs. 4.2%, P＜0.001), more chronic GVHD (cGVHD; 22.4% vs. 6.6%, P=0.014) at 1 year, but similar grade III-IV aGVHD (7.9% vs. 2.1%, P=0.157), 3-year estimated overall survival (OS, 89.0% vs. 91.0%, P=0.555) and 3-year estimated failure free survival (FFS, 86.8% vs. 80.3%, P=0.659). Multivariate analysis showed no significant difference in primary engraftment and survival outcomes between two cohorts.
Conclusion
Haplo-identical transplantation using our protocol was proved to be an effective and safe choice in SAA patients who failed previous IST. It could be recommended for those who lack a MRD as higher priority.
Session topic: Non-malignant hematopoietic disorders
Keyword(s): Aplastic anemia, Haploidentical stem cell transplantation
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