Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center
This phase 2, single-center clinical trial evaluated the efficacy and safety of guadecitabine, with or without donor lymphocyte infusion, following allogeneic stem cell transplantation (allo-SCT) in adult patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The study had t...
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| Format: | Article |
| Language: | English |
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Ferrata Storti Foundation
2025-07-01
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| Series: | Haematologica |
| Online Access: | https://haematologica.org/article/view/12186 |
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| author | Betül Oran Peter Thall Amin Alousi Gheath Al-Atrash Rohtesh Mehta David Marin Partow Kebriaei Uday Popat Roland Bassett Qaiser Bashir Jin Im Amanda Olson Jessica Jewell Portia Smallbone Elizabeth Shpall Richard Champlin |
| author_facet | Betül Oran Peter Thall Amin Alousi Gheath Al-Atrash Rohtesh Mehta David Marin Partow Kebriaei Uday Popat Roland Bassett Qaiser Bashir Jin Im Amanda Olson Jessica Jewell Portia Smallbone Elizabeth Shpall Richard Champlin |
| author_sort | Betül Oran |
| collection | DOAJ |
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This phase 2, single-center clinical trial evaluated the efficacy and safety of guadecitabine, with or without donor lymphocyte infusion, following allogeneic stem cell transplantation (allo-SCT) in adult patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The study had three treatment cohorts based on disease status post-transplant. Cohort 1 included patients with hematological relapse after transplant (n=13). Cohort 2 consisted of patients with minimal residual disease (MRD) detected after transplant (n=18). Cohort 3 compromised of patients in remission without MRD within 100 days post-transplant (n=24). The primary objectives were to achieve morphological complete remission (CR) in cohort 1 and MRD eradication in cohort 2 within six cycles of guadecitabine. Cohort 3 patients received 12 cycles to improve relapse-free survival (RFS).
In cohort 1, 21.4% of patients achieved morphological CR. In cohort 2, 47.1% achieved MRD eradication. Those who cleared MRD had a 2-year RFS of 62.5%. Cohort 3 patients had a 2-year RFS rate was 62.5% with a median follow-up of 48 months.
No unexpected adverse events (AEs) occurred, and no graft failures were observed. Guadecitabine demonstrated efficacy and a favorable safety profile across all cohorts, supporting the investigations of hypomethylating agents.
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| format | Article |
| id | doaj-art-bd8a456b59af415f8299581eb18ed8b9 |
| institution | Kabale University |
| issn | 0390-6078 1592-8721 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | Ferrata Storti Foundation |
| record_format | Article |
| series | Haematologica |
| spelling | doaj-art-bd8a456b59af415f8299581eb18ed8b92025-08-20T03:31:46ZengFerrata Storti FoundationHaematologica0390-60781592-87212025-07-01999110.3324/haematol.2025.287628Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single centerBetül Oran0Peter Thall1Amin Alousi2Gheath Al-Atrash3Rohtesh Mehta4David Marin5Partow Kebriaei6Uday Popat7Roland Bassett8Qaiser Bashir9Jin Im10Amanda Olson11Jessica Jewell12Portia Smallbone13Elizabeth Shpall14Richard Champlin15Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Biostatistics, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Biostatistics, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer CenterDepartment of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center This phase 2, single-center clinical trial evaluated the efficacy and safety of guadecitabine, with or without donor lymphocyte infusion, following allogeneic stem cell transplantation (allo-SCT) in adult patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The study had three treatment cohorts based on disease status post-transplant. Cohort 1 included patients with hematological relapse after transplant (n=13). Cohort 2 consisted of patients with minimal residual disease (MRD) detected after transplant (n=18). Cohort 3 compromised of patients in remission without MRD within 100 days post-transplant (n=24). The primary objectives were to achieve morphological complete remission (CR) in cohort 1 and MRD eradication in cohort 2 within six cycles of guadecitabine. Cohort 3 patients received 12 cycles to improve relapse-free survival (RFS). In cohort 1, 21.4% of patients achieved morphological CR. In cohort 2, 47.1% achieved MRD eradication. Those who cleared MRD had a 2-year RFS of 62.5%. Cohort 3 patients had a 2-year RFS rate was 62.5% with a median follow-up of 48 months. No unexpected adverse events (AEs) occurred, and no graft failures were observed. Guadecitabine demonstrated efficacy and a favorable safety profile across all cohorts, supporting the investigations of hypomethylating agents. https://haematologica.org/article/view/12186 |
| spellingShingle | Betül Oran Peter Thall Amin Alousi Gheath Al-Atrash Rohtesh Mehta David Marin Partow Kebriaei Uday Popat Roland Bassett Qaiser Bashir Jin Im Amanda Olson Jessica Jewell Portia Smallbone Elizabeth Shpall Richard Champlin Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center Haematologica |
| title | Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center |
| title_full | Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center |
| title_fullStr | Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center |
| title_full_unstemmed | Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center |
| title_short | Guadecitabine improved relapse-free survival in high-risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant: phase II results from a single center |
| title_sort | guadecitabine improved relapse free survival in high risk acute myeloid leukemia and myelodysplastic syndrome patients after transplant phase ii results from a single center |
| url | https://haematologica.org/article/view/12186 |
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