In a nutshell
This study aimed to investigate the outcomes of allogeneic transplantation in patients with core-binding factor acute myeloid leukemia in second complete remission.
This study concluded that transplant was effective in a large proportion of these patients.
Some background
Core binding factor acute myeloid leukemia (CBF-AML) is one form of AML. There are two subtypes of CBF-AML based on cytogenetic abnormalities (changes in chromosomes that contain DNA). One subtype contains cytogenetic abnormalities of t(8;21)(q22;q22) and the other subtype contains cytogenetic abnormalities of inv(16)(p13q22)/t(16;16)(p13;q22).
Chemotherapy is used for treatment of CBF-AML and a long-term response is usually achieved. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is considered for the treatment of patients who relapse (disease returns) and achieve second complete remission (no signs of cancer after a second-line treatment). Allo-HSCT is a procedure in which a person receives blood-forming stem cells from a genetically similar, but not identical, donor. This is often a sister or brother but could be an unrelated donor.
It was not known how safe and effective allo-HSCT would be for patients in second remission with CBF-AML.
Methods & findings
This study involved 631 patients with CBF-AML who were in second complete remission. The outcomes of allo-HSCT in these patients were measured.
The leukemia-free survival (LFS) probability at 2 years was 59.1%. The LFS probability at 5 years was 54.1%. The overall survival (OS) probability at 2 years was 65%. The OS probability at 5 years was 58.2%.
The frequency of relapse (cancer returns) at 2 years was 19.8%. This rate at 5 years was 22.5%. The frequency of non-relapse mortality (NRM – death from a source other than relapse) at 2 years was 20.9%. The frequency of NRM at 5 years was 23.3%.
The most important unfavorable factors that influenced LFS and OS were leukemia with t(8;21), presence of 3 or more additional chromosomal abnormalities and Karnofsky performance score <80. A reduced-intensity conditioning (less chemotherapy than normal) was also associated with a higher risk of relapse. Measurable residual disease (a small number of cancer cells that remain after treatment) before the transplant was associated with an increased risk of relapse and lower LFS.
The bottom line
This study concluded that allo-HSCT in patients with CBF-AML in second complete remission was able to improve the outcomes of a large proportion of patients.
The fine print
This study was based on medical records. Information might have been missing. This may influence the findings.
Published By :
Haematologica
Date :
Aug 22, 2019