Optimizing hypomethylating agents in myelodysplastic syndromes

Abstract

The indication and practical use of HMAs in MDS so far remain those of phase III registration studies, but will hopefully be modified with future results of ongoing clinical and translational research.

Spliceosome-related gene mutations in myelodysplastic syndrome can be used as stable markers for monitoring minimal residual disease during follow-up

“…Epigenetic therapy by using azacitidine is the first drug therapy to show a significant survival advantage in patients with higher-risk MDS and can prevent or delay the hematologic relapse and MRD after HSCT [33,34]. The responsiveness to hypomethylating agents may be associated with the presence of mutations affecting epigenetic regulators [35,36]. In addition to its association with responsiveness to drug therapies, the gene mutations reported in MDS patients are associated with specific clinical features and as predictors of overall survival [37].…”

Establishment and Validation of an Updated Diagnostic FCM Scoring System Based on Pooled Immunophenotyping in CD34+ Blasts and Its Clinical Significance for Myelodysplastic Syndromes

“…The combination of early scoring, advanced scoring and the percentage of CD34+ blasts could contribute to the diagnosis of some patients who are shifting from RCMD to RAEB-1. Considering that the disease stage is an important component of selecting particular treatments such as immunosuppressive therapy and decitabine treatment [27], [28], the degree of early scoring or advanced scoring may also be helpful for treatment selection. As shown in this study, low-risk MDS patients with high early scoring and low advanced scoring may be candidates for immunosuppressive therapy, whereas those with high advanced scoring and low early scoring may be more suitable for decitabine treatment.…”

Decitabine as a conditioning regimen in haploidentical stem cell transplantation for refractory acute myeloid leukaemia

“…Because of the relative lack of organ toxicity, significant myelosuppression and possibly favourable immunologic effects, decitabine is potentially an excellent candidate for use in combination with standard preparative regimens for patients undergoing HSCT therapy. Use of single‐agent decitabine is efficient and tolerable for the treatment of patients with relapsed leukaemia and MDS after undergoing allogeneic HSCT . Recent literature shows that decitabine can be used as a bridge therapy prior to allogeneic HSCT in patients with refractory/relapsed AML .…”

“…Thus, novel strategies to improve the efficacy of conditioning regimens are required . Decitabine, a pyrimidine analogue with significant antileukaemic activity, is known to be effective in the treatment of patients with refractory/relapsed leukaemia . Decitabine is effective as a preparative regimen in allogeneic HSCT in patients with high‐risk acute leukaemia .…”