Pb2020: outcomes of patients with high risk myelodysplastic syndromes

Topic: 10. Myelodysplastic syndromes - Clinical Background: In high-risk myelodysplastic syndromes (MDS), the goal of therapy is to prolong survival and reduce the risk of transformation into acute leukemia, but only a few drugs are currently available for treatment. Aims: We report our therapeutics results of high - risk MDS ‘patients. Methods: This is a retrospective study included patients with high risk MDS classified according to the International Prognostic Scoring System (IPSS) as intermediate-2 or high-risk groups, and treated in Hedi Chaker Hospital in south of Tunisia, between 2015 and 2021. Results: A total of 43 patients were included with a median age of 57 years (24-83), and a sex ratio of 1.1. Five patients (<45 years) underwent an allogeneic hematopoietic stem-cell transplantation (AHSCT), with unfortunately an early toxic death in 3 patients, a relapsed disease in one patient after one year, and one patient is en complete remission after 48 months. Sixteen patients were treated by Azacitidine. 93 cycles were administered with a median duration of therapy of 6 cycles (1-18 cycles). Four patients had achieving a partial response with hematologic improvement, 6 patients had a stable disease and 6 patients had a disease progression. The median of survival of this group was 15 months; overall survival (OS) at 12 and 24 months was 57% and 15 % respectively. Two patients who having a deletion of the long arm of chromosome 5 received Lenalidomide allowing to obtain in a transfusion independence in both patients, with a transient cytogenetic response and after that a progression to acute leukemia in 24 months in one patient, and persistent hematologic response after 48 months of follow up in the other patient. The remains of patients (20 patients) received best supportive care with an (OS) of 24% at 12 months and 9% at 24 months. The overall survival (OS) of the whole cohort was respectively 42% and 13% at 12 and 24 months. Summary/Conclusion: AHSCT remains the only curative treatment when, but this concerns a minority of patients. Azacitidine improve the survival of patients compared to those received supportive care. The current challenge in the management of higher risk MDS is to improve outcome by combining classical hypomethylating agents with novel drugs. Keywords: MDS