Abstract

Myelodysplastic syndromes (MDS) and AML are rare blood cancers existing on a continuum, with poor clinical outcomes. This retrospective cohort study obtained real-world data on rates of transformation from MDS to AML, treatment patterns in AML, and survival outcomes for patients with AML in England. This study analysed retrospective data from the English national CAS registry and included all adult patients with MDS or AML treated with systemic anti-cancer therapy (SACT) from January 2013 to December 2018, with follow-up to December 2020. Three study cohorts were generated: non-transformed patients with MDS of any risk category (cohort A), patients with AML who transformed from MDS of any risk category (cohort B), and all other patients with de novo and secondary AML (cohort C). Patient characteristics, rates of transformation to AML and SACT treatments received after azacitidine were described; overall survival (OS) was estimated using Kaplan-Meier methods. Cohorts A, B and C included 6,549, 368 and 6,936 patients (total N=13,853), with median age at diagnosis 67, 71, 65 years, respectively. The majority of patients were male (58.54%) and of white ethnicity (87.58%). Transformation rates of patients with MDS of any risk category (total cohorts A and B) were 4.48-11.23% depending on year of diagnosis and available follow-up; median time to transformation was 13 months. Treatment sequences following azacitidine were similar in cohorts B (N=56) and C (N=1,572); the majority of patients did not receive further SACT (66.07%; N=37 and 68.58%; N=1,078, respectively). Median OS was 2.7 months from AML transformation for cohort B and 16 months from SACT initiation for cohort C. This nationwide study highlights the poor survival for patients with AML in England, particularly for patients who have transformed from MDS to AML. Treatment options are limited, with significant unmet need for new effective therapies.

Full Text
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