Epidemiology of myelodysplastic syndromes: Why characterizing the beast is a prerequisite to taming it

Decitabine induces change of biological traits in myelodysplastic syndromes via FOXO1 activation

“…The incidence of MDS in the elderly gradually increases with age, which is one of the main factors that threaten the quality of life and survival of the elderly. Although 3 therapies targeting MDS have been approved since 2004, the overall 5-year survival rate remains relatively poor at approximately 31% without a clear temporal improvement in outcomes [1]. The heterogeneous nature of MDS demands a complex and personalized variety of therapeutic approaches.…”

Good but not good enough: Clinical trial participation of patients with myelodysplastic syndromes

“…6 For example, the hypomethylating agent azacitidine (AZA) has been the only agent shown to have a mortality benefit in the treatment of patients with higher risk myelodysplastic syndromes (MDS). 7 However, the median overall survival observed with AZA in the landmark randomized phase 3 trial AZA-001 has not been reproducible at the population level in the United States or other countries. [8][9][10][11][12] Therefore, understanding the factors underlying the low rates of and disparities in clinical trial enrollment among patients with MDS is essential for counseling patients appropriately on the likely benefit of a particular treatment.…”

“…Although cancer is more prevalent among older patients, MDS tends to occur among the oldest of patients with a median age of 77 years at diagnosis in the United States. 7 Therefore, many patients with MDS have limited social and family support; these are factors often needed to cope with the added requirements of clinical trial participation. Although the 23% clinical trial participation rate reported by Brierley et al 13 appears favorable in comparison with the rate of less than…”

“…19 With an annual age-adjusted incidence rate of 4 per 100,000 persons in the United States, MDS is a rare disease, and clinical trial recruitment requires concerted efforts. 7 How could greater clinical trial participation rates be achieved, and is this even necessary? With a poor 5-year overall survival rate of 31%, MDS has a prognosis worse than most solid malignancies, and this highlights the need for better therapies and clinical trials.…”

“…With a poor 5-year overall survival rate of 31%, MDS has a prognosis worse than most solid malignancies, and this highlights the need for better therapies and clinical trials. 7 If we extrapolate from other cancers, options to achieve this goal include 1) liberalizing the inclusion and exclusion criteria of clinical trials with respect to age and comorbidities (eg, focusing on disease-specific factors such as genetically defined disease subtypes for efficacy analyses while using the broader patient population for safety analyses), 2) providing logistical and potentially financial support for clinical trial participants, 3) involving community providers more, and 4) making changes to the regulatory and reimbursement landscape (Fig. 1).…”