Evaluation of the outcomes of newly diagnosed patients with high-risk myelodysplastic syndrome according to the initial therapeutical strategies chosen in usual clinical practice

Montoro, Maria Julia; Pomares, Helena; Coll, Rosa; Bernal del Castillo, Teresa; Tormo, Mar; Jiménez, Ana; Brunet, Salut; Casaño, Javier; Oiartzabal, Itziar; Díez-Campelo, María; Ramos, Fernando; Romero, Rafael; Salido-Fiérrez, Eduardo; Pedro Olive, Carme; Bargay, Joan; Muñoz-Novas, Carolina; López, Rocío; Rafel, Montserrat; Valcárcel, David; ERASME study group

doi:http://dx.doi.org/10.1080/10428194.2022.215460

Evaluation of the outcomes of newly diagnosed patients with high-risk myelodysplastic syndrome according to the initial therapeutical strategies chosen in usual clinical practice

Citació

Montoro MJ, Pomares H, Coll R, Bernal Del Castillo T, Tormo M, Jiménez A, Brunet S, Casaño J, Oiartzabal I, Díez-Campelo M, Ramos F, Romero R, Salido-Fiérrez E, Pedro C, Bargay J, Muñoz-Novas C, López R, Rafel M, Valcárcel D; ERASME study group. Evaluation of the outcomes of newly diagnosed patients with high-risk myelodysplastic syndrome according to the initial therapeutical strategies chosen in usual clinical practice. Leuk Lymphoma. 2023 Mar;64(3):679-90. DOI: 10.1080/10428194.2022.215460

Resum

Myelodysplastic syndromes (MDS) are a heterogeneous group of diseases without a care standard and show variability in treatment outcomes. This Spanish, observational, prospective study ERASME (CEL-SMD-2012-01) assessed the evolution of newly diagnosed and treatment-naïve high-risk MDS patients (according to IPPS-R). 204 patients were included: median age 73.0 years, 54.4% males, 69.6% 0-1 ECOG, and 94.6% with comorbidities. Active treatment was the most common strategy (52.0%) vs. stem cell transplantation (25.5%) and supportive care/watchful-waiting (22.5%). Overall (median) event-free survival was 7.9 months (9.1, 8.3, and 5.3); progression-free survival: 10.1 months (12.9, 12.8, and 4.3); and overall survival: 13.8 months (15.4, 14.9; 8.4), respectively, with significant differences among groups. Adverse events (AEs) of ≥3 grade were reported in 72.6% of patients; serious AEs reported in 60.6%. 33.1% of patients died due to AEs. Three patients developed second primary malignant neoplasms (median: 8.2 months). Our study showed better outcomes in patients receiving active therapy early after diagnosis.