Analysis of Real-World Outcomes in First-Line Treatment Among Patients With Myelodysplastic Syndromes

A Global, Retrospective Observational Cohort Study

There is a need for improved first-line clinical management strategy for treating patients with lower-risk (LR) myelodysplastic syndromes (MDS), according to a global, retrospective, observational cohort study presented at the Society of Hematologic Oncology (SOHO) 2023 Annual Meeting in Houston, Texas. 

“As new treatments for MDS become available, it is important to understand real-world clinical practice patterns to tailor management to patients’ needs,” wrote Argiris Symeonidis, MD, University of Patras Medical School, Patras, Greece and colleagues. “We aimed to describe characteristics, [first-line] treatment patterns, and outcomes of lower-risk MDS patients in a US and [European] cohort.”

This analysis included adult patients receiving frontline systemic treatment for lower-risk MDS. A total of 421 patients of a median age of 75 years were included, with 351 in the US and 70 in Europe. The patients included in the study had <5% blasts in bone marrow, erythropoietin <500 U/L, and Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 2. Patients receiving erythropoiesis-stimulating agents (ESAS) in the first-line or any non- erythropoiesis-stimulating agents as first-line treatments were included as subgroups in the study. 

The majority of the patients included (81%) had ECOG PS ≤1. Most patients received ESAs in the first-line, at a mean of 20.7 months. The median pre-baseline hemoglobin was 9.2 grams per deciliter (g/dL). During their frontline treatment, 42% of patients achieved a hemoglobin increase of  ≥1.5 g/dL. The median time to hemoglobin increase was 1.9 months, while the median duration of response was 8.9 months.

Study results indicated that 10% of all patients progressed to high-risk MDS and/or acute myeloid leukemia (AML) a median of 13.7 months after starting first-line therapy. Of those who received ESA in the first-line, 9%, at a median 16.0 months, and of those who received other first-line treatment, 10% at a median 7.6 months. Additionally, 52% of patients required second-line treatment: of those who received ESA in the first-line, 56% at a median 7.1 months; and of those who received other first-line treatment, 37% at a median 3.7 months. Among patients treated with ESA in the first-line who received second-line treatment, 74% received an ESA-containing regimen, of whom 78 (58%) received ESA monotherapy. 

“Patients with [lower-risk] MDS in the US and [Europe] had similar baseline characteristics and received [first-line] treatment for >1.5 years, although most did not achieve clinically meaningful [hemoglobin] improvement,” Symeonidis and coauthors concluded. “Patients who discontinued ESAs in [the first-line] often received ESAs in [the second-line]. These findings suggest a need for more effective [first-line] clinical management approaches in LR MDS.”

Source:

Symeonidis A, Yucel A, Deshpande GA, et al. First‑Line (1L) treatment patterns and outcomes among patients with newly diagnosed myelodysplastic syndromes (MDS): A global, retrospective observational cohort study. Presented at 2023 SOHO Annual Meeting; September 6-9, 2023; Houston, TX. Abstract MDS-067