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. 2024;20(27):1993-2004.
doi: 10.1080/14796694.2024.2379228. Epub 2024 Aug 14.

Treatment characteristics and outcomes in lower-risk, non-del(5q) myelodysplastic syndromes: findings from a medical record review in the USA, Canada and Europe

Maria Diez-Campelo  1 , Aylin Yucel  2 , Ravi K Goyal  3 , Rohan C Parikh  3 , Elizabeth Esterberg  3 , Maria Jimenez  3 , Martina Sluga-O'Callaghan  4 , Dimana Miteva  5 , Hong Xiao  2 , Ulrich Germing  6
Affiliations

Treatment characteristics and outcomes in lower-risk, non-del(5q) myelodysplastic syndromes: findings from a medical record review in the USA, Canada and Europe

Maria Diez-Campelo et al. Future Oncol. 2024.

Abstract

Aim: To assess treatment patterns and outcomes in patients with non-del(5q) lower-risk myelodysplastic syndromes.Methods: Patient medical records were reviewed in the USA, Canada (CAN), UK and the EU.Results: Analysis included 119 patients in the USA/CAN (median age, 61.5 years) and 245 patients in the UK/EU (median age, 67.3 years). Most patients received erythropoiesis-stimulating agents (ESAs) as first-line (1L) therapy (USA/CAN: 89.0%; UK/EU: 90.2%). A substantial proportion of 1L erythropoiesis-stimulating agent-treated patients were transfusion dependent before 1L (USA/CAN: 37.1%; UK/EU: 51.2%); a small percentage of these patients achieved transfusion independence during 1L therapy (USA/CAN: 2.8%; UK/EU: 14.4%).Conclusion: These findings highlight an unmet need for more effective treatments among patients with non-del(5q) lower-risk myelodysplastic syndromes.

Keywords: hematologic outcomes; lower-risk myelodysplastic syndromes; non-del(5q); real-world data; treatment patterns.

Plain language summary

[Box: see text].

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Conflict of interest statement

RK Goyal, RC Parikh, E Esterberg, M Jimenez and M Sluga-O'Callaghan are full-time employees of RTI Health Solutions, an independent nonprofit research organization that was retained by Bristol Myers Squibb to conduct the research that is the subject of this manuscript. Their compensation is unconnected to the studies on which they work. A Yucel, D Miteva and H Xiao are employees of Bristol Myers Squibb and may hold shares and/or stock options in the company. M Diez-Campelo is a full-time employee of University Hospital of Salamanca, Salamanca, Spain and has received speaker honoraria from Bristol Myers Squibb and Novartis, and served on advisory boards for Agios, Blueprint Medicines, Bristol Myers Squibb, GlaxoSmithKline, Hemavant, Keros, Novartis, and Syros, and has received support for attending meetings and/or travel from Gilead. U Germing is a full-time employee of the University Hospital of Dusseldorf, Dusseldorf, Germany, and has received speaker honoraria from Bristol Myers Squibb, Janssen and Novartis, and consulting fees from Bristol Myers Squibb, as well as grants or contracts from Bristol Myers Squibb and Novartis. The authors have no other competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript apart from those disclosed.

Figures

Figure 1.
Figure 1.
Overview of study design. ESA: Erythropoiesis-stimulating agent; G-CSF: Granulocyte colony-stimulating factor; MDS: Myelodysplastic syndromes; RBCT: Red blood cell transfusion.
Figure 2.
Figure 2.
Transfusion dependence before 1L treatment. Transfusion dependence before 1L treatment among a subgroup of patients with non-del(5q) LR-MDS treated with 1L ESA-containing regimens. Transfusion dependence was defined as a RBCT requirement of ≥2 units and was measured within the 8-week period before treatment initiation. Sample sizes (n) refer to the total sample size in each country or pooled geographic region. 1L: First-line; CAN: Canada; ESA: Erythropoiesis-stimulating agent; LR-MDS: Lower-risk myelodysplastic syndromes; RBCT: Red blood cell transfusion; TD: Transfusion dependent.
Figure 3.
Figure 3.
Hematologic improvement achieved during 1L ESA therapy. (A) RBCT reduction. Hematologic improvement based on the threshold for RBCT reduction among a subgroup of patients with non-del(5q) LR-MDS treated with 1L ESA-containing regimens. The threshold for hematologic improvement was defined as a decrease of ≥50% in the RBCT need at any point during follow-up compared with diagnosis or treatment initiation. Sample sizes refer to the total sample size in each country or pooled geographic region. (B) Increased hemoglobin levels. Hematologic improvement based on the threshold for increased hemoglobin levels among a subgroup of patients with non-del(5q) LR-MDS treated with 1L ESA-containing regimens. The threshold for hematologic improvement was defined as an increase in hemoglobin level of ≥1.5 g/dl or 15 g/l. Sample sizes refer to the total sample size in each country or pooled geographic region. Assessed among patients with known hemoglobin level at treatment initiation. 1L: First-line; CAN: Canada; ESA: Erythropoiesis-stimulating agent; LR-MDS: Lower-risk myelodysplastic syndromes; RBCT: Red blood cell transfusion.
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