Treatment characteristics and outcomes in lower-risk, non-del(5q) myelodysplastic syndromes: findings from a medical record review in the USA, Canada and Europe
Creators: name: Diez-Campelo, Maria
name: Yucel, Aylin
name: Goyal, Ravi K
name: Parikh, Rohan C
name: Esterberg, Elizabeth
name: Jimenez, Maria
name: Sluga-O'Callaghan, Martina
name: Miteva, Dimana
name: Xiao, Hong
name: Germing, Ulrich
Publisher: name: Taylor & Francis
Year: 2024
Subjects: subject: Medicine
subject: Pharmacology
subject: Immunology
subject: FOS: Clinical medicine
subject: Cancer
subject: Science Policy
subject: Infectious Diseases
subject: FOS: Health sciences

description: <b>Aim:</b> To assess treatment patterns and outcomes in patients with non-del(5q) lower-risk myelodysplastic syndromes. <b>Methods:</b> Patient medical records were reviewed in the USA, Canada (CAN), UK and the EU. <b>Results:</b> 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%). <b>Conclusion:</b> These findings highlight an unmet need for more effective treatments among patients with non-del(5q) lower-risk myelodysplastic syndromes. Patients with lower-risk myelodysplastic syndromes (LR-MDS) often present with anemia and frequently require red blood cell transfusions (RBCTs). Transfusion dependence is associated with reduced survival and poor quality of life. Therefore, the goal of LR-MDS treatment is to manage anemia and reduce transfusion burden. In patients with non-del(5q) LR-MDS, erythropoiesis-stimulating agents (ESAs), with or without granulocyte colony-stimulating factor, are an established treatment for anemia in patients who are ESA-naive. Treatment options are limited for patients who are unresponsive to ESAs. Currently, little is known about the real-world treatment patterns and clinical outcomes of patients with non-del(5q) LR-MDS. This retrospective medical record review aimed to describe patient characteristics, treatment patterns and hematologic outcomes among patients with non-del(5q) LR-MDS in the USA, Canada (CAN), UK and EU (France, Germany, Spain). Data were abstracted for 119 patients with non-del(5q) LR-MDS in the USA/CAN and 245 patients in the UK/EU. Most patients initiated first-line (1L) therapy (USA/CAN: 109 [91.6%]; UK/EU: 225 [91.8%]) with a mean time from MDS diagnosis to initiation of 1L treatment of 10.1 months in the USA/CAN and 9.6 months in the UK/EU. The majority of patients received ESAs as 1L therapy (USA/CAN: 97 [89.0%]; UK/EU: 203 [90.2%]) and the median duration of 1L ESA treatment was 25.8 months in the USA/CAN and 31.8 months in the UK/EU. Prior to 1L treatment, 36 patients (37.1%) treated with 1L ESA-containing regimens were transfusion dependent (TD) in the USA/CAN, while 104 patients (51.2%) were TD in the UK/EU. Among these patients who were TD before 1L, only a small proportion (2.8% USA/CAN; 14.4% UK/EU) achieved transfusion independence during 1L. An RBCT reduction was achieved by 41.7% and 52.9% of 1L ESA-treated patients who were TD at treatment initiation in the USA/CAN and UK/EU, respectively. A hemoglobin improvement was achieved by 32.1% of 1L ESA-treated patients in the USA/CAN and 44.8% in the UK/EU. These findings highlight an unmet need in anemia management among patients with non-del(5q) LR-MDS and a requirement for new and more effective treatment options.