Treatment characteristics and outcomes in lower-risk, non-del(5q) myelodysplastic syndromes: findings from a medical record review in the USA, Canada and Europe
Bibliographic record
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. 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.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.006 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.004 | 0.010 |
| Science and technology studies | 0.001 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".