A natural history of lower-risk myelodysplastic syndromes with ring sideroblasts: an analysis of the MDS-CAN registry.
Rena BucksteinLisa ChodirkerLee MozessohnKaren W L YeeMichelle GeddesNancy ZhuApril ShamyHeather A LeitchGrace ChristouVersha BanerjiLeber BrianDina KhalafEve St-HilaireNicholas FinnThomas NevillMary-Margaret KeatingJohn StorringRobert DelageAnne ParmentierAksharh ThambipillaiMohammed SiddiquiChristopher WestcottChris CameronAlexandre MamedovPaul SpinDerek TangPublished in: Leukemia & lymphoma (2022)
Patients with lower-risk (LR) myelodysplastic syndromes (MDS) with ring sideroblasts (RS) have better prognosis than those without RS, but how they fare over time is not fully understood. This study's objective was to assess the natural history of LR MDS with RS ≥5% using MDS-CAN registry individual data. Kaplan-Meier estimates and generalized linear mixed models were used to describe time-to-event outcomes and continuous outcomes, respectively. One hundred and thirty-eight patients were enrolled; median times from diagnosis to enrollment and follow-up were 6.6 and 39.6 months, respectively. Within 5 years of enrollment, 65% of patients had ≥1 red blood cell transfusion dependence episode. Within 5 years of diagnosis, 59% developed iron overload, 38% received iron chelation therapy, 14% progressed to acute myeloid leukemia, and 42% died. Patients exhibited inferior health-related quality of life trends. These first real-world data in LR MDS-RS in Canada indicate a high level of morbidity and mortality over a 5-year period. Clinical Trial Registration: ClinicalTrials.gov Identifier: NCT02537990.
Keyphrases
- end stage renal disease
- ejection fraction
- clinical trial
- newly diagnosed
- acute myeloid leukemia
- chronic kidney disease
- prognostic factors
- red blood cell
- peritoneal dialysis
- healthcare
- type diabetes
- stem cells
- randomized controlled trial
- patient reported outcomes
- machine learning
- mesenchymal stem cells
- insulin resistance
- phase ii
- sickle cell disease