Patterns of Treatment among Patients with Recently-Diagnosed Myelodysplastic Syndromes in a National Registry, 2006–2008
Authors: Van Bennekom Carla M. , Abel Gregory , Anderson Theresa , Stone Richard M. , Kaufman David W. 
Published: October 2019

Abstract
Background: Patterns of treatment among patients with recently-diagnosed myelodysplastic syndrome (MDS) are of particular interest with the recent introduction of disease-modifying treatments (DMT), and have not been characterized on a population basis.
Methods: The “Patient Registries at Slone: MDS” is a national disease-based observational registry conducted by Boston University. All patients with MDS diagnosed within 4 months of enrollment are eligible for inclusion. Patients obtain information about the registry from their treating physicians or the internet and self-enroll. Information on treatment, clinical events, and quality of life is obtained by questionnaire and medical record review at baseline and at six-month intervals. We report on 290 patients residing in 44 states who were enrolled from June 2006 through June 2008 and completed a baseline questionnaire within six months of diagnosis.
Results: A total of 71 of the 290 patients (24%) had received DMT since diagnosis, including azacitidine (9%), decitabine (7%), lenalidomide (6%), or multiple agents (2%). In contrast, 167 (58%) reported receiving supportive therapy such as erythropoesis-stimulating agents (ESAs; 43%), myeloid growth factors (12%), iron chelators (1%), or antibiotics (16%)(categories not mutually exclusive). Patients from New England had the lowest prevalence of DMT (6%). Overall, lack of prescription drug coverage was associated with a lower rate of DMT (11% vs. 25% in covered patients). Patients who received DMT were also more likely to receive supportive therapy: 55% were treated with ESAs compared to 39% of patients without DMT (p=0.03); the corresponding proportions for myeloid growth factors were 32% vs. 5% (p<0.0001), and for antibiotics, 32% vs. 11% (p<0.0001). Among the 164 patients for whom WHO subtyping and IPSS were available to date, none with RA or RARS received DMT, and the prevalence of DMT increased with increasing IPSS (from 9% among low risk to 60% among high risk patients). Among low/intermediate-1 risk patients combined, the prevalence was 15% compared to 45% among intermediate-2/high risk patients (p=0.0002). Two of 12 patients treated with azacitidine and 5 of 10 treated with decitabine were classified as intermediate-1 risk. There were 16 patients with 5q-, of whom 5 received lenalidomide (31%) compared with 7 of the 146 non-5q- patients (5%) (p=0.002). Of the 12 patients who received lenalidomide, 7 (58%) had intermediate-2 disease.
Conclusions: Our results suggest that in the modern treatment era, the majority of newly-diagnosed MDS patients in the United States receive supportive therapy soon after their initial diagnosis, while relatively few receive DMT. In our cohort, those who did receive DMT were much more likely to receive supportive treatment as well, perhaps to ameliorate the hematologic side effects of treatment. It is noteworthy that nearly one-third of patients treated with a DNA hypomethylating agent were classified as intermediate-1. A sizable proportion who received lenalidomide either did not have 5q- or were classified as intermediate-2, which are outside the drug’s approved indications.