Abstract
BackgroundSuboptimal use of hypomethylating agents (HMAs) among higher-risk myelodysplastic syndrome (HR-MDS) patients can translate into worse health outcomes and economic burden. We estimated the direct medical costs associated with HMA treatment nonpersistence among HR-MDS patients. Patients and MethodsUsing the Surveillance, Epidemiology, and End Results–Medicare linked database, a retrospective cohort of patients diagnosed with refractory anemia with excess blasts (RAEB), a diagnosis that substantially overlaps with HR-MDS, between January 2011 and December 2015 was analyzed. Patients who had ≥ 1 year of continuous Medicare enrollment before diagnosis and who did not receive stem cell transplant or lenalidomide in the follow-up period were included. Patients receiving HMAs were stratified into HMA persistent (≥4 HMA cycles) and HMA nonpersistent (<4 cycles or a gap of ≥ 90 days between cycles) groups. Healthcare resource use and costs during the follow-up period were reported descriptively as total and per patient per month (PPPM). Weighted generalized linear models (GLM) were used to compare estimated healthcare resource use and costs between HMA groups. ResultsAmong the 664 patients with RAEB, 295 (44.4%) were HMA nonpersistent and 369 (55.6%) HMA persistent. On the basis of weighted GLM analysis, the HMA nonpersistent group incurred significantly (P < .05) higher total PPPM costs compared to the HMA persistent group ($18,039 vs. $13,893), particularly for hospitalization ($3,375 vs. $2,131), and emergency room ($5,517 vs. $2,867) costs. ConclusionThere is a substantial economic burden associated with early discontinuation of guideline-recommended HMA therapy in RAEB patients. The study findings necessitate closer care management in this population in order to improve outcomes and reduce healthcare spending.
Highlights
Hypomethylating agents (HMAs) are guideline recommended therapeutic options for patients with higher-risk myelodysplastic syndromes (HR-MDS).[1]
A total of 664 patients diagnosed with HR-MDS (RAEB) who initiated treatment with HMA were included in the study (Figure 1)
Patient characteristics between HMA-persistent and -nonpersistent groups were similar; compared to those with HMA persistence, those with nonpersistence were older at HR-MDS diagnosis, and a lower proportion of patients were married and initiated therapy with azacitidine (Table 1)
Summary
Hypomethylating agents (HMAs) are guideline recommended therapeutic options for patients with higher-risk myelodysplastic syndromes (HR-MDS).[1]. HMA treatment is provided in multiple cycles, with each cycle comprising 5 to 7 consecutive days of azacitidine or decitabine administered intravenously or subcutaneously in the widely recommended schedules for patients with HR-MDS. There have been reported difficulties ensuring treatment cycles are administered as scheduled.[5] In their study among patients with MDS and other hematologic cancers, Tendas et al[6] reported a delay in 31% of the scheduled azacitidine cycles. The 7-day treatment schedule of HMA, which requires weekend administration, has been noted to be challenging for both patients and treatment centers.[7] In order to elicit clinical response, HMA treatment is recommended to be provided to patients for least 4, or even 6, treatment cycles.[8] Premature treatment discontinuation or suboptimal treatment is likely to affect clinical and economic outcomes. There is some evidence to suggest that underperformance (loss of response or inability to achieve a primary response) of HMAs is linked to premature termination of treatment cycles[9]; the economic impact of premature HMA treatment discontinuation is currently unknown
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