Direct Medical Costs Associated With Treatment Nonpersistence in Patients With Higher-Risk Myelodysplastic Syndromes Receiving Hypomethylating Agents: A Large Retrospective Cohort Analysis


Suboptimal 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 methods

Using 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.


Among 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.


There 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.

AuthorsN Joshi, H Kale, S Corman, T Wert, K Hill, AM Zeidan
JournalClinical Lymphoma Myeloma Leukemia
Therapeutic AreaOncology
Service AreaReal-World Evidence
LinkClick Here

Share this post