Date of Award

1-1-2020

Document Type

Open Access Thesis

Degree Name

Master of Public Health (MPH)

Department

School of Public Health

First Advisor

Xiaomei Ma

Second Advisor

Nikolai Podoltsev

Abstract

Background: Current treatments for Myelofibrosis (MF) are largely palliative, with ruxolitinib being the breakthrough treatment approved for higher-risk patients by the Food and Drug Administration in November of 2011. Little is known about the “real-world” patterns of care and outcomes of MF patients since the introduction of ruxolitinib. Patients and Methods: The SEER-Medicare database was used to identify older patients diagnosed with MF from 2007 through 2015. Treatment patterns were assessed using Medicare part B and D claims, and multivariate cox proportional hazards regression models were used to assess the effect of ruxolitinib use on survival in MF patients. Results: A total of 773 patients were identified. The median age of our study population (N=773) was 76 years (IQR, 70, 80), and 88.9% of the population was non-Hispanic white. Of 342 patients who were diagnosed during 2012-2015 (i.e., the ruxolitinib era), 127 (37.1%) were ruxolitinib users. The median duration of ruxolitinib use was 16.21 months. Most ruxolitinib users started with doses of 5 (23.6%), 10 (27.0%), or 20 (23.6%) mg twice a day (BID). Among the patients who started treatment at 5 mg BID, 56.6% were never able to increase the dose above 5 BID. Only 3.1% were able to use the maximum dose of 25 mg BID. 54.3% of patients were taking hydroxyurea or prednisone during the same time period that they took ruxolitinib. Medications to manage anemia were used more commonly by MF patients diagnosed before 2012. Among patients diagnosed in 2012-2015, there was no difference in survival between ruxolitinib users and non-users. Conclusion: Older MF patients treated with ruxolitinib had similar survival when compared to patients who did not receive this medication but possibly belonged to the lower disease risk group. For many ruxolitinib users, the dose of ruxolitinib could not be escalated, additional medications were used concurrently, and the drug was discontinued quickly after initiation. Optimization of ruxolitinib use may be necessary to accomplish better outcomes. Furthermore, development of new drugs which may be used together with ruxolitinib or after its discontinuation is needed.

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