Date of Award

Fall 1-10-2020

Degree Type

Dissertation

Degree Name

Doctor of Philosophy (PhD)

Department

Public Health

First Advisor

Richard Rothenberg

Second Advisor

Matthew Hayat

Third Advisor

Christine Kempton

Abstract

Hemophilia B is an inherited, X-linked, bleeding disorder caused by a mutation of the clotting factor 9 (FIX) gene. The mutation reduces the amount of FIX protein and results in spontaneous and trauma-related bleeding episodes. In 1994, approximately 2,800 men with hemophilia B (MWHB) were treated at hemophilia treatment centers (HTCs) in the United States (US). To date, studies examining health outcomes for MWHB in the US have not been compared across disease severities. Treatment of MWHB has become more complex with changes in prophylaxis practices in the US and the introduction of novel treatment products. Observational studies that describe health outcomes among MWHB and current treatment practices are important to inform future clinical practices. These cross-sectional analyses used data from MWHB enrolled in the Community Counts surveillance Registry (Registry) from 2014 to 2018. The first paper compared the sample of MWHB in the Registry to the population of MWHB who received treatment in HTCs and described the demographic, clinical factors, and health outcomes across disease severities. From 2014-2018, the population of MWHB who received care in HTCs included 4,816 MWHB, of which 2091 participated in the Registry. The second paper examined demographic, clinical factors, and health outcomes associated with treatment regimen, prophylaxis versus episodic; and used a marginal model. The final model included ethnicity, health insurance, history of a joint bleed, and interactions between severity by chronic pain as well as age by history of central venous access device utilization. The third paper examined demographic, clinical factors, and health outcomes associated with treatment product type utilization, standard half-life products versus extended half-life products, among MWHB on continuous prophylaxis; and used a marginal model. The final model included disease severity, enrollment year, HTC region, and percent of missed treatment dose. The second and third paper demonstrated that patient-level treatment outcomes were clustered by the HTCs where they received care. Future studies should examine the treatment dosage and frequency of administration of treatment products for MWHB on prophylaxis and replicate these studies for hemophilia A to determine if the factors associated with treatment are similar for all men with hemophilia.

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