Semester

Spring

Date of Graduation

2012

Document Type

Dissertation

Degree Type

PhD

College

School of Pharmacy

Department

Pharmaceutical Sciences

Committee Chair

S Suresh Madhavan

Abstract

Multiple sclerosis (MS) is a chronic inflammatory demyelinating neurological disorder with no known cure. Disease modifying drugs (DMDs) used in the treatment of MS reduce the frequency relapses and delay the progression of the disease. Although DMD therapy is the mainstay of treatment of MS, it is associated with variety of side effects and severe adverse events. A majority of currently available DMDs are injectable, except one which is available in the pill form. Therapeutic decisions in MS are challenging due to the inconvenience of administration, frequency of administration, longer duration of therapy, side effects, and risk of adverse events associated with DMDs. The purpose of this series of studies was to better understand different aspects of DMDs from patients' point of view. A total of three studies were conducted using qualitative and quantitative research methods. Data for the first study were collected using focus group interviews among eighteen MS patients attending neurology clinic affiliated to a teaching hospital. Study two and study three were performed using a web-based survey questionnaire in a sample of MS patients residing in the United States using a cross-sectional study design. The specific objectives are the three studies were: 1) To explore patients' experiences, opinions, and expectations related to DMDs used in MS, 2) to estimate preference weights and relative importance of attributes for DMDs used in MS treatment using conjoint analysis, and 3) to assess disease and treatment related factors associated satisfaction with treatment among MS patients. Participants in the first study had an understanding of importance of DMDs in the therapeutic management of MS. MS patients reported to adapt themselves to available DMD choices, but had greater expectations from emerging DMDs. Participants reported that the convenience of DMD administration, occurrence of relapses, delay of disability progression were important factors related to DMD; however, most were concerned about the side-effects that affected day-to-day functioning and the adverse events associated with DMDs. The results of the second study revealed that attributes indicating risks associated with DMDs (progressive multifocal leukoencephalopathy and severe liver dysfunction) were the most important attributes followed flu-like symptoms, delaying disability progression, frequency of relapses, and mode of DMD administration. Strength of evidence on treatment outcomes expressed in number of years was the least important attribute to patients. The results of the third study indicated that factors such as type of current DMD used, relapses experienced, disability status, total number of MS symptoms experienced, and past experiences with DMDS were associated with lower treatment satisfaction scores.

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