Michael Stevenson
AMAG Pharmaceuticals
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Publication
Featured researches published by Michael Stevenson.
Expert Review of Cardiovascular Therapy | 2017
Michael Davidson; Michael Stevenson; Andrew Hsieh; Zahid Ahmad; Caroline Crowson; Joseph L. Witztum
ABSTRACT Background: Familial Chylomicronemia Syndrome (FCS) is a rare genetic disorder that is caused by a decrease or an absence of lipoprotein lipase activity. FCS is characterized by marked accumulation of chylomicrons and extreme hypertriglyceridemia, which have major effects on both physical and mental health. To date, there have been no systematic efforts to characterize the impact of chylomicronemia on FCS patients’ lives. In particular, the impact of FCS on the burden of illness (BoI) and quality of life (QoL) has not been fully described in the literature. Methods: IN-FOCUS was a comprehensive web-based research survey of patients with FCS focused on capturing the BoI and impact on QoL associated with FCS. Sixty patients from the US diagnosed with FCS participated. Patients described multiple symptoms spanning across physical, emotional and cognitive domains. Results: Patients on average cycled through 5 physicians of varying specialty before being diagnosed with FCS, reflecting a lengthy journey to diagnosis Nearly all respondents indicated that FCS had a major impact on BoI and QoL and significantly influenced their career choice and employment status, and caused significant work loss due to their disease. Conclusion: FCS imparts a considerable burden across multiple domains with reported impairment on activities of daily living and QoL.
Expert Review of Clinical Pharmacology | 2017
Zahid Ahmad; Rob Halter; Michael Stevenson
Familial chylomicronemia syndrome (FCS), also referred to as type 1 hyperlipoproteinemia or lipoprotein lipase deficiency, is a rare genetic lipid disorder characterized by severe elevations in cir...
Expert opinion on orphan drugs | 2018
Valerie Salvatore; Alan Gilstrap; Karren Williams; Swati Thorat; Michael Stevenson; A. R. Gwosdow; Andrew Hsieh; Brant C Hubbard; David Davidson
ABSTRACT Background: Familial chylomicronemia syndrome (FCS) is a disease caused by impaired lipoprotein lipase function and characterized by chylomicronemia, reduced quality of life (QoL) and risk of pancreatitis. The aim of the current study is to assess if QoL can be improved by patients being connected to other patients. Methods: Respondents (N = 50) categorized into 3 groups (actively connected, passively connected and non-connected) self-reported their current or comparative assessments of QoL before and after connection with FCS-focused support organizations using a customized retrospective web-based survey. Results: Connected respondents showed significantly improved perceptions of overall health, disease severity, motivation to take care of health and emotional well-being (p ≤ 0.05). Any level of connection produced noticeable benefits, but active connection in the form of regular interaction with other patients reported the greatest improvements. Additionally, respondents reported higher levels of satisfaction with their primary treating physician after being connected. The majority of patients (62%) reported joining support groups following referrals from their physicians. Conclusions: Similar to other disease states, connecting with other patients with FCS had a positive impact on aspects of quality of life. Physicians may play a central role in referring their patients with FCS to support groups.
Expert Review of Cardiovascular Therapy | 2018
Marcello Arca; Andrew Hsieh; Handrean Soran; Paul D. Rosenblit; Louis O’Dea; Michael Stevenson
ABSTRACT Background: Volanesorsen, an investigational inhibitor of apoC-III synthesis, significantly reduced triglyceride levels in clinical trials in patients with familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by marked chylomicronemia leading to a spectrum of symptoms, including recurrent abdominal pain and episodes of potentially fatal acute pancreatitis (AP). Objective: To determine the effect of volanesorsen on burden of disease on patients with FCS Methods: ReFOCUS was a retrospective global web-based survey open to patients with FCS who received volanesorsen for ≥3 months in an open-label extension study. The survey included questions about patients’ experiences before and after volanesorsen treatment. Results: Twenty-two respondents had received volanesorsen for a median of 222 days. Volanesorsen significantly reduced the number of symptoms per patient across physical, emotional, and cognitive domains. Significant reductions from baseline were reported for steatorrhea, pancreatic pain, and constant worry about an attack of pain/AP. Respondents reported that volanesorsen improved overall management of symptoms and reduced interference of FCS with work/school responsibilities. Reductions in the negative impact of FCS on personal, social, and professional life were also reported. Conclusions: Treatment with volanesorsen has the potential to reduce disease burden in patients with FCS through modulation of multiple symptom domains.
Journal of Clinical Lipidology | 2018
Michael Davidson; Michael Stevenson; Andrew Hsieh; Zahid Ahmad; Jeanine E. Roeters van Lennep; Caroline Crowson; Joseph L. Witztum
Blood | 2012
Saroj Vadhan Raj; Andrew Hsieh; William Strauss; Michael Stevenson; Kristine Bernard; Lee F. Allen
Journal of Clinical Lipidology | 2018
Michael Davidson; Michael Stevenson; Andrew Hsieh; Zahid Ahmad; Jeanine Roeters van Lennep; Joseph L. Witztum
Journal of Clinical Lipidology | 2018
Marcello Arca; Andres Digenio; Andrew Hsieh; Paul D. Rosenblit; Michael Stevenson
Journal of Clinical Lipidology | 2018
Valerie Salvatore; Alan Gilstrap; Karren Williams; Andrew Hsieh; A. R. Gwosdow; Michael Stevenson; David Davidson
Atherosclerosis Supplements | 2018
Michael Davidson; Andrew Hsieh; Zahid Ahmad; Jeanine Roeters van Lennep; Michael Stevenson