Systemic Sclerosis and Vascular Dysfunction
“Systemic Sclerosis (SSc) Vasculopathy: Improved Clinical Monitoring and Treatment”
Systemic sclerosis (SSc; scleroderma) is a multi-organ systemic disease characterized by activation of immune cells, which results in vascular dysfunction (vasculopathy) and subsequent scarring (fibrosis). SSc has a higher than expect prevalence in the US military. On a national level there are 5,766 SSc patients (ICD-9 710.1) presently cared for in the Veterans Health Administration (VHA). While there is no cure for SSc, studies of therapeutics that can help slow disease progression are valuable to our Veterans. This proposal addresses the solicitation for projects with attention to SSc requested by President Obama after reviewing potential contamination of water at Camp Lejeune. This proposal is a patient-centered outreach for our Veterans with SSc to inform and prevent catastrophic endstage vascular abnormalities, including digital ulcers, pulmonary arterial hypertension (PAH) and scleroderma renal crisis in SSc. The study proposes a novel application of a therapeutic for this disease. A better understanding of the initiating insult and natural progression of SSc vasculopathy is needed in order to develop therapeutics with a goal of curing/treating the underlying disease. This project has the potential to impact not only Veterans with SSc, but also those with vascular abnormalities including digital ulcers, PAH, and renal crisis. This proposal represents a potential major therapeutic advance for our Veterans with SSc.
Drug - BH4
BH4 is between 5-20 mg/kg/day given to a total of 32 SSc patients (16 complicated, 16 uncomplicated)
Non-invasive technique, flow mediated dilatation (FMD) to define vasculopathy in SSc.
Drug - Placebo
This will be given to a total of total of 32 SSc patients (16 complicated, 16 uncomplicated)
Systemic Sclerosis (SSc) Vasculopathy: Improved Clinical Monitoring and Treatment