News for Patients


Patient-derived stem cells shed light on pulmonary hypertension

Author: Leigh MacMillan
Date Published: September-2014
Source: Vanderbilt University

Pulmonary arterial hypertension (PAH) – high blood pressure in the lungs – has been linked to both heritable and idiopathic (spontaneous) causes. The underlying vascular changes that cause PAH are unclear and previous attempts to study changes in patient tissues have been limited by end-stage disease effects.

 

New Targets For Pulmonary Arterial Hypertension Treatments Identified By Researchers

Author: Patricia Inacio
Date Published: September-2014
Source: Pulmonary Hypertension News

A new study published in September’s issue of the American Journal of Respiratory and Critical Care Medicine, entitled, “The Sphingosine Kinase 1 / Sphingosine-1-Phosphate Pathway in Pulmonary Arterial Hypertension” reports the discovery of two novel therapeutic targets to treat pulmonary arterial hypertension.

 

Editorial: update on scleroderma and other fibrosing syndromes.

Author: Soumya Chatterjee
Date Published: September-2014
Source: Current Opinion in Rheumatology

With the turn of the century, the knowledge of the pathogenesis of fibrosing disorders has revolutionized, largely because of better understanding of the molecular mechanisms of fibrosis. This section update gives us a glimpse of the recent advances in the field from internationally renowned experts in scleroderma (SSc) and other fibrosing syndromes.

 

Scleroderma: the role of serum autoantibodies in defining specific clinical phenotypes and organ system involvement.

Author: Robyn Domsic
Date Published: September-2014
Source: Current Opinion in Rheumatology

Purpose of review: To discuss recent advances in serologic testing for systemic sclerosis (SSc)-associated antibodies with respect to the diagnosis and prognosis of the disease. Recent findings: The importance of SSc antibodies for diagnosis has become increasingly recognized, as evidenced by incorporation into the 2013 American College of Rheumatology/the European League Against Rheumatism clinical classification criteria for SSc.

 

Mast Therapeutics Announces Positive Top Line Results From Phase 2 Study Of AIR001 For Pulmonary Arterial Hypertension And AIR001 Clinical Development Plan

Author: PipelineReview.com
Date Published: September-2014
Source: PubMed

Mast Therapeutics, Inc. (NYSE MKT: MSTX) today reported top-line results from a Phase 2 study of AIR001 (sodium nitrite) inhalation solution for the treatment of pulmonary arterial hypertension (PAH). Mast obtained the rights to the AIR001 program through its acquisition of privately-held Aires Pharmaceuticals, Inc. earlier this year.

 

Emerging cellular and molecular targets in fibrosis: implications for scleroderma pathogenesis and targeted therapy.

Author: Castelino FV, Varga J.
Date Published: September-2014
Source: Current Opinion in Rheumatology

PURPOSE OF REVIEW: To summarize the recent advances in understanding the novel cytokine pathways, intracellular signaling molecules, cell-fate decisions, cellular aging and senescence, and the cross-talk of effector cells and the extracellular matrix (ECM) in the pathogenesis of systemic sclerosis (SSc) fibrosis. RECENT FINDINGS: Studies from the animal models and human beings implicate novel molecular pathways such as Wnts, the chemokines, chemokine (C-X-C motif) ligand 4 and chemokine (C-C motif) ligand 2, and the lipid mediators lysophosphatidic acid and sphingosine-1-phosphate in the pathogenesis of SSc. These signals, coupled with the mesenchymal cell-fate decisions, contribute to aberrant fibroblast activation and myofibroblast accumulation.

 

Update on scleroderma-associated interstitial lung disease.

Author: Fan MH, Feghali-Bostwick CA, Silver RM.
Date Published: September-2014
Source: Current Opinion in Rheumatology

PURPOSE OF REVIEW: Systemic sclerosis (SSc), or scleroderma, is a heterogeneous and complex autoimmune disease characterized by varying degrees of skin and organ fibrosis and obliterative vasculopathy. The disease results in significant morbidity and mortality, and to date, available treatments are limited. Lung involvement is the leading cause of death of patients with SSc. Over the past year, significant advances have been made in our understanding of SSc-associated lung disease, and this review attempts to encapsulate these most recent findings and place them in context.

 

Recent advances in scleroderma-associated pulmonary hypertension.

Author: Kristen Highland
Date Published: September-2014
Source: Current Opinion in Rheumatology

Purpose of review: This review summarizes recent advances in pulmonary hypertension, a leading cause of morbidity and mortality in scleroderma (SSc). Recent findings: Although WHO Group I pulmonary arterial hypertension (PAH) is the most common cause of pulmonary hypertension, all WHO Groups can occur. PAH is now a criterion for the diagnosis of SSc. Results of recent research have resulted in greater insight into the epidemiology of SSc-pulmonary hypertension with regard to prevalence, incidence and clinical risk factors.

 

First-Line Combination of Ambrisentan and Tadalafil Reduces Risk of Clinical Failure Compared to Monotherapy in Pulmonary Arterial Hypertension Outcomes Study

Author: Gilead
Date Published: September-2014
Source: BusinessWire

Gilead Sciences, Inc. (Nasdaq:GILD) today announced results from the AMBITION study (a randomized, double-blind, multicenter study of first-line combination therapy with AMBrIsentan and Tadalafil in patients with pulmonary arterial hypertensION), which was conducted in collaboration with GlaxoSmithKline (GSK). In AMBITION, first-line treatment of pulmonary arterial hypertension (PAH) with the combination of ambrisentan 10 mg and tadalafil 40 mg reduced the risk of clinical failure by 50 percent compared to the pooled ambrisentan and tadalafil monotherapy arm (hazard ratio = 0.502; 95 percent CI: 0.348, 0.724; p=0.0002).

 

FDA Grants Arena Pharmaceuticals’ PAH Investigational Drug Orphan Drug Status

Author: Leonor Mateus Ferreira
Date Published: September-2014
Source: Pulmonary Hypertension News

The U.S. Food and Drug Administration has granted APD811, an investigational drug candidate internally developed by Arena Pharmaceuticals, orphan drug status. APD811 is an oral agonist of the prostacyclin (IP) receptor, which is designed for the treatment of vasospastic diseases, such as pulmonary arterial hypertension (PAH). “The FDA Office of Orphan Products Development (OOPD) evaluates scientific and clinical data submissions from sponsors to identify and designate drug candidates that could potentially treat rare diseases to help advance the evaluation and development of such products,” explained Craig M. Audet, Ph.D., Arena’s senior vice president of operations and head of global regulatory affairs.

 

New Study Outlines Potential PH Therapy Targets

Author: Patricia Inacio
Date Published: August-2014
Source: Pulmonary Hypertension News

Pulmonary hypertension is a condition caused by increased pressure in the pulmonary arteries. In advanced cases, its symptoms (shortness of breath, tiredness, chest pain) worsen and may limit all physical activity. Now, researchers from University of California, Los Angeles have developed a successful treatment to rescue advanced forms of pulmonary hypertension in experiments involving rodent models.

 

Kidney involvement in systemic sclerosis.

Author: Steen VD.
Date Published: August-2014
Source: Press Medical

Kidney involvement in systemic sclerosis (SSc) is primarily manifested by scleroderma renal crisis (SRC). Formerly, it was the most severe complication in scleroderma and was the most frequent cause of death in these patients. More than 30years ago, with the development of angiotensin converting enzyme (ACE) inhibitors, SRC became a very treatable complication of scleroderma. Although there are still many patients who do not survive and have poor outcomes, early diagnosis of renal crisis and prompt therapeutic intervention can achieve excellent outcomes. Renal abnormalities independent of renal crisis have been noted, but can usually be attributed to other problems.

 

Update in systemic sclerosis-associated pulmonary arterial hypertension

Author: Gashouta MA, Humbert M, Hassoun PM
Date Published: August-2014
Source: Press Medical

Pulmonary arterial hypertension (PAH) is one of the leading causes of death in systemic sclerosis (SSc). Despite advances in treatment options for PAH, long-term prognosis remains poor for scleroderma-associated PAH (SSc-PAH). Although prompt diagnosis and treatment of PAH may have significant impact on survival rates, early detection of the syndrome continues to be challenging in SSc due to several factors ranging from limitations of the current screening tools and the complexities of the disease. In comparison with other PAH subgroups, SSc-PAH patients respond poorly to conventional forms of PAH therapy.

 

First-Line Combination of Ambrisentan and Tadalafil Reduces Risk of Clinical Failure Compared to Monotherapy in Pulmonary Arterial Hypertension Outcomes Study

Author: Bene MD, Pozzi MR, Rovati L, Mazzola I, Erba G, Bonomi S
Date Published: August-2014
Source: PubMed

Digital ulcers (DUs) occur in up to 50% of patients with Systemic Sclerosis (SSc). DUs are painful, recurring and lead to functional disability. Management of DUs includes pharmacologic and local therapy, the healing process is slow and the ulcer can become infected or evolve to gangrene. Autologous fat grafting (AFG) is a technique used to promote tissues repair. We used AFG to treat DUs refractory to conventional treatment to enhance healing process.

 

Fat Cell Transplants Boost Hand Function in SSc

Author: Rita Baron-Faust
Date Published: August-2014
Source: Rheumatology Network

Transplanted into their fingers, their own fat cells produce improvements in hand disability, pain, swelling and Raynaud’s phenomenon in as little as two months among patients with scleroderma, according to preliminary results from a small clinical trial in France. In the open-label, single-arm trial, autologous adipose-derived stromal/stem cells (ADSC) were aspirated from the abdomen, purified, and injected into the fingers of 12 women with systemic sclerosis (SSc), with no serious adverse consequences.

 

Joint and tendon involvement predict disease progression in systemic sclerosis: a EUSTAR prospective study

Author: J. Avouac, U. Walker, E. Hachulla, G. Riemekasten, G. Cuomo, et al
Date Published: August-2014
Source: Annals of the Rheumatic Diseases

Objective To determine whether joint synovitis and tendon friction rubs (TFRs) can predict the progression of systemic sclerosis (SSc) over time. Patients and methods We performed a prospective cohort study that included 1301 patients with SSc from the EUSTAR database with disease duration ≤3 years at inclusion and with a follow-up of at least 2 years. Presence or absence at clinical examination of synovitis and TFRs was extracted at baseline. Outcomes were skin, cardiovascular, renal and lung progression. Overall disease progression was defined according to the occurrence of at least one organ progression.

 

Sclerosis: Autologous Cell Transfers May Help Patients

Author: Jennifer Garcia
Date Published: August-2014
Source: Medscape Medical News

Injections of autologous stromal vascular fraction (SVF) may improve hand function and decrease pain among patients with systemic sclerosis (SSc), according to a new study published online August 11 in the Annals of the Rheumatic Diseases. The phase 1, open-label study enrolled 12 female patients with SSc and a Cochin Hand Function Scale score higher than 20/90. Patients receiving vasodilator or immunosuppressive therapy in the 3 months before or 6 months after enrollment were excluded from the study. Patients were administered subcutaneous injections of autologous SVF into each finger of both hands and were evaluated over the course of a 6-month period.

 

4 keys to appealing a rejected insurance claim

Author: Tom Murhpy
Date Published: August-2014
Source: Associated Press

Keep calm and take notes. Stay true to this principle and you can improve your odds of successfully fighting a health insurer's claim rejection. Experts who help with the appeals process say patients have a 50 percent chance or better of prevailing. They say a winning argument may require heavy doses of research and persistence, but the end result is a decision that can stave off thousands of dollars in medical bills.

 

Pharmacologic Therapy for Pulmonary Arterial Hypertension in Adults: CHEST Guideline and Expert Panel Report

Author: D. Taichman, MD, PhD, FCCP; J. Ornelas, MS; L., MD; J. Klinger, MD, FCCP; et al
Date Published: August-2014
Source: Chest

OBJECTIVE: Choices of pharmacologic therapies for pulmonary arterial hypertension (PAH) are ideally guided by high-level evidence. The objective of this guideline is to provide clinicians advice regarding pharmacologic therapy for adult patients with PAH as informed by available evidence. METHODS: This guideline was based on systematic reviews of English language evidence published between 1990 and November 2013, identified using the MEDLINE and Cochrane Library databases. The strength of available evidence was graded using the Grades of Recommendations, Assessment, Development, and Evaluation methodology. Guideline recommendations, or consensus statements when available evidence was insufficient to support recommendations, were developed using a modified Delphi technique to achieve consensus.

 

Low Blood Pressure Increases Risk of Death in Patients with Systemic Sclerosis-Associated Pulmonary Arterial Hypertension

Author: Lara C. Pullen, PhD
Date Published: July-2014
Source: The Rheumatologist

Patients with pulmonary arterial hypertension (PAH) associated with systemic sclerosis (SSc-APAH) have a higher mortality rate than non-SSc-APAH patients with idiopathic disease as well as those with other connective tissue diseases (CTD-APAH). In a recent study, researchers pinpoint specific predictors that put patients with SSc-APAH at higher risk of death.