New Criteria Could Lead to Earlier Diagnosis of Scleroderma
(National Pain Report) A new classification criterion for systemic sclerosis, a chronic autoimmune disease also known as scleroderma, could help physicians diagnose and begin treating patients sooner. The new criteria, developed by a joint committee formed by the American College of Rheumatology (ACR) and the European League Against Rheumatism, are published in the ACR journal, Arthritis & Rheumatism. They are the first changes in the criteria since 1980. Read article
Saturday, October 19, 2013 4:14:00 PM
Eleventh PH Drug Approved, The Second in Just Ten Days
Today the U.S. Food and Drug Administration (FDA) approved the eleventh pulmonary arterial hypertension (PAH) treatment in the U.S., Actelion Pharmaceutical’s macitentan (commercially known as Opsumit), an oral dual endothelin-receptor antagonist. This comes just ten days after the approval of riociguat (commercially known as Adempas) for the treatment of PAH and some cases of chronic thromboembolic pulmonary hypertension (CTEPH). These two new drugs are the first in five years to be approved by FDA for treating PH.
According to Healio, the phase 3 SERAPHIN drug trial for macitentan was the first to test a PAH drug for its effects on morbidity and mortality. Patients who took the drug showed lower rates of morbidity and mortality as compared to patients who received a placebo, showed improved six-minute walk tests and more showed improvement in their WHO functional class.
Acknowledging the achievement of adding an eleventh drug to the choices for PAH patients in a rare disease state, PHA President Rino Aldrighetti said, “Of the 7,000 rare diseases, only three have as many or more treatments available. We are fortunate to have a strong and growing research community with great communication and cooperation. That has helped foster industry interest in exploring new options for patients.”
PHA itself has committed more than $12.5 million to support cutting-edge research focused on pulmonary hypertension. PHA has been able to leverage such substantial funding through partnerships with the National Heart, Lung, and Blood Institute (NHLBI), American Thoracic Society (ATS), American Heart Association (AHA), and Pfizer.
We know that your ultimate wish is for a cure, and that is why PHA will continue to support the work of researchers until the day when no one suffers from PH.
Learn more: FDA press release | macitentan study findings | PH treatments in the U.S. | PH treatments worldwide | PHA Research Program
Friday, October 18, 2013 4:11:00 PM
Researchers Identify Possible Treatment Avenues for Scleroderma
(Science Codex) Using mice, lab-grown cells and clues from a related disorder, Johns Hopkins researchers have greatly increased understanding of the causes of , showing that a critical culprit is a defect in the way certain cells communicate with their structural scaffolding. They say the new insights point the way toward potentially developing drugs for the disease, which affects approximately 100,000 people in the United States. Read article
Friday, October 18, 2013 4:03:00 PM
New Drug Approved for PAH and CTEPH Patients in U.S.
On Tuesday, the U.S. Food and Drug Administration (FDA) approved Bayer’s riociguat pill for the treatment of pulmonary arterial hypertension (PAH) and for chronic thromboembolic pulmonary hypertension (CTEPH). Riociguat, commercially known as Adempas, will be used for CTEPH patients after pulmonary thromboendarterectomy (PTE) surgery or if surgery is not possible. This will be the first drug available for CTEPH patients and tenth for PAH.
PHA President Rino Aldrighetti says, “Treatment opportunities for patients are growing rapidly. Eighteen years ago there were no treatments for pulmonary arterial hypertension. Twelve years ago there was one. Today there are ten, with a possible eleventh later this year (Actelion’s macitentan). Of the 7,000 rare diseases, only three have as many or more treatments available. We are fortunate to have a strong and growing research community with great communication and cooperation. That has helped foster industry interest in exploring new options for patients.”
Better Prognosis for PH Patients
The strong progress in the field of pulmonary hypertension over the past two decades has led to a better prognosis for PH patients. Whereas physicians treating PH once delivered terminal diagnoses, they now expect their patients to live with their condition. Many patients survive for years -- even 10-20 years or more -- with proper PH treatment.
Patients Not Receiving Treatments in a Timely Manner
“The problem we now face is getting these treatments to patients sooner. While we have made huge strides over the past 20 years, the time it takes to reach a correct PH diagnosis has not changed,” explains PHA Vice President for Community Engagement Mollie Katz. “With missed or late diagnosis, the disease continues to get worse, affecting patients’ survival and quality of life. Many patients will see three or more different physicians over a three year period before they are properly diagnosed with PH, and almost three-fourths have advanced PH by the time they are diagnosed. Because PHA sees early diagnosis as a huge priority, second only to a cure, we are working with medical professionals and patients to improve diagnosis through our early diagnosis campaign, Sometimes it’s PH.
Not There Yet
While there are 10 treatments for pulmonary arterial hypertension (PAH) and now one for CTEPH, there are none approved for pediatric patients. PHA has launched the only pediatric PH research program in the United States, the Robyn Barst Pediatric Research and Mentoring Fund.
And, says Rino, “Ultimately, our goal is a cure. With increasing numbers of researchers engaged in more studies every year, we are hopeful and encouraged as we see that hope turning into action.”
Learn more: FDA decision | riociguat study findings | PH treatments in the U.S. | PH treatments worldwide | Sometimes it’s PH website
Thursday, October 10, 2013 4:08:00 PM
Galectin-3 Strong Prognostic Marker in Patients with Pulmonary Hypertension
(Healio) Patients with pulmonary hypertension and elevated galectin-3 were more likely to have a higher mortality rate at 4 years compared with patients with lower levels of the biomarker, according to data presented at the Annual Scientific Meeting of the Heart Failure Society of America. Read article
Monday, October 07, 2013 4:16:00 PM