Challenges of pulmonary hypertension make it an ideal candidate for DM

TODAY'S APPROACH to interventions that address chronic disease and conditions is both broader and deeper than ever before. But there is another group of patients who rarely, if ever, directly benefit from these programs. According to Alan Wright, MD, former chief medical officer of TheraCom, these are patients who might use more than $100,000 a year in pharmaceutical spending and/or resources, patients who have a disease that is progressive with no known cure. These are the patients who have a rare disease, such as pulmonary arterial hypertension (PAH), a rare blood vessel disorder characterized by increased pressure in the pulmonary artery.

Many primary care physicians are unfamiliar with the symptoms of PAH and therapies, and all too often do not consider the presenting symptoms of a typical PAH patient to be potentially life-threatening. In its earliest stages, PAH symptoms are very similar to those of asthma, allergic reaction, or other diagnoses. As a result, many patients lose precious time in the early stage of the disease, navigating the complexities of the system and looking for answers.

Characterized by vague and erratic symptoms, patients might complain of swelling of extremities, shortness of breath, dizziness, fainting and tiredness. With declining lung function, patients die from right-side heart failure. Patients might develop the disease idiopathically or genetically. Physicians who have patients with existing sickle cell disease, scleroderma, lupus, HIV or liver disease should know that these patients are at high risk for developing secondary PAH and should be monitored closely for signs of PAH symptoms. Patients are currently treated based upon the level of severity of symptoms using the New York Heart Assn. (NYHA) Class Indication of Severity.

"This disease is very often overlooked, and patients typically experience two years of frustrating physician visits before being correctly diagnosed," Aldrighetti says.

Not until 1995 was a pharmaceutical therapy commercially available, and today there are medications in three classes of drugs used to treat PAH patients: prostacyclin analogues (Flolan, Remodulin,Ventavis), endothelin receptor antagonists (Tracleer), phosphodiesterase-5 inhibitor (Revatio), and more in the development pipeline. While great strides have been made in drug research, PAH specialists echo what the Pulmonary Hypertension Assn. has been saying for several years: Early diagnosis is still the most critical factor for improved outcomes.

However, physicians cannot easily diagnose a disease when they are unfamiliar with the typical symptoms, or have never encountered a case in their practice.

"When you have a young, generally healthy female patient who presents with the symptoms of PAH-dizziness, shortness of breath, fatigue-your first thought will probably not be, 'This is a rare disease,' but rather, 'This is most likely asthma, or another of the many common respiratory problems,' " says Gary Owens, MD, vice president of medical management and policy for Philadelphia-based Independence Blue Cross Blue Shield.

Despite the low-reported incidence, "the nature of PAH origins, its disease progression and associated care management challenges clearly suggest the need for some form of disease management," says Steve Schelhammer, the founder and now retired CEO of Accordant, a disease management company specializing in rare, high-cost, chronic diseases.

"Because of the relatively low incidence of this disease, it is difficult to justify the cost, and thus the business case, for developing and operating a high-touch, nurse-driven, disease management program in the traditional sense, in a managed care setting," Schelhammer says.

Compounding these challenges is the reality that health services for patients with PAH are delivered almost exclusively by specialists in immunology/rheumatology, cardiology and pulmonary specialty practices. For some patients, this care is only available at Centers of Excellence, requiring great driving distances, overnight stays, and sometimes even relocation. Coordinated care is available, but only after patients are diagnosed as their medication needs are managed through specialty pharmacies.