Briefly describe a specific question, or set of related questions, about a health care test or treatment that this program should consider.

The American College of Chest Physicians (ACCP) proposes to update the medical therapies section of 2007 updated publication on Medical Therapies for Pulmonary Arterial Hypertension: ACCP Evidence-Based Clinical Practice Guidelines.1

1. Medical Therapy for Pulmonary Arterial Hypertension: Updated ACCP Evidence-Based Clinical Practice Guidelines. Chest 2007; 131 (6):1917-28

Does your question include a comparison of different health care approaches? (If no, your topic will still be considered.)

yes

If yes, explain the specific technologies, devices, drugs, or interventions you would like to see compared:

2 new drugs have now been approved for the treatment of PAH:

• inhaled treprostinil
• ambrisentan

Comparisons should be made between these new drugs and the older ones, listed here by class:

• calcium channel antagonists (nifedipine, diltiazem, amlodipine, but not verapamil)
• prostanoids (epoprostenol, treprostinil, iloprost)
• endothelin antagonists (bosentan, ambrisentan)
• phosphodiesterase inhibitors (sildenafil)

Furthermore, there is now some support in the literature for combination therapies. It would be ideal to include combinations in the CER effort.

What patients or group(s) of patients does your question apply to? (Please include specific details such as age range, gender, coexisting diagnoses, and indications for therapy.)

Patients with pulmonary arterial hypertension, defined as a mean pulmonary artery pressure ?25 mm Hg with a pulmonary capillary wedge pressure ?15 mm Hg measured by cardiac catheterization.

Are there subgroups of patients that your question might apply to? (For example, an ethnic group, stage or severity of a disease.)

No exclusions for age, gender, ethnicity, or co-morbidities

Treatment might be severity dependent and should be defined by functional class

Describe the health-related benefits you are interested in. (For example, improvements in patient symptoms or problems from treatment or diagnosis.)

Control of pulmonary artery pressure, and prevention of right ventricular dysfunction, right heart failure, and mortality. Other benefits include improvement in dyspnea, functional class, and quality of life.

Describe any health-related risks, side effects, or harms that you are concerned about.

Side effects of various drugs include but are not limited to liver function abnormalities, elevated serum aminotransferase concentration, headache, flushing, epistaxis, dyspepsia, diarrhea, peripheral edema, nausea, nasal congestion, dizziness, increased INR or prothrombin time.

Appropriateness for EHC Program

Does your question include a health care drug, intervention, device, or technology available (or likely to be available) in the U.S.?

yes

Which priority area(s) and population(s) does this topic apply to? (check all that apply)

EHC Priority Conditions (updated in 2008)

• Cardiovascular disease, including stroke and hypertension
• Functional limitations and disability
• Pulmonary disease/asthma

AHRQ Priority Populations

• Low income groups
• Minority groups
• Women
• Children
• Elderly
• Individuals with special health care needs, including individuals with disabilities or who need chronic care or end-of-life health care

Federal Health Care Program

None

Importance

Describe why this topic is important.

PAH may occur either in the setting of a variety of underlying medical conditions or as a disease that uniquely affects the pulmonary circulation. Usually, PAH does not become manifest until extensive vascular damage has already resulted. It can be progressive, sometimes resulting in death.

What specifically motivated you to ask this question? (For example, you are developing a clinical guideline, working with a policy with large uncertainty about the appropriate approach, costly intervention, new research you have read, items in the media you may have seen, a clinical practice dilemma you know of, etc.)

Since the 2007 update of the medical therapies section of the 2004 guidelines, 2 new drugs have been approved by the FDA and the literature is showing some support for treatment with combination therapies.

Does your question represent uncertainty for clinicians and/or policy-makers? (For example, variations in clinical care, controversy in what constitutes appropriate clinical care, or a policy decision.)

yes

If yes, please explain:

This is a highly complex disease with a limited number of experts. Patients are usually referred to specialists for treatment but even the experts are unable to keep up with the rapidly evolving literature. The promise of combination therapies has been tempting but there is no known CER that included such protocols.

Potential Impact

How will an answer to your research question be used or help inform decisions for you or your group?

The ACCP intends to develop evidence-based clinical practice guidelines on this topic which will be submitted to the journal CHEST for consideration for publication. Additional dissemination efforts will facilitate implementation of these recommendations in various media and venues.

Describe the timeframe in which an answer to your question is needed.

ACCP can be more flexible with this topic than with certain others, but we hope to have the final evidence tables or profiles by November 1, 2012.

Describe any health disparities, inequities, or impact on vulnerable populations your question applies to.

PAH patients who are not able to obtain care from experienced PAH clinicians, (whether due to geographic location, mobility restrictions, costs, or access) may not receive the latest evidence-based treatments resulting in disparities and inequities.

Nominator Information

Other Information About You: (optional)

Please choose a description that best describes your role or perspective: (you may select more than one category if appropriate)

The ACCP intends to develop evidence-based clinical practice guidelines on this topic which will be submitted to the journal CHEST for consideration for publication. Additional dissemination efforts will facilitate implementation of these recommendations in various media and venues.

Are you making a suggestion as an individual or on behalf of an organization?

Organization

Please tell us how you heard about the Effective Health Care Program

ACCP has partnered with the AHRQ Effective Health Care Program many times previously. This particular topic was discussed with the Duke Evidence-Based Practice Center, who has been selected by AHRQ to complete pulmonary and cardiovascular CERs.