Key points

Step therapy for treatment of asthma has proven a successful means of guiding practitioners through marked changes in management strategy and of gaining better control over this difficult disease process.

In the middle to late twentieth century, asthma therapy was not providing satisfactory control of the disease. Physician visits, self-reported reactive airway disease prevalence, and deaths due to asthma increased between 1960 and 1990. Even more troubling was that a percentage of the deaths were attributed to the bronchodilator therapy, specifically exacerbations of disease related to long-acting beta2-agonist (LABA) use. Even without these concerns, bronchodilator therapy did not seem to be the definitive therapy to manage asthma.

Scientific work in the 1960s included the discovery of IgE and a better delineation of the role of inflammation in reversible airway disease. In combination with recently developed inhaled corticosteroids, this provided a better understanding of the disease as well as a pathway that could make a substantial positive impact on the quality of life for a patient with asthma.

Several challenges surrounded the acceptance of this radical algorithm change. The first was the need to educate and change practice patterns of the wide range of practitioners treating asthma, which required informing them and gaining their acceptance of the strategy. Unlike a new medication or adjunctive therapy, the concept of inflammation-based therapy for asthma represented a marked philosophic change. Because physicians tend to gather their continuing education from the medical journals and societies related to their specific field, there was a need to reach out to several disparate societies and journals to get information to the practitioners regarding changes in the therapy of asthma.

Asthma therapy is also complicated by the variability of the disease from life-threatening exacerbations to a completely normal quality of life between episodes. This disease process does not lend itself to simply starting a medication and continuing it routinely for years, as in the case of hypertension or types of cardiac disease. There is a legitimate concern among physicians and patients about overmedication or undermedication of asthma. Without guidelines, it was unclear when it would be most appropriate to start medications. Unfortunately, with the medications involved, there were side effect profiles that made overuse undesirable. Steroid therapy, which represented a critical new role in asthma care, is known to have side effects that include growth retardation, decreased immunocompetence, and multiple system-based complications. All branches of medicine recognize a strong desire to limit steroid use, which complicates acceptance of the new regimen. In asthma, these limitations must be balanced against the risk of progression of disease and the inability to stabilize the airway by other means.

Beta agonists have a somewhat diminished but still vital role in the more modern therapy approach. These agents also suffer from reported complications, including electrolyte disorders, cardiac dysrhythmias, and sudden death.

This change in asthma management occurred at a time when clinical practice guidelines were less well understood and certainly less trusted. Current data on the value of evidence-based guidelines were obviously not available and the physicians of the time were concerned that guidelines were intended to restrict testing and control medical practices for the benefit of third-party payers.

It was in this medical environment that the National Asthma Education and Prevention Program (NAEPP), under the auspices of the National Heart, Lung, and Blood Institute, performed a thorough literature review and created a guideline pathway for asthma that was published in 1991. The guidelines have been revised in 1997, 2002, and most recently in 2007. The guideline divided asthma into categories of intermittent or persistent, with the latter category being subdivided into mild, moderate, or severe. This categorization then provided the clinician with a step therapy based on the severity of the asthma. These pathways have largely succeeded in changing the way asthma is managed, starting in the last decade of the twentieth century. The intervening time has allowed statistical analysis to identify actual improvement in asthma management as measured by fewer emergency visits and deaths.

From 1991 to 2006, progress in the field, new medications, and updated data resulted in the need for changes to the asthma management algorithm. The stepwise approach has grown necessarily more complicated, but the overall principles remain. The guideline continues to provide some rigidity but allows physician judgment and insight. In the current version, the guidelines include patient education and some patient responsibilities. Although some of the elegant simplicity of the 1991 algorithm is lost, the present update allows better active consideration of allergic components of the disease. In addition, there are more options for effectively allowing medication use to step up when needed and step down when possible.

The now-familiar four categories of asthma remain unchanged. The step approach for asthma therapy has increased these to six steps. The care recommendations are stratified for patient ages of 0 to 4 years, 5 to 11 years, and 12-plus years to allow for better treatment of pediatric medication variances.

The initial step, as before, is the diagnosis of asthma. The goal is to identify asthma, evaluate and exclude other diagnoses, and determine the severity of the disease. Patient history, spirometry (in patients older than 5 years), and the exclusion of other diagnoses is important in arriving at the correct diagnosis. The severity of the disease is important in determining how to initiate therapy. The control aspect of disease is vital in monitoring and controlling therapy. Severity is defined as the intrinsic intensity of the disease process. This parameter is important for the initiation of therapy ( Table 1 ).

The initial assessment is the first and arguably the most critical step, because it has a role in both diagnosis and the direction of initial therapy. The steps of the initial assessment are illustrated in Table 1 . Note that the diagnosis includes a history of symptoms and types of precipitating factors, not only spirometry. Spirometry is nevertheless required in patients 5 years and older. These historical data combined with the results of spirometry assist the clinician in making the diagnosis of asthma and provides for patient stratification into a defined level of asthma severity.

Because this clinical information is vital to the correct categorization of the patient’s level of asthma, the clinician should be familiar with Table 1 when taking the asthma history. In addition to categorization into intermittent versus persistent, and as mild, moderate, or severe, the patients are also grouped according to age. Based on the level of findings, the clinician is directed to a subsequent algorithm that indicates the recommended medical therapeutic selection ( Table 2 ).

Critical changes in the third revision

The updated guidelines reflect a identified need to monitor the control of asthma. Whereas the initial evaluation of the patient with asthma is to determine at which step to initiate therapy, the reassessments are geared to determine the control of therapy and the need to adjust medications. The evaluation with ongoing therapy is to assess whether the patient’s quality of life has improved by the control of symptoms as outlined in the written physician-patient plan. The evaluation should allow a determination of whether medication use should increase (step up) or decrease (step down). This permits the best quality of life with minimal medication use.

In addition to the medication therapy, the guidelines make education a central component to therapy. This includes assuring that the patient understands self-monitoring of asthma control and can identify signs of acute exacerbation or generalized worsening of disease. Education also encompasses the correct way of physically taking the medications and the decision process for the use of rescue medications. Finally, education extends to the lifestyle changes vital to minimizing asthmatic episodes.

Emphasis is placed on a written asthma plan, which is to be created individually for the patient and reviewed regularly between the patient and physician.

Allergic disease also receives recognition and therapeutic recommendations in the 2007 version of the guidelines. The second expert panel acknowledged the inflammatory component of asthma and the genetic disposition to atopic disease as a major factor. However, the most recent guideline takes the singular step of advocating allergy immunotherapy in cases where a clear connection exists “between symptoms and exposure to an allergen to which the patient is sensitive.” This is an important comment for several reasons. Patients with asthma have a higher risk of adverse response to immunotherapy. There have been questions in the literature about whether allergy immunotherapy is sufficiently helpful in the control of asthma to make it worth the risks of adverse reaction. The inclusion of immunotherapy by the expert panel seems to address these concerns, approving the use of immunotherapy in mild to moderate persistent disease, at least in cases in which a direct link to asthma can be appreciated.

Recommendations that are more specific are possible when patients are grouped into 0 to 4 year olds and 5 to 11 year olds. The latter group was represented as a portion of the adult recommendations in earlier versions of the expert panel recommendations.