Single Patient-Reported Outcome System for Adults with Congenital Heart Disease

Patient-reported data revealed 6 symptoms that are important to adult patients across the spectrum of congenital heart disease.

A single disease-specific patient-reported outcome may be useful for quality and outcome assessments, according to results from a patient survey on symptoms associated with congenital heart disease activity.

Adults with congential heart disease (ACHD) is a patient population that has grown significantly in the last 2 decades, according to the researchers, and yet little data on disease activity exists. Therefore, they sought to examine whether patient-reported symptoms could be utilized to create a patient-reported outcome measure. Their results, published in Circulation: Cardiovascular Quality and Outcomes, identified 6 symptoms relevant to patients across the congenital heart disease spectrum.

“We identified a group of symptoms of particular importance to patients with ACHD and examined how the frequency and importance of these symptoms varied across the spectrum of ACHD,” the authors wrote. “Unexpectedly, the symptoms of greatest significance to ACHD patients are largely similar and independent of underlying cardiac diagnosis.”

Researchers created a 39-item survey using data from physicians specializing in ACHD to assess symptoms that patients associate with disease activity. A total of 124 patients (mean age: 42.7 ± 14.2; 67% female) completed the survey, and an additional 40 patients took the survey as part of a confirmatory cohort to confirm the validity of the initial data.

Researchers analyzed the survey results based on underlying anatomy and disease complexity. When data was grouped by anatomy, the researchers observed significant differences in disease-related symptom rankings in only 6 out of 39 symptoms. They determined 6 symptoms that were of particular significance to patients regardless of underlying anatomy, including trouble sleeping, fatigue, low energy levels, anxiety or stress due to health concerns, depression or feelings of hopelessness, and concern over the cost of medical care (eg, medications and tests).

Patients with anatomy of greater complexity experienced overall greater symptom severity than those with low or moderate complexity. This resulted in a higher ranking of 5 symptoms related to self-image, physical capacity, perceived health status, or overall psychological state. 

The confirmatory cohort had symptom experiences similar to the original cohort, with differences observed in only 5 of the 39 symptoms. For example, researchers observed that while sleepiness and headaches were identified as “important symptoms” in the initial cohort, the confirmatory cohort found them to be of “greater importance.” Interestingly, “defibrillator or implantable cardiac defibrillator firing” was identified as “below average importance” in both groups.

“Based on these findings, we think that a single PRO [patient-reported outcome] can be developed for patients with ACHD,” the authors concluded. “In future work, we will employ focus groups and cognitive debriefing exercises with the goal of reducing the number of items and developing a PRO with good content validity.”


Cedars AM, Stefanescu A, Broberg C, et al. Adult Congenital Heart Disease Patients Experience Similar Symptoms of Disease Activity. Circ Cardiovasc Qual Outcomes. 2016. doi: 10.1161/circoutcomes.115.002154.