Hereditary angioedema burdens include quality of life, health care costs
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Key takeaways:
- Approximately 35% of respondents had poorly controlled disease.
- Quality of life drops substantially during attacks.
- Costs total €22,764 per year, primarily due to medications.
Patients with hereditary angioedema experience significant burdens in disease control, quality of life, treatment satisfaction and societal costs, reported a study published in The Journal of Allergy and Clinical Immunology: In Practice.
Additional prophylactic strategies may mitigate these burdens, Lauré M. Fijen, MD, PhD, internal medicine resident, department of vascular medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, and colleagues wrote.
The researchers surveyed 69 adults (mean age, 45.8 years; 60.9% women) treated for hereditary angioedema (HAE) at Amsterdam UMC, which is the national reference center for these patients, in 2021.
Primary locations of angioedema included the abdomen for 28 patients (40.6%) and the extremities for 18 patients (26.1%). Also, 59% were using prophylactic treatment, 28% only used on-demand treatment for attacks, and 13% were not on any treatment.
The 59 patients with an Angioedema Activity Score (AAS28) had an average of 16.61 (standard deviation [SD], 29.55) and a median of 0, indicating that most of them did not have an attack during the study period.
The mean total score for the angioedema-specific health-related quality of life questionnaire (AE-QoL) was 30.99 (SD, 20.18), with fatigue and mood problems representing the greatest struggles (37.54; SD, 22.37) and the lowest scores appearing in the nutrition domain (20.29; SD, 28.04).
With a mean Angioedema Control Test (AECT) score of 10.83, the researchers said, 64.71% of the patients had well-controlled disease.
Also, treatment global satisfaction had a mean of 69.4 (SD, 21.88), with limited variation between domains. Patients who experienced an attack during the follow-up period and those who did not experience an attack had similar treatment satisfaction scores.
AAS28 scores included 3.08 (SD, 7.48) for the 37 patients with well controlled HAE and 39.36 (SD, 37.18) for the 22 patients with poorly controlled HAE.
Based on responses to a generic health-related quality of life instrument (EQ-5D-5L) with five dimensions, pain and discomfort presented the most problems, and self-care presented the fewest problems.
Average EQ-5D-5L scores included 0.873 on days without an attack, which the researchers called almost identical to the average utility of the Dutch general population (0.869), and 0.42 on days with an attack for the 22 patients who experienced an attack during the study period.
Costs totaled 1,897 Euros per patient per month or €22,764 per patient per year, although costs varied with 16% of patients reporting no costs at all and other patients reporting costs of up to €20,000 per month. Medical costs accounted for 74% of total costs, with an average of €1,406 per patient per month and a range of €0 to €18,069.
With 68% of these costs related to HAE medicines, plasma-derived C1-inhibitor concentrates (n = 10) represented the largest share of these medications. Non-HAE medicines accounted for an average of €170 in costs, and other health care costs tallied €284.
Averaging €133 per patient, hospital admissions absorbed the largest share of other health care costs, with 27 patients (39%) admitted to the hospital and four patients who required emergency care at least once.
With approximately €468 per 4 weeks in total productivity costs per patient, short-term absenteeism cost €113, long-term absenteeism cost €191, presenteeism cost €55 and unpaid work cost €109. Also, productivity costs included €344 for patients with well controlled HAE and €686 for patients with poorly controlled HAE.
Informal care such as domestic help, help with self-care and practical help like transportation cost an average of 24 per patient per month, with a range of 10 to 1,213 for the seven patients who required informal care. Average time demands included 1.88 hours for domestic help, 0.53 hours for help with self-care and 1.17 hours of practical help.
Costs for patients with well-controlled HAE compared with those for patients with poorly control included €566 vs. €2,883 for total medical costs, €122 vs. €2,412 for HAE medication and 1 vs. 64 for informal care.
Based on these findings, the researchers concluded that a subset of patients with HAE do not experience sufficient improvements in disease control with their current prophylactic strategies, so additional strategies may be needed to improve disease outcomes as well as quality of life in addition to mitigating the financial impact of HAE.
Perspective
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These findings are significant. They underscore the complexity of HAE management as well as the patients’ immense need.
I think there is likely a greater percentage of patients on prophylaxis in the United States, especially in centers of excellence that treat HAE on a regular basis. From most that I know who treat this, we have closer to 75% to 80% on prophylaxis and 20% to 25% on acute, on-demand therapy only. All patients, no matter what, should have acute, on-demand therapy. This study quotes 59% on prophylaxis and 13% without any treatment at all. This is unacceptable now.
In my practice, we have far fewer hospitalizations, fewer missed days off work and overall better control. Our biggest issue is insurance barriers. Insurance continues to make access of care and medicine very difficult for patients and extremely time-consuming and costly to treating physicians. These barriers are a cause of HAE exacerbations and worsening control with increased burden to the patient.
This is such a significant problem, I have coined a new subset category in diagnostics for this patient population: HAE exacerbations due to insurance barriers. This may be different in the United States as opposed to the Netherlands since there are differences in the delivery of health care, but I believe access to appropriate prophylactic and acute emergency medications is consistent.
This is pertinent. As the study stated, “These results suggest that current prophylactic strategies provide insufficient improvements on disease control in a considerable subset of patients with HAE. For this reason, additional prophylactic strategies appear to be needed to improve disease control in a large subset of patients with HAE.”
More patients need to be on better prophylactic regimens. The reasons they are not on these regimens are multi-factorial, but insurance is the top reason, in my opinion. While insurance companies are putting up barriers to medication access to save their margins, they are costing patients and the system elsewhere and worsening their burden and quality of life.
We must keep fighting for our patients. Ultimately, there must be better checks and balances on insurance and government programs and their medical decision making with patients. They are deciding outcomes without a medical license, and there is currently little repercussion to them. The medical decision-making needs to be in a shared decision-making process between those interested in actual medical care — the doctor and patient — with minimal interference from those who are motivated to deny care — insurance.
An in-depth and detailed analysis of all private and government insurance companies and their denial and prior authorization processes needs to occur. There must be greater transparency in their policies, standard operating procedures, and communications with doctors and patients. The onus of burden should be placed on the insurance company to prove a patient does not need treatment with the company explaining its case in detail to the doctor and patient instead of the other way around, where the burden of proof lies solely on the doctor and patient as to why they need it.
Insurance companies must be scrutinized, with evidence supporting their systems. I feel they contribute greatly to the disease state burden and lack of quality of life in HAE patients. The stress caused by worrying about access to lifesaving medication creates more attacks, loss of work and school productivity, and depression/anxiety by itself, and I believe it is an independent risk factor in this group.
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