Pnd110 Symptoms, Delayed Diagnosis and Quality of Life (Qol) Deterioration - Exploring the Burden of Amyotrophic Lateral Sclerosis (Als)

Abstract

Objectives: To understand the economic and humanistic burden attributed to myasthenia gravis (MG). Methods: We conducted a literature search (January 2009 to April 2019) in the biomedical database Embase (including Medline), focusing on English language observational studies and economic models evaluating patients with MG in the United States. Key search terms focused on costs, healthcare resource utilization, and quality of life (QoL). Results: A total of 18 studies were identified that included economic or humanistic burden data. Median length of hospitalization in patients with MG ranged from 2 days to 8 days, with costs per admission, using National Inpatient Sample data, ranging from a median of $16,000 in 1 study to a mean of nearly $99,000 in another analysis. Patient factors including MG crisis, presence of respiratory failure, and complex medication regimens (including multiple immunosuppressive and conventional therapies) worsened the burden, increasing length of stay from a median of 4 -6 days to 6 -10 days. Costs of admission increased from $16,000 to $26,600 for patients hospitalized for MG (not MG crisis), up to almost $54,000 for patients experiencing an MG crisis and receiving plasma exchange. Among patients treated with multiple immunosuppressants, presence of continued functional disability contributed to a worse QoL. One study found that QoL was worse in women than men, but the difference was eliminated for women who had undergone a thymectomy. Conclusions: The economic and humanistic burden associated with MG is considerable, particularly for patients with severe complications, including MG crisis. Understanding the risk factors associated with MG-related complications, and the potential impact on treatment modalities, is crucial to improve the medical management of these patients. Additionally, there is a clear unmet need in this patient population for new treatment options to improve disease course and prevent complications.

Health-related quality of life across disease stages in patients with amyotrophic lateral sclerosis: results from a real-world survey

“…In the recent systematic review of QoL deterioration in ALS by Forsythe and colleagues [ 11 ], a severe decline in health status and HRQoL was found to correlate with increased disease severity, with EQ-5D utility values ranging from 0.79 to 0.65 at stage 1, 0.67–0.53 at stage 2, 0.71–0.35 at stage 3, and 0.50–0.12 at stage 4 on the King’s staging system [ 30 , 45 – 47 ]. The results of the present investigation, with adjusted mean EQ-5D-5L utility scores ranging from 0.65 at King’s stage 1 to 0.11 at stage 4, are therefore in line with previous findings of QoL changes in pALS and are well below general population norms in the UK and other countries (0.83–0.92) [ 48 ].…”

“…Physical impairment and progression rate of physical deterioration in ALS have a significant impact on emotional well-being and there is a correlation between disease severity and/or decreasing physical function and declining health-related quality of life (HRQoL) [ 9 , 10 ]. A recent systematic literature review investigating the burden of ALS confirmed that pALS experienced poor QoL and loss of function, which deteriorated with disease progression [ 11 ]. The burden of symptoms was high, with patients experiencing various debilitating symptoms, with fatigue, depression, and pain being the most frequently reported [ 11 ].…”

“…A recent systematic literature review investigating the burden of ALS confirmed that pALS experienced poor QoL and loss of function, which deteriorated with disease progression [ 11 ]. The burden of symptoms was high, with patients experiencing various debilitating symptoms, with fatigue, depression, and pain being the most frequently reported [ 11 ]. On the other hand, there are reports in the literature of relatively well-maintained QoL despite physical function decline [ 12 – 14 ].…”

“…Depending on the severity of the disease, care partners of pALS (cALS) who are not providing care under a professional contract may spend most of the day providing care, resulting in substantial humanistic and economic burden, with associated depression and reduced QoL [ 11 , 16 ]. A cross-sectional survey in Germany revealed that costs of informal care (i.e., care provided by non-professional care partners, mainly family members) represented nearly half of all costs of illness in ALS [ 17 ].…”