ABSTRACT
INTRODUCTION: This study assessed the clinical burden of refractory myasthenia gravis (MG), relative to nonrefractory MG. METHODS: Rates of myasthenic crises, exacerbations, inpatient hospitalizations, and emergency room (ER) visits over a 1-year period were measured for 403 refractory, 3,811 nonrefractory, and 403 non-MG control patients from two administrative health plan databases. RESULTS: Compared with nonrefractory patients, a significantly greater percentage of refractory patients had at least one myasthenic crisis (21.3% vs. 6.1%; P < 0.001) and at least one exacerbation (71.2% vs. 32.4%; P < 0.001) over a 1-year period. Refractory patients were also significantly more likely to be hospitalized and/or have an ER visit than nonrefractory patients and non-MG controls (P < 0.001 for all). DISCUSSION: Refractory MG patients have significantly greater clinical burden and are more likely to utilize intensive healthcare resources than nonrefractory patients. Furthermore, refractory patients may be at greater risk of crises throughout the disease course than previous studies have suggested. Muscle Nerve, 2018.
ABSTRACT
BACKGROUND: Asthma is a common disorder that affects approximately 8% of the U.S. POPULATION: Treatment guidelines indicate inhaled corticosteroids (ICS) as the mainstay treatment, yet poor asthma control is common among ICS-treated patients. Treatment escalation (ICS dose increase and other controller therapy add-ons) is used to manage symptoms. Real-world studies of postescalation outcomes may inform treatment decisions. OBJECTIVES: To (a) describe characteristics and treatment patterns among asthma patients who escalated treatment and (b) assess outcomes (exacerbations, uncontrolled asthma, and health care resource utilization [HCRU]) after escalation. METHODS: The study cohort was identified from a large U.S. administrative claims database via ICD-9-CM codes for asthma (493.xx on ≥ 2 dates) and initiation (defining index date) of long-term (> 1 fill) ICS-containing treatment between January 1, 2009, and September 30, 2014. One year of continuous enrollment was required before and after the index date. Escalation was defined as ≥ 1 of the following: ICS dose increase; a switch between ICS, long-acting beta-2 agonists (LABA), or leukotriene modifiers (LTRM) to a different ICS, LABA, or LTRM; or add-on of controller medications (e.g., antibody biologic). Escalation patterns were examined. Rates of exacerbation (defined by inpatient admission, emergency department [ED] visit, or office visit with a pharmacy claim for an oral corticosteroid [OCS] within 7 days) and occurrence of uncontrolled asthma (defined by > 4 fills for a short-acting beta agonist [SABA] in a 1-year period, ≥ 1 OCS fill, or ≥ 1 asthma-related ED visit or inpatient admission) were calculated. Per-patient-per-year (PPPY) HCRU was estimated. RESULTS: Among 35,126 patients (mean [SD] age 38 [16] years) who initiated long-term ICS-containing treatment, 5,044 (14%) patients escalated their index regimens at 136 (105) days post-index (i.e., pre-escalation period). The most frequent changes, alone or in combination, included ICS dose increase (68%) or LABA (27%) or LTRM (25%) add-ons. Before escalation, the exacerbation rate was 1.60 (5.10) PPPY, and 1,108 (22%) patients experienced exacerbation. During the postescalation period of 251.6 (138.9) days, the exacerbation rate was 0.75 (2.9) PPPY, and 1,038 (21%) patients experienced exacerbation. A majority (> 85%) of exacerbations in the periods before and after escalation were associated with an office visit plus an OCS pharmacy claim within 7 days. Uncontrolled asthma was experienced by 41.5% and 41.0% of patients before and after escalation, respectively. Ambulatory care visits were common before (mean [SD] 24.0 [26.7] all-cause and 8.5 [13.4] asthma-related PPPY) and after escalation (19.3 [21.3] all-cause and 4.6 [8.1] asthma-related PPPY). CONCLUSIONS: Among asthma patients who initiated a long-term ICS-containing regimen, approximately 14% escalated therapy within a year of initiation. Yet, 21% of those patients had ≥ 1 exacerbation, and 41% of patients had uncontrolled asthma within 1 year after treatment escalation. The results demonstrate an unmet need among asthma patients who escalated their ICS-containing treatment. DISCLOSURES: This study was sponsored and funded by Boehringer-Ingelheim, which contracted with Optum to conduct the research. The sponsor collaborated with Optum on the preparation, writing, revision, and approval of the manuscript. Bengston, Cao, Hulbert, Wolbeck, Elliott, and Buikema are employees of Optum. Yu and Wang are employed by Boehringer-Ingelheim. Study concept and design were contributed by Bengston, Yu, and Wang. Cao, Hulbert, and Wolbeck collected the data, and data analysis was performed by Bengston, Yu, and Wang. The manuscript was written by Bengston, along with Yu and Wang, and revised by Bengston, Yu, and Wang, along with the other authors.