Health-related quality of life impairment among patients with severe chronic rhinosinusitis with nasal polyps in the SINUS-24 trial

Purpose: Chronic rhinosinusitis with nasal polyps (CRSwNP) is a predominantly type 2 inflammatory disease with a high symptom burden. Data are lacking on the comparative health status of patients with CRSwNP. This analysis compared baseline physical and mental health-related quality of life (HRQoL) and overall health status of patients with severe CRSwNP enrolled in a Phase 3 clinical trial with general population norms and with other chronic diseases. Methods: In this post hoc cross-sectional analysis of baseline data from the SINUS-24 study (NCT02912468), HRQoL was measured using the 36-item Short Form (SF-36) questionnaire and general health status was measured using the EuroQol-5 Dimension visual analog scale (EQ-VAS). Analyses included the intention-to-treat (ITT) population and subgroups defined by prior sinonasal surgery, systemic corticosteroid use, and coexisting asthma or non-steroidal anti-inflammatory drug-exacerbated respiratory disease (NSAID-ERD). Scores were compared with published values for population norms (50 for SF-36 physical component summary (PCS) and mental component summary (MCS), 70.4−83.3 for EQ-VAS) and for rheumatoid arthritis, type 2 diabetes, and asthma. Results: In the ITT population (n=276), mean SF-36 physical component summary (PCS), SF-36 mental component summary (MCS), and EQ-VAS scores were below general population norms (46.4, 48.6, and 66.0, respectively). Mean SF-36 PCS and EQ-VAS scores were below population norms across all subgroups; mean SF-36 MCS scores were below the population norm in all subgroups except no prior surgery. SF-36 PCS and MCS scores from SINUS-24 were generally similar to other chronic diseases, except SF-36 PCS which was lower in rheumatoid arthritis. EQ-VAS scores in SINUS-24 were lower than in other chronic diseases. HRQoL scores weakly correlated with objective measures of disease severity. Conclusion: In patients with severe CRSwNP, including those with coexisting asthma/NSAID-ERD, HRQoL was worse than population norms and as burdensome as diseases such as type 2 diabetes, asthma, and rheumatoid arthritis

Impact of Natalizumab on Patient-Reported Outcomes in a Clinical Practice Setting: A Cross-Sectional Survey

Objective: To assess multiple sclerosis (MS) patients' experience with natalizumab (TYSABRI, Biogen Idec, Inc. and Elan Pharmaceuticals, Inc.) in a clinical practice setting. Abstract: Methods: MS patients who were enrolled in the TOUCH (TYSABRI Outreach Unified Commitment to Health) prescribing program and who had received their third natalizumab infusion participated in this study. Patient-reported measures included an overall quality-of-life (QOL) assessment, an adapted version of the Multiple Sclerosis Impact Scale-29 (MSIS-29), and pre-/post-disease level and functional status scores. MSIS-29 responses were modified to measure patient-perceived change since initiating natalizumab. Paired t-tests assessed pre-/post- changes in disease level and functional status, where negative change indicated improvement. Abstract: Results: Results from 451 patients in this study indicated that 73% were female and, on average, were diagnosed with MS >11 years previously. Almost all (96%) patients had used one or more MS drugs prior to natalizumab initiation. After receiving natalizumab, 97% of all patients reported an improvement or remained stable in their overall QOL. Despite the short treatment duration, there were significant improvements (mean ± SD change) in disease level (-0.26 ± 0.99, paired t-test - 5.47; p < 0.001) and functional status (-0.33 ± 0.73, paired t-test - 9.40; p < 0.001) scores. More than 80% of patients reported an improvement in one or more MSIS-29 physical items. The physical item on the adapted MSIS-29 with the highest reported improvement (58%) was 'the ability to do physically demanding tasks'. The physical item with the lowest reported improvement (32%) was 'problems using transport'. Abstract: Conclusion: Overall, the experiences of MS patients with natalizumab were positive in a clinical practice setting. Patients reported improvements in overall QOL, ambulation and functional status as early as after three natalizumab infusions. While preliminary, these early results are suggestive of a beneficial effect of natalizumab in patients with MS and warrant further long-term investigation of the impact of this treatment on patient outcomes.

Pharmacoeconomic comparison of aripiprazole once-monthly and paliperidone palmitate from a head-to-head clinical trial in schizophrenia: a US analysis

Schizophrenia presents a substantial clinical and economic burden to the health-care system. In QUAlity of LIfe with AbiliFY Maintena (QUALIFY), a randomized head-to-head study of aripiprazole once-monthly 400 mg (AOM 400) compared with paliperidone palmitate (PP; 78–234 mg/mo), AOM 400 demonstrated greater improvement in health-related quality of life and functioning in patients with stable schizophrenia. The present analysis used health economics assessment data collected during the QUALIFY study to determine the direct medical and pharmacy costs and the cost-effectiveness associated with each treatment over 6 months. Compared with those receiving PP, patients receiving AOM 400 incurred significantly lower direct total costs ($8908±186 vs$9675±190, p=0.005) and treatment costs ($7967±113 vs$8706±116, p<0.001). Effectiveness results in the subset of patients included in the cost analyses were similar to the overall population: mean (95% CI) improvement in Heinrichs-Carpenter Quality of Life Scale total score was greater with AOM 400 (5.97 [3.87; 8.08]) compared with PP (2.85 [0.56; 5.08]). Likewise, Clinical Global Impression–Severity improved more in the AOM 400 group (–0.59 [–0.71; –0.47]) compared with PP group (–0.37 [–0.46; –0.27]). Therefore, the analysis of data from stabilized patients with schizophrenia in the QUALIFY study indicated that AOM 400 is associated with lower health-care costs and greater effectiveness compared with PP and thus represents the economically dominant strategy

Reduction in inpatient resource utilization and costs associated with long-acting injectable antipsychotics across different age groups of Medicaid-insured schizophrenia patients

Objective: Evaluate utilization of inpatient healthcare resources and associated costs after 12 months of treatment using long-acting injectable (LAI) antipsychotic medications among a large sample of Medicaid-insured patients categorized by different age groups. Method: Adult patients with schizophrenia were identified from the Thomson Reuters MarketScan Research database (1/1/2006–12/31/2010) before initiation of treatment using LAI antipsychotic agents. Utilization of inpatient healthcare resources and associated direct medical costs were compared for 12-month baseline and 12-month follow-up periods. Results: Among 3,094 Medicaid-insured patients with schizophrenia initiating treatment with LAIs, the mean number of all-cause hospitalizations and hospitalization days were reduced by 24% and 31% (p<0.0001) compared with baseline, respectively, with similar significant reductions among all age groups (18–30, 31–40, 41–50, and 51–60 years). During 12-month follow-up with LAIs, mean reductions in all-cause costs were $4,369 (18–30 years, p<0.0001),$3,681 (31–40 years, p<0.0001), $2,051 (41–50 years, p=0.1332), and$4,492 (51–60 years, p=0.0107). Subanalyses separating first-generation and second-generation medication groups resulted in mean reduction in all-cause costs of $3,561 and$3,645, respectively. Conclusions: Results from this large cohort study provide naturalistic real-world evidence of the utility of LAIs in patients with schizophrenia and suggest that these agents may help to reduce the risk of relapse across all age groups (especially among younger patients). Given that relapse prevention is the ultimate goal of antipsychotic treatment, results from this large Medicaid patient population establish the value of LAIs for the management of schizophrenia

Inpatient resource use and costs associated with switching from oral antipsychotics to aripiprazole once-monthly for the treatment of schizophrenia

Background: Schizophrenia is associated with high direct healthcare costs due to progression of disease and frequent occurrence of relapses. Aripiprazole once-monthly (AOM) has been shown to reduce total psychiatric hospitalizations among patients who switched from oral standard of care (SOC) therapy to AOM in a multicenter, open-label, mirror-image study of patients with schizophrenia. Because of the increasing need to improve patient outcomes while containing costs, it is important to understand the impact of AOM treatment initiation on medical costs associated with psychiatric hospitalizations and antipsychotic pharmacy costs. Methods: In the current study, an economic model was developed using data from the AOM mirror-image study to evaluate the psychiatric hospitalization-related medical costs and antipsychotic pharmacy costs during a 6-month period before (retrospective period) and after (prospective period) the AOM treatment initiation. The economic model evaluated cost-saving potential of AOM among all patients (n=433) as well as a subset of patients with ≥1 prior hospitalization (n=165) who switched from oral SOC to AOM. Unit cost data were obtained from publicly available sources. Results: Both hospitalizations and hospital days were reduced following a switch from oral SOC to AOM. As a result, psychiatric hospitalization-related costs were lower during the prospective period when compared with the retrospective period. Furthermore, the increase in antipsychotic pharmacy costs due to switching from oral SOC to AOM was offset by a reduction in psychiatric hospitalization-related medical costs. Per-patient costs were reduced by $1,046 (USD) in the overall population and by$20,353 in a subset of patients who had at least 1 psychiatric hospitalization during the retrospective period. Results were most sensitive to changes in hospitalization costs. Conclusions: AOM is associated with reducing the risk of relapse among patients with schizophrenia. The increase in antipsychotic pharmacy costs due to switching from oral SOC to AOM was offset by a reduction in costs associated with psychiatric hospitalizations, thereby presenting a cost-saving opportunity for health plans

Impact of natalizumab on patient-reported outcomes in multiple sclerosis: a longitudinal study

Abstract Background Natalizumab (Tysabri, Biogen Idec and Elan Pharmaceuticals) significantly reduces the relapse rate and disability progression, and improves health-related quality of life (HRQoL), in patients with relapsing-remitting multiple sclerosis. We investigated the impact of natalizumab on patient-reported outcomes (PROs) in a real-world setting. Methods PRO data were collected from patients enrolled in a longitudinal real-world study using validated measures administered as surveys before the patients initiated natalizumab treatment and after the 3rd, 6th, and 12th monthly infusion. HRQoL, ability to carry out daily activities, disability level, and impact on cognitive functioning and fatigue were assessed. Results A total of 333 patients completed 12 months of assessments. After 12 months of natalizumab treatment, 69% to 88% of patients reported a positive outcome (either an improvement or no further decline) in all PRO measures assessed. Significant improvements in general and disease-specific HRQoL were observed after three infusions, both with physical (p  Conclusions PRO measures were improved with natalizumab in a real-world setting. The improvements were observed as early as after 3 months and sustained over a 12-month period. The improvements in PROs show that, in clinical practice, the clinical benefits of natalizumab are translated into patient-reported benefits.</p

Dupilumab provides early and durable improvement of symptoms in patients with chronic rhinosinusitis with nasal polyps

Abstract Objectives To evaluate within‐patient symptom improvement in the dupilumab SINUS‐24/‐52 studies in patients with severe chronic rhinosinusitis with nasal polyps (CRSwNP) (NCT02912468/NCT02898454). Methods Patients received dupilumab 300 mg or placebo every 2 weeks for 24 (SINUS‐24) or 52 weeks (SINUS‐52) on background intranasal corticosteroids. Patients daily reported symptoms of nasal congestion (NC), loss of smell (LoS) and rhinorrhoea on a scale of 0–3 (0 – no symptoms, 1 – mild, 2 – moderate, 3 – severe symptoms). The proportions of patients with moderate‐to‐severe symptoms (score ≥ 2) at baseline who improved to no‐to‐mild symptoms (score ≤ 1) were determined at Weeks 2, 24 (pooled studies) and 52 (SINUS‐52). Subgroups with prior sinonasal surgery and coexisting asthma were analysed. Results At baseline in the pooled intention‐to‐treat population (n = 724), the proportions of patients with scores ≥ 2 for NC, LoS and rhinorrhoea were 87, 94 and 64%, respectively. Significantly, more patients achieved scores ≤ 1 (no/mild symptoms) with dupilumab vs placebo for each symptom at each time point {Week 2 NC 12% vs 2% [odds ratio 8.9 (95% CI 3.0–26.3)], LoS 5% vs 1% [4.6 (1.3–16.8)], rhinorrhoea 9% vs 2% [4.8 (1.5–15.4)], all P < 0.05; Week 24 NC 54% vs 14% [8.7 (5.6–13.5)], LoS 43% vs 6% [14.4 (7.9–26.0)], rhinorrhoea 53% vs 16% [6.6 (4.1–10.9)], all P < 0.0001}. Results were similar in subgroups with prior surgery and coexisting asthma. Conclusion Significantly, more patients achieved improvement from moderate‐to‐severe symptoms to no‐to‐mild symptoms with dupilumab than placebo, regardless of prior surgery or coexisting asthma. Improvement was observed as early as Week 2 and continued through to Week 52