Background: GSP301 nasal spray is a fixed-dose combination of the antihistamine olopatadine hydrochloride and the corticosteroid mometasone furoate. Objective: To evaluate the efficacy, safety, and tolerability of GSP301 in pediatric patients (aged >= 6 to <12 years) with seasonal allergic rhinitis (SAR). Methods: This double-blind, randomized, parallel-group study randomized 446 eligible patients 1:1 (GSP301 [olopatadine hydrochloride 665 mg and mometasone furoate 25 mg] or placebo) as 1 spray/each nostril twice daily for 14 days. The primary end point was change from baseline in average morning and evening subject-reported 12-hour reflective Total Nasal Symptom Score (rTNSS) over a 14-day treatment period analyzed using mixed-effect model repeated measures. Additional assessments included instantaneous Total Nasal Symptom Score, Pediatric Rhinoconjunctivitis Quality of Life Questionnaire, reflective Total Ocular Symptoms Score, instantaneous Total Ocular Symptoms Score, individual symptoms, Physician-assessed Nasal Symptom Score, and adverse events. Results: GSP301 showed clinically meaningful and statistically significant improvement in rTNSS vs placebo (-0.6; 95% confidence interval, -0.9 to -0.2; P =.001). Statistically significant improvements favoring GSP301 were shown for all individual rTNSS symptoms, instantaneous Total Nasal Symptom Score, and most of its individual symptoms, Physician-assessed Nasal Symptom Score (P =.01), and Pediatric Rhinoconjunctivitis Quality of Life Questionnaire (P <.001). For ocular symptoms, numerical improvements favoring GSP301 were observed, with statistical significance achieved only for reflective "tearing/watering eyes" (P =.04). Treatment-emergent adverse events occurred in 12.0% and 10.4% of patients in the GSP301 and placebo groups, respectively. One subject (0.5%) (placebo group) experienced a serious adverse event (suspected viral meningitis) that was not related to the study treatment and was resolved. Conclusion: GSP301 was well tolerated and efficacious for treating SAR symptoms in pediatric patients and showed a favorable safety profile. (c) 2022 American College of Allergy, Asthma & Immunology.

Objectives Asthma affects over 6 million children in the United States alone. This study investigated the efficacy and long-term safety of mometasone furoate-formoterol (MF/F) and MF monotherapy in children with asthma. Materials and Methods This phase 3, multicenter, randomized controlled trial evaluated metered-dose inhaler twice daily (BID) dosing with MF/F 100/10 mu g or MF 100 mu g in children, aged 5 to 11 years, with a history of asthma for greater than or equal to 6 months and confirmed bronchodilator reversibility, who were adequately controlled on inhaled corticosteroid/long-acting beta-agonist combination therapy for greater than or equal to 4 weeks. After a 2-week run-in on MF 100 mu g BID, eligible patients received 24 weeks of double-blind treatment and were followed for safety up to 26 weeks. The primary efficacy endpoint was the change from baseline in AM postdose 60-minute AUC %predicted FEV1% across 12 weeks of treatment. Results A total of 181 participants received at least one dose of MF/F (n = 91) or MF (n = 90). MF/F was superior to MF across the 12-week evaluation period, with a treatment advantage of 5.21 percentage points (P < .001). Superior onset of action with MF/F over MF was achieved as early as 5 minutes postdose on day 1. Overall, approximately 50% of participants experienced one or more treatment-emergent adverse events, with fewer occurring in the MF/F group. Conclusions In children 5 to 11 years of age with persistent asthma, the addition of F to MF was well tolerated and provided significant, rapid, and sustained improvement in lung function compared with MF alone.

Asthma affects over 6 million children in the United States alone. This study investigated the efficacy and long-term safety of mometasone furoate-formoterol (MF/F) and MF monotherapy in children with persistent asthma.

Introduction Seasonal allergic rhinitis (SAR) symptoms often impair quality of life (QoL). In two randomized, double-blind phase 3 studies, twice-daily GSP301 nasal spray, a fixed-dose combination of olopatadine hydrochloride/mometasone furoate, significantly improved reflective and instantaneous Total Nasal Symptom Scores (rTNSS, primary endpoint; iTNSS, secondary endpoint) vs placebo (presented elsewhere). Results of additional endpoints comparing the efficacy and QoL of GSP301 vs placebo are reported here. Methods In study 1 (NCT02631551; N=1,180) and study 2 (NCT02870205; N=1,176), patients with SAR (≥12 years) were randomized 1:1:1:1 to GSP301 (olopatadine 665μg/mometasone 25μg BID), olopatadine (665μg BID), mometasone (25μg BID), or placebo for 14 days. Mean changes from baseline in Physician-assessed Nasal Symptom Score (PNSS) and Rhinoconjunctivitis Quality of Life Questionnaire–Standardized Activities [RQLQ(S)] for GSP301 vs placebo were analyzed using mixed-effect model repeated measures (P Results GSP301 significantly improved PNSS vs placebo in study 1 (least squares mean difference [95% CI]: -0.82 [-1.26, -0.38], PPPPP Conclusions In two phase 3 SAR studies, twice-daily GSP301 treatment provided significant improvements in nasal symptoms and QoL vs placebo and was well tolerated.

The inhaled corticosteroid MF, as delivered via dry-powder inhaler (DPI) QD in the evening (PM), is approved in the US to treat pediatric asthma. This study evaluated 3 doses of MF as delivered via MDI, in children ages 5-11yr with persistent asthma. This 12-week randomized, double-blind, placebo-controlled study included 5 arms: MF-MDI 50mcg BID, MF-MDI 100mcg BID, MF-MDI 200mcg BID, MF-DPI 100mcg QD PM and placebo, using a double-dummy design. The primary analysis assessed 3 doses of MF-MDI, vs placebo, on the change in %-predicted forced expiratory volume in one second (FEV1) from Baseline to Week-12; a secondary analysis compared MF-MDI 50mcg BID versus MF-DPI 100mcg QD PM. Adverse events (AEs) were monitored throughout the study. All 3 doses of MF-MDI were superior to placebo on %-predicted FEV1 at Week-12; least-squares (LS) mean differences from placebo were 3.87 (P=0.019), 6.29 (P<0.001), and 5.34 (P=0.001) percentage-points for MF-MDI 50, 100, and 200mcg BID, respectively. MF-MDI 50mcg BID was similar to MF-DPI 100mcg QD PM, though the LS mean difference of 1.39 (P=0.368) numerically favored MF-MDI 50mcg BID. AE incidences were similar among all treatment groups. There were no reports of oropharyngeal candidiasis or dysphonia (which were AEs pre-specified for analysis) in the trial. In children ages 5-11yr with persistent asthma, all three doses of MF-MDI (50mcg, 100mcg, and 200mcg) BID demonstrated significant improvement in FEV1 after 12 weeks of treatment. MF was generally well tolerated; no new safety concerns were identified in this trial.

BackgroundThis study aimed to assess the efficacy of MP-AzeFlu (a novel intranasal formulation of azelastine hydrochloride and fluticasone propionate in a single spray) in children with seasonal allergic rhinitis (SAR) and explore the importance of child symptom severity assessment in paediatric allergic rhinitis (AR) trials.MethodsA total of 348 children (4-11years) with moderate/severe SAR were randomized into a double-blind, placebo-controlled, 14-day, parallel-group trial. Efficacy was assessed by changes from baseline in reflective total nasal symptom score (rTNSS), reflective total ocular symptom score (rTOSS) and individual symptom scores over 14days (children 6-11years; n=304), recorded by either children or caregivers. To determine whether a by-proxy effect existed, efficacy outcomes were assessed according to degree of child/caregiver rating. Moreover, total Paediatric Rhinitis Quality of Life Questionnaire (PRQLQ) score was compared between the groups.ResultsA statistically superior, clinically relevant efficacy signal of MP-AzeFlu versus placebo was apparent for PRQLQ overall score (diff: -0.29, 95% CI -0.55, -0.03; p=0.027), but not for rTNSS (diff: -0.80; 95% CI: -1.75; 0.15; p=0.099). However, as the extent of children's self-rating increased, so too did the treatment difference between MP-AzeFlu and placebo; MP-AzeFlu provided significantly better relief than placebo for rTNSS (p=0.002), rTOSS (p=0.009) and each individual nasal and ocular symptom assessed (except rhinorrhoea; p=0.064) when children mostly rated their own symptoms.ConclusionsMP-AzeFlu is an effective treatment for AR in childhood. Caregivers are less able than children to accurately assess response to treatment with available tools. A simple paediatric-specific tool to assess efficacy in AR trials in children is needed.

Summary Objectives Mometasone furoate (MF), delivered via dry‐powder inhaler (DPI) QD in the evening (PM), is a treatment option for pediatric patients with asthma. We evaluated MF delivered via a metered‐dose inhaler (MDI), in children ages 5–11 years with persistent asthma. Methods This was a 12‐week double‐blind, double‐dummy, placebo‐controlled trial. Pateints were randomized to the following treatments: MF‐MDI 50 mcg BID, MF‐MDI 100 mcg BID, MF‐MDI 200 mcg BID, MF‐DPI 100 mcg QD PM, and placebo. The primary analysis assessed MF‐MDI doses versus placebo, on the change in %‐predicted forced expiratory volume in one second (FEV 1 ) from baseline to week‐12; a secondary analysis compared MF‐MDI 50 mcg BID versus MF‐DPI 100 mcg QD PM. Adverse events (AEs) were monitored throughout the trial. Results For change from baseline in %‐predicted FEV 1 at week 12, least‐squares (LS) mean differences from placebo were 3.87 ( P = 0.019), 6.29 ( P < 0.001), and 5.34 ( P = 0.001) percentage‐points for MF‐MDI 50, 100, and 200 mcg BID, respectively. The LS mean difference for MF‐MDI 50 mcg BID versus MF‐DPI 100 mcg QD PM was 1.39 ( P = 0.368). AE incidences were similar among all treatment groups. There were no reports of oropharyngeal candidiasis or dysphonia, which were AEs pre‐specified for analysis,. Conclusions In children ages 5–11 years with persistent asthma, all three doses of MF‐MDI (50, 100, and 200 mcg BID) demonstrated significant improvement in FEV 1 after 12 weeks of treatment. MF was generally well tolerated with no new safety concerns identified in this trial. Pediatr Pulmonol. 2017;52:310–318. © 2016 Wiley Periodicals, Inc.

BACKGROUND Breath-actuated inhalers (BAI) have been developed to simplify the delivery of inhaled medication. OBJECTIVE To evaluate the safety and efficacy of beclomethasone dipropionate hydrofluoroalkane BAI and metered-dose inhaler (MDI) versus placebo in patients who previously used a mid- to high-dose inhaled corticosteroid or inhaled corticosteroid/long-acting beta agonist for persistent asthma. METHODS This phase III study included five treatment groups: placebo, and four beclomethasone dipropionate groups (BAI 320 μg/day, BAI 640 μg/day, MDI 320 μg/day, and MDI 640 μg/day). Efficacy over 12 weeks was assessed by spirometry, peak flow measurements, and other clinical end points. Safety was assessed by adverse events. RESULTS Baseline-adjusted trough morning forced expiratory volume in 1 second area under the effect curve from time 0 to 12 weeks (primary end point) was increased in the BAI 320 and BAI 640 μg/day groups and the MDI 640 μg/day group versus placebo (not significant). Clinically important improvements were noted in morning and evening peak expiratory flow and decreased rescue medications. More patients who received placebo than patients in active treatment groups withdrew due to meeting the stopping criteria for worsening asthma. Patients in the active treatment groups experienced a greater decrease in asthma symptoms than patients in the placebo group. Quality of life and Asthma Control Test scores improved in the active treatment groups compared with the placebo group (p ≤ 0.0074). The most common adverse events (>5% in any group) were oral candidiasis and upper respiratory tract infection. CONCLUSION Clinical benefits for patients who used BAI 320 and 640 μg/day and MDI 640 μg/day were demonstrated. The safety profiles of BAI 320 and 640 μg/day were comparable with that of the MDI. These benefits and the continued need for better symptom control among patients with asthma support the continued development of this controller medication. ClinicalTrials.gov identifier NCT02031640.

Background: Beclomethasone dipropionate (BDP) nasal aerosol (non-aqueous) is approved for management of seasonal and perennial allergic rhinitis (PAR) in adolescents and adults.Objective: To evaluate the efficacy and safety of BDP nasal aerosol at 80 mg/day in children with PAR.Methods: This 12-week, phase 3, double-blinded, placebo-controlled, parallel-group study randomized 547 children (4-11 years old) with PAR to once-daily BDP nasal aerosol at 80 mg/day or placebo. The primary end point was change from baseline in average morning and evening reflective total nasal symptom score (rTNSS) during the first 6 weeks of treatment in patients 6 to 11 years old. Changes from baseline in average morning and evening instantaneous TNSS (iTNSS) in children 6 to 11 years old and average rTNSS and iTNSS in children 4 to 11 years old were assessed during the first 6 weeks of treatment.Results: Improvements were significantly greater with BDP nasal aerosol than with placebo during the first 6 weeks of treatment in children 6 to 11 years old in average morning and evening rTNSS and iTNSS (mean treatment difference -0.66 [ P = .002] and -0.58 [ P = .004], respectively). Improvements in average morning and evening rTNSS and iTNSS also were significantly greater in patients 4 to 11 years receiving BDP nasal aerosol than with placebo during the first 6 weeks of treatment (P - .002 and P - .004, respectively). Similar improvements were seen during 12 weeks of treatment. The safety profile of BDP nasal aerosol was comparable to that of placebo.Conclusion: The BDP nasal aerosol at 80 mg/day in children 4 to 11 years old was well tolerated and effective in controlling nasal symptoms of PAR. (C) 2015 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc.

BDP nasal aerosol (a nonaqueous formulation) is approved for management of seasonal and perennial allergic rhinitis in adolescents and adults. This post hoc analysis evaluated the effectiveness of BDP nasal aerosol by baseline symptom severity in children with SAR. This 2-week, double-blind, placebo-controlled study evaluated children with SAR (aged 6-11 years) randomized to BDP nasal aerosol 80μg/day (n=239), 160μg/day (n=242), or placebo (n=234). Efficacy included change from baseline in patient-reported average AM and PM reflective and instantaneous TNSS (rTNSS and iTNSS) by baseline symptom severity: less severe (baseline rTNSS/iTNSS

BACKGROUND:MK-3641 is a short ragweed sublingual tablet under investigation for immunotherapy of ragweed pollen-induced allergic rhinitis. OBJECTIVE:To characterize the safety and tolerability of a ragweed sublingual tablet (Merck/ALK-Abelló) in ragweed-allergic adults with or without conjunctivitis. METHODS:Data from 4 randomized, double-blinded, placebo-controlled trials of MK-3641 (2 28-day and 2 52-week trials) were evaluated. Pooled analyses examined short-term safety over 28 days from all 4 trials and long-term safety from the 52-week trials. RESULTS:Across all studies, 757, 198, 454, and 1,058 subjects were randomized to placebo or 1.5, 6, or 12 Amb a 1-U of MK-3641, respectively. Treatment-related adverse events were more frequent in the 6- and 12-Amb a 1-U MK-3641 groups than in the placebo group and were primarily local application-site reactions occurring in the first few days of treatment. There was no treatment-associated loss of asthma control or worsening of asthma associated with treatment. No swellings led to airway obstruction or respiratory compromise. No treatment-related anaphylactic shock, life-threatening, or serious treatment-related adverse events were reported for any MK-3641 dose. Of the 1,707 MK-3641-treated subjects, 1 systemic (anaphylactic) reaction was reported (0.06%). The 52-week long-term assessment was generally similar to the safety profile based on the 28-day assessment. CONCLUSION:MK-3641 doses up to and including 12 Amb a 1-U were well tolerated, with no unexpected safety findings. Sublingual immunotherapy risks such as worsening asthma or airway swellings that could cause airway obstruction were not observed. Systemic reactions and use of epinephrine were uncommon. In these studies, after the first dose was administered in a health care setting, self-administration was well tolerated. TRIAL REGISTRATION:clinicaltrials.gov Identifiers: NCT01469182, NCT00783198, NCT00770315, and NCT00978029.

RationaleIntranasal corticosteroids relieve nasal and ocular symptoms associated with seasonal allergic rhinitis (SAR); however, their effects on other bothersome symptoms such as itching of ears/palate have not been extensively evaluated. The effect of beclomethasone dipropionate (BDP) nasal aerosol treatment on relief of itching of ears/palate in patients with SAR is reported here.MethodsPatients (≥12 years of age) enrolled in this double-blind, placebo-controlled study were randomly assigned to once-daily treatment with BDP nasal aerosol 320 μg or placebo. Although the primary efficacy endpoint was change from baseline in average AM and PM patient-reported total nasal symptom score over the 2-week treatment period, change from baseline in average AM and PM patient-reported non-nasal symptom scores (itching/burning eyes, tearing/watering eyes, eye redness, itching of ears/palate) were also evaluated using the non-nasal subpopulation (N=304).ResultsAt week 2, BDP nasal aerosol resulted in a greater improvement in reflective and instantaneous non-nasal symptom scores versus placebo (LS mean treatment difference: –0.79, P<0.001 and –0.70, P=0.002, respectively). The change from baseline in reflective non-nasal symptom score was greater with BDP nasal aerosol compared with placebo on day 2 and from day 4 onward (P<0.05). Moreover, BDP nasal aerosol resulted in a greater improvement versus placebo in all individual non-nasal symptom scores including itching of ears/palate (LS mean treatment difference for itching of ears/palate: –0.21 [reflective], –0.22 [instantaneous]; P<0.001 for both).ConclusionsBDP nasal aerosol provides substantial relief of non-nasal symptoms, including the common and bothersome symptom of itching of ears/palate, in patients with SAR. RationaleIntranasal corticosteroids relieve nasal and ocular symptoms associated with seasonal allergic rhinitis (SAR); however, their effects on other bothersome symptoms such as itching of ears/palate have not been extensively evaluated. The effect of beclomethasone dipropionate (BDP) nasal aerosol treatment on relief of itching of ears/palate in patients with SAR is reported here. Intranasal corticosteroids relieve nasal and ocular symptoms associated with seasonal allergic rhinitis (SAR); however, their effects on other bothersome symptoms such as itching of ears/palate have not been extensively evaluated. The effect of beclomethasone dipropionate (BDP) nasal aerosol treatment on relief of itching of ears/palate in patients with SAR is reported here. MethodsPatients (≥12 years of age) enrolled in this double-blind, placebo-controlled study were randomly assigned to once-daily treatment with BDP nasal aerosol 320 μg or placebo. Although the primary efficacy endpoint was change from baseline in average AM and PM patient-reported total nasal symptom score over the 2-week treatment period, change from baseline in average AM and PM patient-reported non-nasal symptom scores (itching/burning eyes, tearing/watering eyes, eye redness, itching of ears/palate) were also evaluated using the non-nasal subpopulation (N=304). Patients (≥12 years of age) enrolled in this double-blind, placebo-controlled study were randomly assigned to once-daily treatment with BDP nasal aerosol 320 μg or placebo. Although the primary efficacy endpoint was change from baseline in average AM and PM patient-reported total nasal symptom score over the 2-week treatment period, change from baseline in average AM and PM patient-reported non-nasal symptom scores (itching/burning eyes, tearing/watering eyes, eye redness, itching of ears/palate) were also evaluated using the non-nasal subpopulation (N=304). ResultsAt week 2, BDP nasal aerosol resulted in a greater improvement in reflective and instantaneous non-nasal symptom scores versus placebo (LS mean treatment difference: –0.79, P<0.001 and –0.70, P=0.002, respectively). The change from baseline in reflective non-nasal symptom score was greater with BDP nasal aerosol compared with placebo on day 2 and from day 4 onward (P<0.05). Moreover, BDP nasal aerosol resulted in a greater improvement versus placebo in all individual non-nasal symptom scores including itching of ears/palate (LS mean treatment difference for itching of ears/palate: –0.21 [reflective], –0.22 [instantaneous]; P<0.001 for both). At week 2, BDP nasal aerosol resulted in a greater improvement in reflective and instantaneous non-nasal symptom scores versus placebo (LS mean treatment difference: –0.79, P<0.001 and –0.70, P=0.002, respectively). The change from baseline in reflective non-nasal symptom score was greater with BDP nasal aerosol compared with placebo on day 2 and from day 4 onward (P<0.05). Moreover, BDP nasal aerosol resulted in a greater improvement versus placebo in all individual non-nasal symptom scores including itching of ears/palate (LS mean treatment difference for itching of ears/palate: –0.21 [reflective], –0.22 [instantaneous]; P<0.001 for both). ConclusionsBDP nasal aerosol provides substantial relief of non-nasal symptoms, including the common and bothersome symptom of itching of ears/palate, in patients with SAR. BDP nasal aerosol provides substantial relief of non-nasal symptoms, including the common and bothersome symptom of itching of ears/palate, in patients with SAR.

An aerosolized intranasal corticosteroid formulation is desirable for many allergic rhinitis (AR) patients, especially children, who wish to avoid the "wet feeling" and "drip down the throat" associated with aqueous formulations. Beclomethasone dipropionate hydrofluoroalkane (BDP HFA) nasal aerosol, a novel non-aqueous aerosol, has been shown to be safe and effective in adolescents and adults with AR. This study evaluated the efficacy and safety of BDP HFA in children with seasonal AR (SAR). In this 2-week, double-blind, placebo-controlled study, SAR subjects (6-11 years of age) were randomized to once daily treatment with BDP HFA 80mcg (n=239), BDP HFA 160mcg (n=242) or placebo (n=234). The primary endpoint was average AM and PM reflective total nasal symptom score (rTNSS) over the 2-week treatment period. The change from baseline in average AM and PM rTNSS was significantly greater for BDP HFA versus placebo (-0.71 [95%CI: -1.1, -0.3] for 80mcg; -0.76 [95%CI: -1.1, -0.4] for 160mcg; P<0.001 for both). Similarly, the change from baseline in average AM and PM instantaneous TNSS was significantly greater for BDP HFA versus placebo (-0.63 [95%CI: -1.0, -0.3] for 80mcg; -0.73 [95%CI: -1.1, -0.4] for 160mcg; P<0.001 for both). The safety profile of BDP HFA was generally similar to placebo. These results demonstrate that BDP HFA nasal aerosol provides significant nasal symptom relief in pediatric SAR subjects. These results indicate that BDP HFA nasal aerosol should provide a new effective and well-tolerated treatment option for pediatric SAR patients.

An aerosol formulation may be preferred by some allergic rhinitis (AR) patients, to avoid the "wet feeling" and nasal runoff associated with aqueous nasal corticosteroid sprays. Beclomethasone dipropionate (BDP) hydrofluoroalkane nasal aerosol is a recently developed, nonaqueous, nonchlorofluorocarbon formulation of BDP for the treatment of AR. This study was designed to evaluate the efficacy, safety, and quality-of-life benefits of BDP nasal aerosol in subjects with seasonal AR (SAR). Eligible subjects (≥12 years of age) enrolled in this 2-week study were randomized to either BDP nasal aerosol at 320 μg/day (n = 169) or placebo (n = 171). Efficacy assessments included reflective and instantaneous total nasal symptom scores (rTNSS and iTNSS, respectively), Rhinoconjunctivitis Quality of Life Questionnaire (RQLQ) score, reflective and instantaneous total ocular symptom scores (rTOSS and iTOSS, respectively), and physician-assessed total nasal symptom score (PNSS). Safety and tolerability were also assessed. Subjects receiving BDP nasal aerosol showed a significantly greater improvement from baseline in average A.M. and P.M. rTNSS versus placebo (treatment difference, -0.91; 95% confidence interval, -1.3, -0.5; p < 0.001) over 2 weeks of treatment. Greater improvements in rTNSS with BDP nasal aerosol compared with placebo were evident by day 2 and were maintained throughout the treatment period. Similarly, significant improvements were seen with BDP nasal aerosol in iTNSS (p < 0.001) and RQLQ score (p = 0.005) compared with placebo. Treatment with BDP nasal aerosol also resulted in greater improvements in rTOSS (p = 0.002), iTOSS (p = 0.003), and PNSS (p < 0.001) relative to placebo. BDP nasal aerosol was well tolerated and the overall safety profile was similar to placebo. Results from this clinical study indicated that BDP nasal aerosol provided significant AR symptom relief and was well tolerated in patients with SAR with an overall safety profile similar to placebo. Clinicaltrials.gov identifier: NCT01024608.

Background: Cryopyrin-associated periodic syndromes (CAPS) are rare, inherited autoinflammatory disorders associated with considerable hardship to patients. The interleukin-1 inhibitor rilonacept has been shown to be well-tolerated and effective in preventing CAPS symptoms in 2 pivotal studies.Objective: In this study, the long-term effects of rilonacept for improvement in CAPS symptoms and its safety and tolerability were evaluated during extended treatment.Methods: Patients with CAPS entered a 72-week open-label extension (OLE) following 2 sequential placebo-controlled Phase III studies (n = 44), or entered directly into the OLE (n = 57). Adults received weekly subcutaneous rilonacept 160 mg, and pediatric patients received subcutaneous rilonacept 2.2 mg/kg, up to 160 mg/week. Safety was evaluated in all patients, and efficacy was evaluated using a validated composite key symptom score in 56 patients.Results: After rilonacept treatment for 72 to 96 weeks mean key symptom score at OLE Week 72 was reduced from 2.6 to 0, and the mean number of multisymptom flare days was reduced from 7.3 (34.8% of days) at baseline to 0.6 (2.9% of days) at end point. Elevated levels of inflammatory markers (eg, high sensitivity-C reactive protein and serum amyloid A, were normalized. Adverse events were generally mild to moderate, the most common being injection site reactions and upper respiratory tract infections. The incidence of these events was similar to or lower than the rate reported in the pivotal studies.Conclusions: Long-term treatment with rilonacept of up to 96 weeks resulted in improvements in clinical signs and symptoms of CAPS and normalized biomarkers of inflammation. Rilonacept exhibited a generally favorable safety and tolerability profile in adult and pediatric patients with CAPS throughout the extended treatment period. ClinicalTrials.gov identifier: NCT 00288704. (Clin Ther. 2012;34: 2091-2103) (C) 2012 Elsevier HS Journals, Inc. All rights reserved.