Use Of Dupilumab Research Articles

One-Year Insights into the GLOBOSTAD Multinational Prospective Observational Study of Patients Receiving Dupilumab for Atopic Dermatitis.

Currently, limited data are available on long-term use of dupilumab to treat atopic dermatitis (AD) in a multinational real-world setting. The aim of this analysis was to report the interim 1-year data for patients with AD enrolled in the GLOBOSTAD registry, including treatment patterns, dupilumab effectiveness and safety, and healthcare burden. GLOBOSTAD is an ongoing, 5-year, multinational, prospective, observational study of adult/adolescent (aged ≥ 12years at baseline) patients with AD who initiated dupilumab in real-world settings according to their local country-specific prescribing guidelines. Outcomes were evaluated at baseline and at 3, 6 and 12months and included Eczema Area and Severity Index (EASI) total score, SCORing Atopic Dermatitis (SCORAD) total score, percent body surface area (BSA) affected, Patient-Oriented Eczema Measure (POEM), Dermatology Life Quality Index (DLQI) total score for adults or Children's Dermatology Life Quality Index (CDLQI) total score for adolescents and pruritus Numeric Rating Scale (NRS) total score. At the interim 1-year cut-off (March 2023), 955 patients were enrolled in GLOBOSTAD, and follow-up data were obtained from 903 patients. After dupilumab initiation, mean improvements in effectiveness outcome measures from baseline to month 3 were EASI from 25.1 to 6.1, SCORAD 59.3 to 25.3, POEM 19.7 to 8.7, DLQI 13.7 to 5.3, CDLQI 12.2 to 2.7 and pruritus NRS 6.3 to 2.5, with each measure exceeding the minimal clinically important difference. These positive changes in effectiveness outcomes were maintained or further improved through 12months since treatment initiation. AD-related hospitalizations and emergency room or urgent care facility visits decreased from 11.1% to 1.7% from baseline to month 12. In a multinational real-world setting, dupilumab demonstrated rapid, robust and sustained effectiveness in patients with moderate-to-severe AD across multiple disease domains, including AD signs, symptoms, quality of life and emergency/urgent care visits. Safety was consistent with the known dupilumab safety profile. ClinicalTrials.gov Identifier NCT03992417.

Successful novel use of dupilumab for gastrointestinal involvement of idiopathic hypereosinophilic syndrome: case report and review of the literature.

Hypereosinophilic syndrome (HES) is characterized by blood and tissue hypereosinophilia leading to organ damage. Gastrointestinal involvement is the third most common manifestation. We present a patient with idiopathic HES with secondary eosinophilic esophagitis (EoE), gastritis, and enteritis, corticosteroids-dependent, azathioprine- and mepolizumab-refractory. The patient achieved clinical and histopathologic remission following dupilumab treatment. A 28year-old female presented with chronic episodic nausea and emesis since childhood and initial diagnosis of primary eosinophilic gastrointestinal disease (EGID), improved with corticosteroids, refractory to azathioprine. She was found to have peripheral eosinophilia and multifactorial anemia, with iron, B12, and folate deficiencies. Esophageal, gastric, duodenal, and terminal ileum biopsies showed significant eosinophilic infiltrate. Bone marrow biopsy at age 31 confirmed HES diagnosis. By age 32, she became total parental nutrition (TPN)-dependent. She failed trials of benralizumab and mepolizumab [anti-interleukin (IL)-5 inhibitors], and cromolyn (mast-cell stabilizer). After developing new esophageal stricture, we initiated dupilumab (IL-4/13 inhibitor), recently FDA-approved for EoE. After 9weeks, esophageal stricture, gut tissue eosinophilia, and prior intestinal ulcerations resolved. She ceased TPN and is tolerating a non-restricted diet, with complete symptom resolution. Our patient's complete remission with dupilumab shows promise for broadening its use in treating GI involvement in HES, along with primary EGIDs.

Risk of malignancy associated with use of dupilumab versus other treatments in atopic dermatitis patients: a national database analysis

Risk of malignancy associated with use of dupilumab versus other treatments in atopic dermatitis patients: a national database analysis

Real-world efficacy and safety of dupilumab for paediatric atopic dermatitis: a multicentre retrospective study.

Real-world data regarding the use of dupilumab in children with atopic dermatitis (AD) are limited. To evaluate the real-world efficacy of dupilumab in children with moderate-to-severe AD over an extended follow-up period. This was a retrospective study of patients (≤ 18 years) with moderate-to-severe AD treated with dupilumab in four Israeli tertiary centres. Efficacy and safety were assessed using descriptive statistics. In total, 230 patients were included in the analysis [age 9.9 years (SD 4.3), male/female 1 : 1 ratio)]. Of them, 59.6% (137/230) had ≥ 1 atopic comorbidity. The follow-up duration ranged from 2 to 248 weeks, with a median of 52 weeks (interquartile range 24-96). Within 12 weeks of treatment, 41.7% (68/163) of patients had reached Investigator Global Assessment 0-1. The mean body surface area was reduced from 58.0% (SD 20.5%) at baseline to 27.8% (SD 20.2%) at 12 weeks. The average Pruritus Numeric Rating Scale score was reduced from 7.9 (SD 2.2) at baseline to 2.3 (SD 2.8) at 12 weeks. Adverse events, in 210 patients, included conjunctivitis in 34 patients (16.2%), injection-site reactions in 11 patients (5.2%) and dupilumab-associated head and neck dermatitis in 6 patients (2.9%). Overall, 26 of 210 patients (12.3%) discontinued the treatment: 9 of the 26 patients (35%) because of adverse events and 15 patients (58%) because of inadequate efficacy. The overall probability of dupilumab survival at 52 weeks was 94.0%. Real-world data presented here for 230 paediatric and adolescents with moderate-to-severe AD reinforce dupilumab's efficacy and safety and highlight dupilumab's high survival rate after 1 year of treatment in the paediatric population.

Real-world predictors of dupilumab prescription in patients with chronic rhinosinusitis with nasal polyps.

Despite increasing dupilumab use for chronic rhinosinusitis with nasal polyps (CRSwNP), little is known about the factors influencing its use in real-world practice. We aimed to identify factors that may predict dupilumab prescription in CRSwNP patients who have undergone endoscopic sinus surgery (ESS). A single-institution, retrospective cohort study of patients who underwent ESS for CRSwNP between 2015 and 2023 was conducted. Demographics, comorbidities, 22-item sinonasal outcome test (SNOT-22) scores, and dupilumab prescription date were extracted from patient records. Intraoperative nasal mucus cytokine levels were measured using a multiplex bead assay. Univariate logistic regression analysis was performed to identify factors associated with dupilumab prescription, and multivariate logistic regression was used to adjust for surgery date. A total of 299 CRSwNP patients were included, including seventy (23.4%) who were prescribed dupilumab postoperatively. Patients were more likely to be prescribed dupilumab if they had asthma (odds ratio [OR] 2.304), aspirin-exacerbated respiratory disease (AERD, OR 3.375), elevated tissue eosinophils (OR 1.005), and higher 3-month postoperative SNOT-22 scores (OR 1.027). Patients prescribed dupilumab also had greater odds of having elevated mucus interleukin (IL)-5 (OR 1.128) and IL-13 (OR 1.213). When adjusting for surgery date, associated factors included: asthma (OR 2.444), AERD (OR 3.750), allergic rhinitis (OR 1.833), higher tissue eosinophils (OR 1.005), elevated 3-month SNOT-22 scores (OR 1.028), and higher IL-5 (OR 1.123) and IL-13 (OR 1.202) levels. Asthma, AERD, allergic rhinitis, and elevated tissue eosinophil, IL-5, and IL-13 levels are predictive of dupilumab prescription in CRSwNP patients. These may serve as clinical and inflammatory biomarkers and can aid in counseling patients about expected disease trajectory.

DUPILUMAB: A NEW ALLY FOR THE TREATMENT OF EOSINOPHILIC ESOPHAGITIS?

INTRODUCTION: Eosinophilic esophagitis is a chronic, immune-mediated disease of the gastrointestinal tract, resulting from the interaction between genetic, environmental and immunological factors of the affected patient. The clinical picture is characterized by esophageal dysfunction, which may present as dysphagia, precordial pain, upper abdominal pain or food impaction. Treatment aims to improve clinical symptoms and prevent disease progression and subsequent complications. In this context, immunobiologicals are emerging and promising therapies, with Dupilumab being the first specific treatment approved by the FDA. It is an IgG4 monoclonal antibody that targets IL-4R alpha and, from there, reduces protein transphorylation, transcription and the T helper 2-regulated response. The objective of this systematic review is to evaluate the efficacy of this drug in the treatment of eosinophilic esophagitis, in comparison with the options already available on the market. METHOD: PubMed searches were performed using the keywords “eosinophilic esophagitis dupilumab” and “dupilumab treatment esophagitis eosinophilic”. Initially, 60 results were obtained, but after exclusion by title, abstract and full text, 14 articles were included. RESULT: all articles analyzed demonstrated improvement in patients using Dupilumab, with 13 of them indicating a reduction in eosinophil counts, 8 showing clinical improvement in symptoms, 8 indicating histological improvement, 7 indicating improvement in the endoscopic pattern, according to the Endoscopic Reference for Eosinophilic Esophagitis, and 4 indicating a reduction in the Dysphagia Symptom Questionnaire (DQQ) score. DISCUSSION: eosinophilic esophagitis is a rare disease, but with potential for progression, negatively interfering with the patient's quality of life. Therefore, since current drug therapy is nonspecific, the use of Dupilumab presents itself as an innovative and optimistic solution for the future, because it changes the understanding of disease management, expands therapeutic options and improves prognosis. CONCLUSION: the use of Dupilumab in the treatment of eosinophilic esophagitis has proven to be effective. However, for the treatment to be well established with regard to the doses used, frequency of administration and longitudinal monitoring, further studies are still required.

Dupilumab for Chronic Hand Eczema: A Systematic Review and Meta-Analysis.

Chronic hand eczema (CHE) is a distressing and pervasive dermatologic condition, and effective treatment can be challenging. The novel IL-4/13 inhibitor dupilumab has shown clinical efficacy for atopic dermatitis (AD) and is being studied for CHE. PubMed database was searched for terms dupilumab and chronic hand eczema/dermatitis, including CHE subtypes (dyshidrotic, hyperkeratotic, allergic, contact, atopic, occupational, vesicular). Twenty-two study publications met inclusion and exclusion criteria for evaluation. Two multicenter prospective studies, one randomized controlled trial, two prospective observational studies, one retrospective study, nine case series, and seven case reports were included in the analysis with 374 total patients. Any partial response or complete resolution of CHE occurred in 80.3% of patients treated with dupilumab within 4-16 weeks of treatment. The patient response tended to increase throughout the duration of these studies for a significant percentage of patients, and for many it lasted beyond the study duration. Significant improvement in hand eczema severity scores and quality-of-life scores were noted. Efficacy was reported in over half of the cases of hyperkeratotic hand eczema; however, efficacy was decreased compared with relatively high consistent efficacy in other CHE subtypes such as dyshidrotic, vesicular, atopic, and contact CHE. Patients with current or previous AD had increased improvement scores compared to those without. The most common adverse effects were conjunctivitis (9.9%), herpes viral infection (1.4%), and facial erythema/dermatitis (1.3%). Dupilumab shows therapeutic efficacy in treating CHE. Further randomized controlled studies of dupilumab use in CHE subtypes are needed.

Dupilumab Use in Patients With Hypereosinophilic Syndromes: A Multicenter Case Series and Review of the Literature

BackgroundHypereosinophilic syndromes (HES) are defined as hypereosinophilia with eosinophil-related clinical manifestations, some of which overlap in presentation with asthma, atopic dermatitis, eosinophilic esophagitis, and/or chronic rhinosinusitis with nasal polyps (CRSwNP). Dupilumab is approved to treat these conditions but can induce a transient rise in the absolute eosinophil count (AEC) and rare eosinophil-related complications. ObjectiveTo determine whether eosinophil-related complications of dupilumab are increased in HES. MethodsRetrospective chart review of patients with HES treated with dupilumab enrolled on an IRB-approved research protocol at the National Institutes of Health (NCT00001406) or receiving care at Walter Reed National Military Medical Center. Clinical response and treatment-emergent adverse events were recorded. Serum mediators were assessed in a subset of patients before and after dupilumab using stored samples. ResultsAmong the 28 patients (15 male, 13 female; median age 41.5), the most common prescribing indication for dupilumab was CRSwNP (n=11). Twenty-three patients (82%) showed significant clinical improvement on dupilumab. Hypereosinophilia (AEC >1.5x109/L) recurred or worsened in 9/20 patients on dupilumab monotherapy. Moreover, 4/20 (20%) patients developed an eosinophil-related complication with dupilumab discontinuation and/or addition of eosinophil-lowering therapy. None of the 8 patients who received dupilumab while in hematologic remission on an eosinophil-lowering biologic developed hypereosinophilia or an eosinophil-related complication. Serum IgE and eotaxin levels decreased on dupilumab therapy. ConclusionThese data suggest that dupilumab is effective in treating residual symptoms in HES patients but that the incidence of eosinophil-related complications is increased. Concomitant eosinophil-lowering therapy may reduce the risk of eosinophil-related complications during dupilumab therapy in patients with HES.

Dupilumab-Induced Erythrodermic Psoriasiform Dermatitis in Chronic Actinic Dermatitis: Case Report and Innovative Treatment Approach.

The off-label use of dupilumab has proven to be an effective treatment option for severe chronic actinic dermatitis (CAD). We present the first case of CAD presenting with dupilumab-induced erythrodermic psoriasiform dermatitis. A modified, spaced dupilumab dosing regimen, combined with cyclosporine, successfully managed both conditions, offering a promising strategy for managing dupilumab adverse effects while maintaining control over chronic actinic dermatitis.

Maternal, Fetal, and Labour Outcomes of Dupilumab Use for Atopic Dermatitis During Pregnancy: A Systematic Review.

Atopic dermatitis is a chronic complex inflammatory disease that significantly impacts maternal well-being and quality of life during pregnancy, warranting effective therapeutic interventions that prioritize maternal health and fetal safety. Dupilumab is approved for moderate-to-severe atopic dermatitis, but limited data exist regarding its safety during pregnancy. We conducted a systematic review to review and analyze maternal, fetal, and labour outcomes in patients receiving dupilumab for atopic dermatitis during pregnancy. Comprehensive searches were conducted using databases including OVID, Scopus, and Web of Science, covering studies published until May 2024. Our search yielded 285 studies, of which 13 met the eligibility criteria. These studies included 68 patients with 69 pregnancies, revealing 58 live births and 11 spontaneous abortions. Dupilumab therapy was administered continuously throughout pregnancy in 22.2% of cases, while 77.8% received intermittent treatment. Maternal atopic dermatitis outcomes showed significant improvement in disease severity. Most pregnancies (86.3%) progressed without complications. Labour-associated outcomes varied, with 82.4% of women undergoing vaginal deliveries. The majority of births occurred at full term (82.5%), with a mean gestational age of 38.4 weeks. Fetal outcomes demonstrated a normal birth weight in 92.3% of cases, with no reported congenital defects. Our review suggests that dupilumab use during pregnancy is associated with improvement of atopic dermatitis and low or minimal risk of major adverse outcomes in treated patients or their newborns. Prospective studies with long-term follow-up are warranted to confirm the safety of dupilumab in this population.

Dupilumab-related late adverse events in patients with chronic rhinosinusitis with nasal polyps

Background Anti-IL-4 receptor α antibody (dupilumab) has demonstrated favorable sinonasal outcomes for chronic rhinosinusitis with nasal polyps (CRSwNP), which is mainly caused by type 2 inflammation. Although increased blood eosinophil levels and injection site symptoms are frequently observed as acute adverse events (AEs) of dupilumab, limited knowledge is available regarding the late AEs of dupilumab for CRSwNP. Objectives We investigated the late AEs following the initiation of dupilumab treatment for CRSwNP. Material and methods Fifty-one patients with CRSwNP treated with dupilumab for > 3 months were enrolled, and their clinical data were collected from their medical records. Results Six (11.8%) patients experienced late AEs. One case of eczema with pruritus, one case of psoriasis-like dermatitis, two cases of severe rash, one case of malignant lymphoma, and one case of alopecia areata were observed. Skin disorders were the most common late AEs in this study. It is a Th1-inflammatory disease, and its mechanism is thought to be due to the immune imbalance caused by dupilumab. We could not confirm whether malignant lymphoma in our case was caused by dupilumab use. Conclusions and significance Skin disorders are often late AEs associated with dupilumab; therefore, careful monitoring after dupilumab initiation should be considered.

Real-World Effectiveness and Use of Dupilumab in Eosinophilic Esophagitis.

Dupilumab, the first US Food and Drug Administration-approved treatment for eosinophilic esophagitis (EoE), lacks real-world data on use and effectiveness. We conducted a retrospective cohort study of 70 patients with EoE prescribed dupilumab, comparing prescriber type, indication, follow-up, and response. Indications varied with gastroenterologists commonly prescribing for treatment-refractory cases and allergists as first-line therapy. Endoscopic assessment was lacking in 25.9%, but those with follow-up showed high histologic remission (92.3% first-line and 85% previous treatment failure). Dupilumab demonstrates effectiveness across EoE severity, including milder disease without previous treatment failure. Improving follow-up and assessing cost-effectiveness will help clarify its role in the treatment algorithm.

Cutaneous T-Cell Lymphoma and Dupilumab Use: A Multifactorial and Complex Story

Cutaneous T-Cell Lymphoma and Dupilumab Use: A Multifactorial and Complex Story

Rate of dupilumab use and symptom severity of patients with chronic rhinosinusitis with nasal polyposis after Draf 3 frontal sinusotomy.

The indications for endoscopic modified Lothrop procedure (Draf 3) in patients with refractory chronic rhinosinusitis with nasal polyposis (CRSwNP) remain unclear. This study evaluates the effectiveness of Draf 3 for refractory CRSwNP focusing on improvements in disease severity and need for subsequent dupilumab rescue therapy. Retrospective review of patients with CRSwNP undergoing Draf 3 surgery at a tertiary center between 2012 and 2022. Clinicodemographic variables were compared across those who did versus did not require rescue with postoperative dupilumab. Time to postoperative dupilumab rescue was analyzed and longitudinal disease-specific outcomes were measured using the sinonasal outcomes test (SNOT-22). Within 87 patients with CRSwNP, 24.1% had aspirin-exacerbated respiratory disease (AERD). Significant improvement in SNOT-22 score was found in CRSwNP with AERD (p<0.001) and without AERD (p=0.01) up to 24 months postoperative. 14.9% eventually required rescue with a dupilumab. More specifically, of 21 patients with AERD, 24.1% eventually required rescue with dupilumab. Dupilumab rescue was associated with a greater number of prior sinus surgeries (p=0.02), prior aspirin desensitization (p=0.02), and worse preoperative Lund-MacKay scores (p<0.001). No association between biologic rescue and frontal recess antero-posterior diameter was found (p=0.20). Draf 3 surgery in CRSwNP was associated with significant improvement in SNOT-22 score at 24 months. Furthermore, only 14.9% of patients required dupilumab rescue. Patients with AERD were more likely to require rescue with dupilumab even though 75.1% avoided treatment with the biologic over the study period.

Dupilumab treatment in paediatric atopic dermatitis (2 to 18 years): Spanish multicentre retrospective real-life study.

Moderate-to-severe atopic dermatitis (AD) can be difficult to manage in paediatric patients, with few licensed treatments in this age group. Dupilumab is approved for AD in children older than 6 months. To assess the effectiveness and safety of dupilumab in a real-life cohort of paediatric AD patients in Spain. A multicentre, retrospective real-life study on the effectiveness and safety of dupilumab in patients aged 2 to 18 years old with moderate-to-severe AD was conducted. Demographic and clinical characteristics were analysed, and effectiveness (EASI, IGA, DLQI, NRS itch), safety, and drug survival measures were assessed. A comparison of our results with other real-world outcomes and with clinical trials was made. Data from 243 patients from 19 centres was collected, with a mean follow-up of 85 weeks. Dupilumab exhibited significant effectiveness, with marked reductions in severity scores from week 4. By week 16, 79.4% of patients reached EASI75 and 40.5% reached EASI90. Mean percentage reduction in EASI was 79.7%. Increasing improvements were observed until week 52, with 85.8% and 49.6% achieving EASI75 and EASI90, respectively. Forty-three patients developed adverse events (AE) (43/243, 17.7%), being the most frequent ocular surface diseases (20/243, 8.2%), injection site reactions (8/243, 3.3%) and facial redness (7/243, 2.9%). Drug survival was high (96.9% and 93.1% after 1 and 2 years of follow-up, respectively), with only 19 (19/243, 7.8%) patients interrupting treatment: 7 (7/243, 2.9%) due to AE, 2 (2/243, 0.82%) due to secondary failure, 5 (5/243, 2.1%) were lost to follow-up and 5 (5/243, 2.1%) entered remission and stopped treatment. Real-life use of dupilumab in paediatric AD showcased sustained effectiveness, high drug survival, and acceptable safety profiles. Longer-term studies are crucial for AE surveillance and how to manage disease remission.

Real-life management of atopic dermatitis patients with an inadequate response to on-label use of dupilumab

In patients with moderate to severe atopic dermatitis (AD) showing an inadequate response to dupilumab 300mg/2weeks, few real-life studies reported the response to alternative regimen maintaining dupilumab.To assess and analyze the response to an increased dose of dupilumab or its combination with cyclosporin A (CsA), methotrexate (MTX), or itraconazole (ITRA), all adult AD patients from 7 French University Hospitals were retrospectively included if they achieved an inadequate response to dupilumab 300mg/2weeks and were subsequently treated with an increased dose of dupilumab (300mg every 7 or 10 days), or a combination of dupilumab 300mg/2weeks with CsA, MTX or ITRA. The response after 3 months, along with epidemiological, clinical, and therapeutic baseline characteristics, were collected.Overall, 68.75% of the 48 included patients achieved an improved response, including 45.8% of complete response (CR). No strategy proved significantly better. Patients showing an initial no response never achieved a further CR versus 52.4% of patients with an initial partial response (p = 0.025). Digestive intolerance and tachycardia led to MTX and ITRA discontinuation in 3 patients.Increasing the dose of dupilumab or combining it with CsA, MTX, or ITRA could be alternative and safe options, to be evaluated in further medico-economic studies.

Real-world effectiveness of dupilumab versus benralizumab and mepolizumab.

Introduction: In the United States, this real-world study compared the effectiveness of dupilumab, benralizumab, and mepolizumab in reducing exacerbations and systemic corticosteroid (SCS) prescriptions among patients with asthma. Methods: Patients (≥12 years old) who initiated dupilumab, benralizumab, or mepolizumab (index) between November 2018 and September 2020 were identified by using electronic medical record data. Subjects were included if they had ≥ 12 months of data before and after the index date and two or more severe asthma-related exacerbations before the index date. Differences in baseline characteristics were addressed by using inverse probability treatment weighting (IPTW). Pairwise comparisons between dupilumab and benralizumab, or mepolizumab were conducted by using negative binomial regression, adjusting for baseline rates and unbalance characteristics (≥10% standardized differences) after IPTW. Results: Overall, a total of 1737 subjects met all criteria: 825 dupilumab, 461 benralizumab, and 451 mepolizumab initiators. In the postindex period, dupilumab was associated with a 24% and 28% significant reduction in the risk of severe asthma exacerbations versus benralizumab (incidence rate ratio [IRR] 0.76 [95% confidence interval {CI}, 0.67-0.86)] and mepolizumab (IRR 0.72 [95% CI, 0.63-0.82]), respectively. In addition, dupilumab treatment significantly reduced SCS prescriptions by 16% and 25% versus benralizumab and mepolizumab, respectively (p < 0.05). Conclusion: This study represents one of the largest real-world comparisons of biologics (dupilumab, benralizumab, and mepolizumab) for asthma in the United States to date. This analysis shows that the use of dupilumab was associated with a significantly greater reduction in both severe asthma exacerbations and SCS prescriptions compared with benralizumab and mepolizumab.

Use of Dupilumab in Pediatric Patients: A Review

Use of Dupilumab in Pediatric Patients: A Review

Cutaneous T-Cell Lymphoma after Dupilumab Use: A Real-World Pharmacovigilance Study of the FDA Adverse Event Reporting System

Cutaneous T-Cell Lymphoma after Dupilumab Use: A Real-World Pharmacovigilance Study of the FDA Adverse Event Reporting System

Dupilumab Safety and Efficacy up to 1 Year in Children Aged 6 Months to 5 Years with Atopic Dermatitis: Results from a Phase 3 Open-Label Extension Study.

Pediatric patients with moderate-to-severe atopic dermatitis (AD) often experience a high disease burden and have a high risk of persistent disease. Standard-of-care immunosuppressive systemic treatments have been used off-label for AD in pediatric patients despite concerns for suboptimal safety with continuous use and risk of relapse upon discontinuation. The biologic agent dupilumab is the first systemic treatment approved for moderate-to-severe AD in children as young as 6 months. Long-term safety and efficacy data in this patient population are needed to inform continuous AD management. The purpose of this work was to determine the long-term safety and efficacy of dupilumab treatment up to 1 year in an open-label extension (OLE) study [LIBERTY AD PED-OLE (NCT02612454)] in children aged 6 months to 5 years with moderate-to-severe AD who previously participated in the 16-week, double-blind, phase 3 LIBERTY AD PRESCHOOL trial (NCT03346434 part B; parent study) and were subsequently enrolled in PED-OLE. In PED-OLE, patients received dupilumab every 4 weeks according to a weight-tiered regimen (body weight ≥ 5 kg to < 15 kg: 200 mg; ≥ 15 kg to < 30 kg: 300 mg). Data for 142 patients were analyzed, 60 of whom had completed the 52-week visit at time of database lock. Mean age at baseline was 4.1 y [SD, 1.13; range, 1.0-5.9 years]. A majority (78.2%) of patients reported ≥ 1 treatment-emergent adverse event (TEAE), most of which were mild or moderate and transient. The most frequently reported TEAEs were nasopharyngitis (19.7%), cough (15.5%), and pyrexia (14.1%). One TEAE led to treatment discontinuation (severe urticaria, which resolved in 1 day). By week 52, 36.2% of patients had achieved an Investigator's Global Assessment score of 0/1 (clear/almost clear skin), and 96.6%, 79.3%, and 58.6% had at least 50%, 75%, or 90% improvement, respectively, in Eczema Area and Severity Index scores. Consistent with results seen in adults, adolescents, and older children (aged 6-11 years), treatment with dupilumab for up to 1 year in children aged 6 months to 5 years with inadequately controlled moderate-to-severe AD demonstrated an acceptable long-term safety profile and sustained efficacy. These results support the long-term continuous use of dupilumab in this patient population. ClinicalTrials.gov Identifiers: NCT02612454 and NCT03346434 (part B).