Pediatric Drugs最新文献

{"title":"Novel Agents on the Therapeutic Horizon For Paediatric Inflammatory Bowel Diseases: An Analysis of Clinical Trials Registries.","authors":"Dhruv Gupte, Nidhi Rashmikant Suthar, John K MacDonald, Jessica Le, Ruben J Colman, Brian G Feagan, Camilyn Cheng, Jurij Hanzel, Christopher Ma, Vipul Jairath, Eileen Crowley","doi":"10.1007/s40272-026-00766-1","DOIUrl":"10.1007/s40272-026-00766-1","url":null,"abstract":"<p><strong>Background and aims: </strong>Despite considerable advancements in the therapeutic landscape of inflammatory bowel disease (IBD) for adults, long delays exist for paediatric IBD (pIBD) approval, with a median delay of >7 years following adult approval. Our aim is to summarise the landscape of pIBD clinical trials through a review of trial registries.</p><p><strong>Methods: </strong>We conducted a cross-sectional review of ClinicalTrials.gov and ClinicalTrialsRegister.eu to identify investigational and approved therapeutic agents for the treatment of pIBD. All interventional studies involving pIBD (aged <18 years) from database inception to July 2, 2026, were considered for inclusion.</p><p><strong>Results: </strong>Of 3941 records screened, 123 completed trials (77 randomised controlled trials [RCTs] and 46 non-RCTs) and 90 active trials (55 RCTs and 35 non-RCTs) met the inclusion criteria. A majority of completed trials (71%) recruited mixed adult and paediatric populations, whereas nearly half of the active trials recruited exclusively paediatric patients, with most studies enrolling participants aged 2-17 years. Biologics were assessed in 81 trials (42 completed and 39 active), including the approved agents (n = 39), infliximab, adalimumab, golimumab, and ustekinumab, and investigational therapies (n = 42) such as vedolizumab, guselkumab, mirikizumab, risankizumab, duvakitug, afimkibart, and tulisokibart. Nine completed and 11 active trials assess small molecule therapies, including tofacitinib, upadacitinib, filgotinib, etrasimod, ozanimod, and obefazimod.</p><p><strong>Conclusions: </strong>Efforts to expedite approval of new agents in pIBD are warranted to ensure timely access to effective medications. Consideration for novel trial designs alongside continued engagement with regulatory bodies, sponsors, and the academic pIBD community is essential to advance drug approvals for pIBD.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"547-561"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13624012/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148762664","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"AFREZZA^® (Insulin Human) Inhalation Powder: Pediatric First Approval.","authors":"Sheridan M Hoy","doi":"10.1007/s40272-026-00768-z","DOIUrl":"10.1007/s40272-026-00768-z","url":null,"abstract":"<p><p>AFREZZA^® (insulin human) inhalation powder is a drug-device combination product consisting of a dry powder formulation of rapid-acting recombinant human insulin adsorbed onto proprietary Technosphere^® carrier particles, and a breath-powered oral inhaler. The powder is aerosolized and delivered to the lungs by the patient inhaling through the device. AFREZZA^® (insulin human) inhalation powder has been developed by MannKind Corporation to improve glycemic control in patients with diabetes mellitus and received its first approval in the USA in 2014 for this indication in adult patients. More recently (29 May 2026), AFREZZA^® (insulin human) inhalation powder received its first pediatric approval in the USA to improve glycemic control in pediatric patients 6 years of age and older with diabetes mellitus. This article summarizes the milestones in the development of AFREZZA^® (insulin human) inhalation powder leading to this first pediatric approval for diabetes mellitus.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"575-580"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148795698","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Model-Informed Precision Dosing in Pediatric Patients: Current Software Tools and Bedside Guided Application.","authors":"Abdullah Aljutayli, Emna Gaies, Mohammed S Alasmari, Zekra Kamel Aljehani, Abrar Samman, Sara Abudahab, Manal Abouelkheir, Abdullah Alsultan","doi":"10.1007/s40272-026-00763-4","DOIUrl":"10.1007/s40272-026-00763-4","url":null,"abstract":"<p><strong>Background: </strong>Personalized dosing is particularly important in pediatric patients, as age-related physiological maturation, developmental changes, and patient-specific factors contribute to substantial pharmacokinetic variability. Standard dosing approaches often fail to adequately account for this heterogeneity and may lead to subtherapeutic or supratherapeutic drug concentrations. Model-informed precision dosing (MIPD) addresses these limitations by integrating individual patient data with population pharmacokinetic/pharmacodynamic models to optimize dosing at the individual level more accurately.</p><p><strong>Objective: </strong>This review aimed to (1) provide a structured overview of currently available bedside-compatible MIPD software applications for pediatric dosing, (2) systematically summarize published clinical studies evaluating their use in pediatric and neonatal populations, and (3) illustrate practical bedside application through a step-by-step tutorial.</p><p><strong>Methods: </strong>We identified MIPD software tools that incorporate Bayesian forecasting and at least one pediatric module through iterative literature searches using terms for MIPD and dosing software, and by developer verification. For eligible platforms, we reviewed PubMed-indexed clinical studies involving pediatric patients, summarizing key aspects including study design, population, drug, clinical decision support-related objectives, and findings. A representative tutorial was developed using NextDose for neonatal vancomycin dosing.</p><p><strong>Results: </strong>We identified 11 available MIPD software platforms, varying in deployment, model libraries, drug coverage, electronic health record integration, and regulatory status (e.g., CE-marked medical devices vs non-regulated clinical decision support). Review of 26 clinical studies enrolling more than 4000 pediatric and neonatal patients demonstrated that MIPD consistently outperformed conventional methods in predictive accuracy, precision, and pharmacokinetic target attainment, including significant improvements in target area under the curve attainment for key drugs like vancomycin. Benefits included a reduced sampling burden and faster therapeutic exposure, predominantly for antibiotics (vancomycin, tobramycin, gentamicin) and select chemotherapeutics (busulfan).</p><p><strong>Conclusions: </strong>Model-informed precision dosing software tools offer substantial potential to improve precision dosing in pediatrics, with robust evidence of superior target attainment and operational advantages. Gaps remain in prospective randomized trials, direct patient-centered outcome data, cost-effectiveness analyses, non-antibiotic drug coverage, and clinician training.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"447-464"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148796482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"21‑Valent Pneumococcal Conjugate Vaccine V116 (CAPVAXIVE^®): Pediatric First Approval.","authors":"Matt Shirley","doi":"10.1007/s40272-026-00764-3","DOIUrl":"10.1007/s40272-026-00764-3","url":null,"abstract":"<p><p>The 21‑valent pneumococcal conjugate vaccine (PCV) V116 (CAPVAXIVE^®), developed by Merck & Co., has been approved in the USA since June 2024 for active immunization for the prevention of pneumonia and invasive disease caused by Streptococcus pneumoniae in adults, with subsequent approvals in adults in the EU and a range of other countries. With the inclusion of eight S. pneumoniae serotypes that are not included in other currently licensed pneumococcal vaccines, V116 was designed to target residual pneumococcal disease in adults. Noting that some of the unique serotypes in V116 are responsible for significant pneumococcal disease in children and adolescents, the potential value of V116 in also complementing existing pediatric pneumococcal vaccination regimens has been explored. Supported by phase III clinical evaluation in children and adolescents with an increased risk of pneumococcal disease, in March 2026, V116 received its first pediatric approval, in the EU, for active immunization for the prevention of invasive disease and pneumonia caused by S. pneumoniae in children and adolescents 2 to < 18 years of age who previously completed a primary pediatric pneumococcal vaccination regimen. Subsequently, in June 2026, the US approval for V116 was extended to include use in individuals 2-17 years of age who are at increased risk for pneumococcal disease. This article summarizes the milestones in the development of V116 leading to these first pediatric approvals for active immunization for the prevention of invasive disease and pneumonia caused by S. pneumoniae.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"563-567"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148593002","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How Organ Manifestations Beyond the Bone May Guide Risk Stratification and Treatment Decisions in Chronic Nonbacterial Osteomyelitis.","authors":"Giulia Inguscio, Anja Schnabel, Eve Roberts, Christian M Hedrich","doi":"10.1007/s40272-026-00769-y","DOIUrl":"10.1007/s40272-026-00769-y","url":null,"abstract":"<p><p>Chronic nonbacterial osteomyelitis is an autoinflammatory bone disease characterized by pronounced dysregulation of proinflammatory and anti-inflammatory cytokine production and release. Through innate immune dysregulation, patients with chronic nonbacterial osteomyelitis develop bone inflammation, pathological bone remodeling, and associated pain. Notably, secondary activation of adaptive immune mechanisms may contribute to phenotypic variability and the onset of additional tissue and organ involvement (joints, skin, gut). Currently used treatments are empiric, targeting inflammation and/or osteoclasts. While deemed efficacious across clinical cohorts internationally, in the absence of randomized controlled trials, they lack regulatory approval. While chronic nonbacterial osteomyelitis limited to bones frequently responds to anti-inflammatory treatment with naproxen or, in more severe cases, tumor necrosis factor inhibitors and/or osteoclast inhibition with bisphosphonates, patients with additional symptoms may require additional/different treatments targeting effector T cells and associated cytokines (such as interleukin-17 blockers, Janus kinase inhibitors). Thus, changing molecular, cellular, and phenotypic patterns throughout the disease course offer an opportunity for personalized treatment and suggest a 'window of opportunity' preventing the onset of additional organ involvement. However, 'tools' to guide personalized treatment approaches are currently lacking, and treatment is guided by clinical features, following a trial-and-error approach. This article reviews the available literature, deciphering shared immune mechanisms between chronic nonbacterial osteomyelitis and associated diseases that may guide individualized care. It focuses on shared and disease-specific molecular and cellular patterns of chronic nonbacterial osteomyelitis and associated diseases, delivering arguments for disease stage and phenotype specific treatments, including cytokine blockers and synthetic small-molecule inhibitors.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"505-529"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13623998/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148977729","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incorporating Immunotherapies into the Evolving Standard of Care for Pediatric B-Cell Precursor Acute Lymphoblastic Leukemia.","authors":"Andrej Lissat, Erica Brivio, Janine Stutterheim, Friso Calkoen, C Michel Zwaan, Francisco Bautista","doi":"10.1007/s40272-026-00761-6","DOIUrl":"10.1007/s40272-026-00761-6","url":null,"abstract":"<p><p>Survival for pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL) exceeds 90% in high-income countries due to risk-adapted chemotherapy and measurable residual disease (MRD)-guided strategies. However, relapse remains a leading cause of death, and chemotherapy-related toxicities highlight the need for equally effective, less toxic approaches. B-cell-directed immunotherapies targeting CD19 and CD22 have emerged as transformative modalities. This review synthesizes clinical evidence for three major immunotherapeutic classes: CD19/CD3 T cell engagers (blinatumomab), CD22 antibody-drug conjugates (inotuzumab ozogamicin (InO)), and CD19-directed chimeric antigen receptor (CAR)-T cell therapies, evaluating their integration into frontline and relapsed treatment algorithms, safety, resistance mechanisms, and future directions. Randomized pediatric trials demonstrate that blinatumomab improves survival by reducing relapse and treatment-related mortality when incorporated into frontline and first-relapse therapy, primarily in consolidation. InO shows high induction response and MRD-negativity rates in relapsed disease but carries a risk of sinusoidal obstruction syndrome, particularly around hematopoietic stem cell transplantation (HSCT). CD19 CAR-T cell therapy induces MRD-negative remissions in 80-90% of heavily pretreated patients, with durable survival in 40-50% without mandatory HSCT consolidation. Emerging agents and combination strategies aim to overcome antigen escape and improve durability. Challenges remain regarding central nervous system (CNS) disease control, long-term immune effects, sequencing, regulatory disparities, and global access. Immunotherapies are reshaping pediatric BCP-ALL treatment, enabling chemotherapy reduction while maintaining or improving cure rates. Strategic integration and equitable global access are vital to achieving universal cures with reduced toxicity. Post-immunotherapy relapses may exhibit distinct disease characteristics requiring novel treatment approaches.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"465-486"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148562635","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and Efficacy of Oral Ferric Maltol and Ferrous Sulfate for Iron Deficiency Anemia in Infants, Children and Adolescents: A Multicenter Randomized Trial.","authors":"David Gass, Andrew Freiberg, Marcus K H Auth, Richard K Russell","doi":"10.1007/s40272-026-00753-6","DOIUrl":"10.1007/s40272-026-00753-6","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This phase 3, multicenter, randomized, open-label study (NCT05126901) compared oral ferric maltol with ferrous sulfate, across 23 sites in the US and the UK, evaluating the safety, efficacy, pharmacokinetics, tolerability, and palatability of age-specific doses in infants, children and, adolescents with iron deficiency anemia (IDA).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Patient and methods: &lt;/strong&gt;In a 12-week study, a total of 61 children/adolescents aged 2-17 years (median age 14 years; 45 female patients [73.8%] and 16 male patients [26.2%]) with IDA were randomized to ferric maltol or ferrous sulfate, plus 4 infants aged 1 month to &lt; 2 years (median age 12 months; 3 female patients [75%] and 1 male patient [25%]) were assigned to ferric maltol (open label arm). Ferric maltol dosing was age-dependent (0.6 mg/kg/dose twice a day [BID] for infants; 15 mg BID for 2-11 years; 30 mg BID for 12-17 years), while ferrous sulfate dosing was weight-based (3 mg/kg BID). Safety, efficacy (Hemoglobin [Hb], serum iron, transferrin saturation [TSAT], ferritin), PK, and palatability were assessed. Coprimary end points were Hb changes from baseline and safety, including gastrointestinal tolerability at week 12.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A prespecified interim analysis of the primary end point (change in Hb from baseline to week 12) was conducted after 32 ferric maltol patients completed treatment. As the change was statistically significant (p &lt; 0.0001), recruitment was stopped. The interim analysis used a Pocock spending function with a 3.45% two-sided confidence interval (CI). Both the randomized ferric maltol and ferrous sulfate groups showed meaningful increases in Hb versus baseline (descriptive increases of 1.25 and 1.15 g/dL, respectively). Post hoc analyses confirmed that these changes from baseline were statistically significant (p &lt; 0.0001). Ferric maltol increased Hb by a mean of 1.77 g/dL in the assigned infant group over 12 weeks. Similarly, by week 12, both treatments increased serum iron and TSAT (p &lt; 0.02), ferric maltol showed higher peak iron, and ferritin rose more with ferrous sulfate. Adverse events were mild/moderate and similar in frequency between the two treatment groups (29% ferric maltol, 30% ferrous sulfate), and adverse reactions deemed as at least possibly related to treatment were more common with ferrous sulfate. Among children aged 6-17 years, ferric maltol was rated as tasting OK, Good, or Very Good more frequently than ferrous sulfate at two time points. On day 1, 79.3% of patients receiving ferric maltol reported OK, Good, or Very Good taste, compared with 50.0% receiving ferrous sulfate (OR 0.26, 95% CI 0.07-0.97; p = 0.027). By day 28, this difference persisted (75.0% versus 45.8%), favoring ferric maltol (OR 0.28, 95% CI 0.07-1.05; p = 0.046). The significant results for taste were replicated for smell; ferric maltol had significantly better smell ratings than ferrous sulfate on days 1 and 28 (p ≤ 0.003).&lt;","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"531-545"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148456312","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ocrelizumab: Pediatric First Approval.","authors":"Sheridan M Hoy","doi":"10.1007/s40272-026-00765-2","DOIUrl":"10.1007/s40272-026-00765-2","url":null,"abstract":"<p><p>Ocrelizumab, a recombinant humanized IgG1 monoclonal antibody directed against CD20-expressing B-cells, has been developed by Genentech, Inc. for the treatment of multiple sclerosis (MS). Both intravenous infusion and subcutaneous injection formulations of ocrelizumab are approved in the EU and the USA for the treatment of adults with relapsing or primary progressive forms of MS. In May 2026, the intravenous infusion formulation of ocrelizumab (OCREVUS^®) was approved in the USA for the treatment of relapsing-remitting MS (RRMS) in pediatric patients 10 years of age and older who weigh 25 kg or more. This article summarizes the milestones in the development of ocrelizumab leading to this first pediatric approval for RRMS.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"569-574"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148685476","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current Management and Emerging Therapies for Pediatric Vitiligo: A Narrative Review.","authors":"Jade Howard, William Fitzmaurice, Aleena Boby, Benjamin Levin, Nanette Silverberg","doi":"10.1007/s40272-026-00767-0","DOIUrl":"10.1007/s40272-026-00767-0","url":null,"abstract":"<p><p>Pediatric vitiligo (PV) accounts for approximately 30% of all vitiligo cases. PV is often associated with unique treatment challenges, including access barriers, prolonged treatment duration, and significant psychosocial impacts on patients and their families. Standard management typically includes topical therapies aimed at clearance of inflammatory cell infiltrates that attack melanocytes and phototherapy aimed at repigmentation through stimulation of pigment cell activity and movement into the depigmented regions. However, some patients may require more intensive interventions, including systemic therapy, surgical procedures, or depigmentation therapy when repigmentation is not likely or desired. The approval of topical 1.5% ruxolitinib cream for individuals aged 12 years and older, following the demonstration of the drug's efficacy and safety, has prompted increased interest in emerging therapies for vitiligo. This article explores current topical treatments, phototherapy, and novel therapeutic approaches specifically in the pediatric and adolescent populations focusing on available published data. Additionally, we highlight the psychological impact of vitiligo on young patients and their families and emphasize practical considerations when managing this condition in this population.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"487-504"},"PeriodicalIF":3.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149025811","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness and Safety of IL-17A Inhibitors in Pediatric Psoriasis: A Real-World, Multicenter, International Study.","authors":"Vito Di Lernia, Bertille Bonniaud, Eve Puzenat, Christine Bodemer, Morgane Seyler, Marieke M B Seyger, Mathilde Tardieu, Helena Iznardo, Francesca Satolli, Paula C Luna, Olivier Carpentier, Ouafa Hocar, Tiago Torres, Iria Neri, Luca Stingeni, Cristina Bertoli, Alain Beauchet, Emmanuel Mahé","doi":"10.1007/s40272-026-00762-5","DOIUrl":"10.1007/s40272-026-00762-5","url":null,"abstract":"<p><strong>Background: </strong>Secukinumab and ixekizumab are interleukin (IL)-17A inhibitors approved for the treatment of moderate-to-severe plaque psoriasis in children and adolescents. While their efficacy has been established in clinical trials, real-world data on long-term treatment persistence and the representativeness of trial populations are limited.</p><p><strong>Objective: </strong>The aims were to evaluate the 24-month drug survival, effectiveness, and safety of secukinumab and ixekizumab in a real-world pediatric cohort, and to assess the hypothetical eligibility of these patients for pivotal phase 3, randomized controlled trials (RCTs).</p><p><strong>Methods: </strong>This multicenter, retrospective study analyzed pediatric patients (< 18 years) with moderate-to-severe plaque psoriasis treated with secukinumab or ixekizumab across 38 international referral centers. The primary endpoint was the 24-month treatment continuation rate. Secondary endpoints included clinical effectiveness (Physician Global Assessment [PGA] 0/1; Psoriasis Area and Severity Index [PASI] 75, 90, and 100 response rates at months 3, 6, and 12) and safety. Additionally, we assessed the eligibility of this real-world cohort for the pivotal phase 3 RCTs of these treatments.</p><p><strong>Results: </strong>A total of 152 patients (mean age 12.9 years; 53.9% female) received 160 treatments (139 secukinumab, 21 ixekizumab). The 24-month continuation rate was significantly higher for secukinumab compared to ixekizumab (p = 0.036). No significant differences in drug survival were observed based on weight status (p = 0.65) or prior biologic exposure (p = 0.10). PASI 100 was achieved at month 12 by 61.5% of patients on secukinumab and 33.3% on ixekizumab. Higher response rates were observed in bio-naïve patients and those with normal weight. The most commonly reported adverse events (AEs) were eczema and candidiasis in the secukinumab group, and injection site pain in the ixekizumab group, with no serious AEs reported. Only a limited proportion of this real-life cohort would have met the inclusion criteria for pivotal RCTs, primarily due to lower baseline disease severity or specific clinical conditions.</p><p><strong>Conclusion: </strong>Our study confirms the high effectiveness and favorable safety profile of IL-17A inhibitors in pediatric psoriasis. The low rate of hypothetical RCT eligibility highlights a gap between trial populations and clinical practice, underscoring the vital role of daily-practice data in guiding treatment.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148592991","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}