Journal of Pediatric Pharmacology and Therapeutics最新文献

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Chemical Stability of Atomoxetine Hydrochloride and Fluoxetine Hydrochloride Oral Anhydrous Extemporaneously Compounded Suspensions. 盐酸阿托莫西汀和盐酸氟西汀口服无水临时复合混悬液的化学稳定性。
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-00010
Kendice Ip, Courtaney Davis, Christine Vu, Yashoda V Pramar, Maria Carvalho, Daniel Banov
{"title":"Chemical Stability of Atomoxetine Hydrochloride and Fluoxetine Hydrochloride Oral Anhydrous Extemporaneously Compounded Suspensions.","authors":"Kendice Ip, Courtaney Davis, Christine Vu, Yashoda V Pramar, Maria Carvalho, Daniel Banov","doi":"10.5863/JPPT-25-00010","DOIUrl":"https://doi.org/10.5863/JPPT-25-00010","url":null,"abstract":"<p><strong>Objective: </strong>Atomoxetine is indicated for the treatment of attention-deficit/hyperactivity disorder, and fluoxetine is commonly used for treating depression. Variable dosing per body weight is required in children, and the commercially available capsules do not offer the required flexibility and potential for customization. The objective of this study was to evaluate the physicochemical stability of atomoxetine hydrochloride and fluoxetine hydrochloride oral anhydrous extemporaneously compounded suspensions.</p><p><strong>Methods: </strong>An anhydrous suspension of atomoxetine hydrochloride was prepared from commercially available capsules at a 4-mg/mL concentration, and an anhydrous suspension of fluoxetine hydrochloride was prepared from pure drug powder at a concentration of 10 mg/mL. The extemporaneously compounded suspensions were prepared according to standardized formulas and methods of preparation. Samples were stored at refrigerated and controlled room temperatures for 6 months. The physicochemical properties of the samples were analyzed and characterized on days 0, 14, 30, 60, 90, and 180. A robust stability-indicating ultra-performance liquid chromatography assay method was validated and employed.</p><p><strong>Results: </strong>The samples remained consistent in appearance and color, with atomoxetine forming a pale-yellow suspension and fluoxetine yielding an off-white to faint beige suspension. Both anhydrous suspensions remained within the ±10% potency specification. The percent concentration relative to labelled concentration (potency) was at or above 96.5% for atomoxetine and at or above 96.8% for fluoxetine.</p><p><strong>Conclusion: </strong>Beyond-use dates of 6 months at both refrigerated and room temperature conditions have been established for the atomoxetine 4-mg/mL anhydrous suspension and the fluoxetine 10-mg/mL anhydrous suspension.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"598-607"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657040/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149184945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Use of Topical Analgesics in Neonates. 新生儿局部镇痛药的使用。
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-26-00103
Susan Bedwell
{"title":"Use of Topical Analgesics in Neonates.","authors":"Susan Bedwell","doi":"10.5863/JPPT-26-00103","DOIUrl":"https://doi.org/10.5863/JPPT-26-00103","url":null,"abstract":"","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"715-718"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657066/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149185059","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Off-Label Use of Medicines in Pediatrics: Necessary and Not "Off-Knowledge". 儿科药物说明书外使用:必要而非“知识外”。
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-01217
Constance Linardos, Ellie Whitney, Haley Schluterman, Lucy Eletel, Talia Thomas, Gregory L Kearns
{"title":"Off-Label Use of Medicines in Pediatrics: Necessary and Not \"Off-Knowledge\".","authors":"Constance Linardos, Ellie Whitney, Haley Schluterman, Lucy Eletel, Talia Thomas, Gregory L Kearns","doi":"10.5863/JPPT-25-01217","DOIUrl":"https://doi.org/10.5863/JPPT-25-01217","url":null,"abstract":"","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"705-710"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657061/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149185016","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Physicians' and Pharmacists' Awareness, Exposure, and Perception Regarding Predatory Solicitations at a Mother-Child Teaching Hospital. 母婴教学医院医生和药剂师对掠夺性招揽的认识、暴露和感知
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-00145
Juliette Mutin, Michel Lallier, Philippe Dodin, Jean-François Bussières
{"title":"Physicians' and Pharmacists' Awareness, Exposure, and Perception Regarding Predatory Solicitations at a Mother-Child Teaching Hospital.","authors":"Juliette Mutin, Michel Lallier, Philippe Dodin, Jean-François Bussières","doi":"10.5863/JPPT-25-00145","DOIUrl":"https://doi.org/10.5863/JPPT-25-00145","url":null,"abstract":"<p><strong>Objective: </strong>Predatory journals and conferences frequently target researchers through unsolicited emails, exploiting the pressure to publish by offering rapid dissemination at the expense of scientific rigor and ethical standards.</p><p><strong>Methods: </strong>This descriptive cross-sectional study targeted all members of the hospital's Council of Physicians, Dentists, and Pharmacists, including residents in these fields. A 24-item questionnaire was developed and distributed electronically.</p><p><strong>Results: </strong>A total of 140 physicians and pharmacists participated in the study. No responses were received from dentists. A majority, 93% (130/140), reported having presented posters at conferences and 91% (127/140) reported having published scientific articles. Overall, 59% (82/140) of the respondents reported being aware of the phenomenon of predatory solicitations, but only 14% (20/140) were familiar with Beall's list of predatory journals, and 16% (23/140) had used tools to verify the integrity or legitimacy of a journal. Two-thirds of respondents (94/140) reported that they had received predatory solicitations by email. Only 1% (2/140) reported presenting a poster at a conference, later identified as predatory, while 6% (9/140) reported that they had published in a predatory journal.</p><p><strong>Conclusions: </strong>Physicians and pharmacists at the study hospital were exposed to predatory solicitations. The results underscore the urgent need for targeted education and awareness initiatives to safeguard scientific integrity within the pediatric community.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"659-665"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657041/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149184950","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Management of Bupropion Toxicity With Intravenous Lipid Emulsion Therapy in a Pediatric Patient: A Case Report. 静脉脂质乳剂治疗儿童安非他酮毒性的处理:1例报告。
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-00131
Morgan Bonincontri, Bradley Snyder, Marcelo Egea, Bernardo Flasterstein, Brittany Allison
{"title":"Management of Bupropion Toxicity With Intravenous Lipid Emulsion Therapy in a Pediatric Patient: A Case Report.","authors":"Morgan Bonincontri, Bradley Snyder, Marcelo Egea, Bernardo Flasterstein, Brittany Allison","doi":"10.5863/JPPT-25-00131","DOIUrl":"https://doi.org/10.5863/JPPT-25-00131","url":null,"abstract":"<p><p>This case report involves a 2-year-old female with no past medical history who presented after ingestion of an unknown amount of 300 mg bupropion extended-release tablets. Shortly after ingestion, the mother mechanically induced vomiting, which recovered only small pieces of the tablets. The patient remained asymptomatic for a short period, then she began vomiting and had several tonic-clonic seizures, leading to emergency medical service (EMS) being called and ultimately, intubation for airway protection. On arrival at the pediatric intensive care unit (estimated 6 hours post ingestion), the patient was unresponsive with fixed and dilated pupils and no cough, gag, or respiratory effort. An electrocardiogram revealed that the patient had a first-degree heart block with a prolonged QTc interval. The patient remained unresponsive while off sedation with no effort to breathe over the ventilator. A bolus of intravenous lipid emulsion (ILE) 1.5 mL/kg followed by a continuous infusion of 0.5 mL/kg/min was administered (estimated 8 hours post ingestion). A few minutes following the start of the infusion, the patient began to spontaneously breathe over the ventilator. The patient was successfully extubated within 36 hours after ILE, and within 72 hours the patient had recovered to baseline and discharged home. This case displays successful use of ILE for bupropion toxicity in a young child. More studies are needed to fully understand the benefits and potential risks of the use of ILE in this population.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"671-674"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657068/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149184999","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Critical Role of Pharmacists in Addressing the Pediatric Mental Health Crisis: A Pediatric Pharmacy Association Position Paper. 药剂师在解决儿童心理健康危机中的关键作用:儿科药学协会立场文件。
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-00102
Sarah E Kubes, Sylvia Stoffella, Kymberly Buzzeo, Angela Chen, Brian Gallaher, Alán Linh Nguyen, Susan M Butler
{"title":"The Critical Role of Pharmacists in Addressing the Pediatric Mental Health Crisis: A Pediatric Pharmacy Association Position Paper.","authors":"Sarah E Kubes, Sylvia Stoffella, Kymberly Buzzeo, Angela Chen, Brian Gallaher, Alán Linh Nguyen, Susan M Butler","doi":"10.5863/JPPT-25-00102","DOIUrl":"https://doi.org/10.5863/JPPT-25-00102","url":null,"abstract":"<p><p>The significant rise in rates of depression and anxiety in children and adolescents coupled with underdiagnosis and undertreatment poses a serious health threat to the pediatric population. The escalating pediatric mental health crisis has been associated with rising suicide rates and attempts, increased deaths due to drug overdose, higher health care costs, and limited access to adequate care. A shortage of mental health resources and specialized providers in both community and institutional settings further exacerbates these challenges. This paucity of care highlights the need for additional support, and pharmacists, as accessible health professionals, can help bridge the gap in pediatric mental health care. Pharmacists play a vital role in pediatric mental health by advocating for and performing mental health screenings for pediatric patients presenting with acute psychological and behavioral health issues across various care settings, including emergency departments, inpatient units and outpatient pharmacies. Additionally, pharmacists enhance the efficacy and safety of psychotropic therapies in children with diagnosed mental health conditions by minimizing drug interactions, optimizing dosing, monitoring for adverse effects, and providing patient and family education. This article serves as a Position Statement of the Pediatric Pharmacy Association (PPA), which strongly advocates for the inclusion of pharmacists as key members of multidisciplinary teams supporting pediatric patients with mental health challenges, emphasizing their critical role in improving patient outcomes. Additionally, PPA advocates for routine mental health screenings for all pediatric patients and recommends utilization of referrals and standardized workflows for managing pediatric mental health conditions and substance use disorders.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"689-704"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657063/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149185069","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Safety of Intravenous Ibuprofen Administration in the Pediatric Emergency Department. 儿科急诊科静脉注射布洛芬的安全性
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-00160
Silvia Cerezo Corredera, Mikel Olabarri Garcia, Cristina Lillo Díaz, Javier Benito, Santiago Mintegi, Silvia Garcia Gonzalez
{"title":"Safety of Intravenous Ibuprofen Administration in the Pediatric Emergency Department.","authors":"Silvia Cerezo Corredera, Mikel Olabarri Garcia, Cristina Lillo Díaz, Javier Benito, Santiago Mintegi, Silvia Garcia Gonzalez","doi":"10.5863/JPPT-25-00160","DOIUrl":"https://doi.org/10.5863/JPPT-25-00160","url":null,"abstract":"<p><strong>Objective: </strong>Intravenous (IV) ibuprofen has recently been approved for treating moderate acute pain and fever, but evidence in pediatric use remains limited. This study aimed to evaluate the short-term safety of IV ibuprofen in children treated in a pediatric emergency department (PED).</p><p><strong>Methods: </strong>A prospective observational study was conducted including children aged 6 months to younger than 14 years who received IV ibuprofen between January 2021 and September 2023 in a PED of a tertiary hospital. IV ibuprofen was administered for moderate acute pain or fever when oral administration was not feasible due to vomiting or the need for fasting before a potential surgical intervention. Physicians documented patient history, previous analgesia, reasons for consultation and IV ibuprofen administration, vital signs, pain scores (0-10) before and 10 minutes after administration, and any adverse events (AEs).</p><p><strong>Results: </strong>We included 287 patients, with a median age of 9 years (IQR, 6-11). No serious AEs occurred, and no hospital admissions were required due to IV ibuprofen. Mild AEs occurred in 15 patients (5.2%), most commonly transient pain or itching at the infusion site. One patient developed a mild skin rash leading to treatment discontinuation, with complete recovery. Pain scores showed a median reduction of 3 points (IQR, 2-4).</p><p><strong>Conclusions: </strong>Short-term use of IV ibuprofen in the PED was associated with an acceptable safety profile and clinically relevant pain reduction. However, further research is needed to confirm these findings and to evaluate safety with repeated dosing or longer duration of use.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"653-658"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657053/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149185012","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Qualitative Study of Parent and Health Care Provider Perspectives on Polypharmacy in Children With Medical Complexity. 医疗复杂性患儿家长与医护人员对多药治疗观点的质性研究
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-00152
Abigail Musial, Joanna E Thomson, Allison M Wiedeman, Lisa M Vaughn, Katherine A Auger, Andrea Blum, Angela M Statile, Sarah Stiehl, Amanda C Schondelmeyer
{"title":"A Qualitative Study of Parent and Health Care Provider Perspectives on Polypharmacy in Children With Medical Complexity.","authors":"Abigail Musial, Joanna E Thomson, Allison M Wiedeman, Lisa M Vaughn, Katherine A Auger, Andrea Blum, Angela M Statile, Sarah Stiehl, Amanda C Schondelmeyer","doi":"10.5863/JPPT-25-00152","DOIUrl":"https://doi.org/10.5863/JPPT-25-00152","url":null,"abstract":"<p><strong>Objective: </strong>To understand contributing factors and possible solutions to polypharmacy in children with medical complexity (CMC) from the perspective of family caregivers and clinicians.</p><p><strong>Methods: </strong>Using group-level assessment (GLA), a structured large-group qualitative method, family caregiver and clinician participants individually generated responses to semi-structured prompts, analyzed responses in small groups, and reached group consensus on themes. Investigators merged themes across GLA sessions. To substantiate GLA findings, we conducted semi-structured interviews with CMC caregivers. Using a combined inductive/deductive approach based on GLA themes, we integrated interview findings into final themes.</p><p><strong>Results: </strong>We conducted 4 GLA sessions with CMC family caregivers and clinicians (n = 37) and 4 semi-structured interviews with family caregivers. Final themes highlighted how clinicians and families prioritize possible benefits to medications, leading to a willingness to start medications, often without a clear plan to reassess, and a reluctance to stop medications related to fears of disrupting the child's state of health. Challenges communicating between multiple subspecialists and inadequate partnership with CMC caregivers potentiate challenges of polypharmacy.</p><p><strong>Conclusions: </strong>Participants emphasized the cycle of prescribing cascades and hesitancy to deprescribe as contributing to polypharmacy. Findings related to communication challenges and caregiver partnership imply standard medication management processes incorporating family input are warranted.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"644-652"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657057/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149184920","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Pediatric Pharmacotherapy: Why the Fundamentals (Still) Matter. 儿童药物治疗:为什么基础(仍然)很重要。
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-26-00067
Susan M Abdel-Rahman
{"title":"Pediatric Pharmacotherapy: Why the Fundamentals (Still) Matter.","authors":"Susan M Abdel-Rahman","doi":"10.5863/JPPT-26-00067","DOIUrl":"https://doi.org/10.5863/JPPT-26-00067","url":null,"abstract":"<p><p>Pediatric pharmacotherapy has evolved to encompass a broad array of clinical, regulatory, research, and drug development activities. Yet nearly everything done today builds on foundational elements of the discipline. Through examples from a career in pediatric pharmacology and therapeutics, this article challenges readers to 1) consider how often they inspect the foundations that underlie their career activities and 2) reflect on how they might address fundamental deficiencies that they observe when faced with the opportunity. This article recaps the 2026 Sumner J. Yaffe Lifetime Achievement Award lecture presented at the Pediatric Pharmacy Association annual meeting in Arlington, Virginia on April 9, 2026.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"590-597"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657065/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149185014","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Trends and Outcomes of Voxelotor Treatment in Pediatric Patients With Sickle Cell Disease: A Retrospective Study. 儿童镰状细胞病患者体素治疗的趋势和结果:一项回顾性研究
Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2026-10-01 Epub Date: 2026-10-12 DOI: 10.5863/JPPT-25-00166
Grace Kalmus, Carly Nesbitt, Jason Payne, Beatrice Gee, Amy Tang
{"title":"Trends and Outcomes of Voxelotor Treatment in Pediatric Patients With Sickle Cell Disease: A Retrospective Study.","authors":"Grace Kalmus, Carly Nesbitt, Jason Payne, Beatrice Gee, Amy Tang","doi":"10.5863/JPPT-25-00166","DOIUrl":"https://doi.org/10.5863/JPPT-25-00166","url":null,"abstract":"<p><strong>Objective: </strong>Sickle cell disease (SCD) is characterized by hemoglobin S polymerization, leading to anemia, pain, and organ damage. Voxelotor is a therapy for SCD that was withdrawn from worldwide markets in September 2024. We evaluated trends in voxelotor use and its impact on pediatric SCD patients before withdrawal.</p><p><strong>Methods: </strong>A retrospective study was conducted using patient records from 4 Children's Healthcare of Atlanta (CHOA) hospitals. Data on patient demographics, length of time on voxelotor, clinical markers, hospital usage, side effects, and discontinuation reasons were analyzed.</p><p><strong>Results: </strong>Two hundred forty-five patients were taking voxelotor between 2019 and 2024 outside of clinical trials. Of these, 188 (77%) patients were on voxelotor for at least 6 consecutive months, with an average of 25.0 months on treatment. The main reason for discontinuation was due to a side effect (47%) for patients on treatment for less than 6 months and market withdrawal (69%) for those taking 6 months or more. On average, patients had a significant increase in mean hemoglobin (0.5 g/dL, p < 0.001), a decrease in hemolysis markers, and a mean decrease in annualized emergency department visits. At the time of market withdrawal, 123 patients were actively taking voxelotor.</p><p><strong>Conclusions: </strong>Overall, patients had a positive hematologic and clinical response to voxelotor, with the majority remaining on treatment for at least 6 consecutive months. Despite market withdrawal due to safety and efficacy concerns, a subset of patients derived clinical benefits from voxelotor, highlighting the need for continued development of effective therapeutics for SCD.</p>","PeriodicalId":37484,"journal":{"name":"Journal of Pediatric Pharmacology and Therapeutics","volume":"31 5","pages":"608-616"},"PeriodicalIF":0.0,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13657039/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149185027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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