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1.
Pediatr Blood Cancer ; 70(4): e30182, 2023 04.
Artigo em Inglês | MEDLINE | ID: mdl-36715441

RESUMO

BACKGROUND: Mitogen-activated protein kinase enzyme (MEK) inhibitors are used in the treatment of pediatric patients with neurofibromatosis, low grade glioma, and astrocytoma, and may demonstrate a unique side effect profile in this population. Inhibition of MEK has been shown to decrease interleukin (IL)-6 production, a proinflammatory cytokine. The inhibition of IL-6 and other proinflammatory cytokines is thought to decrease muscle wasting and may contribute to weight gain. However, there is limited information on the association of MEK inhibition and weight gain in children and adolescents. This study aimed to characterize and define the incidence of significant weight gain associated with MEK inhibitors in pediatric patients. METHODS: This was a retrospective chart review conducted at a tertiary pediatric hospital. Children 1-18.99 years old were included if they started a MEK inhibitor from July 1, 2013-October 31, 2021, and continued therapy for at least 6 months. Significant weight gain was defined as ≥5% increase in patient's weight-for-age percentile. RESULTS: Sixty-seven patients were included in the analysis. Sixty-two received trametinib and 5 received selumetinib. An increase in weight-for-age percentile ≥5% was seen in 60% of patients receiving selumetinib and 56% on trametinib. The Dunnett's multiple comparisons test revealed a difference in weight-for-age percentile from baseline to end of data collection (p = .0173). Patients who were obese at baseline were more likely to lose weight during treatment, while underweight patients increased in weight-for-age percentiles. CONCLUSIONS: Weight gain may be a notable side effect associated with the use of MEK inhibitors in pediatric patients.


Assuntos
Glioma , Adolescente , Adulto , Criança , Pré-Escolar , Humanos , Lactente , Adulto Jovem , Citocinas , Glioma/tratamento farmacológico , Incidência , Quinases de Proteína Quinase Ativadas por Mitógeno , Inibidores de Proteínas Quinases/efeitos adversos , Inibidores de Proteínas Quinases/uso terapêutico , Estudos Retrospectivos , Aumento de Peso
2.
Pediatr Blood Cancer ; 70(3): e30190, 2023 03.
Artigo em Inglês | MEDLINE | ID: mdl-36602034

RESUMO

The event-free survival of pediatric low-grade gliomas is poor, and patients often require multiple treatment strategies. While MEK and RAF inhibitors are efficacious in early-phase trials, not all patients respond, and many experience progression following completion of therapy. Evaluating combination therapies that may enhance efficacy or prolong disease stabilization is warranted. We report our institutional experience using concurrent trametinib and lenalidomide in the treatment of primary pediatric central and peripheral nervous system tumors. Two of four patients using this combination therapy experienced severe thromboembolic events, necessitating discontinuation of therapy. This combination requires further investigation, and we urge caution if used.


Assuntos
Piridonas , Pirimidinonas , Humanos , Criança , Lenalidomida/efeitos adversos , Piridonas/efeitos adversos , Pirimidinonas/efeitos adversos , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Proteínas Proto-Oncogênicas B-raf
3.
J Clin Pharm Ther ; 47(7): 1081-1087, 2022 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-35304755

RESUMO

WHAT IS KNOWN AND OBJECTIVE: The C5 inhibitor eculizumab is the standard of care for treatment of atypical haemolytic uraemic syndrome (aHUS). Ravulizumab, a next-generation C5 inhibitor, was engineered to have a longer terminal half-life than eculizumab. We describe practical benefits of the advanced ravulizumab 100 mg/mL formulation. COMMENT: Use of ravulizumab results in fewer maintenance infusions per year (25%-50%) compared with eculizumab. Maintenance infusion time of ravulizumab 100 mg/mL is 2-4 times shorter than ravulizumab 10 mg/mL in all weight cohorts and approximately half that of eculizumab for patients weighing <40 kg. Ravulizumab 100 mg/mL requires fewer vials annually than eculizumab in most weight cohorts. WHAT IS NEW AND CONCLUSION: With ravulizumab 100 mg/mL, patients and caregivers experience fewer infusions per year and decreased annual infusion times, improving infusion experience. Infusion centres can expect corresponding decreases in resource utilization.


Assuntos
Síndrome Hemolítico-Urêmica Atípica , Anticorpos Monoclonais Humanizados , Síndrome Hemolítico-Urêmica Atípica/tratamento farmacológico , Cuidadores , Humanos
4.
J Pediatr Hematol Oncol ; 43(6): e897-e899, 2021 08 01.
Artigo em Inglês | MEDLINE | ID: mdl-33273413

RESUMO

BACKGROUND: Patients with malignant diseases are at high risk for refractory Clostridioides difficile infections (CDI). Fecal microbiota transplantation (FMT) restores the gastrointestinal microbiome and may be an effective treatment for patients who fail pharmacotherapy. However, FMT is not commonly used in the oncology population because of risk for donor-derived infection. OBSERVATIONS: The authors report successful use of FMT in a pediatric patient with refractory CDI actively receiving chemotherapy. The patient's symptoms improved 1 day following FMT. He did not experience infectious complications or other adverse effects. CONCLUSIONS: FMT may be a feasible option for treatment of refractory CDI in pediatric oncology patients.


Assuntos
Clostridioides difficile/fisiologia , Infecções por Clostridium/complicações , Colite/microbiologia , Colite/terapia , Transplante de Microbiota Fecal , Adolescente , Infecções por Clostridium/microbiologia , Infecções por Clostridium/terapia , Microbioma Gastrointestinal , Humanos , Masculino
5.
Pharmacotherapy ; 39(6): 645-664, 2019 06.
Artigo em Inglês | MEDLINE | ID: mdl-30793794

RESUMO

Neurodevelopmental disorders (NDDs), a group of disorders affecting ~1-2% of the general population, are caused by changes in brain development that result in behavioral and cognitive alterations, sensory and motor changes, and speech and language deficits. Neurodevelopmental disorders encompass a heterogeneous group of disorders including, but not limited to, Smith-Magenis syndrome, Lesch-Nyhan disease, cri du chat syndrome, Prader-Willi syndrome, pervasive developmental disorders, fragile X syndrome, Rett syndrome, Cornelia de Lange syndrome, and Down syndrome. Self-injurious behaviors (SIBs) are common in children with NDDs; depending on the specific NDD, the incidence of SIBs is nearly 100%. The management of SIBs in this population is complex, and little high-quality data exist to guide a consistent approach to therapy. However, managing SIBs is of the utmost importance for the child as well as the family and caregivers. Behavior therapies must be implemented as first-line therapy. If behavioral interventions alone fail, pharmacotherapy becomes an essential part of management plans. The limited available evidence for the use of common pharmacologic agents, such as second-generation antipsychotics, and less common agents, such as clonidine, n-acetylcysteine, riluzole, naltrexone, and topical anesthetics, is reviewed. Additional data from well-designed studies in children with NDDs are needed to gain a better understanding of this common and troublesome problem including efficacy and safety implications associated with pharmacotherapy. Until then, clinicians must rely on the limited available data, clinical expertise, and ongoing systematic monitoring when managing SIBs in children with NDDs.


Assuntos
Transtornos do Neurodesenvolvimento/tratamento farmacológico , Psicotrópicos/uso terapêutico , Comportamento Autodestrutivo/tratamento farmacológico , Humanos , Transtornos do Neurodesenvolvimento/complicações , Psicotrópicos/efeitos adversos , Comportamento Autodestrutivo/complicações
6.
Pediatr Pulmonol ; 54(8): 1200-1208, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31012285

RESUMO

BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor and lumacaftor/ivacaftor improve the status of existing infections in patients with cystic fibrosis (CF). It is unknown how well these drugs protect patients against incident infections. We hypothesized that CFTR modulator treatment would decrease new infections with Pseudomonas aeruginosa or Staphylococcus aureus. METHODS: We retrospectively studied a single-center cohort of patients with CF during two time periods (2008-2011, Era 1) and (2012-2015, Era 2) based on the January 2012 approval of ivacaftor. Using Kaplan-Meier analysis, we compared the time to any new infection with P. aeruginosa, methicillin-resistant S. aureus (MRSA), or methicillin-sensitive S. aureus (MSSA) that was absent during a 2-year baseline. We stratified the analysis based on whether patients received ivacaftor or lumacaftor/ivacaftor during Era 2. We used the log-rank test and considered P < 0.05 statistically significant. RESULTS: For patients receiving ivacaftor or lumacaftor/ivacaftor in Era 2, there was a statistically significant delay in the time to new bacterial acquisition in Era 2 vs. Era 1 ( P = 0.008). For patients who did not receive CFTR modulators, there was a trend toward slower acquisition of new bacterial infections in Era 2 compared to Era 1, but this was not statistically significant ( P = 0.10). CONCLUSIONS: Patients receiving ivacaftor or lumacaftor/ivacaftor for CF had significantly delayed acquisition of P. aeruginosa and S. aureus after these drugs were released. This method for analyzing incident infections may be useful for future studies of CFTR modulators and bacterial acquisition in CF registry cohorts.


Assuntos
Aminofenóis/uso terapêutico , Aminopiridinas/uso terapêutico , Benzodioxóis/uso terapêutico , Fibrose Cística/tratamento farmacológico , Infecções por Pseudomonas/prevenção & controle , Quinolonas/uso terapêutico , Infecções Estafilocócicas/prevenção & controle , Adolescente , Adulto , Criança , Pré-Escolar , Regulador de Condutância Transmembrana em Fibrose Cística , Combinação de Medicamentos , Feminino , Humanos , Masculino , Pseudomonas aeruginosa , Estudos Retrospectivos , Staphylococcus aureus , Adulto Jovem
7.
Am J Pharm Educ ; 82(4): 6270, 2018 May.
Artigo em Inglês | MEDLINE | ID: mdl-29867238

RESUMO

Objective. To illustrate a method for integrating co-curricular activities, quantify co-curricular activities, and evaluate student perception of achievement of goals. Methods. Throughout a longitudinal course, students engaged in self-selected, co-curricular activities in three categories: professional service, leadership, and community engagement. Hours were documented online with minimum course requirements. Students reflected on experiences and assessed goal attainment. Assignments were reviewed by faculty and feedback was given to each student. Results. From 2010 to 2016, there were 29,341 co-curricular hours documented by 756 students. The most popular events were attending pharmacy organization meetings and participating in immunization clinics. More than half of the students agreed they were able to meet all of their professional goals (mix of career and course goals) while 70% indicated goals were challenging to meet. Conclusion. This method for integrating co-curricular activities using a continuing professional development model demonstrates a sustainable system for promoting professional development through experience and self-reflection.


Assuntos
Currículo , Educação em Farmácia/métodos , Aprendizagem , Farmacêuticos/psicologia , Papel Profissional/psicologia , Estudantes de Farmácia/psicologia , Avaliação Educacional/métodos , Humanos , Profissionalismo
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