The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG
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J Pediatr Pharmacol Ther · Jan 2021
ReviewOnasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy.
Spinal muscular atrophy (SMA) is a debilitating disorder characterized by degeneration of large motor neurons. It is a heterogeneous group of disorders caused by a homozygous deletion in the survival motor neuron (SMN) gene on chromosome 5, resulting in a SMN protein deficiency. Small amounts of SMN protein are also produced by the SMN2 gene, which that differs from SMN1 by a single nucleotide. ⋯ This one-time intravenous injection of this new gene therapy also bears a hefty price tag; however, it may be more cost effective in the long run versus the multiple intrathecal administrations needed with nusinersen. Drug access and use are hindered by drug cost, payer reimbursement issues, and lack of long-term data from clinical studies. Questions also remain regarding the safety and efficacy of repeated drug administration for patients with advanced disease.
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J Pediatr Pharmacol Ther · Jan 2021
Adolescents' Perspectives on Prescription Opioid Misuse and Medication Safety.
This study explored adolescents' perspectives on prescription opioids, opioid safety and misuse, and sources of opioid information. ⋯ Adolescents had some knowledge pertaining to prescription opioids, but they had misconceptions related to safety. Participants were aware of safety risks and negative consequences of misuse. Adolescents obtained medication information from various sources, including health care professionals, family and peers, and online sources. Educational efforts that target adolescents should provide opportunities for addressing misconceptions about safe and responsible use of medications.
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J Pediatr Pharmacol Ther · Jan 2020
ReviewElexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy.
Elexacaftor-tezacaftor-ivacaftor is a newly approved triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulating therapy that contains 2 correctors and a potentiator of the CFTR channel. Its labeled indication for use is for persons 12 years of age and older with at least 1 F508del mutation for the CFTR gene. This drug combination provides potential therapy to many patients who had previously been excluded from CFTR modulation therapy due to the nature of their genetic mutations. ⋯ The most common adverse events seen in clinical trials included rash and headache, and laboratory monitoring is recommended to evaluate liver function. Continued evaluation of patient data is needed to confirm its long-term safety and efficacy. Elexacaftor-tezacaftor-ivacaftor is a monumental and encouraging therapy for cystic fibrosis; however, approximately 10% of the CF population are not candidates for this or any other CFTR modulation therapy.
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J Pediatr Pharmacol Ther · Jan 2020
Case ReportsHome Use of Intranasal Dexmedetomidine in a Child With An Intractable Sleep Disorder.
Sleep disturbance is a crucial issue in pediatric palliative care, with a dramatic impact on the quality of life of children and families. Dexmedetomidine (DEX) is a selective α-2 agonist, with anxiolytic, hypnotic, and analgesic properties, that could play a role in the management of refractory sleep disturbances. We describe the use of intranasal DEX as a sleep inductor in a 10-year-old female with dystrophic epidermolysis bullosa and a severe sleep disorder. ⋯ There was a definite improvement in sleep quality and duration, daytime alertness, pain control, and quality of life. No side effects were reported and the drug retained its effect over time (the patient is currently taking the drug). Intranasal DEX could be a safe and effective strategy to manage refractory sleep disturbances in children in pediatric palliative care.
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J Pediatr Pharmacol Ther · Jan 2020
Modeling and Simulation Analysis of Aprepitant Pharmacokinetics in Pediatric Patients With Postoperative or Chemotherapy-Induced Nausea and Vomiting.
Aprepitant is effective for the prevention of chemotherapy-induced or postoperative nausea and vomiting (CINV/PONV). The aim of this study was to develop a population pharmacokinetic (PK) model of aprepitant in pediatric patients and to support dosing recommendations for oral aprepitant in pediatric patients at risk of CINV. ⋯ The developed population PK model adequately described aprepitant PK across a broad pediatric population, justifying fixed (adult) dosing for adolescents and weight-based dosing of oral aprepitant for children.