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Text
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URL Address
<a href="http://doi.org/10.1002/phar.1290" target="_blank" rel="noreferrer">http://doi.org/10.1002/phar.1290</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
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Pharmacotherapy challenges of Fontan-associated plastic bronchitis: a rare pediatric disease
Publisher
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Pharmacotherapy
Date
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2013
Subject
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Child; Humans; Rare Diseases; Bronchitis; Fontan Procedure
Creator
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Brooks K; Caruthers RL; Schumacher KR; Stringer KA
Description
An account of the resource
Pediatric pharmacotherapy is often challenging due to the paucity of available clinical data on the safety and efficacy of drugs that are commonly used in children. This quandary is even more prevalent in children with rare diseases. Although extrapolations for dosing and administration are often made from available adult data with similar disease states, this translation becomes even more problematic in rare pediatric diseases. Understanding of rare disease pathophysiology is typically poor, and few, if any, effective therapies have been studied and identified. One condition that illustrates these issues is plastic bronchitis, a rare, most often pediatric disease that is characterized by the production of obstructive bronchial airway casts. This illness primarily occurs in children with congenital heart disease, often after palliative surgery. Plastic bronchitis is a highly clinically relevant and therapeutically challenging problem with a high mortality rate, and, a generally accepted effective pharmacotherapy regimen has yet to be identified. Furthermore, the disease is ill defined, which makes timely identification and treatment of children with plastic bronchitis difficult. The pharmacotherapies currently used to manage this disease are largely anecdotal and vary between the use of macrolide antibiotics, mucolytics, bronchodilators, and inhaled fibrinolytics in a myriad of combinations. The purpose of this review is 2-fold: first, to highlight the dilemma of treating plastic bronchitis, and second, to bring attention to the continuing need for studies of drug therapies used in children so safe and effective drug regimens can be established, particularly for rare diseases.
2013-09
Identifier
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<a href="http://doi.org/10.1002/phar.1290" target="_blank" rel="noreferrer">10.1002/phar.1290</a>
Rights
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Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Type
The nature or genre of the resource
Journal Article
2013
Backlog
Bronchitis
Brooks K
Caruthers RL
Child
Fontan Procedure
Humans
Journal Article
Pharmacotherapy
Rare Diseases
Schumacher KR
Stringer KA
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Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
Citation List Month
Backlog
URL Address
<a href="http://doi.org/10.1002/phar.1201" target="_blank" rel="noreferrer">http://doi.org/10.1002/phar.1201</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
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Neurogenetic disorders and treatment of associated seizures.
Publisher
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Pharmacotherapy
Date
A point or period of time associated with an event in the lifecycle of the resource
2013
Subject
The topic of the resource
Humans; Treatment Outcome; Epilepsies; Angelman Syndrome/dt [Drug Therapy]; Anticonvulsants/tu [Therapeutic Use]; Epilepsies; Rett Syndrome/dt [Drug Therapy]; Seizures/dt [Drug Therapy]; Tuberous Sclerosis/dt [Drug Therapy]; Angelman Syndrome/co [Complications]; Anticonvulsants/ad [Administration & Dosage]; Anticonvulsants/ae [Adverse Effects]; KT Synthesis; Myoclonic/co [Complications]; Myoclonic/dt [Drug Therapy]; Rett Syndrome/co [Complications]; Seizures/co [Complications]; Tuberous Sclerosis/co [Complications]
Creator
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Faulkner MA; Singh Sanjay P
Description
An account of the resource
Seizures are a frequent complication associated with several neurogenetic disorders. Antiepileptic medications remain the mainstay of treatment in these patients. We summarized the available data associated with various antiepileptic therapies used to treat patients with neurogenetic disorders who experienced recurrent seizures. A MEDLINE search was conducted to identify articles and abstracts describing the use of antiepileptic therapy for the treatment of various neurogenetic syndromes. Of all the neurogenetic syndromes, only autism spectrum disorders, Angelman syndrome, Rett syndrome, Dravet syndrome, and tuberous sclerosis complex were identified as having sufficient published information to evaluate therapy. Some efficacy trends were identified, including frequent successes with valproic acid with clonazepam for epilepsy with Angelman syndrome; valproic acid, stiripentol, and clobazam (triple combination therapy) for epilepsy with Dravet syndrome; and vigabatrin for infantile spasms associated with tuberous sclerosis complex. Due to a paucity of information regarding the mechanisms by which seizures are generated in the various disorders, approach to seizure control is primarily based on clinical experience and a limited amount of study data exploring patient outcomes. Although exposure of the developing brain to antiepileptic medications is of some concern, the control of epileptic activity is an important undertaking in these individuals, as the severity of eventual developmental delay often appears to correlate with the severity of seizures. As such, early aggressive therapy is warranted. 2013 Pharmacotherapy Publications, Inc.
2013
Identifier
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<a href="http://doi.org/10.1002/phar.1201" target="_blank" rel="noreferrer">10.1002/phar.1201</a>
Rights
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Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Type
The nature or genre of the resource
Journal Article
2013
Angelman Syndrome/co [Complications]
Angelman Syndrome/dt [Drug Therapy]
Anticonvulsants/ad [Administration & Dosage]
Anticonvulsants/ae [Adverse Effects]
Anticonvulsants/tu [Therapeutic Use]
Backlog
Epilepsies
Faulkner MA
Humans
Journal Article
KT Synthesis
Myoclonic/co [Complications]
Myoclonic/dt [Drug Therapy]
Pharmacotherapy
Rett Syndrome/co [Complications]
Rett Syndrome/dt [Drug Therapy]
Seizures/co [Complications]
Seizures/dt [Drug Therapy]
Singh Sanjay P
Treatment Outcome
Tuberous Sclerosis/co [Complications]
Tuberous Sclerosis/dt [Drug Therapy]