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AHRQ Research Studies
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Research Studies is a compilation of published research articles funded by AHRQ or authored by AHRQ researchers.
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1 to 2 of 2 Research Studies DisplayedFeldman AG, Parsons JA, Dutmer CM
Subacute liver failure following gene replacement therapy for spinal muscular atrophy type 1.
This paper reports on two cases of transient, drug-induced liver failure after gene replacement therapy using an adeno-associated virus vector containing the survival motor neuron 1 gene.
AHRQ-funded; HS026510.
Citation: Feldman AG, Parsons JA, Dutmer CM .
Subacute liver failure following gene replacement therapy for spinal muscular atrophy type 1.
J Pediatr 2020 Oct;225:252-58.e1. doi: 10.1016/j.jpeds.2020.05.044..
Keywords: Newborns/Infants, Neurological Disorders, Genetics, Treatments, Adverse Drug Events (ADE), Adverse Events, Medication, Medication: Safety, Patient Safety, Case Study
Krantz MS, Liu Y, Phillips EJ
Anaphylaxis to PEGylated liposomal echocardiogram contrast in a patient with IgE-mediated macrogol allergy.
The authors describe a case of anaphylaxis to PEG 5000-conjugated liposomal perflutren, a perfluorocarbon gas used as echocardiography contrast. They conclude that, given the severity of the patient’s reactions, allergists, cardiologists, and radiologists should become aware of the possibility of hypersensitivity reactions to macrogols to facilitate prompt diagnosis, treatment, and future avoidance. They suggested that use of PEGylated liposomal perflutren and other drugs and devices containing HMW PEG be avoided in favor of alternatives when patients report previous immediate hypersensitivity reactions consistent with severe macrogol allergy. They recommended further studies.
AHRQ-funded; HS026395.
Citation: Krantz MS, Liu Y, Phillips EJ .
Anaphylaxis to PEGylated liposomal echocardiogram contrast in a patient with IgE-mediated macrogol allergy.
J Allergy Clin Immunol Pract 2020 Apr;8(4):1416-19.e3. doi: 10.1016/j.jaip.2019.12.041..
Keywords: Adverse Events, Diagnostic Safety and Quality, Patient Safety, Case Study