CSP - Certified Specialty Pharmacist Clinical Management: Neurology and Rare Disease Questions and Answers Flashcards
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A patient with phenylketonuria (PKU) is prescribed sapropterin (Kuvan). What is the standard method to assess response to this therapy?
Answer: Blood phenylalanine level measured after a 1-month therapeutic trial
Response to sapropterin is determined by measuring blood phenylalanine levels after a 1-month trial; only patients who respond (defined as a ≥30% reduction in phenylalanine) should continue therapy.
Which therapy for Duchenne muscular dystrophy (DMD) requires a specific exon-skipping amenable mutation for eligibility, making genetic testing essential before dispensing?
Answer: Eteplirsen (Exondys 51)
Eteplirsen is indicated specifically for patients with a confirmed mutation amenable to exon 51 skipping; genetic testing is mandatory to confirm eligibility before the specialty pharmacy can process the prescription.
A specialty pharmacist receives a prescription for edaravone (Radicava) for ALS. What is the MOST important logistical consideration for this therapy?
Answer: The therapy requires IV infusion in cycles and coordination with an infusion center or home infusion service
Edaravone is administered as IV infusions in 28-day cycles (10 infusions per cycle initially); the specialty pharmacy must coordinate with infusion centers or home infusion providers for ongoing access.
For patients with Fabry disease receiving agalsidase beta (Fabrazyme), which adverse effect requires pharmacist patient education BEFORE each infusion?
Answer: Infusion-associated reactions including fever, chills, hypertension, and urticaria
Infusion-associated reactions are among the most common adverse effects with agalsidase beta; patients should be premedicated and counseled to report symptoms such as fever, chills, and skin reactions during infusion.
Which of the following best describes the role of a CSP in managing a patient on ozanimod (Zeposia) for relapsing MS who develops macular edema?
Answer: Recommend drug discontinuation and coordinate urgent ophthalmology evaluation, notifying the prescriber immediately
Macular edema is a serious, vision-threatening adverse effect of ozanimod that requires prompt discontinuation and urgent ophthalmology evaluation; the CSP must notify the prescriber immediately.
When a specialty pharmacy is evaluating a prescription for ataluren (Translarna) for DMD, which condition would make the patient INELIGIBLE for this therapy?
Answer: Non-sense mutation in the dystrophin gene
Ataluren works specifically for non-sense (stop codon) mutations in the dystrophin gene; patients without this mutation type would not benefit and are not candidates for therapy.