CSP CSP - Certified Specialty Pharmacist Clinical Management: Neurology and Rare Disease Questions and Answers 2

Question 1

A patient with phenylketonuria (PKU) is prescribed sapropterin (Kuvan). What is the standard method to assess response to this therapy?

Accepted Answer

Blood phenylalanine level measured after a 1-month therapeutic trial

Answer

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.

Question 2

Which therapy for Duchenne muscular dystrophy (DMD) requires a specific exon-skipping amenable mutation for eligibility, making genetic testing essential before dispensing?

Accepted Answer

Eteplirsen (Exondys 51)

Answer

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.

Question 3

A specialty pharmacist receives a prescription for edaravone (Radicava) for ALS. What is the MOST important logistical consideration for this therapy?

Accepted Answer

The therapy requires IV infusion in cycles and coordination with an infusion center or home infusion service

Answer

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.

Question 4

For patients with Fabry disease receiving agalsidase beta (Fabrazyme), which adverse effect requires pharmacist patient education BEFORE each infusion?

Accepted Answer

Infusion-associated reactions including fever, chills, hypertension, and urticaria

Answer

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.

Question 5

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?

Accepted Answer

Recommend drug discontinuation and coordinate urgent ophthalmology evaluation, notifying the prescriber immediately

Answer

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.

CSP - Certified Specialty Pharmacist Practice Test

CSP - Certified Specialty Pharmacist Practice Test

CSP CSP - Certified Specialty Pharmacist Clinical Management: Neurology and Rare Disease Questions and Answers 2