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

Brain MRI at 6 months

Answer

Urine amino acid panel weekly

Answer

Genetic testing to confirm PAH mutation type before continuing

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

Corticosteroids (prednisone)

Answer

Creatine supplementation

Answer

ACE inhibitors for cardiac protection

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

The drug must be administered at a hospital inpatient unit

Answer

The drug is self-administered orally once daily with no special handling

Answer

The prescription requires DEA Schedule II documentation

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

Photosensitivity requiring sunscreen application

Answer

Severe hepatotoxicity requiring liver function monitoring

Answer

Nephrotoxicity requiring dose adjustment based on eGFR

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

Advise the patient to continue the drug and schedule routine ophthalmology follow-up in 1 year

Answer

Switch the patient to a different S1P modulator without prescriber involvement

Answer

Recommend over-the-counter lubricating eye drops and monitor symptoms

Question 6

When a specialty pharmacy is evaluating a prescription for ataluren (Translarna) for DMD, which condition would make the patient INELIGIBLE for this therapy?

Accepted Answer

Non-sense mutation in the dystrophin gene

Answer

Age under 5 years

Answer

Patient is ambulatory

Answer

Concurrent use of oral corticosteroids