CSP - Certified Specialty Pharmacist Clinical Management: Neurology and Rare Disease Questions and Answers Flashcards
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A patient with hereditary angioedema (HAE) is prescribed subcutaneous icatibant (Firazyr) for acute attacks. What is the MOST important self-management counseling point?
Answer: Inject at the first signs of an acute attack and seek emergency care if symptoms do not improve within 6 hours
Icatibant is indicated for on-demand treatment of acute HAE attacks; prompt injection at symptom onset is critical, and patients should seek emergency care if response is inadequate within 6 hours.
Which clinical finding would MOST warrant a CSP recommending a dose interruption or reduction in a patient on siponimod (Mayzent) for secondary progressive MS?
Answer: New onset of symptomatic bradycardia or second-degree AV block
Siponimod, like other S1P modulators, can cause cardiac conduction effects; symptomatic bradycardia or AV block warrants dose interruption and urgent prescriber notification.
A patient with transthyretin amyloidosis (ATTR) is prescribed patisiran (Onpattro). How is this RNA interference (RNAi) therapy administered?
Answer: IV infusion every 3 weeks
Patisiran is administered as an IV infusion every 3 weeks and requires premedication with corticosteroids, acetaminophen, and antihistamines to reduce infusion-related reactions.
For a patient with Pompe disease receiving alglucosidase alfa (Lumizyme), the specialty pharmacist notes that the patient has developed high and sustained antibody titers. What is the MOST appropriate clinical action?
Answer: Notify the prescriber, as high antibody titers can reduce drug efficacy and immune tolerance protocols may be needed
High sustained antibody titers against alglucosidase alfa are associated with reduced clinical response; the prescriber should be notified so immune tolerance induction strategies can be considered.
A CSP is counseling the caregiver of a child with spinal muscular atrophy who will receive onasemnogene abeparvovec (Zolgensma). Which statement about this gene therapy is MOST accurate?
Answer: It is a one-time IV gene therapy that requires corticosteroid pretreatment to prevent liver toxicity
Zolgensma is a single-dose IV gene therapy; corticosteroid pretreatment and monitoring are required due to the risk of acute liver injury associated with adeno-associated virus vector delivery.
Which of the following specialty pharmacy services is MOST critical for ensuring patient access to ultra-high-cost gene therapies such as betibeglogene spartacus (Zynteglo) for beta-thalassemia?
Answer: Outcomes-based contracting facilitation and manufacturer financial assistance coordination
Gene therapies with multi-million-dollar price tags require specialty pharmacies to navigate outcomes-based contracts and manufacturer assistance programs to make therapy financially feasible for payers and patients.