A. Type 1 B. Type 2 C. Type 3a D. Type 3b E. Unsure

A. A deficiency of lysosomes within cells of the bone and bone marrow, resulting in the inability of bone and bone marrow to function B. A deficiency of glucocerebrosidase, resulting in accumulation of glycolipids in macrophage lysosomes C. An overproduction of acid ß-glucosidase, resulting in destruction of organs such as bone, bone marrow, spleen, and liver D. An overproduction of saposin C, resulting in accumulation of glucose in cells causing organ dysfunction E. Unsure

A. Enzyme replacement therapy (ERT) with velaglucerase alfa B. ERT with alglucerase C. Substrate reduction therapy (SRT) with eliglustat D. SRT with miglustat E. Unsure

A. Must be reconstituted with sterile water for injection (SWI) and administered over 2 hours B. Must be reconstituted with SWI, diluted in 0.9% sodium chloride (normal saline; NS) to a final volume of 100 mL and infused over 2 hours C. Must be reconstituted with SWI and diluted with NS to a final volume of 100 mL and infused immediately D. Must be reconstituted with SWI, diluted in NS to a final volume of 100 mL, and administered through a low-protein-binding infusion set and in-line, low-protein-binding 0.2-?m filter over 1 hour E. Unsure

A. AH should continue velaglucerase alfa 30 units/kg IV every 2 weeks because he has achieved improvement in symptoms and is tolerating the infusions well B. AH should continue velaglucerase alfa but the dose should be increased to 45 units/kg IV every 2 weeks because, although he has improvement in symptoms and is tolerating the infusions well, he has not yet met his short-term management goals C. AH should be switched to another ERT product because, although he has improvement in symptoms and is tolerating the infusions well, he has not yet met his short-term management goals D. AH should undergo a splenectomy E. Unsure

A. ERT is a life-long treatment, but, once symptoms are improving, occasional treatment holidays are permitted to improve quality of life B. ERT is a life-long treatment; interruptions in treatment have resulted in worsening symptoms and disease progression, so they should be avoided C. ERT is a life-long treatment, so taking occasional drug holidays is expected and safe D. ERT is a life-long treatment, but treatment holidays of less than 2 months have been found to be safe E. Unsure

A. Bone involvement, such as osteolytic lesions leading to pathologic vertebral compression fractures B. Leukopenia C. Parkinsonism-like symptoms D. Pulmonary hypertension E. Unsure

A. For patients receiving imiglucerase therapy, switching to eliglustat is only recommended if therapeutic goals are not yet met B. For patients who have achieved stable therapeutic goals on imiglucerase therapy, switching to eliglustat is not detrimental to maintaining hematologic function and organ volume C. For patients who have achieved stable therapeutic goals on imiglucerase therapy, switching to eliglustat will improve organ volume and maintain hematologic function D. Outcomes of patients switching between therapies have not been well studied; thus, outcomes are unknown E. Unsure

A. Eliglustat 84 mg by mouth 2 times daily B. Eliglustat 84 mg by mouth once daily C. Eliglustat 84 mg by mouth 3 times daily D. None of these regimens: eliglustat is contraindicated in this patient E. Unsure

A. Coordinating care with home infusion centers for subsequent cycles to allow the patient to receive at-home infusions and improve quality of life B. Monitoring for neurologic symptoms and avoiding use of neurotoxic drugs C. Monitoring for changes in medications to avoid any potential interactions with drugs that affect CYP2D6, CYP3A4, P-glycoprotein, and Class IA and III antiarrhythmics D. Monitoring for diarrhea, as this affects dosing E. Unsure