Anagrelide hydrochloride and Cerdelga

• Generic Name: anagrelide
• Dosage Forms: n.a.
• Other Brand Names: Agrylin

Reduction of elevated platelet counts and associated risk of thromboembolic and hemorrhagic events in patients with thrombocythemia secondary to essential thrombocythemia (ET) and other myeloproliferative disorders. Has been designated an orphan drug by FDA for the treatment of ET.

Management of ET generally based on a risk-stratification approach. Treatment with a cytoreductive agent (e.g., anagrelide, hydroxyurea) usually reserved for patients at high risk (i.e., age >60 years, previous history of thrombosis, and/or platelet count ≥1,500,000/mm3) of developing thromboembolic and/or hemorrhagic complications. Some clinicians also recommend cytoreductive therapy in intermediate-risk ET patients (e.g., 40–60 years of age, platelet count >1,000,000/mm3, and cardiovascular risk factor [smoking, arterial hypertension, hypercholesterolemia, or diabetes mellitus] or familial thrombophilia).

Hydroxyurea (often combined with low-dose aspirin) generally considered drug of choice in high-risk patients with ET because of proven efficacy and infrequent acute toxicity. However, because of potential leukemogenic effects with hydroxyurea when used long term or sequentially with other cytotoxic drugs, anagrelide or interferon alfa suggested as alternative therapy in high-risk patients, particularly younger patients (<40–60 years of age), and in those who do not respond to or cannot tolerate hydroxyurea. Consider cautious use of low-dose aspirin concomitantly with anagrelide based on relative risks of thrombosis and arterial hemorrhage in individual patients. (See Specific Drugs or Foods under Interactions.)

• Generic Name: eliglustat
• Dosage Forms: capsule
• Other Brand Names: Cerdelga

Cerdelga (eliglustat) reduces the formation of a certain protein in the body in people with type 1 Gaucher disease>.

Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials (lipids). Lipids can build up in the body, causing symptoms such as easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or weakened bones that are easily fractured.

Cerdelga is used to treat mild to moderate type 1 Gaucher disease in adults. It is used only if a specific liver enzyme (2D6) in your body breaks down or metabolizes drugs at a certain rate.

Cerdelga may improve the condition of the liver, spleen, bones, and blood cells in people with Type I Gaucher disease. However, eliglustat is not a cure for this condition.