542: Anemias

Antithymocyte Globulin Equine (ATGAM)

Aplastic Anemia

MOA: stimulates hematopoeitic stem cells

AE: local pain/erythema, anaphylaxis

Cyclosporin A

Aplastic Anemia

MOA: calcineurin inhibitor (block activation of T-cells)

AE: nephro/hepatotoxicity, HTN, hyperglycemia, hyperkalemia, alt mental, seizures, blood cancer risk

Eltrombopag

Aplastic Anemia

MOA: TPO-R agonist (increase megakaryocytes)

AE: hepatotoxicity, thrombosis, cataracts, hemorrhage

Crizanlizumab

Sickle Cell Anemia

MOA: mAb against p-selectin (adhesion molecule for endothelial cells)

AE: arthralgia, diarrhea, pruritis, vomiting, chest pain

Decitabine

Sickle Cell Anemia

MOA: cytadine analog (inhibits nucleic acid synthesis)

AE: neutropenia, thrombocytopenia, anemia, fatigue, nausea, petechiae, C/D, hyperglycemia

Folic Acid

Sickle Cell Anemia

MOA: supplements folic acid stores to enable erythropoiesis

AE: allergies

L-glutamine

Sickle Cell Anemia

MOA: increases NAD production to reduce oxidative stress on RBC’s

AE: pain, GI upset

Hydroxyurea

Sickle Cell Anemia / Thalassemia

MOA: decreases binding of HbS to endothelial cells (↓occlusion/pain)

AE: bone marrow suppression, myelosuppresion, GI upset, fetal abnormalities, anorexia, rash, dysuria, malaise/fever

Deferasirox

Thalassemia

MOA: iron chelator (reduces chronic iron elevation)

AE: renal/hepatic toxicity, GI hemorrhage, bone marrow suppression, rash, abd pain, N/V/D

Deferiprone

Thalassemia

MOA: iron chelator (reduces chronic iron elevation)

AE: agranulocytosis, neutropenia, abd pain, red urine, N/V

Deferoxamine

Thalassemia

MOA: iron chelator (reduces chronic iron elevation)

AE: hearing/vision changes, renal toxicity, HoTN, nausea, inj site rxn

Luspatercept

Thalassemia

MOA: fusion protein, blocks TGF in blood to ↑ RBC production

AE: fatigue, bone/joint/muscle pain, GI upset, allergies

Betibeglogene Autotemcel

Beta-Thalassemia

MOA: CD34+ cells encoding B-globin gene

AE: thrombocytopenia

Desmopressin

Von Willebrane Disease

MOA: modified ADH nonapeptide with selectivity for V2-receptor that triggers release of VWF

AE: headaches, nausea, cramps, allergic rxn

Aminocaproic acid

Von Willebrane Disease

MOA: inhibit plasminogen (dissolves clots)

AE: edema, HA, malaise, thromboses, brady, leukopenia, thrombocytopeni

Tranexamic acid

Von Willebrane Disease

MOA: inhibit plasminogen (dissolves clots) 10x more potent than Aminocaproic acid

AE: back pain, abd pain, diarrhea, fatigue, anemia, embolism/thrombosis

Von Willebrand Factor

Von Willebrane Disease

MOA: recombinant VWF, promotes platelet adhesion

AE: hypersensitivity rxn, emolism/thrombosis

Efanesoctocog alfa (Altuviiio)

Von Willebrane Disease

MOA: modified longer acting VWF (adds Fc antibody to protect from body, uses piece of VWF, adds XTEN to protect from degradation)

AE: hypersensitivity rxn, arthralgia, back pain

Emicizumab

Hemophilia

MOA: mAb, binds factors IX and X

AE: embolism/thrombosis

Concizumab

Hemophilia

MOA: mAb, targets tissue factor pathway inhibitor

AE: infusion rxn, nausea, diarrhea

Marsticimab

Hemophilia

MOA: mAb, targets tissue factor pathway inhibitor

AE: infusion rxn, itching

Valoctocogene Roxaparvovec

Hemophilia A

MOA: make FVIII

AE: infusion rxn, ↑ liver enzymes, nausea

Etranacogene Dezaparvovec (B)

Hemophilia B

MOA: make FIX

AE: ↑ ALT, ↑ CK, infusion rxn, flu symptoms

Fitusiran

Hemophilia

MOA: RNAi , inhibits antithrombin

AE: ↑ clots, ↑ liver enzymes