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Red blood cell (RBC) production needs three things: raw materials (iron, vitamin B12, folic acid), a signal from the kidney (erythropoietin), and a working bone marrow. Drugs for anemia replace the missing material or signal. Hematopoietic growth factors also stimulate white cell and platelet production.
| Drug group | Mechanism |
|---|---|
| Iron (oral, IV) | Supplies iron for hemoglobin synthesis; absorbed mainly in the duodenum as ferrous (Fe²⁺) iron |
| Vitamin B12 (cyanocobalamin, hydroxocobalamin) | Needed for DNA synthesis and myelin maintenance; oral B12 normally requires intrinsic factor for absorption in the terminal ileum |
| Folic acid | Needed for DNA synthesis; deficiency causes megaloblastic anemia and fetal neural tube defects |
| Erythropoiesis-stimulating agents (ESAs) — epoetin alfa, darbepoetin alfa, methoxy polyethylene glycol-epoetin beta | Recombinant forms of erythropoietin → marrow makes more RBCs |
| HIF-prolyl hydroxylase inhibitors — vadadustat (oral) | Raise the body's own erythropoietin production |
| Myeloid growth factors — filgrastim, pegfilgrastim (G-CSF), sargramostim (GM-CSF) | Stimulate neutrophil production and release |
| Thrombopoietin receptor agonists — romiplostim (SC), eltrombopag, avatrombopag (oral) | Stimulate platelet production |
| Drug (prototype first) | Key use | Key point |
|---|---|---|
| ferrous sulfate (325 mg tablet ≈ 65 mg elemental iron) | Iron deficiency anemia | Once daily or every other day (may improve absorption and tolerance); continue about 3 months after Hb normalizes |
| ferrous gluconate, ferrous fumarate | Same | Doses are compared by elemental iron content |
| iron sucrose, ferric carboxymaltose, ferric derisomaltose, ferumoxytol, low-molecular-weight iron dextran | Oral intolerance or failure, malabsorption, ongoing losses, CKD, later pregnancy with severe deficiency | IV; hypersensitivity monitoring |
| cyanocobalamin | Pernicious anemia, B12 deficiency (gastrectomy, bariatric surgery, ileal disease, strict vegan diet, long-term metformin or PPI use) | IM or deep SC injections (commonly 1,000 mcg), frequent at first then monthly; lifelong in pernicious anemia; high-dose oral B12 is an alternative for many clients |
| folic acid | Folate deficiency (poor diet, alcohol use disorder, pregnancy, methotrexate, phenytoin, hemolysis) | Commonly 1 mg daily for deficiency; 0.4–0.8 mg daily before conception for prevention of neural tube defects (4 mg after a previous affected pregnancy) |
| epoetin alfa, darbepoetin alfa | Anemia of CKD, chemotherapy-induced anemia, zidovudine-related anemia, reducing transfusion before some surgery | Weekly to monthly SC or IV; need adequate iron |
| vadadustat | Anemia of CKD in clients on dialysis | Oral alternative to ESAs; daprodustat (approved 2023) was withdrawn from the US market in December 2024 |
| filgrastim, pegfilgrastim | Prevent or shorten chemotherapy-induced neutropenia, stem cell mobilization | Filgrastim daily SC; pegfilgrastim once per chemotherapy cycle |
| romiplostim, eltrombopag | Chronic immune thrombocytopenia (ITP); eltrombopag also in aplastic anemia | Aim for a safe platelet count, not a normal one |
Match the drug to the cause: iron deficiency → microcytic; B12 or folate deficiency → macrocytic (megaloblastic); anemia of CKD → low erythropoietin. Always rule out B12 deficiency before giving folic acid alone — folic acid corrects the anemia but lets B12-related nerve damage progress.
| Drug | Key adverse effects |
|---|---|
| Oral iron | Constipation, nausea, epigastric pain, dark green or black stools (expected, harmless), tooth staining (liquid forms) |
| IV iron | Hypersensitivity reactions (rare but can be severe), hypotension, flushing, joint and muscle aches, permanent brown skin staining with extravasation; ferric carboxymaltose → low phosphate; ferumoxytol and iron dextran carry boxed warnings for anaphylaxis; ferumoxytol alters MRI images for up to about 3 months |
| Vitamin B12 | Injection-site pain; hypokalemia early in treatment of severe megaloblastic anemia (rapid new cell production); rare allergy |
| Folic acid | Rarely allergic reactions; masks B12 deficiency |
| ESAs | Hypertension, thrombosis (DVT, dialysis access clotting), stroke and MI, seizures, headache; rare pure red cell aplasia (antibodies). Boxed warning: higher hemoglobin targets increase death, cardiovascular events, and stroke; in some cancers, ESAs shortened survival or increased tumor progression |
| HIF-PH inhibitors | Boxed warning for death, MI, stroke, thrombosis — similar to ESAs |
| Filgrastim, pegfilgrastim | Bone pain (common), splenic rupture (rare — left upper abdominal or shoulder pain), acute respiratory distress syndrome, allergic reactions, sickle cell crisis in sickle cell disease, leukocytosis |
| Romiplostim, eltrombopag | Thrombosis, rebound thrombocytopenia after stopping, marrow fibrosis; eltrombopag boxed warning: hepatotoxicity in all clients (monitor liver tests), and hepatic decompensation in chronic hepatitis C with interferon/ribavirin |
| Test | Adult reference / target |
|---|---|
| Hemoglobin | Anemia (WHO) < 13 g/dL (130 g/L) men, < 12 g/dL (120 g/L) nonpregnant women |
| Reticulocyte count | Rises within about 7–10 days of effective iron, B12, or folate therapy — the earliest sign of response |
| Ferritin | < 30 ng/mL (30 µg/L) = iron deficiency; also watch for iron overload |
| Transferrin saturation | < 20% suggests iron deficiency |
| Blood pressure (ESA) | Controlled before and during therapy |
| ANC (G-CSF) | Neutropenia = ANC < 1,500/µL (1.5 × 10⁹/L); severe < 500/µL (0.5 × 10⁹/L) |
| Platelets (TPO agonists) | Target about ≥ 50,000/µL (50 × 10⁹/L) in ITP to reduce bleeding |
Iron poisoning (a leading cause of fatal poisoning in young children)
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