Hydroxyurea
HYDREA
What it isRibonucleotide reductase inhibitor that raises fetal hemoglobin; sickle cell disease and myeloproliferative disease.
Why this oneIt reduces crises, acute chest syndrome and mortality in sickle cell disease, which supportive care alone does not.
What limits itMyelosuppression requires regular counts. Teratogenic, and it takes months for the fetal hemoglobin rise to show benefit.
FDA-approved for
- sickle cell disease (1998)
- chronic myeloid leukemia, melanoma
Off-label
- thrombocythemia and polycythemia vera
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Neoplastic diseases | Adults | 15 mg/kg QD | Individualized to response/tumor type |
Renal Reduce dose 50% (7.5 mg/kg QD) if CrCl <60 mL/min or ESRD; on dialysis days give after hemodialysis
Hepatic Undefined — no data for adjustment; monitor hematologic parameters closely
Instructions
- Swallow capsules whole; do not open, break, or chew (cytotoxic drug)
- Base dose on actual or ideal body weight, whichever is less
- Prophylactic folic acid recommended
- Monitor blood counts at least weekly; correct severe anemia before starting
Cautions
- Severe myelosuppression — do not initiate if bone marrow markedly depressed
- Hemolytic anemia
- Human carcinogen; secondary leukemia and skin cancer with long-term use
- Embryo-fetal toxicity
- Cutaneous vasculitic toxicities (ulcers, gangrene)
- Avoid live vaccines
- Pancreatitis, hepatotoxicity, neuropathy with concomitant antiretrovirals
- Radiation recall
- Macrocytosis (may mask pernicious anemia)
- Pulmonary toxicity/interstitial lung disease (incl. fatal)
Adverse reactions
- Hematologic (myelosuppression, leukopenia, thrombocytopenia, anemia)
- GI symptoms (nausea, vomiting, diarrhea, stomatitis)
- Anorexia
Pregnancy Can cause fetal harm (teratogenic/embryotoxic in animals); avoid pregnancy, use effective contraception
Lactation Excreted in human milk; discontinue breastfeeding during treatment
⚠ BOXED WARNINGS
- Myelosuppression
- Malignancy
Cost, est. cash$50–$10,000/month or cycle
Generic entry1995
Classes and tags
Clinical profile
Legacy pregnancy categoryD
Defining liabilityMyelosuppression and agent-specific hepatic, cardiac, pulmonary, neurologic or reproductive toxicity require oncology monitoring.
Mechanism of action: Hydroxyurea ballicule
