Cytotoxic / Disease-Modifying Agent
Pregnancy: D
Hydroxycarbamide (Hydroxyurea)
Brand names: Xagrid, Siklos, Hydrea
Adult dose
Dose: Sickle cell: 15 mg/kg/day, titrate up by 5 mg/kg every 12 weeks to max 35 mg/kg/day; ET: 500–1000 mg/day
Route: oral
Frequency: once daily
Max: 35 mg/kg/day
Siklos formulation licensed specifically for sickle cell; increases HbF production; wear gloves when handling if carer
Paediatric dose
Dose: 15 mg/kg
Route: oral
Frequency: once daily
Max: 35 mg/kg/day
Concentration: Siklos 100 mg/mL or 100 mg tablets mg/ml
Sickle cell disease: start 15 mg/kg/day; increase every 8–12 weeks; monitor every 4 weeks; avoid if ANC <2×10⁹/L or Plt <80×10⁹/L
Dose adjustments
Renal
Reduce dose if eGFR <60 ml/min; avoid if eGFR <10 ml/min
Hepatic
Use with caution
Paediatric weight-based calculator
Sickle cell disease: start 15 mg/kg/day; increase every 8–12 weeks; monitor every 4 weeks; avoid if ANC <2×10⁹/L or Plt <80×10⁹/L
Clinical pearls
- Increases HbF — reduces sickling, crisis frequency, and mortality in sickle cell disease (MSH trial: 44% reduction in pain crises)
- Leg ulcers: reversible on dose reduction — monitor lower limbs
- Teratogen: double contraception in both male and female patients
Contraindications
- Pregnancy
- Breastfeeding
- Severe myelosuppression
- Active severe infection
Side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Mucositis
- Leg ulcers (long-term)
- Skin pigmentation
- Secondary leukaemia (rare)
- Teratogenicity
Interactions
- Didanosine/stavudine (HIV) — fatal pancreatitis/neuropathy
- Antiretroviral drugs
- Cytotoxics (enhanced myelosuppression)
Monitoring
- FBC every 2 weeks until stable, then monthly
- LFTs
- Renal function
- HbF levels (sickle cell)
Reference: BNFc; BNF 86; NICE NG143; MSH trial. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Calculators
- Rate-Pressure Product (RPP) · Haemodynamics
- DAPT Score · Coronary Artery Disease
- Mehran Score for Post-PCI Contrast Nephropathy · Coronary Artery Disease
- Aortic Dissection Detection Risk Score (ADD-RS) · Aortic Disease
- RoPE Score for Patent Foramen Ovale · Structural Heart Disease
- Canadian Cardiovascular Society (CCS) Angina Grading · Coronary Artery Disease
Pathways
- Major Haemorrhage / Massive Transfusion · BCSH; RCOA; RCEM; RCS — BCSH Guidelines
- Anaemia Investigation · BSH / NICE
- Splenomegaly Workup · BSH; BMJ Best Practice
- Deep Vein Thrombosis Diagnosis and Treatment · NICE CG144 / NICE NG158
- Sickle Cell Crisis · BSH 2021 / BCSH
- Neutropenic Sepsis · NICE CG151 2012 / ESMO