Mycophenolate mofetil
CELLCEPT
What it isInhibitor of inosine monophosphate dehydrogenase, blocking lymphocyte purine synthesis; transplant and autoimmune disease.
Why this oneLymphocyte-selective, so it spares other marrow lines more than azathioprine, and it has replaced it in most transplant regimens.
What limits itPowerfully teratogenic with a REMS and mandatory contraception. GI intolerance and leukopenia; it is not the same dose as the sodium salt.
FDA-approved for
- prophylaxis of organ transplant rejection (1995)
Off-label
- lupus nephritis
- autoimmune hepatitis
- myasthenia gravis
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Kidney transplant rejection prophylaxis | adult | 1 g BID (PO or IV over ≥2 h) | ||
| Heart transplant rejection prophylaxis | adult | 1.5 g BID (PO or IV over ≥2 h) | ||
| Liver transplant rejection prophylaxis | adult | 1.5 g BID PO or 1 g BID IV over ≥2 h | ||
| Kidney transplant rejection prophylaxis | peds ≥3 months | 600 mg/m² BID | 2 g/day | |
| Heart transplant rejection prophylaxis | peds ≥3 months | 600 mg/m² BID | 900 mg/m² BID | 3 g/day |
| Liver transplant rejection prophylaxis | peds ≥3 months | 600 mg/m² BID | 900 mg/m² BID | 3 g/day |
Renal Kidney transplant with severe chronic graft impairment (GFR <25 mL/min/1.73m²): avoid doses >1 g BID
Hepatic No dose adjustment for kidney transplant patients with severe hepatic parenchymal disease
Doses >1 g BID avoided when GFR <25
Instructions
- Give on an empty stomach (may give with food in stable patients if necessary)
- Do not crush tablets or open/crush capsules; avoid inhaling or skin/mucosal contact with powder
- IV over no less than 2 h — never rapid or bolus injection
- Give initial dose within 24 h of transplant; IV only until oral tolerated, up to 14 days
- Interrupt or reduce dose for neutropenia (ANC <1.3 ×10³/mcL)
Contraindications
- Polysorbate 80 (TWEEN) allergy — IV formulation
Cautions
- Neutropenia and pure red cell aplasia — monitor CBC
- GI bleeding, ulceration, perforation
- Avoid in hypoxanthine-guanine phosphoribosyl-transferase deficiency (Lesch-Nyhan/Kelley-Seegmiller)
- Acute inflammatory syndrome
- Hypersensitivity (angioedema, anaphylaxis)
- Avoid live attenuated vaccines
- No blood donation during and 6 wk after; no semen donation during and 90 days after
Adverse reactions
- Diarrhea
- Leukopenia
- Infection (incl. opportunistic)
- Vomiting
- Anemia
- Hypertension
- Peripheral edema
- Nausea
Pregnancy Increased risk of first-trimester pregnancy loss (45–49%) and multiple congenital malformations; avoid if safer options available
Lactation No human milk data; present in rat milk — risk to breastfed infant cannot be excluded
⚠ BOXED WARNINGS
- Embryo-fetal toxicity
- Malignancies and serious infections
Cost, est. cash$100–$1,500/month
Generic entry2012
Classes and tags
Clinical profile
Legacy pregnancy categoryD
Defining liabilityMajor teratogenicity, myelosuppression and serious infection risk.
Mechanism of action: Mycophenolate mofetil ballicule
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