1 What is this medicine
and what is it used for?
Melphalan is a bifunctional nitrogen mustard alkylating agent
that cross-links DNA strands, preventing cancer cell replication and triggering
apoptosis. It is one of the oldest and most established chemotherapy agents,
with particular efficacy in plasma cell dyscrasias.
Oral melphalan 2 mg tablets are used for:
• Multiple myeloma (MM) — oral
melphalan-prednisolone (MP) regimen, historically as standard therapy and still
used in elderly or frail patients not eligible for novel agents or ASCT.
• Ovarian cancer — palliative treatment
of advanced ovarian carcinoma.
• Breast cancer — advanced or
metastatic breast carcinoma (selected cases).
• Polycythaemia vera — disease control
(less commonly now).
2 How to take this
medicine
Take on an EMPTY STOMACH — at least 1 hour before food. Food
significantly reduces melphalan absorption. The dose and schedule vary by
protocol (e.g. MP: melphalan 0.25 mg/kg/day for 4 days every 6 weeks). Swallow
tablets whole with water. Handle carefully — do not crush (cytotoxic).
|
CYTOTOXIC MEDICINE — HANDLING PRECAUTIONS: Melphalan is a cytotoxic alkylating agent — it
can cause cancer (secondary malignancies) and is mutagenic. Women who are pregnant or planning pregnancy
should not handle melphalan tablets directly — wear gloves. After handling: wash hands thoroughly. Store separately from other household
medicines. |
3 Possible side effects
|
Frequency |
Side Effect |
What to Do |
|
Very Common (>10%) |
Myelosuppression (bone
marrow suppression) |
Dose-limiting
toxicity. CBC before each cycle — hold or reduce if counts too low. |
|
Very Common (>10%) |
Nausea / vomiting |
Take anti-emetics as
prescribed; take on empty stomach. |
|
Very Common (>10%) |
Alopecia |
Usually reversible
after treatment ends. |
|
Common (1–10%) |
Mucositis / stomatitis |
Gentle mouth care;
avoid irritants; mouth rinses. |
|
Common (1–10%) |
Diarrhoea |
Hydration; loperamide
if needed. |
|
Uncommon |
Secondary malignancies
(leukaemia) |
Long-term risk;
monitor with regular blood tests. |
|
Uncommon |
Pulmonary fibrosis |
Report new or
worsening breathlessness. |
|
Seek help urgently |
Febrile neutropenia
(fever > 38°C after chemotherapy) |
Emergency: IV
antibiotics within 1 hour. |
4 Contraindications
Melphalan is contraindicated in: patients with recent
myelosuppressive chemotherapy or radiation without haematological recovery;
known hypersensitivity; and pregnancy (teratogenic — effective contraception
required during treatment and for 6 months after for both males and females).
5 Drug interactions
• Ciclosporin: significantly increases
melphalan nephrotoxicity — use with caution, monitor renal function.
• Live vaccines: contraindicated during
chemotherapy.
• Nalidixic acid: potential increased
risk of haemorrhagic colitis — avoid.
• Carmustine: additive pulmonary
toxicity.
6 Storage
Store at 2–8°C (refrigerate). Original packaging, protect
from light and moisture. Cytotoxic — handle with care, wear gloves. Keep out of
reach of children. Return unused tablets to the pharmacy.
7 Prescription
requirement
|
PRESCRIPTION ONLY MEDICINE (POM) — Specialist
haematology/oncology prescription. Cytotoxic medicine — dispensing requires
cytotoxic-trained pharmacist verification. CBC monitoring before each
treatment cycle is mandatory. |
8 Guidance for patients
& caregivers
Melphalan must be taken on an empty stomach — food can reduce
the amount of medicine absorbed, reducing its effectiveness. Take it at least 1
hour before eating your meal.
Bone marrow suppression is the most important side effect.
Blood counts will be checked before every treatment cycle. If you develop a
fever above 38°C at any time after taking melphalan, go to the emergency
department immediately — this may be febrile neutropenia and requires urgent
treatment.
Wear gloves when handling the tablets if you are a caregiver.
Pregnant women should not handle melphalan.
9 Pharmacist &
prescriber notes
Oral melphalan is erratically absorbed — bioavailability
ranges from 25–89%. Absorption is significantly reduced (by 39–45%) by food;
hence the fasting instruction. For myeloma, intravenous high-dose melphalan
(HDM — 200 mg/m2) is used as conditioning for autologous stem cell transplant —
a separate IV formulation.
The oral 2 mg tablet is used for chronic outpatient treatment
in elderly patients. Complete blood count (CBC) nadir occurs at 14–21 days
after each cycle — monitoring at day 14 and 21 is standard. Secondary
myelodysplasia and acute leukaemia risk increase with cumulative melphalan
exposure — typically after 2+ years of continuous treatment.
10 Frequently asked
questions
If I have a fever, why is it an emergency?
After melphalan, your white blood cell count is at its lowest
around 2–3 weeks after treatment. A fever at this time may indicate a serious
infection (febrile neutropenia) that can be life-threatening without prompt IV
antibiotic treatment.
Is melphalan still used now that newer myeloma medicines
are available?
Yes — melphalan-prednisolone remains an option for elderly or
frail patients with multiple myeloma who cannot tolerate more intensive
regimens. For younger, fitter patients, high-dose melphalan with autologous
stem cell transplant remains the gold standard consolidation approach.
Can I have melphalan as an infusion instead of tablets?
High-dose IV melphalan is used as conditioning chemotherapy
before stem cell transplantation — this is a different, much higher dose than
the oral regimen. Your haematologist will determine which formulation is
appropriate.
Will my hair fall out?
Alopecia (hair loss) can occur with melphalan, though it is
often partial rather than complete. Hair usually regrows after treatment is
completed.
Can a caregiver crush the tablet if I have swallowing
difficulties?
No — melphalan tablets should never be crushed as this
creates cytotoxic dust. If swallowing is difficult, discuss alternatives (IV
formulation or other agents) with your haematologist.