TECHNOLOGY APPRAISAL GUIDANCE
Liposomal cytarabine–daunorubicin
for untreated acute myeloid
leukaemia
The technology
Liposomal cytarabine–daunorubicin
(Vyxeos, Jazz Pharmaceuticals) is ‘indicated
for the treatment of adults with newly
diagnosed, therapy-related acute myeloid
leukaemia or acute myeloid leukaemia
with myelodysplasia-related changes.’
Liposomal cytarabine–daunorubicin
is given by intravenous infusion over
90 minutes according to the following
schedule:
• For induction of remission: daunorubicin
44mg/m 2 and cytarabine 100mg/m 2 on days
1, 3 and 5 for the first course and on days
1 and 3 for subsequent courses, if needed.
• For consolidation (5 to 8 weeks after the
start of the last induction): daunorubicin
29mg/m 2 on days 1 and 3. A subsequent
course of consolidation may be given
when there is no disease progression or
unacceptable toxicity.
The company stated that the list price
of liposomal cytarabine–daunorubicin is
£4581 per 50-ml vial.The company has
a commercial arrangement that makes
liposomal cytarabine–daunorubicin
available to the National Health Service
(NHS) with a discount and it is the
company’s responsibility to let relevant
NHS organisations know details of the
discount.
Recommendations
Liposomal cytarabine–daunorubicin meets
NICE’s criteria for being a life-extending
treatment at the end of life. Using the
most plausible assumptions and the price
discount, the cost-effectiveness estimates
of liposomal cytarabine–daunorubicin
compared with standard chemotherapy
are within the range that NICE normally
considers a cost-effective use of NHS
resources for end-of-life treatments.
So liposomal cytarabine–daunorubicin
is recommended.
Committee discussion
A potential new treatment option
People with acute myeloid leukaemia that
is therapy-related or with myelodysplasia-
related changes would welcome a new
treatment option.
Therapy-related acute myeloid leukaemia
and acute myeloid leukaemia with
myelodysplasia-related changes are high-
risk types of acute myeloid leukaemia
with poor survival outcomes. Patient
experts described that the most common
symptoms include fatigue, feeling
weak or breathless, loss of memory and
concentration, bruising and bleeding, and
nausea or vomiting. They also highlighted
that the diagnosis has an emotional and
financial effect on patients, and their
families and carers. Both the patient and
clinical experts explained that patients
would welcome a treatment that helps
them be well enough to have a stem cell
transplant, which is potentially a curative
treatment. The committee concluded that
people with therapy-related acute myeloid
leukaemia and acute myeloid leukaemia
with myelodysplasia-related changes would
welcome a new treatment that could
improve survival, quality of life, and the
chance of getting a stem cell transplant.
hospitalpharmacyeurope.com | 2019 | 3