NICE has today (Friday, 21 March) recommended ruxolitinib, also
known as Jakavi and made by Novartis, as a new treatment option
for people with acute graft versus host disease (GvHD) that has
not responded to standard steroid treatment.
GvHD is a serious complication after a life-saving bone marrow or
stem cell transplant from another person. It happens when white
blood cells in the donated bone marrow or stem cells attack the
patient's own organs.
The treatment, which is available in the NHS from today, is
recommended for people aged 12 and over and around 300 people in
England could benefit each year.
Currently, about 1,200 patients undergo donor stem cell or bone
marrow transplants annually, with just under 600 people
developing acute GvHD. About half of these cases do not respond
to standard steroid treatment. As such there is a need for
additional treatment options.
Acute GvHD can affect the skin, liver, and gastrointestinal
tract. Symptoms often include abdominal pain or cramps, nausea
and vomiting, and diarrhoea. It can lead to some people
struggling to swallow due to mouth ulcers, which can cause
extreme weight loss, and in severe cases a feeding tube might be
needed.
People with acute GvHD may have to be regularly admitted to
hospital due to infection and, in severe cases, the condition can
be life-threatening.
Recognising the severe nature of the disease NICE's independent
appraisal committee applied a 1.2 severity weighting.
Ruxolitinib, taken as a twice-daily pill, works by blocking
specific enzymes (Janus Associated Kinases) that trigger
inflammation, effectively reducing symptoms of the
condition.
Ruxolitinib is recommended as an alternative to the current
second-line treatments used for acute GvHD that responds
inadequately to steroids.
This treatment provides an important alternative to current
second-line therapies used for steroid-resistant acute GvHD.
Unlike existing treatments that often require hospital visits –
such as extracorporeal photopheresis (where a patient's blood is
removed, treated, and returned to their body) – ruxolitinib can
be taken at home.
This significantly reduces hospital outpatient visits and the
associated stress and inconvenience for patients.
Clinical trial evidence shows that steroid resistant acute GvHD
is more likely to improve with ruxolitinib than with standard
care.
Read the full final draft guidance for Ruxolitinib for treating acute
graft versus host disease refractory to corticosteroids in people
aged 12 and over.
