TREATMENT FOR SICKLE CELL
MAJOR SICKLE CELL BOOST
People affected by sickle cell disease have welcomed news of the first new therapy to be made available for the disease in twenty years…
F
or the first time in 20 years, a new therapy for sickle cell disease is to be made available on the NHS.
Crizanlizumab (Adakveo, Novartis) has been recommended by the National Institute for Health and Care Excellence (NICE) as a treatment option for preventing recurrent sickle cell crises in people aged 16 or over.
It is anticipated that more than 300 people a year will receive the treatment via a Managed Access Agreement (MAA). This figure is anticipated to rise to more than 450 people in subsequent years.
Since there is remains some uncertainty about the long-term effectiveness of the treatment and the cost-effectiveness, however, crizanlizumab has not been recommended for routine use on the NHS at this stage, but will allow patients to access crizanlizumab while additional data is collected through clinical trials to address these uncertainties.
44
scottishpharmacist.com
What is sickle cell disease? Sickle cell disease is the name for a group of inherited a health conditions that affect the red blood cells. The most serious type is sickle cell anaemia.
The disorder is characterised by red blood cells that become sickle-shaped, leading to clumping and blockages in the small blood vessels.
These blockages can cause a loss of blood flow and severe pain in episodes known as vaso- occlusive crises (VOCs), or sickle cell crises. The frequency and severity of VOCs is unpredictable, and each episode can take days or weeks to recover, sometimes requiring hospitalisation.
To date, treatments to prevent sickle cell crises have primarily been a tablet called hydroxycarbamide and regular blood transfusions.
Crizanlizumab is an intravenous treatment option that can be taken on its own or alongside hydroxycarbamide.
Clinical evidence suggests that people treated with crizanlizumab have significantly fewer sickle cell crises in a year than those receiving other standard treatment options.
‘Sickle cell disease can be a debilitating condition that has a huge impact on patients’ quality of life,’ said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE.
‘Yet treatment for sickle cell disease has been limited for years, and there has been a lack of treatments for patients whose lives are affected by the condition. Crizanlizumab is an innovative treatment, which has shown the potential to improve hundreds of lives and we are delighted to be able to recommend it. We don’t yet know whether the benefits will translate into longer-term outcomes, and we look forward to seeing what the data collected through this managed access agreement will uncover about its benefits for the future.’
DISEASE HAS BEEN LIMITED FOR YEARS
Page 1 |
Page 2 |
Page 3 |
Page 4 |
Page 5 |
Page 6 |
Page 7 |
Page 8 |
Page 9 |
Page 10 |
Page 11 |
Page 12 |
Page 13 |
Page 14 |
Page 15 |
Page 16 |
Page 17 |
Page 18 |
Page 19 |
Page 20 |
Page 21 |
Page 22 |
Page 23 |
Page 24 |
Page 25 |
Page 26 |
Page 27 |
Page 28 |
Page 29 |
Page 30 |
Page 31 |
Page 32 |
Page 33 |
Page 34 |
Page 35 |
Page 36 |
Page 37 |
Page 38 |
Page 39 |
Page 40 |
Page 41 |
Page 42 |
Page 43 |
Page 44 |
Page 45 |
Page 46 |
Page 47
