But what is sickle cell disease, what are the symptoms and what is the new treatment crizanlizumab?
Here’s everything you need to know.
What is sickle cell disease?
Sickle cell disease is the name for a group of inherited health conditions that affect the red blood cells. The NHS website notes that the most serious type is called sickle cell anaemia.
People with sickle cell disease produce unusually shaped red blood cells that can cause problems because they do not live as long as healthy blood cells and can block blood vessels.
Sickle cell disease is particularly common in people with an African or Caribbean family background.
The disease is a serious and lifelong health condition, although treatment can help manage many of the symptoms.
However, a recently groundbreaking inquiry into sickle cell disease has found “serious care failings” in acute services, as well as evidence of attitudes underpinned by racism.
The report by the all-party parliamentary group (APPG) on sickle cell and thalassaemia, which was led by Pat McFadden MP, found evidence of sub-standard care for sickle cell patients admitted to general wards or attending A&E departments.
The inquiry also found widespread lack of adherence to national care standards, low awareness of sickle cell disease among healthcare professionals, and examples of inadequate training and insufficient investment in sickle cell care.
What are the symptoms of sickle cell disease?
Sickle cell disease can cause a wide range of symptoms which can start from a few months of age, although many children have few or no symptoms if treatment is started early on.
The main symptoms are:
- painful episodes
- getting infections often
Episodes of pain known as sickle cell crises are one of the most common and distressing symptoms of sickle cell disease, which happen when blood vessels to part of the body become blocked.
The pain can be severe and lasts for up to 7 days on average.
A sickle cell crisis often affects a particular part of the body, such as the:
- hands or feet (particularly in young children)
- ribs and breastbone
- legs and arms
However, how often someone with sickle cell disease gets episodes of pain varies a lot.
What is the new treatment for sickle cell disease?
The new treatment is a drug called crizanlizumab and is made by Novartis. It works by being injected into a vein and can be taken on its own or alongside standard treatment and regular blood transfusions.
During a trial, patients taking the crizanlizumab had a sickle-cell crisis 1.6 times a year on average, compared with nearly three times a year normally.
However, because the trial was small and lasted only a year, it currently remains unknown how long the benefits last for.
The National Institute for Health and Care Excellence (NICE) - which recommends treatments in England and Wales - is recommending the use of crizanlizumab for over-16s, although this will be under a special arrangement as NICE has not yet recommended its routine use in the NHS.
Additional data on the treatment will also be collected through clinical trials.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: "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... has shown the potential to improve hundreds of lives and we are delighted to be able to recommend it as the first new treatment for sickle cell disease in two decades."