A woman with a rare blood disorder that left her drained of energy has hailed a new drug as ‘superwoman juice’.
Jess Ratcliffe, 31, was diagnosed with paroxysmal nocturnal haemoglobinuria (PNH) in 2016, which causes red blood cells to break down.
The condition, which currently only affects 600 to 800 people in the UK, can lead to anaemia, kidney problems and life-threatening blood clots if not managed properly.
Ms Ratcliffe, from Surrey, required blood transfusions at home every two weeks to keep the illness at bay.
But the new therapy ravulizumab – which costs around £300,000 per patient per year – only needs to be given every eight weeks, which Ms Ratcliffe said was ‘life changing’.
She now has normal energy levels for seven weeks rather than 10 days at a time, allowing her to work and live normally. Ms Ratcliffe said the medication will allow her to go on holiday.
The personal development coach told MailOnline: ‘The new drug is life-changing – I call it my superwoman juice!’
Jess Ratcliffe, 31, was diagnosed with Paroxysmal Nocturnal Haemoglobinuria (PNH) in 2016, which causes red blood cells to break down. She said it’s ‘hard to put into words’ how big an impact the treatment has on her life
Ravulizumab – made by Alexion Pharmaceuticals and sold as Ultomiris – is given to PNH patients through an IV infusion every eight weeks and takes two hours to administer. The drug, which costs £300,000 per patient per year, is longer-lasting than the old treatment eculizumab, which had to be administered every two weeks
Ms Ratcliffe was diagnosed with PNH after antibiotics and penicillin failed to ease her ongoing flu-like symptoms and sore throat.
Medics suspected she had a vitamin or iron deficiency, but further tests revealed she had the rare blood condition.
Ms Ratcliffe said she had never heard of the condition – which can leads to an iron deficiency – before her diagnosis.
‘I was just about to go and work in the US so was given an infusion of what should have been five years’ worth of iron to see me through,’ she said.
‘But less than six months later a blood test found that my red blood cells had churned all the iron.’
Ms Ratcliffe then required infusions of eculizumab every two weeks, the only drug used to treat the condition until four months ago.
But the National Institute for Health and Care Excellence (NICE), the NHS’ watchdog, in May said doctors should instead use longer-lasting drug ravulizumab.
The recommendation followed trials of ravulizumab on 400 patients worldwide, which showed it was just as effective as eculizumab, but required less frequent treatments.
More than a tenth of the participants were based at King’s College Hospital and Leeds Teaching Hospital.
What is paroxysmal nocturnal haemoglobinuria?
Paroxysmal nocturnal haemoglobinuria (PNH) is a very rare disorder that causes the immune system to attack the body’s red blood cells.
The process where the red blood cells are destroyed is called ‘haemolysis’ and it is responsible for many of the symptoms of the disease.
PNH affects between 600 and 800 people in the UK and patients have a normal life expectancy.
Some sufferers may have no symptoms and do not need treatment, but others can experience lots of complications.
The illness can cause anaemia – low levels of red blood cells, which are responsible for carrying oxygen around your body – life-threatening blood clots, kidney failure and dependence on transfusions.
Its symptoms include dark or black-coloured urine, anaemia, breathlessness, difficulty swallowing, abdominal pain, erectile dysfunction, fatigue, jaundice, blood clots and kidney damage.
It is diagnosed through a series of tests, including a full blood count and urine samples.
Source: PNH National Service
Ravulizumab – made by Alexion Pharmaceuticals and sold as Ultomiris – is a modified antibody that targets the same protein as the old drug, but has the ability to stay in body and gets recycled.
NICE said the increased time between treatments could improve patients’ quality of life, as well as increase their independence and psychological wellbeing.
Some patients still have the old treatment eculizumab but it is used very rarely and mainly in pregnant patients.
Following the recommendation, Ms Ratcliffe now receives the new long-term treatment six times a year, which takes two hours for a community nurse to administer at her home.
Ms Ratcliffe, who has set up her own business since her treatment frequency has been cut, said: ‘It’s hard to put into words the positive impact that the two-monthly (instead of two-weekly) treatments continue to have on my life and wellbeing as a PNH patient.
‘Unlike some other people with the condition, I thought I hadn’t experienced chronic fatigue; that was until I was given the new treatment and noticed the difference it made to my energy levels.
‘The new drug has given me my freedom back and I’m so grateful.’
Ms Ratcliffe said the treatment will allow her to go on holiday and has massively impacted her energy levels.
When she was receiving the old treatment her energy levels would drop in the days leading up to treatment, which had a significant impact on her quality of life.
She would purposefully not schedule too many activities in the days leading up to her infusion, as she would be too tired for them.
But Ms Ratcliffe now has seven weeks – rather than 10 days – of normal energy levels, meaning she can work and live life more normally.
King’s College Hospital treats around 360 PNH patients. Around 80 are receiving the new therapy, while the rest are participating in clinical trials.
Leeds Teaching Hospitals is the only other place in the country offering the drug.
Dr Austin Kulasekararaj, a consultant haematologist who led the trial at King’s College Hospital, said, ‘At King’s, we pride ourselves on helping to develop new treatments and ground-breaking surgical techniques to help improve patient care.
‘The licensing of this new drug is a big step forward for patients with PNH.
‘It will significantly reduce the amount of time they have to spend in hospital or a home receiving treatment, which will have a profoundly positive impact on their lives.’
Dr Morag Griffin, a consultant haematologist at Leeds Teaching Hospital, said, ‘We are delighted with this new therapy which will have a dramatic effect on the quality of life of so many of our patients. This is a positive step forwards.’