King’s uses new treatment for ultra-rare blood condition

“The new drug is life-changing – I call it my Superwoman Juice!”

Jess Ratcliffe

King’s College Hospital in London has begun using a new longer-lasting treatment for Paroxysmal Nocturnal Haemoglobinuria (PNH), following extensive clinical trials and subsequent rollout by the NHS in England.

Ravulizumab has been approved to treat the ultra-rare condition, whereby red blood cells break down and patients – who can suffer from repeated life-threatening blood clots – are required to have frequent blood transfusions.

The new, highly effective treatment, which underwent extensive testing, only requires six treatments per year compared to the old therapy, which had to be given every two weeks.

Of the 400 trial patients who took part in the study globally, 46 were from two centres in England: King’s College Hospital and Leeds Teaching Hospitals. The first patient enrolled in 2015.

Dr Austin Kulasekararaj, Consultant Haematologist, who led the trial at King’s College Hospital with 32 patients, 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.”

Jess Ratcliffe, 31, from Surrey, was diagnosed with PNH in 2016 after experiencing ongoing flu-like symptoms and a sore throat that wasn’t responding to antibiotics or penicillin. Medical professionals initially suspected a vitamin or iron deficiency but after extensive tests she was diagnosed with the rare condition.

Jess said, “I had never heard of the condition before my 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, but less than six months later a blood test found that my red blood cells had churned all the iron.”

Jess then required two-weekly infusions of medication before she joined the clinical trial at King’s.

She added, “The new drug is life-changing – I call it my Superwoman Juice! 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.”

Now on two-monthly infusions, which are administered over a two-hour period by a community nurse at her home, Jess has made a number of life-changing decisions, including setting up her own business.

Dr Morag Griffin, Consultant Haematologist at Leeds Teaching Hospitals, 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.”

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