A PENSIONER who was refused treatment for a life-threatening blood condition has finally received a "miracle" drug to treat the disease, but only after she suffered a potentially fatal blood clot.
Joyce Juszczak's family say they are angry that it took her admission into hospital with the clot, which destroyed one-third of her kidney, for NHS Greater Glasgow and Clyde to reverse their decision not to prescribe her eculizumab.
The 65-year-old, who appeared at the Scottish Parliament with her daughters to press for access to the drug for paroxysmal nocturnal haemoglobinuria (PNH), had failed in a series of appeals over six months, including a review of her case after an intervention from Health Secretary Nicola Sturgeon.
Her consultant submitted a new application for the drug to NHS GGC last Tuesday after she was admitted to Inverclyde Royal Hospital and the family were told it had been approved on Friday afternoon. She received her first dose of the drug the same day and her family said yesterday that she already looked a lot better.
But they remain angry that it has taken a life-threatening incident to get the health board to change its mind over the life-saving treatment for PNH, a potentially deadly condition which causes red blood cells to explode and leaves victims at risk of kidney failure and potentially fatal blood clots.
One of Mrs Juszczak's daughters, Beverley Hardie, told The Herald yesterday: "I think it is disgraceful she had to have a life-threatening clot before they would agree to fund this treatment. If the clot had gone to her brain, heart or lungs she probably wouldn't have survived."
The decision to give Mrs Juszczak the drug came after one-third of her left kidney developed an infarct, an area of tissue death due to a local lack of oxygen. She needed an emergency blood transfusion and has been in hospital since.
Mrs Juszczak's consultant haematologist, Dr Henry Hambley, who accompanied the pensioner to an initial appeal the day before he retired, said: "I am over the moon she has now been started on eculizumab. As far as I am concerned that should have happened before now.
"Thankfully the infarct occurred in an organ where there is considerable redundancy. If it had been her brain then it would have been a completely different story."
Patient groups, which have used Mrs Juszczak's case to highlight how Scotland is failing more than 350,000 people affected by a rare disease, have demanded an advisory group be set up to deal with specialist medication, similar to that already established in England and Wales.
They have called on Ms Sturgeon to end the "postcode lottery" which allows eculizumab to be prescribed on the NHS in England, Wales and Northern Ireland, while it remains not recommended for use in Scotland by the Scottish Medicines Consortium.
An NHS Greater Glasgow and Clyde spokeswoman said: "This patient has been regularly reviewed by a consultant haematologist and a new Individual Patient Treatment Request process has concluded the patient's condition now meets the clinical criteria for this drug to be prescribed."
Asked why it took a potentially fatal clot before Mrs Juszczak got the drug, the health board said: "The criteria for the prescription for this drug are set by the SMC so we would suggest that you speak with the SMC."
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