Compassionate supply of Soliris to cease following PBS listing – November 17, 2014

Breathing borrowed airMedical receptionist, Sarah, 28, Cairns, had lived her entire life with Cystic Fibrosis (CF) until September, 2013 when she received a double lung transplant.

Following transplantation, Sarah visited hospital for weekly blood tests and follow-ups, with a biopsy confirming her treatment had started to compromise her kidney function. While her lungs had significantly improved, Sarah’s kidneys began to fail.

In April this year, Sarah was diagnosed with the ultra-rare blood disease, atypical haemolytic uraemic syndrome (aHUS), and informed by doctors that she would need to spend the next five weeks undergoing treatment with plasma exchange.

With the long-awaited PBS-listing of Soliris from December 1, 2014, the only treatment available to treat the ultra-rare disease, aHUS, Sarah explains what this means to her.

“I’ve actually already been granted compassionate supply of Soliris but the government won’t give me access because my blood levels don’t prove I have aHUS. I have to be flaring. So at the moment, it’s waiting at the hospital in the fridge,” Sarah said.

“My creatinine levels went down to 97µmol/L [close to the normal range] after being on plasma exchange. In order to prove I have aHUS, my creatinine level needs to be elevated.

“So I stopped plasma treatment 3 weeks ago and I’m now back in kidney failure with a creatinine level of 185µmol/L,” said Sarah.

“I’m on no treatment and can feel the effects. I feel constantly drained and I don’t know how my body will handle it.

“The only other way to prove I have aHUS is through a kidney biopsy, however this has to have been done in the past month, and my last biopsy was in June,” Sarah said.

“I’m scheduled for a kidney biopsy in two weeks’ time and only until then, will I be granted access to Soliris.

“Next week, I’m due to go to Melbourne for my first holiday since my lung transplant and I’m determined not to let anything stop me,” said Sarah.

“The thought of finally having access to Soliris is nerve-wracking. I’ve heard there are a number of side-effects associated with the treatment and I am on a number of medicines from my transplant, which give me terrible side-effects.

“Once that’s over, I’ll be more excited about it,” said Sarah.

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