Since receiving a double lung transplant for cystic fibrosis in 2013, medical receptionist Sarah, 29, Cairns, has been battling the ultra-rare blood disorder, atypical haemolytic uraemic syndrome (aHUS).
On December 17, 2014, following kidney failure after a trial period off her regular maintenance therapy, Sarah received subsidised access to Soliris (eculizumab). Within five weeks, Sarah noticed a ten-fold improvement in her kidney function.
Last week, Sarah’s medical team prepared an application with the Health Department’s newly established Expert Review Group (ERG), for renewed access to Soliris. Sarah was resoundingly rejected, leaving her at risk of kidney failure and potentially, death.
This is Sarah’s story.
“When I was six weeks old, I was diagnosed with cystic fibrosis, a genetic condition that produces extra mucus in the lungs leading to ongoing infections and eventually lung damage.
“In 2013, I was lucky to receive a double-lung transplant, however, during a routine blood test, doctors noticed my kidneys had gone into failure,” said Sarah.
“I was subsequently diagnosed with aHUS, which developed as a direct result of immunosuppressant medication I was receiving as a life-long precautionary measure to protect my transplanted lungs.”
Sarah’s medical team initiated her on standard, maintenance treatment for aHUS, plasmapheresis or plasma exchange, where blood plasma is removed and replaced into the blood system.
“I’d been on plasma exchange, a treatment that requires re-transfusing new plasma. But this is more of a supportive treatment,” Sarah said.
“Once Soliris was subsidized, my doctors applied for access on my behalf. However, it took a while for my application to be approved.
“In order to access treatment, I had to come off plasma exchange, which caused my kidneys to fail very quickly, within two weeks,” said Sarah.
“I also had to have a second kidney biopsy to prove my case, prior to being granted access.
“On December 17, 2014, I received my first subsidised dose of Soliris. Within five weeks, my kidney function reportedly improved ten-fold,” Sarah said.
While on Soliris, Sarah’s shorter, day patient stays have enabled a significant improvement in her quality of life.
“Over the last 12 months I’ve been in hospital once a fortnight for a couple of hours, compared to four or six hours, three times a week while on plasma exchange,” said Sarah.
“I’ve been able to work more frequently, go away on holidays, go camping with my fiancé and friends, and have fun. These may be things people take for granted, but are impossible when on constant plasma exchange.
“Most importantly, I feel a lot healthier since being on Soliris, which is what really matters,” Sarah said.
“I no longer feel exhausted from a failing kidney. I finally feel like me. I went snorkelling this year, at the Great Barrier Reef, which is something I’d always dreamed of doing. I also hiked up to a lighthouse on Fitzroy Island, which was another really amazing experience.”
Sarah was initially placed on weekly Soliris infusion, however, following the success of her treatment, she has been receiving fortnightly infusions which have strongly improved and stabilised her kidney function.
Sarah was preparing to have her ensuing Soliris dose on Tuesday, December 8, 2015, when her doctor rang to advise her renewed access application to the newly formed ERG, had been refused.
“I was shocked and devastated when my doctor rang me. We all believed I had a strong case for renewed access, and, as a double-lung transplant recipient, was well-placed to receive a positive outcome,” said Sarah.
“I’d also visited the Federal Health Minister’s advisor, Alex Best, together will fellow member and aHUS Patient Support Group Australia (aPSGA) Treasurer, Debbie Basham, to plead the need for continuing access to the life-saving treatment, for our entire aHUS community, as for many of us, the initial 12-month continuous access agreement was drawing to an end.
“Last month, Mr Best assured us it was his top priority to address the dire situation,” Sarah said.
“Now I’m being told that I need to come off my immunosuppressants in order to stay on Soliris, and I’m absolutely dumbfounded.
“I need to be on immunosuppressants for the rest of my life. Without access to my immunosuppressants, I will die from lung failure. Without access to Soliris, I will die from kidney failure,” said Sarah.
When placed on Soliris, Sarah began planning her wedding for October, 2016. Now, forced off and rejected from treatment Sarah needs to readjust her life.
“I’m now faced with going back onto plasma exchange. This will have a massive toll on my life. It means I won’t be able to meet my work commitments, and I fear I’m going to go back to being sick all of the time, or too tired to really do anything. Most importantly, I’m afraid my kidneys will fail again,” Sarah said.
“I was looking forward to getting married next year, and to having a have a secure future with my soon-to-be husband.
“The Minister’s ERG is forcing me back into kidney failure, but I’m prepared to continue my fight for life, so that I, and other Australians battling the disease, can secure continuing, lifetime access to Soliris,” said Sarah.
“We will fight, until the Minister and her Department understand what we’re going through.
“We’re not just a dollar figure. We’ve got hopes, dreams and families who’ll be shattered if anything happens to us,” Sarah said.
“Soliris has allowed my kidney function to stabilise, and I want to keep it that way.
“The aPSGA and I urge The Hon. Sussan Ley MP, to review my case again, to disclose all members of the ERG to the public, rescind their authority temporarily and delay any formal implementation of any restrictions for patients accessing Soliris, until after the holiday season, to give aHUS patients and our respective families, peace of mind.”
To support Sarah and share in her trials and tribulations, head to her ‘Breathing Borrowed Air’ Facebook page.