Esme’s story – November 11, 2014

Esme, 5

Little girl living with aHUS granted compassionate access to Soliris, Perth

Esme face paintDiagnosed with the ultra-rare, life threatening blood disease atypical haemolytic uraemic syndrome (aHUS) at nine-months of age, Esme, now 5, Perth was privileged to be granted compassionate access to the only treatment that would ultimately save her life, Soliris (eculizumab) in May 2010, aged one.

This is her story.

Travelling around Singapore with her family in November 2009, then nine-month old Esme started experiencing an upper respiratory infection. This prompted her mother, Yee-Tching to take her to the children’s hospital, where she was diagnosed with bronchiolitis.

While in hospital, doctors noticed Esme’s iron levels were low and advised she undergo blood tests after returning home with her family.

After arriving in Perth a few days later, Yee-Tching took Esme to the Princess Margaret Hospital for Children to undergo what she thought, would be a routine blood test.

Following the tests, doctors identified something much more sinister than low iron levels.

After asking Yee-Tching many questions about her child’s health, which she explained as “fairly healthy prior to the bronchiolitis,” doctors diagnosed Esme with the ultra-rare, life threatening blood disease, aHUS.

“My background is in science, yet I had never heard of aHUS. We were completely unaware of what it was,” said Yee-Tching.

“Esme’s doctor had never seen it before. He had seen HUS, but never an atypical case.

“It was a mystery and he had to contact colleagues internationally to confirm the diagnosis,” Yee-Tching said.

The diagnosis came as a huge shock to both Yee-Tching and her family.

“It was a shock for us to go from a normal life, to Esme being diagnosed with this ultra-rare blood disease.

“I had no idea how long Esme would have to spend in hospital. She became so sick, and ended up spending more than three weeks in the hospital,” said Yee-Tching.

“Following her diagnosis, Esme spent a full week in hospital before being released. She was only out for two days before we had to return to hospital, where she then spent the next three weeks.”

Out of hospital for only a few weeks, on Christmas Eve 2009, Esme returned to hospital for a routine blood transfusion, after which Esme experienced a “Code Blue” Esme purple fairyepisode, during which she stopped breathing.

By this point, Yee-Tching had started to notice Esme wasn’t growing as quickly as a normal child of her age. The aHUS had delayed other aspects of her development, for she was often exhausted and weakened by her disease.

Esme’s renal specialist had heard about a treatment called Soliris (eculizumab) and approached the manufacturer to see if Esme could be included in a clinical trial to access the treatment on compassionate grounds.

In May, 2010, aged one, Esme was granted compassionate access to Soliris.

“All we knew is that Esme was getting access to an experimental treatment, and we had to see how she went, and if it would work,” Yee-Tching said.

“Before getting Soliris, we were never sure of what Esme’s future looked like. It was very uncertain as to what each day held for her.

“It has become such a routine for her and for us now, that she goes in once a fortnight for her Soliris treatment, otherwise she is just a happy, normal five-year old.”

Describing the treatment as having significantly transformed Esme’s life, allowing her to attend school and pursue a pretty normal life, Yee-Tching said, “Within two-to-three months of getting access to Soliris, Esme started growing again and behaving more like a baby of her age.”

Explaining her excitement at the recent news that Soliris will be listed on the Pharmaceutical Benefits Scheme (PBS) on December 1, 2014, Yee-Tching said, “It is really exciting news that Aussies living with aHUS will get Soliris. It has really changed Esme’s life and has allowed her to live a relatively normal life.

“We are pleased for everyone who will be able to now access Soliris, but still unsure of what the conditions might mean for Esme. We are worried to that end,” said Yee-Tching.

For more information about Australians living with aHUS, head towww.ahusaustralia.org.au

To join the conversation:

‘like’ us on Facebook [www.facebook.com/AhusPatientSupportGroupAustralia] and

‘follow’ us on Twitter [@apsga_org].

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