x Abu Dhabi, UAESunday 23 July 2017

Transplant plea for UAE girl, 3, with rare illness

Alia Neama is being treated with Soliris, which costs Dh30,000 per vial.

DUBAI //A three-year-old girl who suffers from a rare genetic disorder needs a kidney transplant in the next two years to save her life.

Alia Neama weighs only 10kg and has been on dialysis for most of her life. The little Emirati girl had the tips of three of her fingers removed after contracting gangrene before she was a year old.

She is being treated with Soliris, a drug that costs Dh30,000 per vial. Alia receives one vial every other week. The costs are covered by Dubai Health Authority.

"Without their support, we wouldn't have our child now," said Alia's father, Abdul Hamid Neama. "She might have passed away within just a few months."

Mr Neama is now desperately seeking a compatible kidney for his daughter. "That is the next challenge," he said.

"I've visited transplant centres in India, and soon in Egypt and other countries. So far, we've had no luck."

In late 2009 Alia was found to have aHUS (atypical haemolytic-uraemic syndrome), which causes kidney failure and blood clotting. When she was nine months old, she travelled to Heidelberg University in Germany for treatment.

However, a delay in having Soliris imported from the United States caused complications and she developed gangrene in both hands and legs.

Although the drug reversed much of the damage, she lost the tips of three of the fingers on her right hand.

The complications were rare enough for the doctors treating her to publish a study, which appeared in the academic journal Pediatrics.

Franz Schaefer, the head of paediatric nephrology who treated Alia at Heidelberg University, said complications that affected Alia were almost unheard of.

"Even for the rare disorder this child has, it was quite unusual," he said. "It's a very rare complication. We had the chance to treat her with this new drug and it stopped the progress of the disorder."

Soliris is a relatively new drug, said Dr Schaefer, but its use is proving promising for other sufferers of aHUS.

"It can block the disease process completely and these children who otherwise have lifelong dialysis or die in early infancy now have a very good prognosis," he said.

"We can keep them on the antibody for extended periods of time, maybe even lifelong. They can have a more or less normal life."

Dr Schaefer said that although the drug can stave off kidney failure, it cannot reverse it, so Alia must undergo a transplant to come off dialysis.

"There was a lot of kidney damage, which was irreversible," he said. "Once she undergoes kidney transplantation, she will need to continue taking the drug to prevent a recurrence of the disease."

Dr Schaefer, who is a visiting doctor in Dubai, said the drug was now available on special prescription in the UAE and is used to treat about seven patients.

Eva Simkova, from the department of paediatrics at Dubai Hospital, said Dubai Health Authority was supporting the cost of Alia's continued treatment and that of other Emirati patients "with some difficulties".

"This drug is extremely expensive," she said. "But it can help those who are suffering from this disease.

"If we can get it early, this treatment can rescue kidneys and they wouldn't have to be on dialysis."

mcroucher@thenational.ae