Small mercies; Small mercies: who gets to grow?

23 OCTOBER 2004

By LINLEY BONIFACE

FEATURE

Despite international recommendations that growth hormone treatment should be made available to children who are born unusually small, an Auckland panel of doctors won’t ask Pharmac to supply more of the necessary drug. Linley Boniface reports on the children who get left behind.

On his fourth birthday, Finlay Forsyth was too weak to blow out the candles on his cake. It was hard to believe Finlay was four. His feet were the same size as his 18-month-old brother’s, and he wasn’t strong enough to take the lid off a milk bottle.

Finlay was shy, nervous and reluctant to try anything new, and found children his age too intimidating to play with.

Eight months on, Finlay seems like a different boy. He has learned to jump, to stand on one leg, to bounce on the trampoline and to pedal his trike. Finlay’s physical growth has been matched by a growth in self-confidence, and he now plays games, makes jokes and has new friends.

But Finlay’s transformation has come at a huge cost. From now till Finlay goes through puberty, his parents expect to spend $600,000 on the daily injections of the synthetic growth hormone he needs to maintain his development. Instead of paying off the mortgage on their Wellington home, Julia and Mark Forsyth are putting every cent into Finlay.

Finlay has a very rare disorder called Russell-Silver syndrome, part of a growth disorder category called small for gestational age (SGA). SGA babies are born small, but 90 per cent of them catch up on their growth by the age of two. The other 10 per cent remain shorter than average, have feeding problems, may suffer from hypoglycaemia (low blood sugar) and may take longer to develop physical skills. They have an increased risk of diabetes and heart disease.

In February, the Forsyths began paying for Finlay to have the injections of growth hormone. Most children Finlay’s age grow five to seven centimetres a year; on growth hormone, Finlay is expected to grow 12 centimetres a year. The average adult male with Russell-Silver syndrome is 1.51 metres (4ft 11ins) tall, but Finlay is now on course to reach a normal adult height.

Studies have found that very short adults find it harder to get jobs and make friends, but Finlay’s parents are not motivated solely by concerns about height. The Forsyths and their doctor believe that if Finlay hadn’t had the injections, he would have continued to suffer from ongoing hypoglycaemia, which can interfere with learning development and, in extreme cases, cause brain damage or death.

Finlay has trouble eating and keeping food down, and has had a gastrostomy tube in his stomach since he was two. At night, a pump feeds him continuously to supply him with calories and keep his blood sugar levels stable. He is often ill, and had pneumonia last year.

It would be easy to assume the Forsyths are paying for Finlay’s injections because New Zealand’s health service cannot afford to fund them. But the New Zealand Growth Hormone Committee, which decides who will receive growth hormone treatment, has not asked Pharmac to increase its annual allocation of growth hormone, and consistently underspends its budget.

Yet Finlay’s doctor, Wellington Hospital paediatric endocrinologist Esko Wiltshire, says Finlay needs the drug. The Forsyths have also consulted three of the world’s leading specialists on Russell-Silver Syndrome, and all agree that Finlay needs the drug. Only the committee, which has never examined Finlay or reviewed his three volumes of medical notes, says he doesn’t.

The committee has rejected two applications for growth hormone for Finlay and one appeal. It originally argued that Finlay was a millimetre too tall to meet the guidelines — even though the margin for error in measuring height is plus or minus 1cm. Six months later, when Dr Wiltshire asked the committee for leave to reapply because Finlay now fell well within the height guidelines, he was told the latest measurement was probably inaccurate and he should wait another six months.

Having failed to meet the guidelines on height grounds, Finlay’s only other chance of being funded for treatment was to be found to be deficient in growth hormone. The standard tests — which aren’t perfect — suggest Finlay doesn’t have growth hormone deficiency. However, he doesn’t make enough growth hormone to cope with physiological stress, like hypoglycaemia.

This is where most SGA children fail to make the cut: studies suggest they are not deficient in growth hormone, but need more of it than the average child. In addition, looking at a child’s bone age — a standard way of calculating adult height — is an unreliable way of predicting an SGA child’s eventual height.

Dr Wiltshire was very surprised Finlay’s applications were turned down, particularly in light of his additional serious medical problems. “Finlay has responded to treatment like a child with growth hormone deficiency, which is the best test of growth hormone deficiency,” he says. “Finlay hasn’t been in hospital with hypoglycaemia since he has been on growth hormone treatment. It has made a big difference to his muscle mass, and therefore to his physical abilities and his overall confidence.”

RICHARD STANHOPE, a paediatric endocrinologist at London’s Great Ormond Street Hospital for children and an international expert on Finlay’s condition, examined Finlay and wrote directly to committee chairman Wayne Cutfield to urge the committee to change its decision.

When told it had ignored his recommendation, he told the Forsyths he felt New Zealand “suffered from severe tunnel vision”.

Finlay is not alone in being rejected by the committee. In 2001, 45 new applications were made to it for growth hormone treatment. All the applications came from expert clinicians, yet 20 of the 45 were rejected. Last year, 20 out of 54 new applications were rejected.

At least nine other families in New Zealand are also paying for growth hormone treatment themselves because they were refused state funding.

Jan Polaschek, chairwoman of the Magic Foundation for children with growth problems, says she is becoming increasingly concerned about the way SGA children are treated. “We’re hearing from parents that doctors aren’t even telling them about growth hormone because they don’t want them to be disappointed.”

The argument that growth hormone treatment is used purely for cosmetic reasons cuts little ice with Ms Polaschek. “Growth hormone treatment isn’t just cosmetic: it helps the heart muscles grow, and it helps children develop an appetite so they have fewer feeding problems. Children who are severely short and weak can suffer lack of confidence and low self-esteem, but growth hormone treatment overcomes lots of these problems,” she says.

“There’s never been any cost-benefit analysis done into how growth hormone treatment saves money on the problems SGA children could develop further down the track. At the very least, we should have a system that considers the international research in a transparent way and has a proper appeals process.”

AFTER their application was rejected, the Forsyths appealed to the exceptional circumstances panel. Dr Wiltshire told the panel that every international paediatric endocrinologist he had talked to about Finlay believed a trial of growth hormone therapy would be the most appropriate way to manage his condition.

The panel agreed Finlay met their criteria of having a “rare and unusual combination of circumstances”. But, though he met the criteria, the panel decided to leave the decision as to whether Finlay needed growth hormone to the committee.

The Forsyths then took their case to the pharmaceutical therapies advisory committee, which reviews medical literature and advises Pharmac on which drugs are effective. They submitted more than 45 international research papers confirming that growth hormone treatment was appropriate for SGA. One of the papers, from a consensus development conference of the International SGA Advisory Board, includes the name of Dr Cutfield. Yet the pharmaceutical therapies advisory committee decided not to recommend any widening of access to growth hormone treatment for SGA children.

When the Forsyths made a final appeal to the health minister, health officials refused to take action – again, after seeking advice from the growth hormone committee.

The committee is made up of three Auckland paediatric endocrinologists: Dr Cutfield, Paul Hofman and Alister Gunn. Their decisions are not subject to peer review. They are contracted by Auckland University Services, which, in turn, holds the contract with Pharmac to administer growth hormone treatment in New Zealand.

Mr and Mrs Forsyth believe there is a clear conflict of interest in expecting the committee to determine the best medical treatment for patients while being contractually accountable for minimising the cost of that treatment. The Forsyths believe the committee should be responsible only for examining the medical case for each application, leaving funding issues to Pharmac.

They want endocrinologists from a range of institutions around New Zealand to be represented on the committee, and for parents to be offered the option of splitting the costs of growth hormone treatment with Pharmac.

The committee declined to comment on this story, referring all questions to Pharmac. Pharmac medical director Peter Moodie declined to discuss Finlay’s case, on the grounds that it would be “unfair and inappropriate”. “I’m no expert in this area, but the people who made this decision are,” he says.

Dr Moodie says he regards the committee members as independent, despite the fact that they work together, and sees no need for a more rigorous appeal process.

“We have the opinion of three very well-respected clinicians in Auckland who run the committee with the best and most ethical of intentions.”

He says the committee’s guidelines aim at helping children who are severely short. Widening the criteria might mean Pharmac couldn’t afford to pay for drugs for people with other conditions.

Asked why the committee never uses up all its allocation of growth hormone, let alone asks for any more, Dr Moodie says there are times when a child on the treatment might not need as much of the drug as expected. The committee has to be very careful to ensure none of the drug is wasted.

For the Forsyths, there are no avenues of appeal left. Ironically, Finlay’s treatment has worked too well for him to ever qualify for state funding in the future.

“Obviously this is a huge amount of money, and it will have a big impact on our lives. But when we see the difference the treatment has made to Finlay, it’s a no-brainer,” says Mrs Forsyth. “The parents I feel sorry for are those who aren’t even being told that their children need this treatment. They’re the ones who are missing out the most.”

Tyler’s story: coming close to reaching his genetic potential

23 OCTOBER 2004

At Karen Wilkinson’s first antenatal scan, she was told she must have mixed up her dates. Her baby had a heartbeat, but was too small to be seen on the monitor. When Tyler was delivered by Caesarean at 31 weeks, he was tiny: the smallest 31-week-old Mrs Wilkinson’s specialist had ever seen.

Karen and Darrin Wilkinson, who live in Churton Park, never found out why their son was so small. They were told he’d probably catch up, but he never has.

Tyler wasn’t hungry, and every mouthful was a battle. At three, he was fitted with a feeding tube through his nose. He gained 1.5 kilograms in three months, and was suddenly full of energy and confidence: his stutter vanished within days. But when the tube was removed, Tyler’s weight plummeted again.

Last year, when he was 4½, Tyler was fitted with a stomach tube. The tube enabled his parents to feed him overnight and took the stress away from meals, but he was still growing more slowly than most children.

Like Finlay (whose story is above), Tyler is among the 10 percent of small for gestational age (SGA) children whose growth fails to catch up with their peers. Unlike Finlay, Tyler does not have the complicating factor of hypoglycemia. However, without growth hormone treatment Tyler was expected to be very short – about 1.57 metres (5ft 2ins) – and his size and lack of energy had a huge impact on his confidence.

BOTH the Wilkinsons and their paediatric endocrinologist believed Tyler would benefit from growth hormone treatment. They knew Tyler was not short enough to meet the NZGHC criteria for treatment, but decided to apply anyway – and were rejected. “We wanted it on record that there are kids out there who need growth hormone and don’t get it,” says Mrs Wilkinson.

For the past 14 months, the Wilkinsons have injected Tyler with growth hormone every night. He grew 11cm in the first year of treatment and is far stronger and more confident than he used to be. “I feel like we’re starting to see the real Tyler now,” says his father.

Tyler, who is five, once avoided playground equipment: now he’s a whiz on the giant slide at Chipmunks indoor playcentre in Tawa. When the Wilkinsons met up with friends every Friday night, Tyler would curl up on the sofa under a blanket while the other children played chasing games: now he joins in. And for the first time, Tyler is able to keep up with his brother, Daniel, four, and his sister, Kelsey, two.

“Tyler is strong enough to unbuckle his car seat now. Daniel recently said to him, ‘Wow, Tyler, you’re so strong – maybe when I’m five I’ll be able to unbuckle my car seat, too’,” says Mrs Wilkinson. “Daniel used to be able to run circles around him. Tyler is getting a real kick out of being able to be a proper big brother.”

Both sets of grandparents are helping to fund Tyler’s treatment, which costs about $11,000 a year and will become more expensive as he gets bigger. The Wilkinsons hope Tyler will come close to reaching his “genetic potential”– his predicted height if he hadn’t been SGA – of about 1.8 metres (5ft 11ins). The Wilkinsons’ view is that Tyler has a medical condition, and that growth hormone therapy is the only effective way to treat it. They believe all parents of short SGA kids should be able to choose whether growth hormone treatment is right for their child, regardless of whether they can afford to pay for the treatment themselves.

Like Finlay, Tyler has been seen by Richard Stanhope, an international expert on SGA children. In a letter to the growth hormone committee chairman, Wayne Cutfield, Dr Stanhope says Tyler would have been treated very differently if his family had lived in “a more enlightened environment, such as the United States, or Europe”.

Dr Stanhope urges New Zealand to begin funding growth hormone treatment for SGA children. This would, he says, “make life very much easier for other families who will follow in the Wilkinsons’ footsteps”.

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