Hopes for sick children who go abroad may be ‘unrealistic’

Some treatments offered abroad “give hope which we believe is unrealistic and without foundation”, say two of Ireland’s leading children’s doctors.

In a frank interview with The Irish Times, consultant paediatric haematologist Prof Owen Smith and consultant paediatric oncologist Dr Michael Capra, both of Our Lady's Children's Hospital, Crumlin, say they fear there can be "an exploitation of hope," involving seriously sick children.

They are worried that parents could, as a result, feel “children in Ireland were being underchanged” by the health service.

The perception that treatments abroad can offer hope to very sick children “is not unique to Ireland. It is an international problem and it has got worse in the past five years because social media is [now] involved,” says Dr Capra.

The doctors say children in Ireland are taking part in a number of international trials and that Ireland is at the forefront of developments in cancer treatment.They also say the HSE has “never” refused them funding for any treatments or drugs that have been shown to increase survival rates.

“We have the same trial portfolio as Great Ormond Street Hospital in London, which is the leading paediatric cancer unit in the UK,” he said.

Dr Capra and Prof Smith are in a team of five consultants at Crumlin hospital, which is the childhood cancer centre for under-16s in the country.

Each year some 170 children are diagnosed and, while some cancer care can be given at home, “the lion’s share of treatment is delivered here”, said Dr Capra.

The overall survival rate for paediatric cancer is between 75 per cent and 80 per cent and the doctors attribute this improvement to “international collaboration in the form of clinical trials, where internationally we all work together rather than as single units”.

“Participation in randomised, peer-reviewed, clinical trials has really stepped up the cure rates by 30 per cent in the past 30 years,” says Prof Smith.

The number of patients needed for the trials mean that Ireland must work with other countries. This allows access to trials in other countries in Europe and in the US.

“We couldn’t do those [trials] on our own; we have to get into bed with other international collaborators. So if a child is diagnosed here with a cancer and there is a clinical trial open or available, that child will be entered into that clinical trial,” he says. They say every parent and patient is spoken to about the trials. “Most of them incorporate chemotherapy that is well known, tried and tested, but [the trial relates to] the way in which it is given, the dose, the sequencing and minor variations in the types of these chemotherapies,” says Dr Capra.

“Some trials include new or novel drugs that have gone through a whole maturation process of trials. A maturation of a trial would be a laboratory where no patients are tested – so just on cells; they then transfer that into an animal-type model, then into the human model, and to different phases of trials,” he says.

Prof Smith and Dr Capra prefer to use the word protocol instead of trial, which is an emotive word.

“Some of our parents say, ‘What are you trialling here? Why are you putting my child on a trial?’

“If we have a result from a protocol that has, for example, an 85 per cent cure rate, the next protocol will ask another two, maybe three questions and it is highly unlikely that the cure rate will fall below the previous trial. If you look at our outcomes, they go up and rarely, if ever, go down,” says Prof Smith.

Trials

Our Lady’s Children’s Hospital is involved in a number of trials in collaboration with other international groups including the predominantly UK-based Children’s Cancer Leukaemia group, the pan-European International Society of Paediatric Oncology (SIOP) and the North America collaborative organisation the Children’s Oncology Group, and it has recently joined with the Innovative Therapies for Children with Cancer consortium, which is European based and uses new agents (see panel).

The latter, by definition, “looks at new or experimental treatment and how to bring it into the clinical arena. “If there is something new, it will predominantly come from that group and the other international groups will follow that with protocols across the world,” says Dr Capra.

This collaboration by Crumlin hospital means Ireland is as much at the forefront of treatment as any other country in Europe.

According to Prof Smith, a benefit of Crumlin’s participation in the large international trials is, “in a big trial these novel agents come free”.

The most common childhood cancers are leukaemia, brain tumours and neuroblastoma. Leukaemia has a survival rate of more than 90 per cent but not every prognosis is that good.

Dr Capra says that if a case of neuroblastoma is diagnosed as “high risk”, the survival rate is between 30 and 50 per cent.

“There is approximately a 60 per cent chance that patients, once cured, will relapse and relapse is invariably fatal.”

“Figures [for survival after relapse] are in the region of 4 per cent, possibly 8 per cent, but that is optimistic and there lies the emotion. If you are dealing with a patient who has a high chance of relapse we know is invariably fatal, patients and families will try to move heaven and earth: understandably.”

Some media coverage about some treatments that are available abroad has raised concerns for them. Dr Capra says, “If I was a parent of a current patient with cancer and I read [those media reports] without the background knowledge, I would feel children in Ireland were being underchanged with service.”

“They believe the grass is greener on the other side and that other side is across the Atlantic, in north America, predominantly. That perception needs to be changed.

“If there is a patient under the care of the one in five of us [consultants] here and the treatment is not available here, but is available overseas, we will fight tooth and nail to get the Government to send that patient . . . to fund that patient to go and get that treatment,” he says.

“Unreservedly,” says Prof Smith.

They say they have never been refused funding in such situations. “Therefore, [for] the patients who are doing this, we generally would not recommend this form of treatment but because they are a vulnerable patient group, we believe that, understandably, there has been an exploitation of hope.

“That is an emotive phrase. When your child of two years of age has a relapsed neuroblastoma, which we know is invariably fatal, then you will seek every opinion you can get.

“And you will listen to the opinion that gives you the greatest hope, but realistically that may not be the best treatment for your child and may do your child more harm with the same outcome.”

Exploitataion

Asked who is doing the exploitation, Dr Capra says, “The parents want the answers so they will speak to 20 doctors in North America and if one doctor says, ‘Yes I can do this operation and give you a cure, of course they are going to go’. ”

Prof Smith says, “If they were in a trial we would say, Hang on, maybe there is a case for this. Most of these experimental treatments are outside of clinical trials. I will not, as an individual, send any of my parents into that zone where it is purely experimental and is not within a clinical trial.”

The difference between the trials in which Crumlin hospital is taking part and the trials that cause these doctors concern is, they say, a crucial question.

Dr Capra explains, “These [other] trials are generally hospital-run; an individual hospital may open its own novel trial. Hence it is not within a big, collaborative, recognised consortium.

“In North America, remember the motivation of some of these institutions: they have a competitive health market, they have a fee for service and therefore the more patients they treat, the more money they earn and they are in competition with the hospital down the road. They are completely different to the national health service we run.”

Renumeration

Dr Capra says in North America medicine can be “highly academically remunerated, so the higher your academic career is, the greater will be your career progression. Therefore, if you do more trials – more academic, nonclinical work – you get more and more remuneration and your international stature goes higher and higher. Hence our frustration.”

Prof Smith recalls a family whose child relapsed after a stem-cell transplant and the prognosis was grave.

The boy’s father visited doctors in North America and “there is one who told him, ‘I can give this experimental drug’. ”

The drug was not part of a clinical trial, says Prof Smith, and “I, as the primary carer and physician, have to inform the parents that the chances of their child coming back alive is less than 1 per cent. They do not want to hear that.

“And that is put back in our face [by the parents],” says Dr Capra. “They say, ‘You give us no hope, those doctors give us hope, where do you think we are going?’ .”

“If there was hope and they were on a clinical protocol, we would break our backs to go out there and get the funding [to travel] if we could not deliver that therapy here,” says Prof Smith.

While both consultants are concerned about the huge sums of money families can feel pressurised to raise, it is not their main cause of concern.

“Forget about the money: look at the parents. Look at the psycho-social aspect of the family; they take their child away with huge hopes because someone somewhere says they can do something. They invariably do not take their child home alive. Their child dies, they have [fundraised] €250,000 and the family is completely wrecked,” says Prof Smith.

“What is worse? Their child dying in a unit where they know nobody, with strangers? It is horrible,” he says.

Dr Capra describes a situation where one parent “can have a child whose prognosis is invariably fatal and has to face that challenge in [another] country with the rest of their family here in Ireland, their other children at school.

“ They are separated from their husband or wife, separated from grandparents . . . think of that position. That is the quality of life for that family.

“Even more importantly, an operation can be performed there – not one we would recommend, but parents do it – and if there are complications, that patient will come home and we have to look after that complication.”

This is not a situation the doctors have seen only in recent times and they say it is an international one.

“North America is the attraction and it has got worse in the past five years because social media has become so effective,” says Dr Capra.

Social media

The doctors do talk to their patients, “but when social media is gathering at such speed, the relationship and trust with us is eroded because of what they read in newspapers from individual families and that is the frustration”.

Both give examples where unidentified parents spoke of going to North America as an option after their child has been given a very poor diagnosis.

These treatments “are giving hope we believe is unrealistic. I say to the parents, ‘It is your decision to go, it is not my recommendation and I have said I believe it is unnecessary. In some of these cases I have said, what you are doing will harm your child.’ At the end of the day, it is their decision and I have to respect their decision,” says Dr Capra.

In relation to the doctors in North America, Dr Capra says, “What they do is medically within internationally renowned hospitals: they have their own ethics committee, their own patient base and they have been approached by the parents, so they have not gone out touting for business.”

Prof Smith always asks parents to think about the future as well as the present. “It has happened to me several times with parents and they looked back, having taken our advice, and their child has died peacefully, lovingly, at home and with our support structures.

“One of my former bosses at Great Ormond Street once said to me, ‘Can a child not die with dignity?’ It is a huge issue for us as some of these children are doomed to start with, because of the biology of their disease.”

Dr Capra says it is part of a trend where parents “believe they can be more in control of life and therefore they will fight everything.

“I am personally hurt by some of the campaigns because the inaccuracies [are] reported in order to arouse the community emotion and generosity to fund their project,” he says.

Prof Smith says there are frustrations and issues in the system but “the real hitters, the drugs . . . when we apply to the HSE they never [refuse] us the drugs”.

“You asked us, are we happy in our work? The answer is, by and large, yes, but there are huge frustrations. The frustrations with the HSE I can cope with. But when parents are misled to go certain ways I get really frustrated as a human being.”

“I think it is exploiting the community goodwill, says Dr Capra, “because anybody who reads those articles is going to say, ‘Yes, of course we are going to give you €10,’ but I think it is exploitation.”

Clinical trials and Crumlin hospital

Our Lady’s Children’s Hospital, Crumlin, has more than 30 standard of care protocols. “These protocols are based on the best available treatments worldwide, which are based on the results of trials that have closed,” according to Dr Cormac Owens, a consultant paediatric oncologist at the hospital.

In addition, it uses 22 treatment trials or protocols for a number of different childhood cancers including acute leukaemia, lymphomas, neuroblastoma, hepatoblatoma and rhabdomyosarcoma.

And, as part of its collaboration with international groups, the hospital says it has 11 different protocols in development.

Dr Owens says one of those groups has begun a particularly important trial. “The Innovative Therapies for Children with Cancer (ITCC) consortium recently opened a trial for relapsed neuroblastoma, which is very exciting.”

It is the Beacon trial and is, he says, “the only trial of its kind in the world and is for children who have run out of conventional options”.

He says Irish children are among the 17 children from across Europe who are on this trial.

Dr Owens says the development of new trials is a constantly evolving process and “[Ireland is] at the table. There are more opportunities than before and Europe has more drug trials open than the US.”

In 2007 the European Medicines Agency introduced the Paediatric regulation, which requires pharmaceutical companies to look at the possible benefits for children of new medicines for which they want licences.

In its five- year review, published in June last year, the EMA said the regulation was “a major catalyst to improve the situation for young patients”.