In 1999 the Department of Health (DoH) designated surgery for biliary atresia to be confined to specialist children’s liver units in England and Wales. In addition, they decided there would a third specialist children’s liver centre established, based in Leeds. This was a game changer for Children’s Liver Disease Foundation (CLDF) but it was not without controversy. Catherine Arkley, former CLDF Chief Executive, writes about the background, what happened and why it was so important to families, children and young people affected by childhood liver disease.
What is Biliary Atresia?
Biliary atresia is a rare, potentially life-threatening liver disease affecting new-born babies. In 2009 the estimate of incidence for England and Wales was 1 in 17,000 live births, equating to approximately 50 births per year¹. The early signs are continued jaundice, yellow urine (it should be colourless) and pale coloured poo. The signs are easy to miss, and the baby often seems entirely well. Treatment is an operation called Kasai-Portoenterostomy (Kasai) which aims to establish bile drainage. If this operation fails, liver damage develops and ultimately a liver transplant is needed for survival. Left untreated, the baby will develop cirrhosis and die within two years.
The Background …..
In 1985 the King’s College Hospital team published a study on the outcome of surgery for biliary atresia in Britain between 1980-82². It reported that the Kasai procedure took place in 16 British hospitals and initial success of the Kasai was not just related to the age at surgery but also to surgical expertise. The data showed only centres operating on more than five cases per year had success rates in excess of 50%. As a result of this publication the Department of Health (DoH) recommended biliary atresia surgery should be performed at King’s College Hospital, London, and The Children’s Hospital, Birmingham only. Both were centrally funded children’s liver units. Despite this decision, paediatric surgeons continued to operate for biliary atresia surgery locally and in 1993 the DoH reversed their recommendation. CLDF was dismayed by this decision.
In 1993 CLDF agreed to fund a survey undertaken by the British Paediatric Surveillance Unit (BPSU) on the incidence and outcome of biliary atresia in the British Isles 1993-1995³. The study, led by Dr Patrick McKiernan from Birmingham Children’s Hospital, identified 15 British centres operating for biliary atresia. The findings showed that initial success and longer-term survival with, or without, liver transplant was related to the number of cases managed by the centre as well as its experience. In short, children managed at more experienced hospitals were four times more likely to survive without a liver transplant than in less experienced centres.
What happened next ………
In 1997 the data and outcomes from the BPSU study were passed to the DoH. The DoH asked for advice from the Royal Colleges of Medicine and Surgery. Both professional bodies, including the British Association of Paediatric Surgeons, rejected the call to confine surgery for biliary atresia to specialist units.
CLDF was extremely disappointed with this response from the professional bodies. In its view, confinement of surgery for biliary atresia to specialist centres was in the children’s best interests.
In August 1998 in Private Eye, MD wrote an article4 on biliary atresia and signed off stating “… So if your child is offered this operation [Kasai] at another centre, insist on going to King’s or Brum”.
Incidentally, MD had been responsible for exposing the scandal of paediatric heart surgery at Bristol Royal Infirmary in 1992, so was no stranger to challenging the medical profession.
In September 1998 MD wrote a further article5 explaining that surgery for biliary atresia still had not been designated to specialist centres despite considerable evidence. MD acknowledged there were surgeons whose personal figures for biliary atresia surgery were comparable to the best. He continued to argue that surgery should be restricted to specialist centres. CLDF has often been accused of “leaking” these data to MD. For the record, it did not.
On 26th February 1999 presenter and TV Doctor Phil Hammond (who is also MD) included an item about surgery for biliary atresia in his BBC Programme “Trust me I’m a Doctor”. CLDF wrote a report summing up the evidence to date and called upon the DoH to restrict biliary atresia surgery to specialist centres. It took part in the programme and a brave family told their child’s story. The programme and CLDF had publicity throughout the day of its airing. The public outcry was significant. Later that day the Royal College of Surgeons reversed their opposition to the restriction of surgery for biliary atresia. It was a momentous day and a feeling of achievement for CLDF, as well as babies with biliary atresia.
Department of Health acts ….
In March 1999 the DoH held a meeting of all interested parties, including CLDF, the outcome being to restrict surgery for biliary atresia to specialist centres in England and Wales. There was a second decision to set up a third centre which would be based in Leeds. It was another momentous day for CLDF and families with a child with biliary atresia; surgical restriction and a third specialist centre.
The backlash ….
Not everyone was happy with the DoH decision. Some units performing more than one and less than 5 biliary atresia operations per year expressed dismay with the data analysis and conclusions. They argued their outcomes were comparable to those of the experienced centres. Further, they argued that the profession, not the media, should assess whether surgery for biliary atresia should be carried out6. Concern was also expressed that in centralising services there was the potential to de-skill regional centres and disadvantage families economically and socially.
It is important to place this story in the context of the era in which all this happened. This was a time well before the internet and social media so public awareness of childhood liver disease and CLDF was extremely low. It was also a time when the patient – family – doctor relationship was very different to how it is now.
My family story is typical of the stories CLDF heard from families since the charity’s inception in 1980 until the release of the results of the BPSU study. Our second son Matthew was born in late 1984 and diagnosed with biliary atresia at a regional hospital where he had his Kasai at 8 weeks old. The hospital saw approximately one case per year. We left hospital two weeks after his Kasai with no information and told to come back to clinic in one month. We were traumatised, frightened and uncertain as to what the future held. We felt ill-informed, ill-equipped and that we were the only family caring for a child with a liver disease, let alone biliary atresia. At the monthly clinic visits with just our surgeon we were always seen at the end of the clinic. We would wait for ages, our anxiety increasing as each minute went by. We found out later this was done so we could be given as much time as needed. In hindsight, this was caring but at the time all it did was reinforce our feelings of isolation. I found an outdated medical textbook which made grim reading. We found out that Kings College Hospital treated children with liver disease. Liver transplantation was in its infancy with Ben Hardwick having only recently become the first UK child liver transplant recipient and sadly dying in March 1985 following complications from his second transplant. We asked our surgeon about transplant and for referral to Kings. We were told that transplant would never take off as a treatment and there was little point in a referral. How wrong that statement has turned out to be! We insisted on referral to Kings which was made, somewhat begrudgingly.
We arrived at Kings with Matthew aged five months old. In the morning Matthew underwent a battery of tests. In the afternoon we went to the liver clinic. We looked around and our child no longer appeared different. We met a representative from CLDF, a lovely man called Barry Barton who was the then Director. He gave us information on biliary atresia, told us about CLDF, smiled, offered us friendship and support and put us in touch with a local family. Our relief was palpable. Suddenly we did not feel quite so alone, felt ever so slightly less scared and our anxiety reduced. So started our life-long commitment and support of CLDF. It was a ray of sunshine in our topsy-turvy world. We met Dr Mowat, as he was then. He told us our son was very ill, had rickets and was malnourished. We learned that our baby needed specialised milk formula, vitamins and other medication. We returned home feeling we could at least look after our baby better. Matthew was referred to Addenbrookes for liver transplant assessment. We met Professor Roy Calne who agreed Matthew was a potential candidate but would be the youngest baby in the world to have a transplant at that time. Matthew died before the last of the transplant assessment tests could be carried out. Our lives were altered forever as a result of the enormous privilege of having Matthew.
Our story was far from unusual at that time; it typified the era. Families told us they felt so isolated in being treated at a regional unit. Most were never told about King’s College Hospital or Birmingham Children’s Hospital. Most were not told about the King’s data published in 1985 in the BMJ or given an option for a referral for Kasai at a specialist centre. Regional units rarely were able to offer specialist nurse and dietary support or at best, it was limited. Families developed strong allegiance to the specialist liver centres, no matter the distance from home. At the liver centres, families felt supported and convinced they were doing the best for their child. So, designation of treatment for biliary atresia to specialist centres was considered by CLDF as offering the best management, in every respect, for the child and their family. Families trusted the advice and information given by the units. The day CLDF walked out of the DoH meeting having played a role in securing surgical designation for biliary atresia and a third liver centre was a real game changer not just for biliary atresia, but potentially all childhood liver diseases.
Impact – the evidence …..
As part of the March 1999 meeting the DoH called for a review of the impact of its decision to centralise surgery for biliary atresia. In 2004 Davenport et al6 reported on the management of biliary atresia in England and Wales from 1999-2002 and concluded that early results suggested surgical outcome for biliary atresia can be improved by centralisation of care. In 2007 Stringer7 noted two further benefits of centralisation. Firstly, children requiring transplant remained under the same team and secondly, centralisation facilitated clinical research into relatively rare diseases. Davenport et al reported further in 20118. The aim of this paper was to review the outcome of the policy change and look to provide a national benchmark. The paper discussed a range of issues pertinent to biliary atresia which are beyond the scope of this article but concluded that “…. consideration of most of the relevant indices would suggest that the national outcome in the particular disease has improved.” The paper also identified two consequences of centralisation: de-skilling within regional units and anecdotal social and economic issues for families, particularly cost and time of travel. The authors observe that the change in practice was not brought about by scientific evidence but mostly by the intense scrutiny of the media and public at a time of heightened interest in the wake of the events surrounding the provision of children’s heart surgery at Bristol.
In the time after centralisation much work has been done to develop shared care between the specialist and regional centres. This has done much to address the issues surrounding de-skilling and building parental confidence in shared care arrangements.
Looking back – a personal reflection….
This story must be looked at in the context of the era in which it happened. The doctor – patient – family relationship then is markedly different to how it is now and has a bearing on what and how things happened.
Another striking aspect of the story is the role of patient and family voices in shaping service provision. I was the only person in the room representing patients or a patient group at the March 1995 DoH meeting. At that time patient representation was not a standard or given. I am convinced CLDF was only present because we had been involved in the media hue and cry. Much was made at the time that change should be brought about by professionals and not by media and public outcry. The lack of dialogue between all groups meant sharing and understanding the differing points of view did not occur and seeking a consensus was not a perceived goal. I suspect many professionals found it difficult to be challenged and called to account by parents and a patient group. In turn, parents and CLDF felt considerable frustration that their views were not taken into consideration by the professional bodies. In the absence of any process to hear patient – family voices working with the media to create awareness of the issue became a valid route. I recognise there may have been bruised egos and professionals who felt misjudged. Notwithstanding, I still believe the outcome was in the best interests of children with biliary atresia. It also led to the referral of patients with other liver conditions over time. Hearing the patient – family voice may not be perfect today, but it has come a long way since those days.
I accept the scientific evidence may not be considered strong. Biliary atresia is a rare disease so gaining numbers to generate statistically significant data will always be challenging. There is also the complication that the Kasai procedure cannot be regarded as a perfect solution for biliary atresia, at best it provides a child with the chance to increase their survival with their native liver. Many experts agree that most biliary atresia patients will need liver transplantation ultimately. Defining and interpreting outcomes thus is difficult and generating strong scientific evidence for biliary atresia will always be challenging. The follow up studies have been important in providing reassurance that decisions made were justified.
CLDF had much anecdotal evidence as to the problems encountered by parents whose child was treated at a regional unit at that time. Parents were not told about the specialist centres and were not able to make informed decisions. Then, and now, I believe this to be wrong. CLDF listened to parents who were incensed on finding out there was choice. Many said they had been denied the opportunity to “maximise” the outcome for their child and would have travelled to have done so. We heard heart breaking stories on a regular basis yet CLDF felt powerless to encourage those in position to bring about change to listen and hear our thoughts as to what “better” could look like. At that time the focus was survival. As outcomes improved, teams turned to looking at the quality of that survival as well. Today the role of psychology is seen as important element within the care of children with liver disease. In hindsight, a research study to capture patient and family stories might have helped to make CLDF’s case be heard. It would have complemented the scientific data to make a more compelling argument perhaps, but social research was not regarded highly at that time.
The argument that regional units reduce the social and psychological pressures on families because they are located nearer to the family home is not wholly convincing. A regional unit may not be near a family’s home. At a specialist centre, families gain significant peer support from meeting other families either on the ward or in clinic and fortunately, technology now helps to reduce the impact of physical distance. Caring for a sick child with a life limiting, long term condition is hugely stressful, when this is superimposed onto being away from family and friends it adds to the psychological impact. But the reality is that caring for a sick child means being away from the family home, normal routine, other siblings and just “being a family” is all thrown up in the air. CLDF’s experience is that families want their child to be seen at the best place possible and have confidence in the care and advice provided. The regional and centralised units have made considerable effort to develop effective shared care arrangements and this has made a huge difference in reducing the social and psychological pressures for families and patients.
The financial impact of a child being in hospital is real and cannot be underestimated or understated. The impact is likely to be greater the further away from home. Austerity has certainly not helped. The NHS continues to centralise its services so there needs to be more pressure placed on decision makers to address providing proper financial support to families of those being cared within a centralised service.
This article has enabled me to reflect. It has allowed me to remember with great fondness the many families I met in the early days of my time as CEO at CLDF. They were my inspiration and driving force and it was a huge honour to have known them. I also remember the many healthcare professionals I encountered at that time. The BPSU story was a game changer for CLDF. I am proud of being involved on behalf of CLDF and the families, past, present and future. I believe it did and has made a difference to the management of children with liver disease, particularly biliary atresia. I’ve asked myself often when writing this, would I do it again? My answer is yes given the same set of circumstances.
- Livesey JL, Cortina Borja M, Sharif K, et al. Epidemiology of biliary atresia in England and Wales (1999-2006). Arch Dis Child Fetal Neonatal Ed 2009:94:F451-5
- McClement JW, Howard ER, Mowat AP. Results of surgical treatment for extrahepatic biliary atresia in United Kingdom 1980-82. Survey conducted on behalf of the British Paediatric Association Gastroenterology Group and the British Association of Paediatric Surgeon. Br Med J 1985;290:345-7
- McKiernan PJ, Baker AJ, Kelly DA. The frequency and outcome of biliary atresia in the UK and Ireland. Lancet 2000;355(9197):25-9
- MD. Doing the Rounds. Private Eye 1998 Aug 21:12
- MD. Doing the Rounds. Private Eye 1998 Sep 4:12
- Davenport M, Ville De Goyet J, Stringer MD, et al. Seamless management of biliary atresia. England & Wales 1999-2002. Lancet 2004;363:1354-7
- Stringer MD, Biliary Atresia: Politics and Progress – A UK Perspective. ANZ Journal of Surgery 2007 PS08 accessed https://doi.org/10.1111/j.1445-2197.2007.04125_8.x
- Davenport M, Ong E, Sharif K et al, Biliary atresia in England & Wales: results of centralization and new benchmark. J Pediatric Surg. 2011;46;1689-1694