2040
History suggests that in twenty years time trials of therapies we see in journals today will be in use and because of this our patient cohort will change. But something’s will inevitably be the same.
Using respiratory as an archetype I am going to give my thoughts as to how things are going to change. I have discussed some of this with our trainees who are already seeing some of these changes.
Technology Dependent Patients
Most tertiary paediatric centres in the UK care for 125-150 respiratory technology dependent children and the numbers are increasing. The numbers of patients with home PN are increasing, as are the numbers of children with gastrostomies. Children may also have renal/peritoneal dialysis. Currently the technology exists for home ECMO (cardio-respiratory bypass, eg whilst awaiting transplant), cardiac support ie internal rhythm generators and soon artificial hearts, and artificial pancreases for diabetics. We are entering an era of “if you can think it, we can build it” as the technology to produce smaller more efficient devices increases.
The number of children who are technology dependent is certainly likely to increase over the next ten years.
From a service point of view, it should be possible to monitor these children remotely leading to fewer clinic visits.
What happens then will depend on the future genetic and medical technologies and lifestyle choices. That is, genetic medicine, Crispr, personalised medicines and social, ethical and moral decisions about health choices, who is resuscitated and health economics will start to affect those who require technology to support survival. It may result in an increase (as a bridge to future medicines and treatment) or a decrease as less people require technological support.
Complex diseases / syndromes
The last 30 years has seen a revolution in the care and support for children with complex diseases and has opened up a new challenges and differences in the way cultures think about health and survival.
Children with trisomy 21 would not have had cardiac procedures in the 1970s. This is now common place, along with gastrostomies and even tracheostomies. Screening was introduced in the last decade,however, as the number of people having later pregnancies has increased and a strong social media campaign promoting T21 the actual number of pregnancies resulting in children in Down’s syndrome has remained static in the UK. In other countries such as Iceland and China, there are no children born with T21.
Other children with syndromes previously undiagnosed and/ or fatal are also surviving for longer, such as Edwards syndrome (T18), trisomy 9, prematurity survivors, children with severe cerebral palsy and other neuromuscular diseases.
These children often have frequent prolonged hospital admissions, often with multiple PICU attendances. Indeed, for simple surgical procedures, these patients often require a PICU bed. This is a change in the use of PICU from supporting the survivors of tragic acute illness or trauma to supporting surgical teams in elective procedures.
If admitted acutely to PICU, this group of patients often have prolonged stays, although mortality remains low, it is at least double that of a child without premorbid comorbidity and risk of readmission to PICU within a year is also much higher.
There are no therapies other than supportive for the above group and they are likely to increase in number, with subsequent increases in care needs and dependencies. They are also likely to be technologically dependent.
Chronic lung disease of prematurity deserves a specific mention here. The number of children born premature is increasing due to a variety of factors, eg maternal lifestyle choices, drug use, smoking, obesity, late/early pregnancies. And the number of survivors are increasing. Children born at 26 weeks gestation and younger have a 50-100% chance of cerebral palsy and there are an increasing number of children born at 23 weeks and a push to resuscitate those born even earlier.
If they reach term they come with multiple medical problems, eg respiratory support, liver/GI failure, developmental delay. It is difficult to predict the long term outcomes of these children, but some of the survivors of the early surfactant era (1990s) are now presenting in their 30s with a COPD type syndrome.
As the numbers in this group increase so will their care needs, increased likelihood of PICU admissions and prolonged admissions.
The hospital has to prepare for this by increasing critical care beds and respiratory medical support.
“Common” childhood chronic conditions, eg asthma, bronchiectasis, diabetes, epilepsy, inflammatory bowel disease
The number of people with asthma appears to have plateaued. However, children continue to die from it. The treatments have remained the same for about 40 years consisting of steroids and bronchodilators, with recently leukotriene receptor antagonists (montelukast) showing some benefit in some people as an add on therapy. Delivery devices have only marginally improved and the only real change has been in the use of combined inhalers. Monoclonal antibodies provide an additional therapy for a small number of patients but are a long term therapy. There are no curative treatments on the horizon, and there is unlikely to be any dramatic changes due to the multifactorial nature of asthma. Indeed, increasing pollution and climate change may result in an increase in exacerbations. There is a suggestion that as physicians get better at phenotyping asthma it should lead to more specific treatment regimens and identifying those who would benefit from monoclonal antibodies better. There is evidence that asthma plans reduce exacerbations, as do increasing clinical reviews.
As far as I am aware it is much the same for many of the above conditions except diabetes, which lends itself to the possibility of treatment through stem cells, transplant, or management through technology, eg the libre pump and similar devices. IBD may be responsive to faecal transplants. Epilepsy may lend itself to technological management, eg electrical stimulation or surgery.
In summary, there may be little overall change, but specific groups may benefit from specific interventions. From a service point of view the hospital has to be prepared to adapt to increasing interventions in this group, so eg surgical needs may increase.
Rare single gene disorders, eg cystic fibrosis, spinal muscular atrophy, some neuromuscular disease, primary ciliary dyskinesia, and cancers
Cancer and Cystic fibrosis has become the archetypes for personalised medicine and using trials to identify best therapies.
CF is the most common autosomal recessive genetic disorder in Caucasians. There are over 2000 mutations in the CF gene of which 200 are disease causing. The mutations affect any part of the pathway from transcription though processing and folding and stability on the cell surface of the CF protein (CFTR).
Now, by identifying specific genes new medicines (CF Transmembrane regulator modulators) can treat the underlying pathology rather than just the symptoms. Similar research is occurring in cancer and nusinersin can be used for types of spinal muscular atrophy, resulting in improved movement and respiratory survival. The main barriers to treatment have now become cost, with CFTR modulators costing £100,000s per patient per year for life, nusinersin is about £600,000 per patient per year for life. Newer therapies with even better outcomes are on the horizon.
Crispr is potentially the holy grail of genetic medicine. It is a technology that has been developed in the last ten years to delete faulty genes and insert functional genes. It has rumoured to have been used in China to create babies resistant to HIV. It has the potential to revolutionalise single gene disorders, offering a potential cure. It will come with both cost and ethical barriers.
From a service point of view the hospital has to look to getting more patients into trials for future medicines to bring down their costs. The NHS will have to balance the costs of these new drugs against the potential benefits to patients.
In summary, the 80s and 90s were the era of monoclonal antibodies and genetic identification; the naughties and tweenies have been about endotyping and personalising medication, the next 20 years will see improved genetic identification, genetic information and the beginnings of true genetic therapy.
History of Medicine in a Snapshot:
Symptoms – treat Symptomatically (Ancient Greeks until Modern Age) = Phenotypes
Symptoms + Biomarkers (eg eosinophils, calprotectin etc) – personalised medicine = Endotypes
We are here.
Symptoms + Biomarkers + Genes – Gene specific medicine = Genotypes
This is beginning
Next:
Genetic studies = Genetic manipulation
If you don’t know about Crispr start here:
https://www.youtube.com/watch?v=ZImVkl8QTW8
And then Google Ted talks Crispr and enter the Rabbit Hole.
“Common Childhood Illnesses” and other infectious diseases
In every paediatric textbook there are a couple of pages on measles, mumps, rubella etc. Due to vaccines it is now rare to see these. Vaccination has led to a dramatic drop in rotavirus diarrhoea. Bacterial meningitis had dropped by about 50% even before the introduction of Men C and B vaccines. The introduction of HBV and improvements in BCG could lead to a similar effect with TB. A HIV vaccine is possible. Varicella (chicken pox) vaccine is on the schedule for the over 65s and in other countries it is given to children. With appropriate political leadership it should be introduced in this country. Prevenar has resulted in a fall in pneumonia, pneumococcal meningitis, otitis media etc.
There are new RSV vaccines on the horizon along with therapies for acute illnesses.
Machine learning and improved technologies should result in better influenza vaccines (this years ‘flu vaccine was about 80% efficacious in children but only ~50% in adults).
This should result in a fall in attendances due to acute illnesses.
But…
All Western countries have seen a rise in the anti-vax movement, supported to some degree, either actively (Trump) or passively (the Blairs not declaring whether Leo was vaccinated) by politicians. The NHS website is not wholly supportive of vaccines and does not prevent anti-vaxers from commenting on the web pages. Further, parents are less supported by close families and more alert to the signs of illnesses. Social media supports the concept of the “missed diagnosis.” This has resulted in an increase in attendance at acute care facilities and parents demanding more investigations.
Public health, schools and the hospital have to provide support and education in helping to determine whether or not their child is well or ill. Smartphone apps and wearable devices may help this.
The Healthy Child, Lifestyle Illnesses, Normo-illness, Psychiatric and Psychosomatic disease
During the 90s and naughties there was a mini boom in childhood wellness. This is demonstrated in the reduction in infectious diseases, eg meningitis before the introduction of vaccines, and the improvement of nutrition and education in children and families. However, levels of inactivity have risen, and good nutrition has resulted in obesity, some of which may have been exacerbated by austerity (cheap food is higher in calories).
Locally patient groups have expressed a desire to increase their knowledge about how to keep their children healthy rather than preventing/treating illness.
Doctors will need to be able to provide this advice. The Hospital should provide information on supporting a healthy lifestyle. Doctors and health care workers should promote the guidance from WHO regarding activity levels, screen time and fruit and vegetable intake.
Obesity is increasing asthma symptoms, GORD, obstructive sleep apnoea and type 2 diabetes. Levels of parental smoking will likely plateau at about 17%, secondary and tertiary smoking is known to increase respiratory exacerbations in children with chronic lung conditions. It is not known what the long term effects of secondary vaping will be. Pollution levels will increase for at least the next ten years resulting in an increase in respiratory exacerbations.
A healthy and active lifestyle should be promoted at all levels.
Activity trackers are promoting anxiety regarding health in adults and children’s activity trackers will do the same. Tracker have largely not yet reached medical grade, although the ECG on the Apple watch is now FDA approved, there are no over the counter approved sleep devices.
Activity trackers will result in an increase in the number of referrals for “normo-illness” which will lead to more investigations to prove wellness. We are already seeing this.
There is an increasing awareness of childhood psychiatric illnesses and mental health problems, somewhat driven by social media. There is likely to be further investment required in pyschiatrists, psychologists and counsellors. Information needs to be available to parents about how to advise their children over the use of social media, and support children and young people in a world where more frequent exams, testing, assessments, constant monitoring, peer pressure to look good and behave right and social media results in increased pressure.
Parenting classes shouldn’t just be for the parent of a newborn. They need to continue into the child’s adolescence.
Psychosomatic disease is difficult to measure. My feeling is that more parents expect ‘normal children’ to be well all the time and any symptom eg cough, dyspnoea on exercise, frequency of stooling, abdominal pain is taken as a sign of illness even though it likely falls with in the spectrum of normal. There is increasing pressure to have “normal” children and a reduction in knowledge of variation of normal.
Doctors will have to have a good concept of normal in order to identify normal and psychosomatic illness. There will be increasing pressure to do investigations to prove wellness.
Summary
In my opinion, based on the evidence I am aware of new technologies and changing demographics, the next 20 years will be result in further changes the population attending hospital. There will be a reduction in acute illnesses and children with single gene disorders and cancer will have better, more effective treatments and potential cures.
This will be offset by an increasing population of children with complex needs, technology dependence and “normal” children presenting with “normal” symptoms or psychiatric/psychosomatic problems.
It is likely that Children’s hospitals will have to adapt by proving more PICU beds, being prepared for more long stay complex patients, more gene therapies and personalised medicines and more surgical interventions.
A Children’s hospital also needs to be pro-active in promoting wellness, a healthy lifestyle, good mental health, and an awareness of what is normal.
Note this was originally written in 2019 and has been edited to be more generic. It does not represent any organisation and my thoughts only.
I look forward to seeing comments ...
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