Paediatric and youth cancers
This chapter examines low survival rate (LSR) cancers that affect
children and young people.
Cancer Australia defines a child 'as a person aged less than 15 years',
and provides the following information about cancers in this age group:
The types of cancers that occur in children, and the way they
respond to treatment, can be different from cancers that occur in adults. They
can also be different from the types of cancers that occur in adolescents and
young adults (aged 15–29 years) – there are often specific protocols and
guidelines for the management of adolescents and young adults with cancer,
which bridge the gap between children’s cancers and adult cancers.
However, Mr Peter Orchard of CanTeen Australia explained that the
definition of a child varies across jurisdictions:
In [Western Australia] there is a hard line drawn that will
come into play in the next few months—when a young person turns 16, they are
then directed to the adult setting even if they have been treated in the
paediatric setting. In Victoria, with the Royal Children's Hospital, there is
more flexibility; they will go up to 18. So there are just two examples of the
Cancer Australia also provided an explanation of why cancer occurs in
In most cases, we don’t know why children get cancer.
Children are too young to have the same risk factors that affect adults (e.g.
environmental exposures, lifestyle, infections). Tumours occasionally develop
as a result of a genetic error made in children’s growing bodies.
In children, age is not a risk factor for cancer, but the
incidence of some cancers varies with age. Some childhood cancers tend to
appear in very young children and others in older children. Family history is
also important because a few childhood cancers run in families.
The following sections examine the most common LSR cancers in this
group, the unique issues and challenges faced by this group of people with LSR
cancer and the difficulties with transitioning from paediatric to adult
treatment and care. Prior to a discussion of these issues, the section below
considers the personal impact of childhood and youth cancers.
The personal impact of childhood and youth cancers
Many parents, relatives and friends of children and young people who
have suffered from or are currently diagnosed with cancer shared their
experiences with the committee. Childhood and youth cancer have a devastating
effect on the child or young person with cancer, their family and their
The impact of a child dying is pervasive. It is not just the
adults who cannot rationalise the injustice of it; it is also the
children—siblings, cousins, and friends. They are all suddenly faced with their
own mortality because something they rationalise as being for the elderly has
happened to one of their peers. While we as adults continue to grieve, so do
the children—nightmares, bedwetting, anxiety, and withdrawal. It goes against
nature. Parents are not supposed to outlive their children. Children are not
supposed to be diagnosed with diseases devoid of survival rates. We should be
able to reassure children that doctors can help them, not have them living in
fear that if they were to get brain cancer they would end up like Tom and the
34 other Australian children who die from it each year.
As a parent of a child who has been diagnosed with brain
cancer – words can be hard to muster to describe how this has impacted our
family. It is devastating. It is all consuming. It is heartbreaking.
Brain cancer seems to offer one blow after another. We don’t
make plans. The plans we do make we often cancel. Life becomes a circle around
appointment times and there is not much left in the way of finances or energy
for normal social life.
We reside in country Victoria and, while I have spent time
with Chloe while she has been in hospital in Melbourne, I have been on constant
call to care for her brother and sister often without notice. I have had to try
and find a way to calm their fears when their sister is so ill and they
desperately want her and their mum and dad to come home. Not only have I had to
watch my precious granddaughter in such pain and going through horrendous
treatments as well as seeing the hurt and worry of her mother and siblings, I
have had to watch helplessly as my younger son struggles through his emotional
pain knowing there is nothing he can do to make his little girl better. This is
heartbreaking for me. A parent is supposed to be able to protect their children
from pain and hurt.
I cannot put into words the suffering our precious daughter
Brooke endured, and now for my wife Olivia and I continue that suffering every
second of every day. We celebrated Brooke being one of the lucky 1 in 5
survivors of Brain Cancer only to have her taken from us by this hideous
disease 10 years later.
When I was 18, I was diagnosed with Gastro Intestinal Stromal
Tumor [sic] (GIST) and was told by my disease had no cure and I was likely to
have about one year left to live. There was no cure in 1996. There is still no
known cure 21 years later. GIST is a rare cancer with low survival rates.
In September 2016 our 13 month old daughter, Isabella, was
diagnosed with brain cancer. She has a grade 3 anaplastic ependymoma. It is an
aggressive cancer; the most aggressive form of ependymoma. This insidious
disease took over ¼ of our daughter’s brain before she was diagnosed. Instead
of our family watching our little girl transition from a baby to a toddler,
witness her first wobbly steps, hear her learning to talk, we watched her
literally fight for her life. Over the course of a week, the longest and most
awful week of our lives, we stood by while Isabella endured 4 brain surgeries.
We watched her suffer countless seizures, the last one requiring a MET call with
staff from the ward, PICU and Emergency attending to assist to try to stabilise
her. We watched as infection racked her body forcing her temperature up to 40
degrees. We watched as a ventilator breathed for her. We waited helplessly
every time she was taken away to the operating theatre, not knowing if she
would return to us. We listened to the neurosurgeon tell us that he had to
abandon the surgery to debulk her tumour because of massive blood loss. We
listened as he told us that they transfused the entire volume of blood in her
body 3 times over before she was able to be stabilised. We cried when she
finally woke up and said “mummy”, “daddy” and “happy” (her 3 favourite words).
We cried when we realised she was paralysed down her right side. We cried when
we realised she could not swallow, could not eat, could not drink and could not
sit up. We cried when she went mute several days after her fourth surgery. We
cried a lot that week. We still cry a lot now.
In addition to the emotional toll of these cancers, there are broader
implications. For example, in respect of brain cancer, Love for Lachie
Most parents will be unable to work when their child is
diagnosed with brain cancer as they need to care for their child fulltime
throughout surgeries, radiation, chemotherapy and other treatments. Brain
cancer is the undisputed most financially costly cancer. Parents can not work
if they have a child diagnosed; adults who are diagnosed can no longer work;
treatment options that are not part of the gold standard treatment plan are
incredibly expensive and for many people become completely financially
prohibitive leaving them to accept their fate with standard ineffective
Some of these broader effects of LSR cancers, such the loss of income, are
discussed in chapter 5.
LSR cancers most commonly affecting children and young people
There are a range of LSR cancers that commonly affect children, for
example, Cancer Australia identified the following cancers: leukaemia, brain
and other central nervous system tumours, Hodgkin disease (Hodgkin lymphoma),
non‑Hodgkin lymphoma, neuroblastoma, soft tissue sarcoma, kidney tumours,
melanoma, bone tumours, germ cell tumours, retinoblastoma and liver tumours.
The committee heard from various submitters and witnesses that brain
cancer kills more Australian children than any other disease,
and while 'the overall survival of some children with brain tumours has
improved' in the paediatric setting, 'the groups of children with poor outcomes
are becoming smaller, and therefore increasingly challenging to study'.
The Australian and New Zealand Children’s Haematology-Oncology Group
(ANZCHOG) made a similar observation:
Childhood cancer comprises less than 1% of the total number
of new cancer diagnoses in Australia each year. This equates to more than 600
children diagnosed with cancer each year. The treatment of childhood cancer is
one of the great success stories of modern medicine. Survival rates have
increased from less than 30% in the 1960s to 80% in the 2000s for all childhood
cancers combined. For Acute Lymphoblastic Leukaemia (ALL), the most common form
of childhood cancer, the cure rate now approaches 90%. Despite these
outstanding successes childhood cancer remains the leading cause of
non-accidental death in children in Australia and many subtypes of childhood
cancer continue to have a very poor prognosis. Unfortunately, the rate of
improvement in survival for children with cancer has plateaued over the past
CanTeen Australia identified that cancer in adolescents and young adults
(AYAs) 'has a distinct biology and responds differently to treatments that are
otherwise successful in paediatric or older adult populations'.
In respect of survival rates for AYAs, CanTeen Australia stated that:
Although overall survival rates are good...at approximately
88%1, this masks poorer outcomes seen in several high lethality cancers for
this age group. Five-year survival for cancers such as Acute Myeloid and Acute
Lymphoblastic Leukaemias and Brain and Bone cancers are still exceptionally low
at between 61.3% and 65.6% with Sarcoma only slightly higher at 76.7%, with
others such as Rhabdomyosarcoma and Lung and Adrenocortical Carcinomas having 5
Year survival rates well below 40%, and Hepatic Carcinoma only 20.6%.
Unique challenges and issues
The committee heard from a number of parents and professional
organisations about the particular challenges and issues faced by children and
young people with cancer.
For example, The Kids' Cancer Project stated that '[t]he challenges of
new anti‑cancer drug development for childhood cancers that are faced
globally are exacerbated in Australia because of our relatively small
These challenges generally arise because of 'the rare nature, smaller population,
limited access to tumour samples, more limited bodies of research knowledge and
therefore reduced funding opportunities'.
The Kids' Cancer Project also noted that '[w]e have seen the improvement
in prognosis of several [childhood] cancers that have had dedicated, focussed
funding from the Federal government', but:
The rarity of several childhood cancers means that they are
not covered by the burden of the population which the current National Health
and Medical Research Council [(NHMRC)] funding model is based on.
The Children’s Cancer Research Unit also discussed challenges arising
from the NHMRC funding model, asserting that:
...characteristics of low survival rate cancers can make it
more difficult for associated research grant proposals to be considered “well
designed (or to have) a near flawless design”. The fact that a particular
cancer is characterised by poor survival rates can reflect a more limited
research base, leading to less scientific knowledge. This can mean a greater
need for more open-ended research grant applications seeking to (for example)
identify treatment targets, or biomarkers of response. However, these more
open-ended proposals can be viewed by grant review committees and reviewers as
“fishing expeditions” that may be less likely to be considered to have
“objectives that are well-defined, highly coherent and strongly developed (and
be either) well designed (or have) a near flawless design”. Similarly, low
survival rate cancers may have fewer experimental models (cell lines, mouse and
other animal models) available for study. It can also be challenging to access
statistically informative and representative sample cohorts, or patient cohorts
for clinical trials. Reduced resources for research could therefore also lead
to reduced “scientific quality” and “significance and innovation” scores for
NHMRC project grant applications, as well as negatively impacting the team’s
Indeed, clinical trials were identified by The Kids' Cancer Project as
'the single most important factor contributing to the dramatic improvements in
survival rates for children with cancer over the past forty years'.
In speaking of access to clinical trials for children, Dr Chris Fraser
of ANZCHOG noted that:
The fact that childhood cancer is relatively rare in one way
assists our ability to conduct clinical trials because the care is very
centralised. Essentially, all of these children are cared for in one of eight
children's cancer centres around the country.
However, ANZCHOG raised a number of obstacles to running clinical
trials, including the expense of clinical trials, reluctance by pharmaceutical
companies to run trials in Australia due to the small population size, and
accessing targeted drugs.
The importance of clinical trials focussed on children and young people was
similarly emphasised by CanTeen Australia, which noted that AYAs face
Compared to paediatric and older adult populations, AYAs have
experienced relatively poorer survival gains and reductions in mortality, in
part driven by poorer access to clinical trials. Embedding clinical research
within standard paediatric care has been the single most important driver of
the dramatic improvements in childhood cancer survival rates seen over the past
40 years. Compared with the approximately 45% of younger children with cancer
in Australia who currently participate in potentially lifesaving clinical
trials, AYA participation rates remain low at approximately 10%.
The rarity of some cancers which disproportionately impact
this age group is another reason for the poorer improvements in length of
survival and mortality. Despite improvements in the diagnosis and treatment of
common cancers that have resulted in dramatic reductions in mortality, early
diagnosis programs for rare cancers have not improved over the last 20 years
and diagnosis often remains slow, resulting in the cancer being diagnosed at a
more advanced stage.
In addition, rare cancer treatments have not advanced at the
same pace as those for common cancers and it is likely that many patients with
rare cancers are receiving suboptimal care; hence a rare cancer diagnosis is
often accompanied by a very poor prognosis. AYAs diagnosed with a rare cancer
are significantly more likely to die from their disease, with these cancers
being responsible for the majority of cancer-related deaths in this age group.
Further, CanTeen Australia submitted that, in circumstances where people
experience paediatric cancers in their 20s:
...ideally they should be able to be part of a paediatric
trial. We forget the fact that it is a paediatric trial; what we do remember is
that it is a trial in this particular topic cancer. If they have got that type
of cancer, they should be able to be part of it.
The difficulty faced by young adults was also noted by ANZCHOG, which
stated that the issue of eligibility for clinical trials for young people
between the ages of 14 and 18 'is a bit of a grey area'.
Dr Fraser elaborated:
Adolescents and young adults have some poorer outcomes in
some types of cancers, and they are not enrolled as frequently on clinical
trials. There is also a discrepancy sometimes between the treatment the same
patient with the same sort of cancer might receive in a paediatric institution
compared to in an adult institution. And there might be discrepancies between
the treatment they might receive in a private adult institutions and a public institutions,
Transitioning to adult treatment
The committee heard that there are particular challenges faced by cancer
patients who transition from paediatric to adult treatment and care. For
example, CanTeen Australia informed the committee about the 'disruption to
treatment' experienced by these patients:
If they are having treatment and then at 16 they have to be
bumped across to a new institution, a whole new team needs to pick them up at
that point. In terms of research, it is that, by definition, they are still a
child but they are not able to be part of a paediatric trial because they are
considered to be too old for a paediatric setting. And the hard rule around
paediatric trials is that they have to happen in a children's hospital that has
been approved by [the Children's Oncology Group (COG)]. They have teams that go
around the world accrediting hospitals for COG trials, but they will not look
at any hospital other than a paediatric hospital. So a 16- or 17-year-old will
not be able to participate in the trial because they cannot attend a setting.
This was also discussed by Professor David Walker:
CHAIR: I understand there is a huge difference, if I
can put it that way, in regard to the way children compared with adults get
treated for exactly the same disease. So if you are moving from the paediatric
area to the adult area it is quite often a bit of a shock. Do you find that?
Prof. Walker: There is no doubt about that. In fact, I
think that is one of the reasons why the outcomes for children's cancers—for
some cancers—have improved to some extent over the years. They get better
coordinated care. Their care is centralised, by the way, so therefore a lot of
the patients are either available for, or have access to, the latest trials.
There is no doubt that there is a greater appetite for coordination of care and
longitudinal care in the paediatric medical community compared to adults.
Prof. Walker: ...even young adults, particularly those
ones transitioning through: they find they are in between and they do not get
either. They do not get the benefit of either.
CHAIR: I understand that when you move from being a
paediatric patient to AYA you do not have the same team. Is that correct?
Prof. Walker: That is true for a lot of things. Kids
who have long-term problems lose contact with the team that has been looking
after them. Team care is far less applied in adult medicine compared with
children's medicine, in a variety of fields. So, yes, it is really quite
difficult when kids get older, whether it be brain cancer or other neurological
problems like spina bifida and things like that—but we are getting off topic.
But that is absolutely true. Absolutely true.
Clinical Associate Professor Nicholas Gottardo of ANZCHOG also informed
the committee that transitioning to adult treatment 'is a bit of an issue',
which varies across states, but that:
...in general, we would not be transitioning a patient during
treatment. If we have taken a patient who is 16 or 17 under our care, we will
complete the therapy that is prescribed for that particular patient. Then a
transition model would be developed with a particular clinician or hospital,
depending where that care was best served. So, generally, we would not be
transitioning a patient [mid-treatment]. That may occasionally happen as a
patient gets well beyond 18 years of age and potentially has a resistant tumour
that is not responding to the treatment that we have delivered up front.
Clinical Associate Professor Gottardo identified the 'wider issue' for
transitioning patients as:
...having a pathway of coordinated care for a child or an
adolescent—or even a child survivor of cancer—into the adult environment, where
they are much more left to their own devices, as opposed to the more
paternalistic paediatric model where we kind of take care of everything. That
type of care can certainly be disjointed. We are now much more aware of this
issue and we are setting up transition clinics et cetera to try and have a
smoother transition between our service and the adult service.
Clinical Associate Professor Gottardo acknowledged the evidence received
by the committee that some children and young adults 'fall between the gaps',
and although it is not a 'major problem' for children up to 16:
...I think the 16- to 18-year-olds fall between the gaps. Often
children's hospitals' business model is younger children, so there are often
restrictions on being able to accept children between 16 and 18. Different
states have different rules on it. It can also depend on whether the child, or
the young adult, ever gets referred to a paediatric centre. Sometimes we just
never find out about them, and we may have a clinical trial available.
Many of our clinical trials with the children's oncology
group go into their early 20s—some of the sarcoma trials go into their 30s—and
we would be able to enrol such patients in a trial. But the adult sector are
not part of those oncology groups and therefore would not be able to and may or
may not have access to trials. But the data certainly suggests that that is the
group that falls between the gaps for enrolling in clinical trials. If they are
admitted to a paediatric centre then there is no difference, but if they are
admitted to an adult centre then they seem to have very low enrolment in an
up-front clinical trial.
Indeed, Mr Robert Perkins—whose son was 17 at the time he was diagnosed
with a GBM malignant tumour and passed away at the age of 21—shared his
experience that his son was too old for a children's hospital, and that '[t]here
was little or no support for adolescents who are dealing with their own
mortality in a hospital system that is mostly dealing with mature adults'.
The committee cannot adequately express its thanks to the individuals
who shared their personal experiences of paediatric and youth cancer. The
devastation of cancer is often compounded when a child or young person—who has
barely commenced their life—is diagnosed. The committee wants to acknowledge
the bravery and resilience of these children and young people, and their
families, who in the face of great personal tragedy strive for knowledge and
solutions not only for their own benefit but also in a quest to spare other
families the same trauma.
Recommendations elsewhere in this report are applicable to the
challenges facing children and young people with cancer; the committee hopes
that action is taken so that all people with LSR cancers face improved
prognoses in the future and that significant in-roads are made to improve the
diagnosis and treatment of all LSR cancers. In particular, the committee hopes
that greater financial support for innovative clinical trials, increased
flexibility in clinical trial design and access, and improved ethical and
governance approvals will see more research into LSR cancers affecting children
and young people.
The committee is concerned about the transition from paediatric to adult
oncology care where it appears, at least in some settings, that children are
abruptly removed from paediatric oncology services and moved to adult oncology
The committee notes that this change from paediatric to adult oncology
services is the responsibility of the state and territory health systems. The
committee encourages the states and territories to consider their current
arrangements for transitioning children and young people from paediatric to adult
oncology services, and ensure that this occurs in a consistent and co-ordinated
way that ensures continuity and quality of care in the best interests of each
The committee recommends that, through the Council of Australian
Governments Health Council, the Australian government leads a process to ensure
that arrangements for transitioning children and young people from paediatric
to adult oncology services occurs in a consistent and co-ordinated way that
preserves continuity and quality of care in the best interests of each
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