The contrast of ITP in children and its impact on families and caregivers

Immune thrombocytopenia (ITP) is somewhat different in children than it is in adults.

As a paediatric haematologist, I see the vast variety of journeys walked by families of children with ITP. I see the uncertainty ITP brings, to have a child with risk of severe life-threatening bleeding, with no ability to accurately predict when and if the illness will resolve. I see families given a whole heap of statistics, and not know which will be relevant to their child, which category they will fall in. I see families given a diagnosis that is not a “severe illness”, it is not cancer, it is very “benign”; but in many many cases it puts their life on hold and causes dramatic changes to their day-to-day life.

I often hear adult haematologists be somewhat dismissive of paediatric ITP. Sometimes they generalise, and say paediatric ITP is more favourable to adult ITP because “it goes away”. Many paediatric patients and their families would not consider ITP a particularly favourable diagnosis. In many ways paediatric ITP is a different disease process, a completely different entity to that of adults, and whilst many children do in fact spontaneously recover quickly, certainly not all children do. In children more than 80% are said to resolve before it becomes chronic ITP by definition.

However, that leaves a reasonable proportion who go on to develop chronic ITP, which equates to up to several hundred children per year in Australia. Thus, chronic ITP is not uncommonly seen in paediatric haematology, and children do graduate paediatric centres to go to adult centres still with their ITP. Yet other paediatric patients who evolve to chronic ITP do recover over the course of several years.

When children are given a diagnosis of ITP, they and their families spend the next few months to years, patiently waiting to see if they will resolve early or be in it for the long haul. During that time of waiting, families often watch their child in trepidation – will the toddler jump off the climbing gym and knock their head that little bit too hard, will the teenager get struck by a ball to the head, will the nose bleed stop, can I leave my child at daycare, can they go to camp, should they stop all sports, what will happen with their next menstrual period? Many affected paediatric patients cannot talk properly yet, and cannot tell their caregivers what they are experiencing, making it all that bit harder.

Like adult ITP, we describe treatment options are first line or second line. First line treatments, namely steroids or IVIg , are also called “rescue therapies” or “temporizing therapies”. These therapies aim to temporarily dull the immune response and cause a transient rise in the platelet count. They are not cure. About 75-80% of patients will respond to each in some way. Some patients respond to both, some patients respond to neither. Some patients get a transient response for weeks, and some last only a couple of days. Some patients get a better response when given both steroids and IVIg together. It is impossible to predict who will do better with which therapy.

This demonstrates one of the many layers of uncertainty faced in ITP. And to top it off, the response a patient has from one treatment option is not always replicable the next time they have the same treatment.

But why aren’t these treatments a cure?

In my time during my haematology fellowship in Canada, a close colleague of mine used to refer to ITP as being like the pilot light of a gas heater. Growing up in Queensland, I had little use of a gas heater but quickly caught on to the analogy. These treatments do not turn off the pilot light, they merely turn off the gas temporarily. The pilot light is still on, waiting for ammunition. For how long does the pilot light stay on? Our best guess is no better than a crystal ball in the majority of cases.

Many paediatric patients with persistent ITP, often with concerning bleeding symptoms, or poor response to temporising agents, are offered second line therapies. In some instances, a bone marrow aspiration and trephine is performed first. Pending on how long the patient has had ITP, and specific circumstances, either second-line immunosuppressive agents (such as rituximab amongst others) or more recently, thrombopoietin receptor agonists (TPORAs) such as romiplostim, eltrombopag and avatrombopag). The introduction of TPORAs has certainly rapidly evolved the treatment of chronic ITP, and in many cases improved quality of life outcome measures in children, and could be the basis of a whole other article.

The dilemmas I see families struggle with are similar to many other paediatric disease processes; families and legal guardians are forced to make decisions for their children, not knowing if they are doing the best thing for their child and desperately wanting to do so. Families are given options, more statistics, a list of side effects, some guidance and recommendations, and asked to choose which therapy they would prefer, if any. Side effects sometimes happen. Bleeding sometimes happens. Regardless, guilt often ensues.

Severe bleeding rates in paediatric ITP varies amongst the studies, with one large study quoting 3% of children with ITP sustaining severe bleeding, but only a fraction of those being life-threatening bleeding. Some patients present with their acute ITP with severe bleeding, whether it be an intracranial bleed, or a severe bleed requiring a blood transfusion such a nose bleed. A statistic of “less than 5% of patients will have severe bleeding” suddenly becomes very irrelevant when you fall into that minority, and that is your reality.

Many of the recent paediatric ITP guidelines in managing ITP, focusses on managing quality of life. I feel this is crucial to the management of paediatric ITP. With all these layers of uncertainty, we must focus on each individual patient and their circumstances. Not only must patients be managed in a way that minimises the risk of severe or life-threatening bleeding, identifying those at most risk and recommending prompt temporising and/or second line agents in such patients, but other factors must be assessed. Such factors include, but are not limited to, patient sporting preferences, recreation and hobbies, care arrangements, distance from hospitals, co-morbidities, menstrual cycles, parent/carer comorbidities and life commitments.

All of these aspects should be continually addressed when considering treatment advice for paediatric patients, and many such things are dynamic and constantly changing.

The treatments ideally focus on encouraging patients and their families to be able to live as normal as possible, whilst minimising the risk of severe bleeding and medication side effects. It is possible to be a professional sports player with ITP, and we need to support this desire if that is what is important to the patient.

In summary, to those families with a child with ITP, we see it often has a huge impact on your life. ITP is in most cases a low-risk illness when considering severe morbidity and mortality, but no one wants to be within the small subset of patients who comes to significant adversity. Quality of life is so important – being able to play your chosen sport safely, to attend the school camp, to go on a rollercoaster with your mates, whatever it is that is important to you. It is important as physicians managing ITP we recognise the priorities to the individual patient and family, and make treatment decisions and advice guided by each individual scenario. Paediatric ITP is different to adult ITP, but the layers of uncertainty over the course of the illness certainly unifies them together.

Paediatric ITP is different from adult ITP, but the layers of uncertainty over the course of the illness certainly unify them.

Learn more about the diagnosis and treatment of ITP in children with the ANZCHOG Guidelines in Patient Resources.