Advances in Pediatric Rheumatology: Biologics Transform Juvenile Arthritis Treatment

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• Evolution of Pediatric Rheumatic Diseases
• Systemic Juvenile Idiopathic Arthritis
• The Biologic Revolution in Treatment
• Targeted Cytokine Therapies
• Dramatically Improved Patient Outcomes
• Full Transcript

Evolution of Pediatric Rheumatic Diseases

Dr. Randy Cron, MD, explains that pediatric rheumatic diseases have seen significant changes in outcomes over the past two decades. The actual diseases themselves remain largely the same. However, treatment approaches and patient prognoses have transformed dramatically.

One notable change involves increased reporting of rare complications. A small subset of children with systemic juvenile idiopathic arthritis now present with severe lung disease. This condition, pulmonary alveolar proteinosis (PAP), was less commonly reported in the past.

Systemic Juvenile Idiopathic Arthritis

Systemic juvenile idiopathic arthritis affects approximately 10% of children with JIA in the United States. Dr. Randy Cron, MD, notes that these patients experience more than just arthritis. They present with high fevers, distinctive rashes, and internal organ inflammation.

These children face a high risk of developing macrophage activation syndrome (MAS). This serious complication involves excessive immune system activation. A very small subset may also develop pulmonary alveolar proteinosis, a potentially fatal lung condition.

The Biologic Revolution in Treatment

The introduction of biologic agents represents the most significant advancement in pediatric rheumatology. Dr. Randy Cron, MD, describes these as manufactured designer drugs that target specific immune pathways. This approach embodies precision medicine in autoimmune disease treatment.

Biologics have radically changed care for both children and adults with rheumatic conditions. These medications specifically target cytokines like IL-1, IL-6, and TNF. The biologic revolution continues to evolve with newer drug classes emerging regularly.

Targeted Cytokine Therapies

Different cytokine blockers work for specific autoimmune conditions. TNF inhibitors work exceptionally well for polyarticular juvenile idiopathic arthritis. However, they show limited effectiveness for the systemic features of systemic JIA.

Dr. Randy Cron, MD, explains that IL-1 and IL-6 blockers demonstrate superior efficacy for systemic JIA. This specificity highlights the importance of targeted treatment approaches. The interview with Dr. Anton Titov, MD, reveals how treatment selection depends on disease subtype and presentation.

Dramatically Improved Patient Outcomes

Biologic medications have transformed quality of life for children with arthritis. Dr. Randy Cron, MD, emphasizes that most patients now participate fully in sports and physical activities. Children can play basketball, soccer, and football without limitations from their disease.

These treatments generally have favorable side effect profiles. Dr. Randy Cron, MD, notes the need to monitor for specific risks like tuberculosis in patients on TNF inhibitors. The overall benefit-risk ratio remains strongly positive for most pediatric rheumatology patients.

Full Transcript

Dr. Anton Titov, MD: Are pediatric rheumatic diseases today different from 20 years ago, and if so how?

Dr. Randy Cron, MD: The outcomes certainly have changed. The actual diseases, it's hard to say. One thing that comes to mind is a very small subset of children with systemic juvenile idiopathic arthritis.

In the United States, at least about one in 1000 children will get a form of chronic arthritis called juvenile idiopathic arthritis. So it's not common, it's about one in 1000. And about in this country, at least, about 10% to maybe systemic juvenile idiopathic arthritis (JIA).

In addition to having arthritis, they have high fevers, rash, sometimes internal inflammation of their liver and other organs. And that's been true for a long time.

However, there's been increased reporting of children who get a rare lung disease called PAP, Pulmonary alveolar proteinosis. A proteinaceous material ends up in your lungs. That's highly fatal.

Even systemic JIA itself, as we talked about earlier, those children are at very high risk for developing macrophage activation syndrome. And a subset of those who are at high risk for developing macrophage activation syndrome, a very small subset, but a real subset can also get this severe lung disease that can lead to things even like clubbing of your fingers, which we see in bad lung diseases like cystic fibrosis, for example.

Anyway, we see a lot more, or we're seeing a lot more of that reported than we had in the past. And no one really knows why. Some people have hypothesized that some of the medicines that we're using to treat these conditions, including IL-1 and IL-6 blockers. Maybe that's why, it's very hard to know.

Cause and effect are difficult here. And it may not be the medicine itself that's triggering it, but maybe how it alters the immune response. It's entirely unclear.

But that's one change. I think that we may be seen, or maybe those children just died in the past and didn't get reported. It's unclear.

But that's one thing. I think that is different, but probably more importantly, in the last 20 years, with the introduction of biologic agents, these are manufactured designer drugs.

We get into the Precision Medicine going after specific cytokines often, whether it's IL-1, IL-6 or TNF. This has radically changed the care not just for kids, but adults too, with some of the more common rheumatic conditions like rheumatoid arthritis, for example.

I mean, that's made a huge impact on their ability to give people often relatively normal lives, which was not the case 20 years ago. And so this biologic revolution, which just keeps getting better and better.

We are getting newer classes of drugs. We're learning more about the immune system both from our patients and from experiments in the lab, whether it's an animal models are in vitro. We just keep learning more and more about even the plasticity or the ability of the immune system to go from one state to another in terms of different types of inflammation and different cell types that can lead to that.

But being able to now block some of what we think are these more dangerous cytokines in certain situations. And it's complicated.

So some of these work very well for psoriasis, for example. But biologics also work for arthritis, or some work for inflammatory bowel disease, but not for psoriasis. And so we're learning as we go.

But it really, these agents have really revolutionized the care, particularly for children with juvenile idiopathic arthritis. It's the TNF inhibitors in particular, and there are other drugs as well, I don't want to short shoot the other ones.

But that's what we've had the most experience with for the longest period of time. They overnight, like in the year 2000, basically revolutionized the care of children with bad arthritis, chronic arthritis, to the point that most of our kids now are playing basketball, soccer, football, whatever - because they can.

These medicines allow them to do that. They don't have a lot of side effects. They're not completely benign, we need to worry about tuberculosis in particular, for example. If you're on a tumor necrosis factor inhibitor, and for the kids was systemic GIA TNF inhibitors didn't work so much for the systemic aspects of disease.

But it turns out that blocking IL-1 or IL-6 works really well for them. And so even those kids have had major benefits from this biologic age of targeted cytokine approaches.