Modern Pediatric Rheumatology: Reversing the Treatment Pyramid for Better Outcomes

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• Reverse Pyramid Approach in Pediatric Rheumatology
• Limitations of Traditional Treatment Methods
• Benefits of Early Biologic Medication Use
• Cost Considerations and Long-Term Benefits
• The Window of Opportunity Concept
• Philosophy of Aggressive Early Treatment
• Full Transcript

Reverse Pyramid Approach in Pediatric Rheumatology

Dr. Randy Cron, MD, describes a fundamental shift in pediatric rheumatic disease treatment strategy. The traditional pyramid approach began with mild medications like NSAIDs. Doctors then slowly escalated treatment over months or years if patients did not improve sufficiently.

Dr. Cron advocates for reversing this treatment pyramid entirely. He recommends starting with the most effective medications first for severely affected patients. This modern approach often begins with TNF inhibitors, sometimes combined with methotrexate, right at diagnosis.

Limitations of Traditional Treatment Methods

The conventional treatment approach often led to significant disease progression. Dr. Randy Cron, MD, explains that starting with non-steroidal anti-inflammatory drugs like ibuprofen had limitations. While relatively safe short-term, chronic NSAID use could cause kidney, liver, and gastrointestinal problems.

Dr. Randy Cron, MD, notes that the slow step-up approach frequently resulted in accrued damage. Patients might spend a year or two on inadequate therapy before receiving effective treatment. This delay allowed irreversible joint damage and disease progression to occur.

Benefits of Early Biologic Medication Use

Early aggressive treatment with biologic medications offers significant advantages. TNF inhibitors work quickly to control inflammation and prevent damage. Dr. Randy Cron, MD, emphasizes that these medications have favorable side effect profiles despite their potency.

Dr. Cron's strategy involves using the most effective drugs with the best benefit-to-side-effect ratio first. The goal is to achieve rapid remission, after which medication dosages might be reduced. This approach can sometimes allow patients to eventually discontinue methotrexate while maintaining disease control.

Cost Considerations and Long-Term Benefits

Dr. Randy Cron, MD, acknowledges that newer biologic medications come with higher upfront costs. Insurance companies often prefer starting with cheaper traditional drugs. However, Dr. Cron argues that early aggressive treatment provides long-term societal cost savings.

Preventing joint damage and disability reduces future healthcare expenses and productivity losses. Dr. Randy Cron, MD, emphasizes that despite initial cost concerns, biologic medications represent a wise investment. They help patients maintain function and avoid the cumulative damage that requires more extensive care later.

The Window of Opportunity Concept

Dr. Randy Cron, MD, discusses the important concept of a "window of opportunity" in rheumatic disease treatment. Early in the disease course, the immune system may be more responsive to intervention. Aggressive treatment during this critical period can lead to better long-term outcomes.

Dr. Randy Cron, MD, explains that slow, stepped treatment approaches may miss this optimal treatment window. The immune system might not respond as well to medications introduced later in the disease process. This concept reinforces the importance of early diagnosis and immediate effective treatment.

Philosophy of Aggressive Early Treatment

Dr. Randy Cron, MD's treatment philosophy centers on the belief that disease is often worse than medication side effects. He developed this approach during training with influential physicians like Dr. Carol Wallace and Dr. David Sherry. Their relatively aggressive treatment strategies demonstrated better patient outcomes.

Dr. Randy Cron, MD, emphasizes that while medications carry some risk, uncontrolled rheumatic disease causes definite harm. His personal strategy involves treating early and aggressively to prevent damage accumulation. This philosophy has proven effective in managing various pediatric rheumatic conditions while minimizing long-term complications.

Full Transcript

The future just keeps looking better and better for us. We just have to be smarter about which patients should get which drug, the timing of that, and how aggressive to be.

There used to be what was called a pyramid approach to treating chronic rheumatic diseases, both adults and kids. You would start out with something like Motrin, ibuprofen, for example, a non-steroidal anti-inflammatory drug. For the most part, you worry about a ton of side effects, but again, it chronically can bother your kidneys, your liver, your gut, for example.

You would start there and the kid wasn't much better in three months. So then you would add what we call a disease-modifying antirheumatic drug, because the non-steroidal anti-inflammatory drugs don't disease-modify something, for example, like low dose methotrexate.

You would try that for a while, and they get a little bit better, maybe, but they weren't good enough. And then you raise the dose of that and see how they're doing, a year or two into the disease. They're still not doing great, and you've accrued a lot of damage along the way.

Dr. Randy Cron, MD: But now, we're trying to flip that pyramid upside down. So if you're sick enough, maybe at diagnosis, we're going to start a TNF inhibitor, maybe in conjunction with methotrexate, for example.

Maybe we try to take the methotrexate away over a year or two, if we can get them into remission. So using what works best with the least side effects.

And that's not always popular with third-party payers, because they tend to be expensive medicines compared to their other drugs.

Dr. Randy Cron, MD: But in terms of long-term benefit to society, they definitely save money.

Dr. Randy Cron, MD: But we still kind of struggle with that. But flipping the pyramid so that we're using the drugs with the highest benefit, the side effects ratio that makes the most sense.

Dr. Randy Cron, MD: But when you see a patient in front of you and you don't have randomized, double-blinded, gold standard, placebo-controlled trials to tell you what to do.

My personal strategy has been as the medicines have gotten better, that the disease is almost always worse than the potential for side effects of medicines that work really well and quickly.

Medicines are not free from side effects but they have low risk of side effects. My approach early on has been to flip this pyramid.

And that, in part, comes from some of my early training with people like Dr. Carol Wallace and Dr. David Sherry, who had a relatively aggressive approach to treating many of our rheumatic diseases. Again, because the disease is often far worse than the therapies.

So, in general, that's been my approach to many of our diseases. It is to treat them early and aggressively.

And there's even a concept of, quote, a 'window of opportunity' in which the disease may be much more amenable to treatment.

Dr. Randy Cron, MD: But if you kind of slow step in, that may not be the case, the immune system may or may not respond as well with that approach, as opposed to an early aggressive approach.

It's more of a philosophy, but I think it's worked rather well.