“Infection is probably the biggest morbidity and mortality risk early on, and steroids dramatically increase the risk of infection."
Joyce Chang, MD MSCE is a pediatric rheumatologist at Boston Children's Hospital. Her main interest is in treating children and teenagers with lupus or lupus-related conditions. There are some 200,000 individuals in the US with lupus and between 10 and 20% of them have disease onset before the age of 18.
Dr Chang herself was diagnosed with lupus at the age of 20 while studying medicine and at the time thought rheumatology would be the last sub-specialty she would pursue because she “didn’t want to read about worst-case scenarios.”
"When I started my pediatric training during my residency, I encountered quite a few young lupus patients and being able to help them convinced me to go into pediatric rheumatology instead of primary care.”
Dr Chang recently published an article in Arthritis and Rheumatology[1] that indicated prospects for pediatric patients with lupus have greatly improved when compared to just 15 years ago. She points out that overall survival rates for pediatric lupus in the US are approximately 97%, although “in terms of morbidity and quality of life, children suffer quite a lot.”
“Part of that is the burden of care and the burden of illness, and another part of it is the treatment – even though the intention is to prevent organ damage and make sure they can reach their full potential and adulthood.
Steroid-toxicity is an even bigger problem for children than it is for adults. On top of the side effects experienced by adults, steroids can permanently impact a young person’s growth and pubertal development.”
She adds that almost every pediatric lupus patient with clinical symptoms will receive some amount of steroids.
“Infection is probably the biggest morbidity and mortality risk early on, and steroids dramatically increase the risk of infection."
Avoiding steroid-related weight gain is also particularly important for young people with lupus as the disease itself elevates the risk of cardiovascular disease and additional weight gain further exacerbates these risks.
“To make matters worse, steroids can cause a range of neuropsychiatric problems. At a time when adolescents are trying to develop their own self-identities and peer relationships, it can be very distressing for them to go through some of the physical changes that come with steroid-toxicity, and then still be able to feel like they can function in a normal social environment.”
Dr Chang says that even though there are now alternative drugs available to clinicians, widespread uptake can take 10 years for them to be utilized in practice, and even then, they could be used more effectively.
“We do have a lot of steroid-sparing agents available, but we are perhaps not using them as effectively as we could, in part because so many of these are relatively new to the market. In general, I think clinicians are still used to the standard practice when these options weren’t available and that leads us to prescribe higher doses of steroids than necessary.
There has been a lot of effort to try to standardize steroid dosing regimens for pediatric lupus, and get away from the older consensus plans that used massive amounts of steroids for long periods of time. The newer plans use lower amounts of steroids and taper far sooner, but perhaps still do not feature as low doses or as fast tapers as they could.”
When she does find herself needing to prescribe steroids to patients, she makes sure to provide a balanced picture that while the steroids will help the disease symptoms, they need to be proactive about managing the side effects.
“I tell my patients they are going to have a love-hate relationship with steroids. While they will make some symptoms feel better, I warn them the side effects are going to be challenging and that they need to contact me if they experience any problems.
We do encourage parents to request a 504 plan so that if the patient falls behind at school there is already a plan in place - and we know that education is so important for future health outcomes."
Dr Chang sees the introduction of the Steritas pediatric Glucocorticoid Toxicity Index (pGTI) as being beneficial in helping to develop even more effective treatment protocols.
“The introduction of a truly objective measure of steroid-toxicity such as the pGTI will help not only identify new steroid-sparing treatments but could also help clinicians be more mindful about how much steroid they are prescribing. It could also trigger clinicians to consider alternative disease-modifying antirheumatic drugs (DMARDs) that could be used to keep toxicity to a minimum.
In my own research, I’m very interested in comparative effectiveness studies, such as which steroid-sparing agents to choose and, in that setting, having an objective measure of steroid-toxicity as a clinical endpoint is really important.”
Dr Chang has recently published research investigating whether newer treatment paradigms have reduced or worsened racial disparities in patient outcomes.[1] Her research shows there is a pressing need to close a significant gap in outcomes for patients of different ethnicities.
If patients (or their parents) can’t get to appointments due to location, costs or time away from work, they end up on steroids for longer and tapers are slower, as the clinician needs to be involved in that process.
“From a health-equity standpoint, the pGTI could prove to be an effective measure to monitor how well a health system is looking after the population it treats. It could be used to determine whether the treatment trajectory of your entire population of lupus patients is headed in the right direction and whether there are marginalized groups that are experiencing higher steroid-toxicities.”
Dr Chang stresses that ensuring equitable access to steroid-sparing treatments and DMARDs will be critical if health systems want to continue to improve outcomes for pediatric lupus patients.
References
- Chang, J.C.,et al. Racial Disparities in Renal Outcomes Over Time Among Hospitalized Children With Systemic Lupus Erythematosus. Arthritis Rheumatology, 2022, 74: 1430-1439. https://doi.org/10.1002/art.42127
Dr Chang is a pediatric rheumatologist and an Assistant Professor of Pediatrics in the Division of Immunology at Boston Children’s Hospital (BCH) and Harvard Medical School. She completed her medical degree at the University of Pennsylvania in 2012, followed by pediatric residency and rheumatology fellowship at the Children’s Hospital of Philadelphia, and a Master of Science in Clinical Epidemiology (MSCE) degree at the University of Pennsylvania in 2018. Upon joining the faculty at BCH, she was appointed Director of Lupus Research for the Lupus Program and serves as principal investigator of the BCH Lupus Registry. She has dedicated her career to studying and improving outcomes of childhood-onset systemic lupus erythematosus and other pediatric rheumatic conditions.