Paget: It is a pleasure to introduce to you today Dr. Thomas Lehman, an internationally recognized specialist in pediatric autoimmune and rheumatologic disorders. Dr. Lehman, what proportion of children develop arthritis or other types of musculoskeletal problems?
Lehman: Arthritis is actually relatively common in childhood. It is about 2 per 1000 in any given year. Musculoskeletal problems are extremely common -- probably 10 to 20 children per 100 have some kind of significant musculoskeletal complaint over any given year. In most of them. it is minor injuries, minor over-use in athletics, and with gentle reassurance and appropriate education, the problem resolves. For children with arthritis, however, they need good care. Too many of the children who have arthritis are dismissed as simply having an overuse injury or muscular problem. Then they don't get the proper care.
In childhood, as the body is growing, if something like arthritis is allowed to go on undetected or improperly treated, it produces permanent problems that can last well into adulthood.
Paget: What are the typical symptoms or complaints that a child or parent may have as they go to their primary care pediatrician?
Lehman: Often, parents go to the doctor and say "He seems to be limping" or "He looks a little stiff when he wakes up in the morning." Because children with arthritis frequently have morning stiffness that resolves over a period of time, the pediatrician will examine the child quickly, not recognize the child's limitation in motion, and say "Everything looks fine to me - it must be growing pains". But children should never have growing pains during the day. Growing pains are a night-time phenomenon. Sometimes they may wake the child up in the middle of the night, but when that child wakes up in the morning, he should be fine. If he isn't fine when he wakes up in the morning, it's not growing pains.
Paget: What are the more typical types of medical problems and joint problems that a child would have, and are they differentiated with regard to age?
Lehman: Children with arthritis have a large variety of conditions. Although we used to talk about juvenile rheumatoid arthritis to refer to any child under the age of 16 with arthritis, we now know that there are 15 to 20 different conditions that cause arthritis in childhood. Some, like pauciarticular juvenile arthritis, frequently start in young children, little boys and little girls with a swollen knee, who often just seem too stiff or don't walk right when they first get up in the morning, but they are fine later. Often, it takes a number of months before the parents realize there is really a problem when someone points out their child's knee is swollen.
Other examples are the older children who often come in and they've been told previously that they are clumsy. Or they've had multiple sprained ankles or multiple injuries and they've been told it's just because they are bad athletes or they don't loosen up. But they have one of the spondyloarthropathies. When you recognize a diagnosis and give them the appropriate treatment, they feel much better and they do much better.
Throughout the whole spectrum of rheumatic disease, each age group has its most common presentations - its most common diseases - and each of those has a different outcome and a different best treatment.
Paget: What should primary care pediatricians do on a regular basis to make sure that they don't miss these problems?
Lehman: The most important advice for primary care pediatricians is "Listen to mothers." We don't expect the primary care pediatrician to understand all of the different diagnoses of childhood rheumatic disease, and we don't need them to know which one it is, but if the mother comes in and says "My child doesn't walk right in the morning," or "My child has a problem," the primary care pediatrician shouldn't be dismissing it as growing pains without a proper evaluation. If that mother comes in and mentions pain in the child's joints more than once, that child should be referred to a rheumatologist so that somebody can properly reassure the mother there isn't a problem or properly diagnose what is going on.
Paget: What are some of the danger signs that the mother and the pediatrician should keep in mind?
Lehman: I think pain and stiffness are the two biggest ones. Children don't normally complain of pain during the day. They don't complain of back pain and knee pain unless something is going on. Stiffness -- there is no good reason a child should be stiff when he wakes up in the morning or walk funny when he wakes up in the morning unless he has an underlying condition. Physicians and parents need to be attentive to these kinds of problems and make sure that children with these problems are appropriately treated.
Paget: What about other symptoms, such as fever, weight loss, failure to thrive?
Lehman: When you see children, especially the older child, you have to be very concerned about what I call adolescent failure to thrive. The child is not doing well. The child has been losing weight. The child is losing interest in school, or has had rashes, fevers or night sweats. Those can be symptoms of many different chronic diseases. The rheumatic diseases, such as lupus, scleroderma, and dermatomyositis, all may start with those conditions. So can tuberculosis, leukemia, and Hodgkin's disease. A child who is failing to thrive, for whatever reason in that older age group, should be referred to an appropriate specialist for a complete diagnostic work-up.
Paget: Why is it important to make a diagnosis as early as possible?
Lehman: In every one of the childhood rheumatic diseases, it doesn't just go away. They don't just grow out of it. There is a small proportion of young children with pauciarticular JRA who do, in fact, get well. All the other rheumatic diseases progressively cause problems over time. The sooner you recognize the disease, the faster you can start appropriate therapy, put that fire out and prevent damage from being done and prevent continued problems from occurring.
Paget: Let's take a few specific disorders. What is your diagnostic, clinical and therapeutic approach to what is called juvenile rheumatoid arthritis?
Lehman: The first thing to understand is that a "juvenile rheumatoid arthritis" doesn't exist anymore. We now have "juvenile inflammatory arthritis or "juvenile idiopathic arthritis." The reason is that juvenile rheumatoid arthritis has nothing to do with rheumatoid arthritis in adults. It is not rheumatoid factor positive; it should be rheumatoid factor negative. So it's a different disease - it's JIA. Within that, there are a number of different conditions. If you take a young child who is stiff when she wakes up in the morning, has a sore knee, that child probably has typical pauciarticular JIA. With appropriate therapy, we can prevent her from having leg length discrepancies, which can come from uncontrolled inflammation. We can prevent her from developing contractures and get her back as a fully functional young adult or young child at that point.
More importantly, many of these children have painless and unsuspected eye disease. They may have eye disease picked up early when they were referred to an ophthalmologist and, when properly diagnosed, we can fix their vision. If that goes undiagnosed for a long period of time, these children can end up blind. We need to catch these children; we need to figure out what is going on, and we need to get them appropriately taken care of.
In the older child, I frequently see children with what I call sausage digit - one big swollen finger. People think "Well, she recurrently stubs her finger" or sometimes it's a toe. "She must recurrently stub her toe or maybe it's always basketball injury, they are chronically doing it." These children often have arthritis in multiple joints that everyone has been dismissing. This is a chronic form of arthritis that is going to cause significant disability in adult life if it is not detected.
Once you detect the disease, you can get the child on appropriate disease-modifying agents and, instead of having somebody who grows up to life-long complaints, - "Yeah, I've always had back problems" or "Yeah, my knees are bad - I must have played too much football or I must have done something skiing," you restore them to good health. They get their strength back, and you can make sure they grow up to be good-function adults.
With proper care today, the vast majority of my patients will walk right past you on the street and you won't realize that they are children who have had arthritis. In the old days, you used to walk into the JRA clinic and there would be kids in wheelchairs. We don't have wheelchairs anymore. Proper care keeps these children mobile, productive members of society, out there working, having their own families. They are fully functional individuals that you don't see.
Paget: You mentioned the term "disease-modifying". Could you tell the website audience what that means?
Lehman: Sure, disease-modifying drugs are the drugs that will change how the outcome of the disease is going to be. One of the key concepts in rheumatic disease right now is that, with uncontrolled disease, the damage index continues to go up at a relatively constant slope over time. If you treat with a disease-modifying agent, you change the slope of that damage index so that, over time, instead of having increased damage and being an increasingly dependent member of society, the child can keep functioning at a higher, employable working level. And, when we actually look at what happens to the children now, they tend to come out above average. They do very well in their social and their economic outcome. They are a little bit more focused because they are not out there as much as the others, and they do very well.
Paget: What kind of medications are you talking about?
Lehman: Virtually every medicine that is used by the adult rheumatologist is used by the experienced pediatric rheumatologist. Long ago, we used to have worry that they hadn't been specifically tested in children. But if you held back until every drug that we used in adults had been specifically tested in children, you wouldn't be able to give them any of the latest therapy. And you would be depriving these children -- who are growing and at the stage where their joints are the most vulnerable to most long-term deformity -- of the best possible medicine.
So right now we go through everything that is available for adult rheumatology and, in the appropriate child, with very careful monitoring, we use those drugs. And we are getting excellent outcomes.
Paget: That includes what medications specifically?
Lehman: That includes all of the nonsteroidal anti-inflammatory drugs. For severe arthritis, we use a lot of sulfasalazine. Methotrexate is routinely used in pediatric rheumatology. Enbrel, the new biologic modifier, is being used. Kineret is being used. All of these drugs now, especially with the new emphasis by the FDA on pediatric approvals, have been tested in children. Although we don't wait for the official results to be out, we wait until it has been proven safe for children, and we then can show that these are very effective drugs and that we can alter the outcome for these children.
Paget: Let's move to a different disorder - systemic lupus erythematosus. Do the children present in different ways than adults with that disorder, and how do you treat them?
Lehman: Children with systemic lupus are a very important sub-group. As you know, we have a big center for children with systemic lupus erythematosus here and tremendous experience with that disease. Sometimes they present explosively, but far more often, they present as that childhood or adolescent failure to thrive. We see a child who is just not doing well, seems to be withdrawing from school, isn't acting right, seems to be a little depressed, losing weight. Sometimes they are having rash, sometimes they are having fever, but often the family doesn't really recognize that rash and fever as coming from lupus. They don't know what they are coming from. Often the child has arthritis, but the family can't recognize it; the kid just hurts all over.
Once we see those children, they get started on an appropriate low dose of corticosteroids. Many of them get much better with just an appropriate low dose. When they don't, when it appears that they are going to require moderate or high doses of steroids for any period of time, we switch them over to immunosuppressive therapy with consistent use of systematic intravenous cyclophosphamide. We have a large number of children who have biopsy-proven diffuse glomerulonephritis who now look normal. They are off medications, they are productive young adults on no medicine at all, they are not Cushingoid, they don't have avascular necrosis. All of those long-term steroid side effects, that cause so much morbidity, we can avoid by getting them off the steroids and on appropriate immunosuppressive drugs.
We have been extremely successful in keeping those children. So instead of saying "I hated the doctor, the steroids gave me acne, the steroids made me fat, the steroids gave me stretch marks," they come to us when they are on the intravenous Cytoxan and say "I don't even remember that I have lupus except when I have to come to my doctor appointments".
Paget: How important is the partnership among the child, the parents, and the physician in defining a good outcome?
Lehman: It's crucial. You have to have the faith of the child and the family in the physician. A treating team of physician, parent and child working together is going to get a good outcome. If you have three poles, each pointing in a different direction, you can't go anywhere. Whenever you are working in pediatric rheumatology, one of our key issues is recognizing that we are dealing with children, who don't necessarily have the same attitude towards illness and responsibility as adults have. We are also dealing with parents, who may have conflicting opinions about what should be done for a child and an incomplete understanding of the importance of the medication. And, we are dealing with ourselves.
Pediatric rheumatologists are primarily educators. We have to educate that child to know what to expect and how to respond. We have to educate the parents so that they believe in the therapy that we are giving to their child. When the three of us work together then we can get an excellent outcome. If one part of the team is running off in another direction, it is never going to work out as well. One of the most important aspects of being a pediatric rheumatologist is knowing you have to be the coach, you have to nurture the team, and keep the team together. That is very different from being an adult rheumatologist, where it is just you and that patient. I have to keep dad in the picture, I have to keep mom in the picture, I have to keep the patient believing in what we are doing, and I have to worry about what grandma is saying at home.
Paget: Things have gotten so much better, but they clearly need to go further. What can people expect for the future with regard to progress?
Lehman: We have made tremendous progress in pediatric rheumatology over the past 20 years. I have been doing this for 25 years now and, having started off walking into clinics full of children in wheelchairs, we have virtually banished wheelchairs from this hospital. The biggest obstacle right now is that there aren't enough pediatric rheumatologists and there are too many children who aren't getting the care early enough in their disease. They are being dismissed as having growing pains, dismissed as having a minor injury from sports, and not getting to the physician soon enough to fix the problems.
If they get to us early, we can put out the small fires, and we can get excellent results. If they come to us after they have arthritis for 10 or 15 years, we are going to replace their joints and make them functional again. But we can't restore them the way we can if we got them the proper therapy early in the disease. Then we can make them perfectly functional adults.
Paget: Thank you, Dr. Lehman.
Dr. Lehman was interviewed by Dr. Stephen A. Paget, Physician-in-Chief Hospital for Special Surgery