Even though it is best known as an affliction of elderly women that often leads to hip and spinal fractures, confinement to bed, and death, bone-thinning osteoporosis also affects older men. Saddest of all, it can even begin its insidious course in children afflicted with rheumatoid diseases such as lupus and juvenile rheumatoid arthritis. "People often don't realize that kids can get rheumatic diseases," says pediatrician and rheumatology expert Emily von Scheven. While childhood cases of these diseases are rare, rarer still are physicians like von Scheven who specialize in understanding and treating young patients stricken with them.

"Most of these conditions are chronic diseases with varied symptoms and unpredictable outcomes," von Scheven says. "I get to know the families and patients very well. We continually try to control the underlying disease and help them with the inevitable ups and downs, which obviously have a huge impact on psychosocial issues, family functioning and schooling."

Children and lupus

As one of just 200 or so pediatric rheumatologists in the nation (most at academic medical centers), von Scheven sees patients from throughout Northern California, Nevada and Oregon. But her goal is not only to apply current treatment regimens, but also to better understand the natural history of these diseases in children, get a clearer picture of the benefits and risks of treatment and improve treatment so that it is tailored to each child's disease and stage of growth.

Currently, her major focus is lupus, an autoimmune disorder that attacks many different organ systems. "That's part of why I like rheumatology," von Scheven says. "Many medical subspecialties are very specific for particular organ systems, but I really get to take care of the whole child."

Lupus is the most deadly childhood rheumatoid disorder. It is treated with substantial doses of steroids, and it is often associated with osteoporosis and bone loss. "Lupus used to kill people by causing kidney damage and life-threatening infections and brain damage," von Scheven says. "Now, because our drugs are so much better, patients are surviving until adulthood." But survival with medication, particularly steroids, carries its own risks: early onset of atherosclerosis, infertility, cancers, osteoporosis, stunted growth, diabetes, glaucoma, cataracts and weight gain. Indeed, finding the balance between risk and benefit, especially in mild cases of pediatric rheumatoid disease in which both the disease and the treatment may contribute to less-than-optimal bone growth, is one of von Scheven's most uncertain tasks.

To learn more about the natural history of lupus in children -- especially the disease's impact on growing bones -- von Scheven is collecting clinical data, information from physical examinations and radiographic bone-density measurements. To conduct the study, she first had to evaluate various techniques for gauging bone density -- primarily dual X-ray absorptiometry, but also CT scans -- because the techniques had been developed and calibrated for adults. No one had standardized ways of measuring bone density and interpreting theresults in normal children, let alone children whose illnesses put them at risk for osteoporosis.

Bone mass accrues during childhood until about age 20, at which time, von Scheven says, "You have your bank of bone for life." Adults whose childhood ailments leave them with less-than-normal peak bone mass early on are at a disadvantage as the inevitable bone loss associated with aging progresses.

Unlike adults, children with lupus rarely have mild disease. When lupus strikes children, it often does so between the age of seven and the onset of puberty. Within this pediatric and adolescent age range there does not appear to be a dramatic difference in the severity of lupus, von Scheven says. But there may be dramatic differences in how the disease impacts growth and development in children of different ages.

Whether osteoporosis and the stunting of growth are more severe with earlier onset of lupus is not known, and it is something von Scheven is trying to answer through her research. She suspects that children who are diagnosed young and whose diseases are brought under control before puberty may have a chance to catch up and grow normally. Similarly, older teens whose skeletons are already mature before they experience any lupus symptoms are likely to already have attained near normal peak bone mass. For those whose disease hits at an in-between age, just when they would be growing taller and accumulating bone mass, the consequences may be far worse.

Other pediatric researchers and clinicians stand to benefit, not only from what von Scheven learns about the natural history of the disease and about treatment side effects, but also from the techniques she has developed for measuring bone density in normal and sick children. "All the information gained about how to measure bones in kids and how to account for other changes in the skeleton when interpreting the bone density measurements has application to any childhood chronic illness, because any chronic childhood illness is going to interfere with the normal process of bone acquisition," she says.

Considering the child

Von Scheven has been at UCSF seven years and is completing a master's degree in clinical research design. While her research program is now gathering steam, with support from the UCSF Pediatric Clinical Research Center and the UCSF Osteoporosis and Arthritis Research Center, she was not always on the fast track. She entered Colby College, a small liberal arts school in Maine, with a well-developed interest in life sciences. She became curious about medicine while participating in a program that enabled undergraduates to get the flavor of different medical specialties by doing "rotations" accompanying physicians at a local hospital. She nonetheless spent two years doing laboratory research before deciding to attend medical school and to practice pediatrics.

Having a background in research at the molecular level is useful, von Scheven says, but she finds clinical research to have closer connections to other aspects of both her patients' lives and her own. Von Scheven is married to an attorney and has two young children. "My desire to have a healthy family impacts my career choices," she says. In pediatric rheumatology she has found her niche.

"A child is a very dynamic organism," she says. "And I'm very interested in how these diseases affect such an organism. When you develop an intervention that reduces mortality or morbidity in young people, it can have a huge impact over the course of a lifetime for each child who benefits. There is a tremendous opportunity to really make a difference."