Lupus Research Improves Kids' Lives

The Division of Rheumatology at Cincinnati Children’s is working with other specialists at the medical center and with researchers around the world to improve outcomes for children with lupus.  

Preserving fertility

Cytoxan (cyclophosphamide) is used to suppress the immune system in children with severe lupus. However, this drug is known to lead to infertility.

Researchers here and at nine other medical centers recently completed a five-year, FDA-sponsored clinical trial of a drug called triptorelin, sold under the brand name Trelstar to treat men with prostate cancer. The goal: to determine whether this GnRH agonist also can protect ovaries in patients with lupus. Study results have not yet been published.

Child-focused dosing

The immunosuppressant mycophenolate mofetil (CellCept) is used to combat kidney complications from lupus. However, this drug is expensive, and when two children of the same weight take the medication, studies show more than 50 percent variation in drug levels in the blood.

Hermine Brunner, MD, MSc, and colleagues are working with Alexander Vinks, PharmD, PhD, director of the Pediatric Pharmacology Research Unit at Cincinnati Children’s, to develop customized tests to help doctors determine the correct dose of CellCept for a particular child.

Kidney damage biomarkers

Some of the most important lupus research at Cincinnati Children’s is related to reducing potential kidney damage. Scientists are testing whether a panel of lupus nephritis biomarkers – developed at Cincinnati Children’s – can detect signs of kidney disease in children with lupus before serious
damage occurs.

“Currently we detect kidney lupus at the time when there’s already kidney damage,” Brunner says. The test could help tailor medication doses, and minimize side effects. It also could reduce the need for repeated kidney biopsies.“Biopsies are expensive, invasive and not very popular among the patients,” Brunner says.

Protecting the brain

In children, brain inflammation from lupus can lead to life-long learning disabilities. In fact, as many as 40 percent of children with lupus have “soft” cognitive impairment, Brunner says. But detecting early signs of cognitive dysfunction is difficult in clinical settings.

Now researchers at Cincinnati Children’s are conducting functional imaging studies to map out how lupus affects the brain. They are also co-developing a software program, together with the Center for the Study of Human Operator Performance (C-SHOP) and the Department of Defense to assess whether children with lupus can benefit from a computer-based screening tool that measures cognitive function among soldiers injured by roadside bombs.

“Currently, cognitive testing is a four- to five-hour test that requires analysis. Insurers rarely pay,” Brunner says. “And there’s a shortage of neuropsychologists who can do the testing.”

Setting new care standards

Brunner and colleagues published a paper in June 2010 in Arthritis Care & Research about efforts to develop global criteria to define lupus flare-ups. More details will emerge in months to come.

“Protocols for treatment plans can lead to better outcomes,” Brunner says. “We are coming up with benchmark measures for lupus and piloting methods to improve adherence.”