Snuffing Out Inflammation

Drug Shows Promising Results in Treating Localized Scleroderma in Children, UNM Study Shows

Armani Sanchez was a toddler when her mother Alma discovered a small brown spot underneath her left arm. A doctor told her it was eczema and said it would go away as the girl grew older - but the problem grew worse.

"I noticed where it started spreading to the upper arm," Alma says. "I ended up buying the eczema creams, shampoos, lotions - nothing was helping."

For Alma and her husband José it was the start of an odyssey of medical visits in search of a diagnosis. Armani was seen in Las Cruces and Silver City before a doctor in Albuquerque confirmed a diagnosis of localized scleroderma - scarring and hardening of the skin that results from inflammation.

"When she saw Armani and saw how bad it was, she said, 'I need to send you to somebody who's really good with this,'" Alma says. "That's when we were transferred to Dr. Kalampokis."

Ioannis Kalampokis, MD, PhD, assistant professor in The University of New Mexico Division of Pediatric Rheumatology, sees hundreds of children from all over the state with autoimmune and inflammatory conditions - many of them rare.

As it happened, he was already using a new therapy for treating localized scleroderma in children.

Doctors don't know what causes scleroderma. There is no cure, so they focus on controlling the inflammation with medication. The standard treatment is a combination of steroids and methotrexate, a cancer drug, but it doesn't work for everyone.

Kalampokis was using abatacept off-label in children with localized scleroderma. Abatacept is a drug that interferes with the activation of T cells thought to play a key role in triggering inflammation as part of the body's immune response. Studies in Europe have shown it is effective in adults with localized scleroderma, but it has seldom been used in children with the condition.

Kalampokis served as lead author of a landmark paper recently published in the journal Seminars in Arthritis and Rheumatism with colleagues Aimee Smidt, MD, professor and chair in the Department of Dermatology, and Belina Yi, DO, a resident in the Department of Pediatrics, that describes the successful use of abatacept in eight young UNM patients, including Armani.

"This is the first published paper on abatacept as a treatment for localized scleroderma in children in the world, and the first on either children or adults in the United States," Kalampokis says.

Abatacept, which previously had won FDA approval to treat juvenile arthritis, has a good safety profile, he says. In children with localized scleroderma, it snuffs out the inflammation that damages the skin. "Once you put out the inflammatory response with steroids early in the treatment, the challenge is to actually keep it down so it never comes back," he says.

Kalampokis started prescribing abatacept for scleroderma patients referred to him by dermatologists. "They had basically failed all other treatments," he says. "I used it in a few patients who had failed previous treatments and observed good outcomes, then I started using it on new patients."

Dermatology chair Aimee Smidt says localized scleroderma is often a very challenging disorder, and patients often see both dermatologists and rheumatologists.

"Dermatologists are taught to use select immunomodulatory medications, but for many patients these are simply not enough," she says. "This study is truly a landmark, in that it will share our experience at UNM successfully treating children with scleroderma in this safe and novel way, and will likely help children with this rare disorder around the country."

The study was a retrospective review of treatments that were provided as part of regular care in the UNM pediatric rheumatology clinic. Kalampokis stresses that further studies will be needed to fully assess the therapeutic potential of abatacept in localized scleroderma.

By the time Kalampokis saw Armani Sanchez in March 2018, her scleroderma had spread to other parts of her body.

The scarring from the disease had reached the point where she couldn't make a fist, touch her shoulder or stretch out her arm. Kalampokis started her on monthly infusions of abatacept, as well as an immunomodulatory drug called mycophenolate and infusions of a steroid.

Armani responded to the regimen immediately, her mother says.

"From the first day when she got the medication, her skin tone changed," Alma says. Her skin lesions stopped progressing, and with the help of physical therapy she gained a greater range of motion in her left wrist and elbow.

Alma credits Kalampokis with turning her daughter's life around and communicating his findings effectively.

"I am so thankful that we found him," she says. "He took every question that I needed to be answered. He still does it to this day."