Stem cell transplant safely eases scleroderma symptoms for teen
Report supports ASCT as 'important therapeutic option' for SSc
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Autologous stem cell transplant (ASCT), a procedure that essentially aims to reset the immune system, was effective for controlling scleroderma in a teenager with severe disease that wasn’t responding well to medications.
“ASCT may represent an important therapeutic option, particularly perhaps in patients who develop severe disease at a young age,” the researchers wrote.
The report, “Autologous stem cell transplant for severe, progressive juvenile systemic sclerosis,” was published in Stem Cells Translational Medicine.
Scleroderma, also called systemic sclerosis (SSc), is an inflammatory disorder characterized by abnormal scarring that can affect the skin as well as organs throughout the body. Inflammatory disorders such as SSc are marked by dysregulation of the immune system, which normally helps protect the body from infections.
ASCT basically intends to replace a patient’s stem cells. After intensive treatments such as chemotherapy and radiation wipe out overly reactive immune cells, the collected stem cells are then transplanted back into the patient to grow a new immune system. The aim is essentially to reset the immune system’s activity so it won’t drive inflammatory disease.
Teen showed ‘dramatic’ improvement after 1 year
Here, researchers described the case of a 17-year-old boy with juvenile SSc who underwent ASCT. The boy had been diagnosed with SSc at age 13 when he was experiencing complications such as widespread rash, pain in both knees, and muscle weakness. In the years since, he’d tried several immune-suppressing therapies, with mycophenolate mofetil added to the treatment regimen due to increasing weight loss, skin tightening, and difficulty breathing associated with lung disease.
Despite treatment, he was still experiencing substantial symptoms and progressive disease. After carefully going over the potential benefits and risks, the patient, his family, and his care team decided to try ASCT. By pre-transplant assessment, he was experiencing severe malnutrition and skin tightening, but his organ function still enabled the transplant.
The procedure was intensive — ASCT always is, usually requiring at least a few weeks spent in the hospital — and involved radiation and immunosuppressants, but it was completed without any major complications. Lab tests showed the boy’s immune system had started to regrow within two weeks of the transplant.
At follow-up just over a year later, he showed notable easing of symptoms, with less skin tightness and better breathing, joint mobility, and endurance. The researchers said he has not required any additional treatment with disease-modifying antirheumatic drugs.
“He did not experience any clinically significant transplant related complications and showed prompt immune-reconstitution. He showed dramatic skin improvement within a year of ASCT with stabilization of interstitial lung disease (ILD), which is expected to improve with nutritional status and ongoing physical therapy,” the researchers concluded, noting that this case adds to a large body of literature supporting ASCT as an option for young people with SSc.
