'The cure is in front of you': The case for stem cell transplants in systemic sclerosis
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DESTIN, Fla. — Stem cell transplantation, accompanied by total body radiation, is the only “cure” for progressive systemic sclerosis currently available, according to a speaker at the Congress of Clinical Rheumatology East annual meeting.
“Mycophenolate doesn’t do much. Cytoxan doesn’t do much. Tocilizumab’s not going to cure systemic sclerosis. Rituximab will not,” Dinesh Khanna, MD, MSc, professor of rheumatology at the University of Michigan, told attendees. “If you have systemic sclerosis that looks like it’s progressive, the cure is in front of you, which is total body radiation and stem cell transplantation.”
Khanna asked audience members to raise their hands if they had ever had a patient with scleroderma undergo the procedure. Noting only four hands in the air, he urged that hematopoietic stem cell transplantation be considered more often. Acknowledging that the total body radiation involved can be “scary,” he outlined data supporting its benefit.
In the SCOT trial, patients with scleroderma were randomized to Cytoxan (cyclophosphamide, Bristol Myers Squibb) or to transplant. The results showed a significantly lower rate of event-free survival over 5 to 10 years in the Cytoxan group compared with the transplant group.
According to Khanna, the data demonstrate that “transplant does not cure everybody,” though it remains “far, far better than giving high-dose chemotherapy” such as Cytoxan. He added that the benefits of Cytoxan and mycophenolate mofetil do not pan out in the long term.
“It makes you feel good, because we are doing something,” Khanna said. “But when you look at Cytoxan patients at the end of 10 years, 60% to 70% of the patients have a relapse of their disease.”
That said, Khanna acknowledged several limitations of stem cell transplantation for patients with scleroderma, including its associated toxicity and potentially limited applicability.
“We do 12 to 15 myeloablative transplants every year. We see more than 2,000 patients. Why is that?” Khanna said. “Because you need the right patient with the functioning heart, with functioning kidneys, with a functioning liver, just in the perfect window of opportunity to be able to transplant.”
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Khanna D. What is New in Systemic Sclerosis. Presented at The Congress of Clinical Rheumatology East; May 9-12, 2024; Destin, Florida (hybrid meeting).
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