Researchers found that peg-interferon-alfa2a (peg-IFN-α) offers a promising disease-modifying approach for preventing bone damage in non-advanced systemic mastocytosis (SM) with refractory bone involvement, potentially by reducing mast cell burden, according to a recent study published in Hematology Reports.
Peg-IFN-α is an immunotherapy used to treat blood cancers and other conditions. Prior research had indicated it may be effective in mast cell–related osteoporosis.
What is a mast cell?
Mast cells are specialized cells of the immune system that mediate inflammatory responses and allergic reactions. They are found in the body’s connective tissue.
The authors aimed to examine low-dose peg-IFN-α in a cohort of 12 patients with indolent or smoldering SM and osteoporosis. The patients were treated with 90-135 µg of peg-IFN-α a week over a median of 59 months. They were also given bisphosphonates, a medication used to treat osteoporosis.
The researchers assessed the treatment’s effect on fracture prevention, bone density changes, tryptase reduction and tolerability over long-term follow-up.
Following treatment, only one patient had a new fracture. Osteolytic lesions (areas of dense bone growth) stabilized in five patients, and three patients had a regression of the osteoporosis pattern. Patients reported they felt less bone pain and had better physical function.
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The treatment was largely well-tolerated, though some patients had mild flu-like symptoms and fatigue.
One patient’s SM progressed during the study, and their treatment plan was changed accordingly.
“These outcomes suggest that peg-IFN-α may play a protective role in halting skeletal deterioration in non-advanced SM, likely by reducing mast cell burden and suppressing the release of bone-resorptive mediators,” the authors noted. “These preliminary findings support the potential of peg-IFN-α as a disease-modifying strategy to prevent progression of bone damage in selected patients with ISM and SSM who exhibit refractory or high-risk bone involvement.”
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