A new research review presented at the American Society of Hematology’s recent annual meeting evaluates the efficacy and safety of neonatal Fc receptor (FcRn) inhibitors in adults with immune thrombocytopenia (ITP). The results showed encouraging outcomes, suggesting that this novel class of immunotherapy may represent an important advancement in treating conditions like ITP and, potentially, fetal and neonatal alloimmune thrombocytopenia (FNAIT).
Similar to FNAIT, ITP is caused by antibodies that mistakenly target platelets. While the research focuses on ITP, it highlights how therapies that reduce harmful antibodies could one day benefit conditions like FNAIT, where antibody-mediated platelet destruction occurs before birth.
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FcRn inhibitors work by blocking the neonatal Fc receptor (FcRn), a protein that normally protects immunoglobulin G (IgG) antibodies from being broken down. When FcRn is blocked, IgG antibodies are cleared from the bloodstream more quickly, including the antibodies responsible for platelet destruction. This approach does not shut down the immune system as a whole, but instead targets the antibodies causing the problem.
In the studies reviewed, adults receiving FcRn inhibitors experienced higher platelet counts compared with those receiving a placebo and a reduced need for emergency treatments. The treatments were generally well tolerated with little increased risk of adverse events.
Researchers say these results are a meaningful step toward expanding the therapeutic options for ITP. For FNAIT, this raises the possibility that FcRn inhibitors could one day be studied as an alternative or additional approach to current treatments such as IVIG during pregnancy.
“These findings support FcRn blockade as a promising and well-tolerated treatment strategy,” the study authors concluded. “Further large-scale, long-term trials are warranted to define its role in standard treatment algorithms.”
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