Researchers from Mass General Brigham have discovered that the drug inebilizumab significantly reduces symptoms in patients with immunoglobulin G4–related disease (IgG4-RD). The findings, from an international phase 3 clinical trial, were published in the prestigious New England Journal of Medicine.
Breakthrough in Rare Disease Treatment
The study, led by Dr. John Stone, a rheumatologist at Massachusetts General Hospital, revealed that inebilizumab reduced the risk of disease symptoms by 87% compared to a placebo. “This is a huge day in the history of this disease,” said Dr. Stone. “We are thrilled to have worked so closely with patients on a trial focused on their specific condition, with hopes that this therapy will soon be available for them.”
IgG4-RD is a rare condition that affects fewer than 200,000 people in the U.S. First identified as a unique disease in 2003, it actually dates back to descriptions in medical literature as early as the late 1800s. The disease is characterized by a build-up of immune cells that produce the IgG4 antibody, which can impact any organ, often affecting multiple organs simultaneously. Commonly involved areas include the pancreas, bile ducts, salivary glands, eyes, lungs, and abdominal tissues.
Complex Symptoms and Challenges in Diagnosis
Symptoms of IgG4-RD vary based on which organs are involved. For example, eye involvement may cause swelling and double vision, while pancreatic involvement can lead to pancreatitis and diabetes. Bile duct issues can result in jaundice. Patients often face a long diagnostic journey, with many being misdiagnosed with cancer and undergoing unnecessary surgeries before receiving the correct diagnosis.
Like many immune-mediated diseases, IgG4-RD causes recurrent flare-ups that require treatment. The standard approach has been the use of steroids, which come with a range of side effects such as weight gain, osteoporosis, anxiety, and a higher risk of infections. Steroids are especially problematic for IgG4-RD patients whose pancreases are affected, as this can exacerbate diabetes due to poor insulin production. “Steroid treatment in these patients often leads to diabetes,” noted Dr. Stone, highlighting the need for better treatment options.
Study Results: Inebilizumab’s Efficacy
In the clinical trial, 135 adults with IgG4-RD were randomly assigned to receive either inebilizumab or a placebo. Over the course of a year, 59.7% of the placebo group experienced disease flare-ups, compared to only 10% in the inebilizumab group. This dramatic reduction underscores the drug’s potential as a new treatment option for IgG4-RD patients.
Inebilizumab, developed by Amgen, targets CD19-expressing B cells, which play a crucial role in IgG4-RD. By depleting these cells, the drug effectively reduces disease activity. However, there are concerns about long-term safety, as B-cell depletion may increase the risk of infections. Patients on this treatment could have reduced responses to vaccines, potentially increasing their risk for severe illnesses like COVID-19 and the flu.
Moving Forward with Caution
To mitigate these risks, researchers recommend that patients receive vaccinations prior to starting inebilizumab and that the drug is used cautiously, guided by blood markers of inflammation. Further studies are needed to confirm the long-term safety profile of inebilizumab, but its initial success in reducing flare-ups offers new hope for those suffering from this challenging condition.
This study marks a significant step forward in the treatment of IgG4-RD, providing a promising alternative to traditional steroid therapy and paving the way for improved patient outcomes.
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