Customize your JAMA Network experience by selecting one or more topics from the list below.
A drug initially approved in 1997 for non-Hodgkin lymphoma recently got the green light to treat patients aged 2 years or older who have granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). Rituximab is the first drug approved for children with these rare forms of vasculitis. It was approved for adults with GPA or MPA in 2011.
In an international clinical trial, 25 patients aged 2 to 18 years with GPA or MPA were treated with intravenous methylprednisolone before starting a 6-month remission induction phase of rituximab infusions. All patients completed the remission induction phase; 24 of 25 completed 18 months or more of follow-up. Investigators reported that 56% of patients achieved remission within 6 months; 100% were in remission within 18 months.
Rituximab’s safety profile was similar in children as in adults. In the pediatric trial, infusion-related reactions were the most common adverse events. Others included headache, nausea, upper respiratory tract infection, and upper abdominal pain. Clinicians are advised to monitor patients for tumor lysis syndrome, cardiac adverse reactions, renal toxicity, and bowel obstruction and perforation.
A boxed warning draws attention to increased risks of fatal infusion reactions, potentially fatal severe skin and mouth reactions, hepatitis B virus reactivation, and progressive multifocal leukoencephalopathy.
Voelker R. Children With Rare Vasculitis Have New Treatment Option. JAMA. 2019;322(17):1644. doi:https://doi.org/10.1001/jama.2019.17531
Create a personal account or sign in to: