Interactive Session Addresses Challenging Cases of Pediatric Lupus

Experts discussed two challenging cases of young patients with lupus during Interactive Challenging Pediatric Lupus Cases: Antiphospholipid Syndrome and Lupus Nephritis on Saturday, Nov. 16. The session will be available on-demand to all registered ACR Convergence 2024 participants after the meeting through Oct. 10, 2025, by logging into the meeting website.

Deborah Levy, MD, Professor of Pediatrics at the University of Toronto, Canada, discussed challenges in the management of antiphospholipid syndrome (APS), an autoimmune thromboinflammatory disease with diverse manifestations.

APS and secondary APS are common in patients with childhood-onset lupus. According to the 2023 ACR/EULAR APS classification criteria, at least one documented clinical criterion plus a positive antiphospholipid antibody test must be present to be classified as APS. However, to be classified as having APS for research purposes, patients must have at least three points from the clinical domains and three points from the laboratory domains. While this classification is useful, it still needs to be validated in pediatric patients.

There are few clinical studies to direct the management of APS in pediatric patients. Thus, management is often based on studies done in adults.

“I like to think of management as three-pronged,” Dr. Levy said. “One is primary thrombosis prevention. That’s really preventing the first clot, and most of the time, that’s before you’ve made a diagnosis of APS syndrome. Second would be secondary thrombosis prevention — treating the acute clot and preventing further clots or new clots. And third would be treatment of the microvascular or other nonthrombotic manifestations.”

Primary prevention in adults focuses on addressing modifiable risk factors and optimizing the management of the underlying autoimmune disease. Patients with systemic lupus erythematosus (SLE) should be treated with hydroxychloroquine, and aspirin should be considered in patients with cardiovascular disease risk factors.

For secondary thrombosis prevention, anticoagulation therapy should be initiated with either a vitamin K antagonist or low molecular weight heparin. Patients with thrombotic APS require long-term anticoagulation therapy. However, 30% of patients continue to experience recurrent thromboses even when they are on anticoagulants.

For the third prong of management, it is important to understand that nonthrombotic manifestations are quite common in APS and will require treatment. However, pulmonary hemorrhage (microvascular APS) is a rare manifestation that requires immunosuppression therapy.

Stacy Ardoin, MD, a board-certified pediatric and adult rheumatologist at Nationwide Children’s Hospital, discussed challenges in the management of severe lupus nephritis (LN), including incomplete renal response, prolonged time to renal response, renal flares, steroid toxicity, tolerability and adherence, and the lack of data in the pediatric population.

“One of the most important things that I think we need to address is, what is our goal when we’re treating these patients with lupus nephritis? Of course, No. 1, our goal is to improve quality of life, limit steroid toxicity, and avoid toxicity from other medications. But from a renal standpoint, what are we trying to achieve?” Dr. Ardoin said.

Despite existing therapies for proliferative LN, renal outcomes remain inadequate for adults and children. Physicians may question when to repeat a renal biopsy when a patient is not responding to treatment. Dr. Ardoin said this approach may be useful to provide more information on disease activity, chronicity, and alternate disease processes.

According to the 2024 KDIGO guidelines, patients with LN should be treated with steroids. Following this, there are several therapeutic options. Traditional treatments include mycophenolate and cyclophosphamide.

The efficacy of standard LN therapies is low, with only 25% of patients having a complete response; however, several new therapeutic options, including belimumab, voclosporin, and obinutuzumab, have shown promise in the treatment of LN.