Efficacy of belimumab for severe childhood-onset systemic lupus erythematosus with diffuse proliferative...

Therapy of childhood-onset systemic lupus erythematosus (cSLE) with drugs is unsatisfactory. Some new drugs such as belimumab and rituximab may improve the course of severe cSLE, although there are few reports on treatment efficiency for these new drugs, especially belimumab.

Here we report on a 16-year-old girl who was diagnosed with cSLE at the age of 13. After several immunosuppressive treatments, which included high-dose steroids, hydroxychloroquine sulfate, cyclophosphamide, etc for blood system damage, she showed little clinical improvement and developed severe pericarditis. Induction treatment with a combination of intravenous high-dose steroids, methylprednisolone, and cyclophosphamide was started, but, after 55 days, the patient developed lupus encephalopathy, lung infection, and lupus nephritis. After using high-dose steroids, cyclophosphamide, plasma exchange, gamma globulin, and appropriate anti-pulmonary inflammation drugs, treatment with tacrolimus was attempted but poorly tolerated by the patient and withdrawn. Eventually, in December 2019, belimumab was initiated on an off-label basis as a last resource to treat lupus nephritis. Belimumab was well tolerated by the patient and resulted in a rapid and marked improvement in clinical symptoms and reduction in proteinuria, serum complement levels and anti-double strand DNA antibodies titer; of note, the patient developed no infectious complications.

Treatment with belimumab could result in prompt remission of severe cSLE with multiple organ damage without the pulmonary infection side effects for children deemed intolerant to conventional and second-line induction therapies. Belimumab should be considered as a potentially efficacious treatment in patients in severe childhood-onset systemic lupus erythematosus.