Key result
Belimumab with immunosuppressants normalizes triglycerides and eliminates GPIHBP1 autoantibodies in autoimmune hypertriglyceridemia.
Case Report (n=1)
Belimumab combined with immunosuppressants effectively reversed rare severe autoimmune hypertriglyceridemia caused by GPIHBP1 autoantibodies in a pediatric patient with SLE.
Hypothesis-generating for belimumab in SLE-related GPIHBP1 autoimmunity; prospective trials needed before clinical adoption.
Glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1 (GPIHBP1) is critical for transporting lipoprotein lipase (LPL) to the capillary lumen, where LPL breaks down triglycerides in triglyceride-rich lipoproteins. We herein report a 12-year-old Chinese girl who presented with severe hypertriglyceridemia and a recent diagnosis of systemic lupus erythematosus (SLE). She was first noted to have severe hypertriglyceridemia at 8.5 years old, complicated by three episodes of acute pancreatitis within 2 years. Between these episodes, her plasma triglycerides remained elevated, but at lower levels. Next-generation sequencing for primary hypertriglyceridemia yielded no significant findings. Investigations for secondary causes, to include fasting glucose, HbA1c, and thyroid function testing, were unrevealing. Given the fluctuating triglyceride levels and negative genetic testing for primary hypertriglyceridemia in the background of SLE, autoimmune hypertriglyceridemia was suspected. The diagnosis of GPIHBP1 autoantibody syndrome was confirmed by an elevated GPIHBP1 autoantibody titer and a low LPL mass in her serum. Her SLE was well controlled with immunosuppressants and belimumab. Fenofibrate and omega-3 fatty acids, which were initially prescribed for her hypertriglyceridemia, were later discontinued. The GPIHBP1 autoantibody and LPL mass normalized 2 years after diagnosis. This case illustrates hypertriglyceridemia caused by a rare disease entity associated with autoantibodies against the GPIHBP1 protein. This entity is worth considering after excluding genetic and common secondary causes of hypertriglyceridemia, particularly in a patient with a history of autoimmune disease.
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Lai et al. (2026) conducted a case report in GPIHBP1 autoantibody-related hypertriglyceridemia and Systemic Lupus Erythematosus (n=1). Belimumab and immunosuppressants was evaluated on Triglyceride levels, GPIHBP1 autoantibody titer, and LPL mass. Treatment with belimumab and immunosuppressants normalized triglyceride levels, eliminated GPIHBP1 autoantibodies, and restored LPL mass after 2 years in a girl with SLE and autoimmune hypertriglyceridemia.
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