Treatment with high-dose sebelipase alfa twice a week can be well tolerated and effective in patients with severe Wolman disease, a rapidly progressive form of lysosomal acid lipase deficiency (LAL-D), according to study results presented at the 26th European Association of Hospital Pharmacists (EAHP) Annual Congress. However, the authors added that it would be necessary to maintain treatment for life.

Sebelipase alfa is a human recombinant enzyme replacement therapy approved by the US Food and Drug Administration (FDA) to treat the underlying cause of LAL-D, and it is marketed under the brand name Kanuma®. Normally, the treatment should be started at a low dose once a week and increased gradually to avoid hypersensitivity reactions. 

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Here, a team of researchers led by Irene Zarra Ferro from Complejo Hospitalario Universitario de Santiago de Compostela in Spain described the safety and efficacy of high-dose sebelipase alfa administered twice a week to treat a 3-month-old baby with aggressive and severe Wolman disease. The patient also had secondary hemophagocytic syndrome, anemia, thrombopenia, hyperferritinemia, altered liver function, and massive hepatosplenomegaly.

The researchers reported that the patient was treated with 11 doses of sebelipase alfa over 35 days, the first dose being 3 mg/kg and the following doses being 5 mg/kg twice a week. After the start of treatment, the patient needed mechanical ventilation, continuous hemodialysis, and repeated red blood cell and platelet transfusions. 

Treatment led to decreases in ferritin, transaminases, and bilirubin. After a month, hepatosplenomegaly was also reduced. There were no adverse effects associated with the treatment.

“After the diagnosis of [Wolman disease] with aggressive and severe presentation, treatment with high-dose twice-weekly [sebelipase alfa] has been an effective and well-tolerated treatment so far in our case,” the researchers wrote.

Reference

Balado AC, de Castro López MJ, Pintos PS, et al. Efficacy and safety of high-dose twice-weekly sebelipase alfa in severe-onset Wolman disease: a case report. Paper presented at: 26th European Association of Hospital Pharmacists (EAHP) Annual Congress; March 23-25, 2022; Vienna, Austria.