Desensitization of olipudase alfa-induced anaphylaxis in a child with chronic neurovisceral acid sphingomyelinase deficiency

• DOI: 10.1016/j.ymgmr.2024.101120
• Publication Date: 2024-07-17T02:53:46Z
• Authors: Laura Fiori; Veronica Maria Tagi; Chiara Montanari; Mirko Gambino; Veronica Carlevatti; Carmela Zizzo; Enza D'Auria; Dario Dilillo; Elvira Verduci; Gianvincenzo Zucc...
• Keywords: Acid sphingomyelinase

Olipudase alfa is indicated for the non-central nervous system manifestations of Acid sphingomyelinase deficiency (ASMD). Anaphylaxis a very rare and life-threatening adverse reaction described this drug. Here, we report case 2-year-old boy affected by chronic neurovisceral ASMD who experienced signs hypersensitivity reactions to olipudase since administered dose 1 mg/kg during escalation proper anaphylactic second administration target therapeutic 3 mg/kg. The treatment was stopped 15 weeks then 7-step desensitization protocol with infused 0.03 applied. Subsequent gradual resumed, successfully reaching 0.3 Moreover, biochemical, radiological disease indexes, which were increased discontinuation, have gradually improved restart treatment. However, at 0.6 mg/kg, patient another drug facial urticarial rash bronchospasm, requiring adrenaline, methylprednisolone, inhaled salbutamol. This highlights need customize according individual tolerance raises issue achieving established in most sensitive children.