Patients with lysosomal storage diseases may require modifications to standard drug desensitization protocols; personalized medicine as well as development of new treatment options are needed.
We present the case of a patient with Morquio A syndrome, with a history of allergic rhinitis and asthma who presented with elosulfase alfa induced anaphylaxis after weekly enzyme replacement therapy since 17 years of age. Desensitization was attempted by using 2 available protocols (found in Guvenir et al and Arroabarren et al). Unfortunately, the patient was unable to tolerate the previously published protocols, leading to unsuccessful desensitization. The patient’s quality of life and health were suboptimal during the period she was unable to receive treatment. Weekly enzyme replacement therapy with elosulfase alfa (Vimizim) is the only Food and Drug Administration–approved treatment of patients with Morquio A syndrome. Before implementing our 4-bag, 18-step modified desensitization protocol, the patient was started on omalizumab, and her asthma and allergic rhinitis were controlled. She also received premedications 30 to 60 minutes before starting the protocol and cetirizine before starting a new bag. This protocol allowed her to successfully reach the final dose needed without any reactions. The patient remained on the protocol for 3 months before removing 1-step per week to achieve the manufacturer’s standard dosing protocol of 1-bag and 7-steps. Our case highlights the importance of personalized medicine and that patients with lysosomal storage diseases might require modifications to standard drug allergy desensitization protocols. It also highlights the need for further research and development of treatment options for patients with lysosomal storage diseases.