Abstract:
Olipudase alfa is indicated for the non-central nervous system manifestations of Acid sphingomyelinase deficiency (ASMD). Anaphylaxis is a very rare and life-threatening adverse reaction described for this drug. Here, we report the case of a 2-year-old boy affected by chronic neurovisceral ASMD who experienced signs of hypersensitivity reactions to olipudase alfa since the administered dose of 1 mg/kg during dose escalation and a proper anaphylactic reaction during the second administration of the target therapeutic dose of 3 mg/kg. The treatment was stopped for 15 weeks and then a 7-step desensitization protocol with the infused dose of 0.03 mg/kg was applied. Subsequent gradual dose escalation was resumed, successfully reaching the dose of 0.3 mg/kg. Moreover, biochemical, and radiological disease indexes, which were increased during treatment discontinuation, have gradually improved since the restart of treatment. However, at the second administration of the dose of 0.6 mg/kg, the patient experienced another adverse drug reaction with facial urticarial rash and bronchospasm, requiring the administration of adrenaline, methylprednisolone, and inhaled salbutamol. This case report highlights the need to customize the olipudase alfa desensitization protocol according to individual tolerance and raises the issue of achieving the established therapeutic target in the most sensitive children.
UNASSIGNED: We report a case of anaphylaxis to olipudase alfa in a child affected by chronic neurovisceral Acid sphingomyelinase deficiency (ASMD) and describe a 7-step desensitization procedure. This procedure, with the total administered dose of 0.03 mg/kg, followed by gradual dose escalation, allowed to reach the dose of 0.3 mg/kg without adverse reactions; however, at the second administration of the dose of 0.6 mg/kg our patient presented another adverse reaction suggesting the need of a different desensitization strategy.
摘要:
寡核苷酸α用于酸性鞘磷脂酶缺乏症(ASMD)的非中枢神经系统表现。过敏反应是该药物描述的非常罕见且危及生命的不良反应。这里,我们报道了一例2岁男孩患有慢性神经内脏ASMD的病例,该男孩在剂量递增期间服用1mg/kg剂量后出现对脂酶α的超敏反应,在第二次服用目标治疗剂量3mg/kg期间出现适当的过敏反应.停止治疗15周,然后应用7步脱敏方案,输注剂量为0.03mg/kg。随后的剂量逐渐增加恢复,成功达到0.3mg/kg的剂量。此外,生物化学,和放射性疾病指数,在停止治疗期间增加,自重新开始治疗以来已逐渐好转。然而,在第二次施用0.6mg/kg的剂量时,患者出现了另一种药物不良反应,包括面部荨麻疹皮疹和支气管痉挛,需要服用肾上腺素,甲基强的松龙,吸入沙丁胺醇.该病例报告强调需要根据个体耐受性定制脂酶α脱敏方案,并提出了在最敏感的儿童中实现既定治疗目标的问题。
■我们报告了一例患有慢性神经内脏酸性鞘磷脂酶缺乏症(ASMD)的儿童对脂酶α的过敏反应,并描述了7步脱敏程序。这个程序,总给药剂量为0.03mg/kg,随后逐渐增加剂量,允许达到0.3mg/kg的剂量而无不良反应;然而,在第二次给药0.6mg/kg时,我们的患者出现另一个不良反应,提示需要采用不同的脱敏策略.