Rockman-Greenberg Cheryl, Josse Robert, Francis Mira, Mhanni Aziz
University of Manitoba, Winnipeg, Manitoba R3E 3P4, Canada.
St. Michael's Hospital, Toronto, Ontario M5B 1W8, Canada.
Bone Rep. 2022 Aug 30;17:101617. doi: 10.1016/j.bonr.2022.101617. eCollection 2022 Dec.
Asfotase alfa is a human recombinant enzyme replacement therapy for hypophosphatasia. We describe 6 adults who were treated with asfotase alfa for 61-68 months in a clinical trial (NCT01163149), after which asfotase alfa was discontinued for 15-48 months. The patients experienced clinical deterioration and, when treatment was restarted, showed improvement. Patients with hypophosphatasia should be closely monitored if asfotase alfa is stopped as clinical decline is likely. Clinical practice guidelines are needed.
阿法骨化醇酶是一种用于治疗低磷性骨软化症的重组人酶替代疗法。我们描述了6名成年人，他们在一项临床试验（NCT01163149）中接受了61 - 68个月的阿法骨化醇酶治疗，之后停用阿法骨化醇酶15 - 48个月。患者出现了临床病情恶化，而重新开始治疗后病情有所改善。如果停用阿法骨化醇酶，低磷性骨软化症患者很可能出现临床病情下降，因此需要密切监测。临床实践指南是必要的。
