de Los Reyes E, Lehwald L, Augustine EF, et al.
Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis
Type 2 Disease: Clinical Practice Considerations From US Clinics [published
online ahead of print, 2020 May 4]. Pediatr Neurol. 2020;S0887-8994(20)30149-1.
doi:10.1016/j.pediatrneurol.2020.04.018
Abstract
Background: Neuronal ceroid lipofuscinosis type 2 or CLN2
disease is a rare, autosomal recessive, neurodegenerative lysosomal storage
disorder caused by tripeptidyl peptidase 1 deficiency. Cerliponase alfa, a
recombinant human tripeptidyl peptidase 1 enzyme, is the first and only
approved treatment for CLN2 disease and the first approved enzyme replacement
therapy administered via intracerebroventricular infusion.
Methods: A meeting of health care professionals from US
institutions with experience in cerliponase alfa treatment of children with
CLN2 disease was held in November 2018. Key common practices were identified,
and later refined during the drafting of this article, that facilitate safe
chronic administration of cerliponase alfa.
Results: Key practices include developing a
multidisciplinary team of clinicians, pharmacists, and coordinators, and
institution-specific processes. Infection risk may be reduced through strict
aseptic techniques and minimizing connections and disconnections during
infusion. The impact of intracerebroventricular device design on port needle
stability during extended intracerebroventricular infusion is a critical
consideration in device selection. Monitoring for central nervous system
infection is performed at each patient contact, but with flexibility in the
degree of monitoring. Although few institutions had experienced positive
cerebrospinal fluid test results, the response to a positive cerebrospinal
fluid culture should be determined on a case-by-case basis, and the
intracerebroventricular device should be removed if cerebrospinal fluid
infection is confirmed.
Conclusions: The key common practices and flexible practices
used by institutions with cerliponase alfa experience may assist other
institutions in process development. Continued sharing of experiences will be
essential for developing standards and patient care guidelines.
No comments:
Post a Comment