TY - JOUR
T1 - Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease
T2 - Clinical Practice Considerations From US Clinics
AU - de los Reyes, Emily
AU - Lehwald, Lenora
AU - Augustine, Erika F.
AU - Berry-Kravis, Elizabeth
AU - Butler, Karen
AU - Cormier, Natalie
AU - Demarest, Scott
AU - Lu, Sam
AU - Madden, Jacqueline
AU - Olaya, Joffre
AU - See, Susan
AU - Vierhile, Amy
AU - Wheless, James W.
AU - Yang, Amy
AU - Cohen-Pfeffer, Jessica
AU - Chu, Dorna
AU - Leal-Pardinas, Fernanda
AU - Wang, Raymond Y.
N1 - Publisher Copyright:
© 2020 The Authors
PY - 2020/9
Y1 - 2020/9
N2 - 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.
AB - 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.
KW - Batten disease
KW - CLN2 disease
KW - Cerliponase alfa
KW - Enzyme replacement therapy
KW - Intracerebroventricular
KW - Late infantile neuronal ceroid lipofuscinosis
KW - Neuronal ceroid lipofuscinosis
UR - http://www.scopus.com/inward/record.url?scp=85087935907&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85087935907&partnerID=8YFLogxK
U2 - 10.1016/j.pediatrneurol.2020.04.018
DO - 10.1016/j.pediatrneurol.2020.04.018
M3 - Article
C2 - 32684372
AN - SCOPUS:85087935907
SN - 0887-8994
VL - 110
SP - 64
EP - 70
JO - Pediatric Neurology
JF - Pediatric Neurology
ER -