An Immune Tolerance Approach Using Transient Low-Dose Methotrexate in the ERT-Naive Setting of Patients Treated with a Therapeutic Protein: Experience in Infantile-Onset Pompe Disease

Authors

Zoheb Kazi
Ankit Desai
R. Bradley Troxler
David Kronn, New York Medical CollegeFollow
Seymour Packman
Marta Sabbadini
William B. Rizzo
Priya S. Kishnani

DOI

10.1038/s41436-018-0270-7

Journal Title

Genetics in Medicine

First Page

887

Last Page

895

Document Type

Article

Publication Date

April 2019

Department

Pediatrics

Keywords

Pompe disease, alglucosidase alfa, antidrug antibodies, methotrexate, prophylactic immune tolerance induction

Disciplines

Medicine and Health Sciences

Abstract

PURPOSE: To investigate immune tolerance induction with transient low-dose methotrexate (TLD-MTX) initiated with recombinant human acid alpha-glucosidase (rhGAA), in treatment-naive cross-reactive immunologic material (CRIM)-positive infantile-onset Pompe disease (IOPD) patients. METHODS: Newly diagnosed IOPD patients received subcutaneous or oral 0.4 mg/kg TLD-MTX for 3 cycles (3 doses/cycle) with the first 3 rhGAA infusions. Anti-rhGAA IgG titers, classified as high-sustained (HSAT; >/=51,200, >/=2 times after 6 months), sustained intermediate (SIT; >/=12,800 and <51,200 within 12 months), or low (LT;

Recommended Citation

Kazi, Z., Desai, A., Troxler, R., Kronn, D., Packman, S., Sabbadini, M., Rizzo, W., & Kishnani, P. (2019). An Immune Tolerance Approach Using Transient Low-Dose Methotrexate in the ERT-Naive Setting of Patients Treated with a Therapeutic Protein: Experience in Infantile-Onset Pompe Disease. Genetics in Medicine, 21 (4), 887-895. https://doi.org/10.1038/s41436-018-0270-7