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

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