Long-term effects of enzyme replacement therapy in an elderly cohort of late-onset Pompe disease

Abstract

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Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review

“…In addition, we checked the reference lists of included publications and identified one additional publication that could be included. In the end, 17 publications fulfilled our selection criteria and were thus included in the review ( Figure 1 ) [ 3 , 5 , 7 , 29 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 ]. Three publications were included from the updated search on 21 June 2022.…”

“…The characteristics of the included publications ( n = 17) are summarised in Table 1 [ 3 , 5 , 7 , 29 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 ]. Included publications were: randomised controlled trials ( n = 2) [ 2 , 41 ], case reports ( n = 5) [ 29 , 33 , 34 , 36 , 39 ], and cohort studies ( n = 10) [ 3 , 35 , 37 , 38 , 40 , 41 , 42 , 43 , 44 , 45 ]. Two publications had an overlap of at least one patient [ 29 , 41 ].…”

“…Several publications reported on the main parameters of interest for this study, anti-rhGAA antibody titres, and/or dosing at a group level [ 3 , 5 , 7 , 38 , 40 , 42 ]; others reported this on a patient level only [ 29 , 33 , 34 , 36 , 37 , 39 , 43 ]. Four publications reported on these parameters on both a patient level and group level [ 35 , 41 , 44 , 45 ]. Hence, we have split the data into group level data ( Table 2 ) and patient level data of patients with a high antibody titre ( Table 3 ).…”

“…The publications included in this review had different definitions of a high antibody titre and/or a HSAT; in addition, the time point(s) at which antibody titres were measured differed between studies. Of the 10 publications [ 3 , 5 , 7 , 35 , 38 , 40 , 41 , 42 , 44 , 45 ] that reported data on a group level, seven publications gave their definition of a high titre [ 5 , 7 , 35 , 38 , 41 , 42 , 45 ], with the definition of a high titre ranging from ≥12,800 [ 5 ], 25,600–819,200 [ 7 ], ≥1:31,200 [ 41 ], ≥1:31,250 [ 45 ], >1:31,250 [ 42 ], to ≥1:51,200 [ 35 , 38 ]. Eleven publications reported data on an individual patient level [ 29 , 33 , 34 , 35 , 36 , 37 , 39 , 41 , 43 , 44 , 45 ]; five of these publications gave a clear definition of a high titre [ 35 , 37 , 41 , 43 , 45 ], with the definition of a high titre ranging from ≥1:31,200 [ 41 ], ≥1:31,250 [ 45 ], >1:31,250 [ 43 ], to ≥1:51,200 [ …”

Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy

“…Conversely, other studies indicate that long-term ERT in LOPD patients leads to high ADA titer, but the immune responses to rhGAA following treatment do not significantly affect its efficacy ( 21 ). The diverse characteristics and varied treatment responses among LOPD patients complicate the assessment of ERT effects and ADA’s influence on this group ( 22 ).…”