An international multicenter efficacy and safety study of IqYmune in initial and maintenance treatment of patients with chronic inflammatory demyelinating polyradiculoneuropathy: PRISM study

Abstract

This prospective, multicenter, single-arm, open-label phase 3 study aimed to evaluate the efficacy and safety of IqYmune in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Patients received one induction dose of 2 g/kg and then seven maintenance doses of 1 g/kg at 3-week intervals. The primary endpoint was the responder rate at the end of study (EOS), defined as an improvement of ≥1 point on the adjusted inflammatory neuropathy cause and treatment (INCAT) disability scale. The responder rate was compared with the responder rate of a historical placebo group (33.3%). Secondary endpoints included changes from baseline to EOS of adjusted INCAT disability score, grip strength, Medical Research Council (MRC) sum score, Rasch-modified MRC sum score, Rasch-built overall disability scale score and the clinical global impression. Forty-two patients, including 23 Ig-naïve and 19 Ig-pre-treated, were included in the efficacy set. The overall response rate at EOS was 76.2% (95% confidence interval [60.5%-87.9%]). The superiority of IqYmune compared to the historical placebo control was demonstrated (P < .0001). The responder rate was numerically higher in Ig-pre-treated than in Ig-naïve patients but confidence intervals were overlapping (84.2% [60.4%-96.6%] vs 69.6% [47.1%-86.8%]). All secondary endpoints confirmed this conclusion. The median time to response was 15 weeks [8.9-19.1 weeks]. A total of 156 adverse events including five serious were considered related to IqYmune, 87.2% were mild. Neither hemolysis nor signs of renal or hepatic impairment were observed. These results demonstrate that IqYmune is an effective and well-tolerated treatment in patients with CIDP.

European Academy of Neurology/Peripheral Nerve Society guideline on diagnosis and treatment of chronic inflammatory demyelinating polyradiculoneuropathy: Report of a joint Task Force—Second revision

“…The ICE randomized controlled trial showed that 94% of patients responded to 2 g/kg induction treatment and two subsequent treatments of 1 g/kg at 3 weeks intervals 161 . The open PRIMA and PRISM studies indicated that a treatment response sometimes may only be observed after three to five infusions of 1 g/kg every 3 weeks 162,163 . Alternatively, clinical experience indicates that most patients respond objectively to no more than two initial courses of 2 g/kg 164 .…”

“…161 The open PRIMA and PRISM studies indicated that a treatment response sometimes may only be observed after three to five infusions of 1 g/kg every 3 weeks. 162,163 Alternatively, clinical experience indicates that most patients respond objectively to no more than two initial courses of 2 g/ kg. 164 It is not well known whether an objective response following only after several treatments is due to a delayed treatment response or to the requirement of a different treatment regimen.…”

European Academy of Neurology/Peripheral Nerve Society guideline on diagnosis and treatment of chronic inflammatory demyelinating polyradiculoneuropathy: Report of a joint Task Force—Second revision

“…The ICE randomized controlled trial showed that 94% of patients responded to 2 g/kg induction treatment and two subsequent treatments of 1 g/kg at 3 weeks intervals [161]. The open PRIMA and PRISM studies indicated that a treatment response sometimes may only be observed after three to five infusions of 1 g/kg every 3 weeks [162,163]. Alternatively, clinical experience indicates that most patients respond objectively to no more than two initial courses of 2 g/kg [164].…”

Minimal important differences and self‐identifying treatment response in chronic inflammatory demyelinating polyneuropathy

“…However, improving the use patient‐relevant outcome measures, is necessary for optimal care, and this requires involvement of patients themselves. In this regard, other avenues for clinical practice may involve consideration of use of latest disease‐specific quality of life measures 31 and Patient Global Impression of Change evaluations 32 . In addition to measuring improvement, outcome measures are also of vital importance in verifying stability in patients with CIDP being weaned off treatment, 33 and further similar studies, in that role, are also warranted.…”