Mariëlle H J Pruppers1, Ingemar S J Merkies, Nicolette C Notermans. 1. aDepartment of Neurology, Maastricht University Medical Center, Maastricht bDepartment of Neurology, Spaarne Hospital, Hoofddorp cDepartment of Neurology, Rudolf Magnus Brain Center, University Medical Center Utrecht, Utrecht, The Netherlands.
Abstract
PURPOSE OF REVIEW: This review aims to provide an overview of all randomized trials performed in IgM Anti-Myelin Associated Glycoprotein related peripheral neuropathy (MGUSP) with emphasis on the applied outcome measures including the rationale for their choice and possible limitations, emphasizing new advances in modern clinimetrics. RECENT FINDINGS: All clinical trials performed in patients with MGUSP have been negative, which raises many unanswered questions: were the sample sizes too small, the duration of the trials too short, the chosen medical interventions not aggressive enough, the definition of being a responder inadequate, and, last but not least, the outcome measures used improper? Only recently has attention been directed towards the possibility of using suboptimal outcome measures as a potential factor that may have contributed to the negative results in MGUSP. SUMMARY: Since there is no international consensus on how to assess and treat patients with MGUSP, the current study addresses new advances in the field of modern clinimetrics and recommendations for future outcome measure studies in MGUSP before proceeding with new interventional trials.
PURPOSE OF REVIEW: This review aims to provide an overview of all randomized trials performed in IgM Anti-Myelin Associated Glycoprotein related peripheral neuropathy (MGUSP) with emphasis on the applied outcome measures including the rationale for their choice and possible limitations, emphasizing new advances in modern clinimetrics. RECENT FINDINGS: All clinical trials performed in patients with MGUSP have been negative, which raises many unanswered questions: were the sample sizes too small, the duration of the trials too short, the chosen medical interventions not aggressive enough, the definition of being a responder inadequate, and, last but not least, the outcome measures used improper? Only recently has attention been directed towards the possibility of using suboptimal outcome measures as a potential factor that may have contributed to the negative results in MGUSP. SUMMARY: Since there is no international consensus on how to assess and treat patients with MGUSP, the current study addresses new advances in the field of modern clinimetrics and recommendations for future outcome measure studies in MGUSP before proceeding with new interventional trials.