Chafic Karam

Vasculitic neuropathy: clinical features, diagnostic approach, and neurological outcomes in a single-center cohort

Sevinch Rakhmonova, Chafic Karam

Background Vasculitic neuropathy (VN) is a diverse group of immune-mediated peripheral neuropathies that result in systemic or non-systemic vasculitic neuropathy. There are limited studies that characterize the neurological outcome of VN patients. We aimed to describe the clinical features, diagnostic workup, and …

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Use of Biologic Therapy in Seropositive Generalized Myasthenia Gravis in a Single Cohort (P11-9.005)

Anne Taylor, Holly Elser, Shawn Bird, Sami Khella et autres

To characterize the indications and experiences of patients with seropositive generalized myasthenia gravis (gMG) treated with biologic therapy.

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Polyneuropathy in p.V142I hereditary transthyretin amyloidosis: Diagnostic challenges and clinical considerations

John E. Dvorak, Sami Khella, Brian Drachman, Rebecca Row et autres

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Prevalence and attribution of polyneuropathy in p.V142I (V122I) hereditary transthyretin amyloidosis

John E. Dvorak, Sami Khella, Brian Drachman, Rebecca Row et autres

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Disease Prognosis and Progression in Transthyretin Amyloidosis

Joban Vaishnav, Dimitrios Bampatsias, Brian C. Boursiquot, Yevgeniy Brailovsky et autres

Transthyretin amyloidosis (ATTR) is a rapidly evolving disease with a rising prevalence and improving prognosis. With an increasing number of patients diagnosed across a broad spectrum of disease stages, disease prognostication is pivotal to guiding therapeutic decisions. Similarly, with varying phenotypes identified …

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Prognostic factors and progression biomarkers in AL amyloidosis: mapping current knowledge and critical gaps

Rajshekhar Chakraborty, Yevgeniy Brailovsky, Mazen Hanna, Ronald Witteles et autres

ABSTRACT: The therapeutic landscape for systemic immunoglobulin light chain (AL) amyloidosis has been revolutionized by daratumumab-based regimens, achieving 76% 5-year overall survival in the landmark ANDROMEDA trial. However, the current prognostic models were developed using patient populations treated with now-suboptimal therapies, creating …

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Real-world treatment utilization in adults with chronic inflammatory demyelinating polyneuropathy in the United States

C. Blein, Chafic Karam, C Arvin-Berod, Deborah E. Gelinas et autres

Background: Management of chronic inflammatory demyelinating polyneuropathy (CIDP) is challenged by heterogeneity in severity, comorbidities, potential adverse effects, and treatment accessibility. This study aimed to elucidate treatment utilization among patients with CIDP in the United States (US) to identify potential unmet needs. …

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BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial

Tuan Vu, Hacer Durmuş, Michael H. Rivner, Sheetal Shroff et autres

Myasthenia gravis (MG) is driven by the secretion of autoantibodies from pathogenic B cell maturation antigen (BCMA)-expressing plasma cells. In this phase 2b randomized, controlled, double-blind trial, we evaluated Descartes-08, an autologous BCMA-directed mRNA chimeric antigen receptor T cell therapy, in patients …

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Acquired and Genetic Amyloid Neuropathies

Chafic Karam, Mamatha Pasnoor, Richard J. Barohn, Mazen M. Dimachkie

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Treatment Impact of Efgartigimod PH20 SC on I-RODS Daily Activity Assessment in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: Post Hoc Analysis of the Registrational ADHERE Study (P10-11.025)

Jeffrey A. Allen, Jie Lin, Mark Stettner, Ingemar S. J. Merkies et autres

Assess the effect on functionality of subcutaneous (SC) efgartigimod PH20 (co-formulated with recombinant human hyaluronidase PH20) in chronic inflammatory demyelinating polyneuropathy (CIDP).

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Hereditary Transthyretin Amyloidosis Polyneuropathy

Taha Qarni, Orly Moshe‐Lilie, Michelle Kaku, Chafic Karam

Tc-PYP scan) are increasingly being used in place of tissue biopsies for confirmation of disease. Novel treatments now include antisense oligonucleotide and RNA interference drugs, whereas new applications such as CRISPR and amyloid antibodies are being studied for potential use in the …

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Treatment characteristics of patients with hereditary transthyretin amyloidosis: a cohort study

Taha Qarni, Felipe J.S. Jones, Brian Drachman, Sami Khella et autres

BACKGROUND: There are novel medications approved for the treatment of hereditary transthyretin amyloidosis (ATTRv), classified as transthyretin (TTR) stabilizers or gene silencers. While many patients may be on both classes of medications, there is no data available on the safety and efficacy …

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