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Neuromuscular Disorders Patient-reported Outcomes—An Emerging Cornerstone of Effective Intravenous Immunoglobulin Therapy Report of a Satellite Symposium Convened at the 61 st Annual Meeting of the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM), Savannah, Georgia, October 29–November 1, 2014 Expert review by: Nicholas Silvestri, 1 Shafeeq Ladha, 2 Tahseen Mozaffar, 3 Gretchen Ayer 4 and Lisa M Betts 5 1. Assistant Professor of Neurology, University of Buffalo, New York, US; 2. Associate Professor of Clinical Neurology, Barrow Neurological Institute, Phoenix, Arizona, US; 3. Professor of Neurology, University of California, Irvine, US; 4. Director Business Development, Walgreen Co., Deerfield, Illinois, US; 5. Program Director, Walgreen Co., Deerfield, Illinois, US Abstract Intravenous immunoglobulin (IVIG) therapy is increasingly important in the management of various immune-mediated neuromuscular d ­ isorders including chronic inflammatory demyelinating polyneuropathy (CIDP), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and other neuromuscular disorders. Administrative burden, quality of life (QoL) concerns, adverse event prevention, economic pressures, and logistical factors are driving greater IVIG use into the home setting where it is administered by nurses. Patient-reported outcome measures (PROMs) are self- assessment instruments designed to measure a patient’s disability, QoL, or their perceptions of health status in relation to specific diseases. PROMs may be a valuable means of monitoring disease status and treatment efficacy in patients receiving IVIG at home. Case reports and small clinical studies show that various specific and general purpose PROMs, such as the 15-item MG-specific QOL (MG-QOL15) and the Myasthenia Gravis Activities of Daily Living scale (MG-ADL), can provide valuable information for patient monitoring at home. PROMs may help to alert physicians that earlier follow-up or treatment regimen changes are needed. PROM data recording systems such as Walgreens’ PartnerPoint Clinical Management SM maintain regular reporting to the physician and enable efficacy and adverse events to be tracked. Pilot studies of patients with neuromuscular disease receiving IVIG at home demonstrate a strong correlation in PROM scores between assessments administered by pharmacy clinical staff and those administered by physicians indicating the reliability and suitability of PROMs for remote patient management. Further work to validate additional commonly used PROMS for autoimmune disease is needed if they are to be useful when administered outside the physician clinic. Keywords Patient-reported outcome measures, IVIG therapy, neuromuscular disease, improving care continuum Disclosure: Gretchen Ayer and Lisa M Betts are employees of the Walgreens IG Program. Tahseen Mozaffar, Nicholas Silvestri, and Shafeeq Ladha are all consultants for Walgreens IG program and have no other disclosures. Open Access: This article is published under the Creative Commons Attribution Noncommercial License, which permits any noncommercial use, distribution, adaptation, and reproduction provided the original author(s) and source are given appropriate credit. Acknowledgments: The content of the forum presentation was developed and reviewed by the Walgreens IG Program PartnerPoint Clinical Management SM Medical Advisory Board (Lisa Christopher-Stine, Tahseen Mozaffar, Nicholas Silvestri, Yuen T So, Shafeeq Ladha) and Ted Burns. Editorial assistance was provided by James Gilbart of Touch Medical Media, London, UK. This article reports the proceedings of a sponsored satellite symposium and as such has not been subject to the journal’s usual peer-review process. Received: February 6, 2015 Accepted: February 20, 2015 Citation: US Neurology, 2015;11(1):40–6 Correspondence: Lisa M Betts, Walgreens Infusion Services, 2050 S Finley Rd, Suite 20, Lombard, IL 60148-4837, US. E: lisa.betts@walgreens.com Support: This article was supported by Walgreens Infusion Services. Patient-reported Outcome Measures The primary aim of treating disease is to give patients freedom from symptoms, disability and side effects, and the freedom to function. Objectively and reliably measuring disability and quality of life (QoL) can be challenging but using patient-reported outcome measures (PROMs), in addition to clinical examination by physician, has proved a valuable means of capturing patient status and response to treatment. 1–3 In disease management, PROMS can be used to estimate symptoms (severity/tolerability of dysfunction or symptom), 40 function, and health-related QoL [HRQoL], such as psychologic well-being, social, and physical functioning). The US Food and Drug Administration (FDA) encourages validated PROMs as parameters that can support labeling requirements. They can be used as endpoints in clinical studies and in regular clinical use. 4 The FDA industry guidelines support the use of outcomes measures for specific neuromuscular disorders such as use of the Rasch-built Overall Disability Scale (R-ODS) in the management of chronic inflammatory demyelinating polyneuropathy (CIDP). 5 Touch ME d ica l ME d ia