New study in Patient-reported Outcomes—An Emerging Cornerstone of Effective Intravenous Immunoglobulin Therapy

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European Neurological Review, a peer-reviewed, open access, bi-annual neurology journal publish cutting-edge article by Nicholas Silvestri, Shafeeq Ladha, Tahseen Mozaffar, Gretchen Ayer and Lisa M Betts.

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Intravenous immunoglobulin (IVIG) therapy is increasingly important in the management of various immune-mediated neuromuscular disorders including chronic inflammatory demyelinating polyneuropathy (CIDP), multifocal motor neuropathy (MMN).

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.

In disease management, PROMS can be used to estimate symptoms (severity/tolerability of dysfunction or symptom), 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. 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).

Case Example 1
A 70-year-old male with a 25-year history of MMN showed right ulnar neuropathy at onset and subsequently showed left foot drop, right radial, and upper trunk symptoms with episodes occurring every 6–8 months at which time a course of 1 g/kg IVIG was prescribed. Following IVIG, he reported a dramatic improvement in addition to better scores on MMT. He regained ability to perform exercises including lifting weights, his hands returned to near normal function, and he reported that the greatest improvement was in his balance. Approximately 4 months after each treatment he started to weaken again and so was treated, as needed, every 6 months. The patient was asked to assess his condition at intervals before and after treatment using the form shown in Figure 1; his resultant scores are shown in Figure 2.

PROMS are also valuable for investigation of the natural history of neuromuscular disease. An example is the 15-item MG-specific QoL scale (MG-QoL15) that was developed from a larger set of 60 items and reduced to a core set.6 In a Japanese study that included 640 consecutive patients with MG, MG-QOL15 scores correlated with prednisolone dose and diseaseseverity. MG-QOL15 scores for patients receiving ≤5 mg/day prednisolone were the same as those receiving no prednisolone whereas scores were significantly worse for those receiving >5 mg/day prednisolone.

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Barney Kent
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