Quantitative Assessment of Response to Long‐Term Treatment with Intravenous Immunoglobulin in Patients with Stiff Person Syndrome

Abstract

BackgroundStiff Person Syndrome (SPS) is an autoimmune condition involving antibodies against several components of the inhibitory synapse in the spinal cord, with Glutamic Acid Decarboxylase (GAD) antibodies being the predominant immune marker. SPS affects approximately 1 patient per million population per year. The effect of IVIG has been established but studies on long term efficacy of regular IVIG are limited. ObjectivesReview clinical details and long-term treatment response using a patient-reported questionnaire in stiff person and related syndromes. MethodsPatients were identified from a tertiary neuroimmunology clinic based on classical clinical symptoms, autoimmune profile, and neurophysiological changes (Dalakas criteria). They were followed up after treatment to assess the response to IVIG. Results patients fulfilled the selection criteria. Patients' demographic profiles and clinical

Intravenous immunoglobulin in the management and outcome of Stiff‐Person syndrome: A systematic review

“…Along with anti‐GAD antibodies, antibodies against glycine receptors were existent in 14 patients 22,28,30,32 . Among 59 patients who were anti GAD antibodies‐negative, 17 had antiglycine receptor antibodies 20,28,30 …”

“…This scoring system was used for 38 out of 95 SPS patients 22,25,28,31,32 . Bose et al used the Birmingham Response to Immunomodulatory Therapy (BRIT) scoring system consisting of a functional ability score which was assessed by mRS and a quality of life (QoL) score, and mentioned significant improvement in 19 SPS patients receiving IVIG 20 . A randomized, double‐blinded, placebo‐controlled, crossover trial of monthly IVIG showed a notable reduction in stiffness, as assessed by the use of scores of the distribution‐of‐stiffness index and the heightened sensitivity scale from baseline (first month) to the second and third month of each treatment phase, which stabilized during washout and increased again on switching to placebo 23 .…”

“…23 Three studies comprising 44 patients used previously defined criteria by Dalakas et al consisting of clinical symptoms, autoimmune profile, and neurophysiological changes for the confirmation of SPS. 20,24,32 Eight SPS patients were classified into classical SPS if lower-extremity and lumbar stiffness and spasms were present, focal SPS if stiffness was restricted to axial, lower-extremity, or upper-body musculature, and PERM if stiffness and spasms involved the whole body and were accompanied by a rapidly progressive encephalomyelopathy. 22 Diagnostic criteria as defined by Lorish et al was used for nine patients in two papers.…”

One in a Million: A Case Report of Stiff Person Syndrome

“…SPS is notoriously difficult to diagnose as it is rare, uncommonly considered, and symptoms evolve over time. A retrospective cohort of 23 patients with SPS spectrum disorders reported that patients were diagnosed on average 3.5 years after initial symptoms and first began immunomodulatory treatment with IVIG more than 5 years after symptom onset [ 5 ]. The initial presenting symptoms, as assessed by Tsiortou et al and colleagues in a retrospective cohort of 57 patients, were slowly progressive proximal leg stiffness which then progressed to muscle spasms, stiffness in the thoracolumbar spine, muscle rigidity, and hyperreflexia [ 3 ].…”

“…When IVIG was stopped, the patients experienced a rebound worsening of their stiffness [ 7 ]. Reports of long-term follow-up of patients on IVIG therapy show that the patients remained stable and continued to improve clinically even after 3–5 years of therapy [ 5 ].…”