Improving Ambulation and Minimizing Disability with Therapeutic Plasma Exchange in a Stiff-person Syndrome Patient with Recurrent Falls

Sanchez, Kyle; Ullah, Aqsa; Waler, Alexandria; Chakfe, Yassar

doi:10.7759/cureus.6209

Cited by 7 publications

(4 citation statements)

References 20 publications

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“…In most severe cases, patients can experience respiratory failure requiring admission to intensive care units [17,18]. The management of SPS is based on immunosuppressants and antispastic drugs for chronic use, and IVIg and plasma exchange as rescue therapies [1,19]. IVIg have been demonstrated as a safe and effective therapy to treat SPS exacerbations, but there are few data due to the rarity of the disease [2,20].…”

Section: Discussionmentioning

confidence: 99%

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome

Di Stefano,

Alonge,

Rini

et al. 2023

J Neurol

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Stiff-person syndrome (SPS) is a rare autoimmune neurological disorder characterized by high titers of antibodies against glutamic acid decarboxylase (GAD) causing impaired GABAergic inhibitory neurotransmission. To date, there is not a defined therapy for such condition, but immunomodulating therapies, such as plasma exchange, intravenous immunoglobulins, and rituximab, have been widely used in clinical practice. However, the efficacy and tolerability of these treatments is not well established. Efgartigimod, a new neonatal Fc receptor (FcRn) blocker, is a human IgG1 antibody Fc fragment engineered with increased affinity for FcRn binding, leading to a reduction in IgGs levels, including pathogenic IgG autoantibody showing promising results in neurological autoimmune disorders and has been approved for the treatment of AChR-seropositive generalized myasthenia gravis (MG). In this study, we report and describe the first data on treatment with efgartigimod in three patients affected by both AChR-seropositive generalized MG and anti-GAD-seropositive SPS. Patients were followed since the start of efgartigimod and for the whole treatment period (12 weeks). MG symptoms were assessed with the “MG activity of daily living score” and the Quantitative Myasthenia Gravis score, while SPS ones were assessed with the “SPS activity of daily living score”; muscle strength was assessed with the Medical Research Council Sum score; the overall disability from MG and SPS was assessed by the modified Rankin Scale. All patients showed an improvement in symptoms of both SPS and MG after 2 cycles of treatment. Our data suggest that efgartigimod may be considered as a candidate drug for SPS and other autoantibody-mediated neurological disorders.

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Section: Discussionmentioning

confidence: 99%

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome

Di Stefano,

Alonge,

Rini

et al. 2023

J Neurol

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“…There are less robust data on the use of other types of immunotherapies in SPSD [3,9]. However, there are some case series and studies that provide supportive evidence for the use of corticosteroids, subcutaneous immunoglobulins (SCIg) [36,37], plasmapheresis/plasma exchange [1,[38][39][40], rituximab [1,31,41], cyclophosphamide [1], and even possibly (though controversially) autologous hematopoietic stem cell transplantation [42,43]. The treatment approach to individuals with SPSD is more varied in practice [9].…”

Section: Discussionmentioning

confidence: 99%

Expanding clinical profiles and prognostic markers in stiff person syndrome spectrum disorders

Wang,

Hu,

Aljarallah

et al. 2023

J Neurol

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Objective To describe the clinical features of a cohort of individuals with stiff person syndrome spectrum disorders (SPSD) and identify potential early predictors of future disability. Background There is a need to better understand the full spectrum of clinical and paraclinical features and long-term impact of SPSD. Design/Methods Observational study from 1997 to 2022 at Johns Hopkins. Clinical phenotypes included classic SPS, partial SPS (limb or trunk limited), SPS-plus (classic features plus cerebellar/brainstem involvement), and progressive encephalomyelitis with rigidity and myoclonus (PERM). Outcome measures were modified Rankin scale (mRS) and use of assistive device for ambulation. Multivariate logistic regression was used to assess significant predictors of outcomes. Results Cohort included 227 individuals with SPSD with mean follow-up of 10 years; 154 classic, 48 SPS-plus, 16 PERM, and 9 partial. Mean age at symptom onset was 42.9 ± 14.1 years, majority were white (69.2%) and female (75.8%). Median time to diagnosis was 36.2 months (longest for SPS-plus and PERM) and 61.2% were initially misdiagnosed. Most had systemic co-morbidities and required assistive devices for ambulation. Female sex (OR 2.08; CI 1.06–4.11) and initial brainstem/cerebellar involvement (OR 4.41; CI 1.63–14.33) predicted worse outcome by mRS. Older age at symptom onset (OR 1.04; CI 1.01–1.06), female sex (OR 1.99; CI 1.01–4.01), Black race (OR 4.14; CI 1.79–10.63), and initial brainstem/cerebellar involvement (OR 2.44; CI 1.04–7.19) predicted worse outcome by use of assistive device. Early implementation of immunotherapy was associated with better outcomes by either mRS (OR 0.45; CI 0.22–0.92) or use of assistive device (OR 0.79; CI 0.66–0.94). Conclusions We present the expanding phenotypic variability of this rare spectrum of disorders and highlight potential predictors of future disability.

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“…Assim, a plasmaferese torna-se uma opção quando existe doença refratária à imunoglobulina humana endovenosa. 39…”

Section: Plasmafereseunclassified

Síndrome de Stiff-Person: Revisão

Azevedo¹

2024

Sinapse

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A síndrome de stiff-person é uma doença autoimune neurológica rara, que se manifesta por rigidez muscular progressiva e espasmos dolorosos, com envolvimento predominante dos músculos axiais e proximais dos membros. A maioria dos doentes apresenta anticorpos dirigidos à descarboxilase do ácido glutâmico, o passo limitante na produção do neurotransmissor inibitório ácido gama-aminobutírico. A síndrome de stiff-person paraneoplásica associa-se frequentemente a anticorpos anti-anfifisina e a carcinoma da mama. O tratamento com fármacos que aumentam o tónus GABAérgico, incluindo a classe das benzodiazepinas, combinado com imunoterapia pode melhorar os sintomas neurológicos, no entanto, o prognóstico é imprevisível e a recuperação espontânea é rara.A literatura relativa a este assunto ainda é escassa e, por se tratar de uma entidade rara, é pouco reconhecida e provavelmente subdiagnosticada. Este artigo de revisão descreve as principais atualizações e sistematiza a informação existente acerca deste tema.

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Improving Ambulation and Minimizing Disability with Therapeutic Plasma Exchange in a Stiff-person Syndrome Patient with Recurrent Falls

Cited by 7 publications

References 20 publications

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome

Expanding clinical profiles and prognostic markers in stiff person syndrome spectrum disorders

Síndrome de Stiff-Person: Revisão

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