Subcutaneous immunoglobulin for maintenance therapy in stiff-person syndrome: One-year follow-up in two patients

Fileccia, Enrico; Rinaldi, Rita; Minicuci, Giacomo; D’Angelo, Roberto; Bartolomei, Luigi; Liguori, Rocco; Donadio, Vincenzo

doi:10.1016/j.nmd.2020.09.024

Cited by 10 publications

(9 citation statements)

References 17 publications

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“…Although second‐line therapeutics (eg, rituximab, cyclophosphamide, mycophenolate) can provide an increased immunosuppression, but only at the cost of an increased risk of adverse events like opportunistic infections and neoplasms 15 . The fact that the symptoms of SPS like spasms and stiffness are mediated by the binding of specific antibodies that bring about the excitation of lower motor neurons forms the biological basis of immunomodulatory treatment and should be considered in most patients with SPS 4,60 …”

Section: Discussionmentioning

confidence: 99%

“…15 The fact that the symptoms of SPS like spasms and stiffness are mediated by the binding of specific antibodies that bring about the excitation of lower motor neurons forms the biological basis of immunomodulatory treatment and should be considered in most patients with SPS. 4,60 The European Federation of Neurological Sciences recommends IVIG for patients with SPS who have a significant disability, including frequent falls and whose response to diazepam and/or baclofen as an initial therapy is unsatisfactory. 48,[61][62][63][64] The recommended dose of IVIG is 2 g per kg body weight over 2-5 d. 62 Several mechanisms of IVIG that can alter the pathogenesis of underlying neuromuscular disorders has been described.…”

Section: Discussionmentioning

confidence: 99%

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

Aryal

Shrestha

Homagain

et al. 2022

Clinical & Exp Neuroim

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Background: Stiff Person Syndrome (SPS), a rare immune-mediated neuromuscular disorder, is characterized by rigidity, stiffness, and intermittent spasms of axial and extremity muscles. Various immunotherapies including intravenous immunoglobulin (IVIG) have been used for this autoimmune condition. Here we aim to review the role of IVIG in the treatment of SPS along with its outcome.Methods: A systematic literature search of PubMed and Embase was conducted to identify the relevant published articles against the predefined criteria using suitable keywords combinations till September 20, 2021. Data were extracted to produce descriptive information of SPS patients on demographics, diagnostics, treatment with IVIG, and outcome.Results: Twelve studies with 216 patients were included in the review and 63.89% of them had classical SPS. Glutamic acid decarboxylase (GAD) autoantibodies were present in 72.68% of the patients and 57.89% in whom electromyography (EMG) was performed had continuous motor activity. IVIG therapy was given to 95 patients in different regimens in various studies and varying scoring systems were used to assess the outcome, and 83.16% showed some form of improvement, 14.74% showed no improvement, while 2.10% worsened. None of the included studies mentioned an adverse effect of IVIG in the patients. Conclusion: IVIG may benefit patients with SPS along with other medications. Owingto the rarity of the disease and insufficient studies on the assessment of immunotherapy in SPS, longitudinal studies with a sizable number of patients are required to clarify clinical course, treatment, and outcome in SPS with the use of IVIG.anti-glutamic acid decarboxylase antibodies, immunotherapy, intravenous immunoglobulin, stiff limb syndrome, stiff person syndrome | INTRODUCTIONStiff Person Syndrome (SPS) is a rare and incapacitating neuromuscular disorder characterized by muscle rigidity, stiffness, and intermittent spasms involving the muscles of axial and extremities. 1,2 Activation of agonist and antagonist muscles results in stiffness, due to which patients have frequent falls, muscle spasms, and chronic muscle pain. 3,4 Originally described by Moersch and Woltman, the etiology of SPS is not known but upcoming evidence suggests a likely autoimmune cause. 2,5 It is associated with a high level of anti-glutamic acid decarboxylase (GAD) antibodies, an enzyme controlling the formation of gamma-amino butyric acid (GABA). 6,7 With no structural damage

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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

Aryal

Shrestha

Homagain

et al. 2022

Clinical & Exp Neuroim

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“…43 Because subcutaneous immunoglobulin given weekly provides a steady-state serum IgG level, it is especially useful for those patients with an early wearing-off effect to ensure sustained benefit. [43][44][45] Long-term Monthly Maintenance Therapy With IVIg…”

Section: Intravenous Immunoglobulinmentioning

confidence: 99%

Therapies in Stiff-Person Syndrome

Dalakas

2023

Neurol Neuroimmunol Neuroinflamm

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Among the glutamic acid decarboxylase (GAD)-antibody–spectrum disorders, the most common phenotypic subset is the stiff-person syndrome (SPS), caused by impaired GABAergic inhibitory neurotransmission and autoimmunity characterized by very high titers of GAD antibodies and increased GAD-IgG intrathecal synthesis. If not properly treated or untreated because of delayed diagnosis, SPS progresses leading to disability; it is therefore fundamental to apply the best therapeutic schemes from the outset. This article is focused on the rationale of specific therapeutic strategies based on the SPS pathophysiology targeting both the impaired reciprocal GABAergic inhibition to symptomatically improve the main clinical manifestations of stiffness in the truncal and proximal limb muscles, gait dysfunction, and episodic painful muscle spasms and the autoimmunity to enhance improvement and slow down disease progression. A practical, step-by-step therapeutic approach is provided, highlighting the importance of combination therapies with the preferred gamma-aminobutyric acid–enhancing antispasmodic drugs, such as baclofen, tizanidine, benzodiazepines, and gabapentin, that provide the first-line symptomatic therapy, while detailing the application of current immunotherapies with intravenous immunoglobulin (IVIg) plasmapheresis, and rituximab. The pitfalls and concerns of long-term therapies in different age groups, including children, women planning pregnancy, and especially the elderly considering their comorbidities are emphasized, also highlighting the challenges in distinguishing the conditioning effects or expectations of chronically applied therapies from objective meaningful clinical benefits. Finally, the need for future targeted immunotherapeutic options based on disease immunopathogenesis and the biologic basis of autoimmune hyperexcitability are discussed, pointing out the unique challenges in the design of future controlled clinical trials especially in quantifying the extend and severity of stiffness, episodic or startle-triggered muscle spasms, task-specific phobias, and excitability.

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“…After stabilising patients with IV immunoglobulins, they were switched to subcutaneous therapy. Symptom stabilisation was achieved during a 1-year follow-up (Fileccia et al, 2020). A study published in 2017 assessed the efficacy of rituximab.…”

Section: Treatmentmentioning

confidence: 99%

“…Opisano dwa przypadki zastosowania podskórnego (subcutaneous, s.c.) immunoglobulin w celu uniknięcia hospitalizacji związanych z podaniem i.v. Na początku leczenia pacjentów ustabilizowano, stosując leczenie i.v., następnie wdrożono terapię s.c. W czasie rocznej obserwacji u pacjentów uzyskano stabilizację objawów (Fileccia et al, 2020). Skuteczność rituksimabu oceniano w badaniu opublikowanym w 2017 roku.…”

Section: Leczenieunclassified

Stiff-person syndrome with sensorimotor polyneuropathy – case report

Jastrzębski¹,

Romanowska²,

Rek-Pacześ³

et al. 2021

Aktualn Neurol

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Stiff-person syndrome (SPS) is a rare disorder with an estimated prevalence in the general population of 1–2 cases/1,000,000. It is 2–3 times more common in females, with symptom onset at the age of 20–50 years in most cases. Although stiff-person syndrome is associated with antibodies against glutamic acid decarboxylase and amphiphysin, their presence is not necessary for the diagnosis. The treatment should be multidirectional and include immunomodulation, symptomatic treatment as well as monitoring and treatment of overlapping autoimmune, and surgery. We present a case report of a patient diagnosed with stiff-person syndrome overlapping with axonal and demyelinating sensorimotor polyneuropathy. The diagnostic workup indicated diabetes-related polyneuropathy. About 30% of patients diagnosed with stiff-person syndrome also have diabetes. Polyneuropathy alone is rarely reported to overlap with the disorder. In our opinion, polyneuropathy may have a beneficial effect on the clinical presentation of stiff-person syndrome.

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Subcutaneous immunoglobulin for maintenance therapy in stiff-person syndrome: One-year follow-up in two patients

Cited by 10 publications

References 17 publications

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

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

Therapies in Stiff-Person Syndrome

Stiff-person syndrome with sensorimotor polyneuropathy – case report

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