Treatment of epidermolysis bullosa acquisita with intravenous immunoglobulin in patients non‐responsive to conventional therapy: clinical outcome and post‐treatment long‐term follow‐up

Ahmed, A. Razzaque; Gürcan, Hakan M.

doi:10.1111/j.1468-3083.2011.04205.x

Cited by 30 publications

(16 citation statements)

References 41 publications

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“…These earlier drugs were withdrawn over a 5–9-month period, and IVIG was continued as monotherapy for a total duration of 30–52 months. In all 10 patients, a satisfactory clinical response was observed, and during followup (29–123 months), no relapse was observed [220]. Collectively, this data strongly supports the assumption that IVIG is an effective treatment option for EBA.…”

Section: Treatment Of Ebasupporting

confidence: 62%

Clinical Presentation, Pathogenesis, Diagnosis, and Treatment of Epidermolysis Bullosa Acquisita

Ludwig

2013

ISRN Dermatology

124

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Epidermolysis bullosa acquisita (EBA) is a chronic mucocutaneous autoimmune skin blistering disease. The pathogenic relevance of autoantibodies targeting type VII collagen (COL7) has been well-documented. Therefore, EBA is a prototypical autoimmune disease with a well-characterized pathogenic relevance of autoantibody binding to the target antigen. EBA is a rare disease with an incidence of 0.2 new cases per million and per year. The current treatment of EBA relies on general immunosuppressive therapy, which does not lead to remission in all cases. Therefore, there is a high, so far unmet medical need for the development of novel therapeutic options. During the last 10 years, several novel in vitro and in vivo models of EBA have been established. These models demonstrated a critical role of the genetic background, T cells, and cytokines for mediating the loss of tolerance towards COL7. Neutrophils, complement activation, Fc gamma receptor engagement, cytokines, several molecules involved in cell signaling, release of reactive oxygen species, and matrix metalloproteinases are crucial for autoantibody-induced tissue injury in EBA. Based on this growing understanding of the diseases' pathogenesis, several potential novel therapeutic targets have emerged. In this review, the clinical presentation, pathogenesis, diagnosis, and current treatment options for EBA are discussed in detail.

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Section: Treatment Of Ebasupporting

confidence: 62%

Clinical Presentation, Pathogenesis, Diagnosis, and Treatment of Epidermolysis Bullosa Acquisita

Ludwig

2013

ISRN Dermatology

124

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“…In this context, both reduced serum IFN-g levels of COL7-immunized mice and a lowered IFN-g expression on human Th1 cells has been found after anti-Hsp90 treatment (Tukaj et al, 2014a(Tukaj et al, , 2014b. In addition, treatment of COL7-immunized mice with intravenous immunoglobulins (IVIG) resulted in ameliorated clinical disease severity, reduced levels of autoantibodies, and a shift toward Th2-mediated nonpathogenic autoantibodies (Hirose et al, 2015), which supports their previously described effective and safe use in patients with autoimmune bullous diseases, including EBA (Ahmed and Gürcan, 2012;Ishii et al, 2010). Although the precise mechanisms are largely unclear, targeting GM-CSF and neutrophil functions could represent further potential treatment options to modulate autoantibody production in EBA patients.…”

Section: Gm-csf and Neutrophilssupporting

confidence: 73%

Epidermolysis Bullosa Acquisita: From Pathophysiology to Novel Therapeutic Options

Kasperkiewicz

Sadik

Bieber

et al. 2016

Journal of Investigative Dermatology

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Epidermolysis bullosa acquisita (EBA) is a prototypic organ-specific autoimmune disease induced by autoantibodies to type VII collagen causing mucocutaneous blisters. In the inflammatory (bullous pemphigoid-like) EBA variant, autoantibody binding is followed by a lesional inflammatory cell infiltration, and the overall clinical picture may be indistinguishable from that of bullous pemphigoid, the latter being the most common autoimmune bullous disease. The last decade witnessed the development of several mouse models of inflammatory EBA that facilitated the elucidation of the pathogenesis of autoantibody-induced, cell-mediated subepidermal blistering diseases and identified new therapeutic targets for these and possibly other autoantibody-driven disorders.

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“…The number of publications as well as the number of reported EBA cases remained relatively constant from 1979 to 2014. An increased number of EBA patients was reported in 1996–1998 [46–48], 2011–2012 [17, 21, 49] and 2016 [50, 51], which was due to the appearance of new diagnostic tools and thus the publication of large EBA patient collections during these time periods (Fig. 1).…”

Section: Resultsmentioning

confidence: 99%

Meta-analysis of the clinical and immunopathological characteristics and treatment outcomes in epidermolysis bullosa acquisita patients

Iwata

Vorobyev

Koga

et al. 2018

Orphanet J Rare Dis

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BackgroundEpidermolysis bullosa acquisita (EBA) is an orphan autoimmune disease. Several clinical phenotypes have been described, but subepidermal blistering is characteristic of all variants. Limited data on clinical and immunopathological characteristics and treatment outcomes in EBA are available. To fill this gap, we collected this information from EBA cases, meeting current diagnostic criteria, published between 1971 and 2016.ResultsWe identified 1159 EBA cases. This number must be, however, interpreted with caution, as it is not possible to check for multiple reporting. The analysis of all cases indicated that EBA affects all age groups (median: 50 years, range: 1 to 94 years) at an equal gender distribution. Non-mechanobullous (non-MB) forms of EBA were observed in 55% of patients, whereas the mechanobullous variant (MB-EBA) or a combination of both variants was described in 38 or 7% of patients, respectively. Type VII collagen (COL7)-specific autoantibodies were primarily of the IgG isotype, but anti-COL7 IgA, IgM and IgE were also documented. Comparison of the 2 clinical EBA types showed a higher frequency of IgA deposits in non-MB EBA as opposed to MB EBA. Mucous membrane involvement was observed in 23% of patients, and 4.4% of cases were associated with other chronic inflammatory diseases. Of note, IgA deposits were more frequently observed in cases with mucous membrane involvement. Our analysis indicated that EBA is difficult to treat and that the choice of treatment varies widely. Chi square was applied to identify medications associated with complete remission (CR). Considering all EBA cases, intravenous immunoglobulin (IVIG, p = 0.0047) and rituximab (p = 0.0114) were associated with CR. Subgroup analysis demonstrated that no treatment was associated with CR for non-MB EBA, while IVIG (p = 0.003) was associated with CR in MB EBA.ConclusionsWithin the limitations of the study, we here document the clinical and immunopathological characteristics and treatment outcomes in a large cohort of EBA patients. The observed associations of single drugs with treatment outcome may serve as a guide to develop clinical trials.Electronic supplementary materialThe online version of this article (10.1186/s13023-018-0896-1) contains supplementary material, which is available to authorized users.

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Treatment of epidermolysis bullosa acquisita with intravenous immunoglobulin in patients non‐responsive to conventional therapy: clinical outcome and post‐treatment long‐term follow‐up

Abstract: The data suggest that IVIg can produce a long-term sustained clinical remission in patients with EBA. In the patients, of this study concomitant therapy could be discontinued and IVIg was used as monotherapy.

Cited by 30 publications

References 41 publications

Clinical Presentation, Pathogenesis, Diagnosis, and Treatment of Epidermolysis Bullosa Acquisita

Clinical Presentation, Pathogenesis, Diagnosis, and Treatment of Epidermolysis Bullosa Acquisita

Epidermolysis Bullosa Acquisita: From Pathophysiology to Novel Therapeutic Options

Meta-analysis of the clinical and immunopathological characteristics and treatment outcomes in epidermolysis bullosa acquisita patients

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