Paraneoplastic autoimmune multiorgan syndrome (PAMS): Beyond the single phenotype of paraneoplastic pemphigus

Amber, Kyle T.; Valdebran, Manuel; Grando, Sergei A.

doi:10.1016/j.autrev.2018.04.008

Cited by 62 publications

(76 citation statements)

References 133 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Both humoral and cellular immune systems have been implicated in the pathogenic spectrum of PNP. 6 This patient's case suggests that antienvoplakin and antiperiplakin antibodies are not by themselves responsible for mucocutaneous lesions, although their pathogenic role remains to be elucidated. In line with this, the removal of antidesmoglein antibodies in mice injected with PNP sera showed the inability of the remaining autoantibodies to produce blister formation.…”

mentioning

confidence: 84%

“…The persistence of antienvoplakin and antiperiplakin antibodies and negativity for antidesmoplakin autoantibodies may explain why IIF on monkey oesophagus showed positive ICS and striated muscle immunostaining whereas rat bladder was negative. Both humoral and cellular immune systems have been implicated in the pathogenic spectrum of PNP . This patient's case suggests that antienvoplakin and antiperiplakin antibodies are not by themselves responsible for mucocutaneous lesions, although their pathogenic role remains to be elucidated.…”

mentioning

confidence: 86%

See 1 more Smart Citation

Persistence of antienvoplakin and antiperiplakin antibodies in a patient with paraneoplastic pemphigus 20 years after remission

et al. 2019

View full text Add to dashboard Cite

et al. Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa. JCI Insight 2019; 4:e126243. 6 Titeux M, Pendaries V, Zanta-Boussif MA et al. SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa. Mol Ther 2010; 18:1509-18. 7 Hovnanian A and GENEGRAFT Partners. Phase I/II ex vivo gene therapy clinical trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector (GENEGRAFT).

show abstract

mentioning

confidence: 84%

mentioning

confidence: 86%

Persistence of antienvoplakin and antiperiplakin antibodies in a patient with paraneoplastic pemphigus 20 years after remission

et al. 2019

View full text Add to dashboard Cite

show abstract

“…The most common variants of pemphigus are PV and PF (including endemic PF; Hübner, Recke, Zillikens, Linder, & Schmidt, ). Rare variants—not addressed herein—include paraneoplastic pemphigus and IgA pemphigus (Amber, Valdebran, & Grando, ; Hashimoto et al., ). In PV, the autoantibody response is directed against dsg3 (mucosal PV) or against dsg1 and dsg3 (muco‐cutaneous PV).…”

Section: Commentarymentioning

confidence: 99%

Translational Use of a Standardized Full Human Skin Organ Culture Model in Autoimmune Blistering Diseases

et al. 2019

View full text Add to dashboard Cite

The full human skin organ culture (HSOC) model is a standardized test system for evaluating pharmacological substances on human skin in vitro. The acantholysis associated with pemphigus vulgaris (PV), a severe and potentially life‐threatening autoimmune skin blistering disease, can be induced in HSOC by injecting a bi‐specific anti‐desmoglein (dsg) 1 and 3 single‐chain antibody variable fragment (scFv). Compared to cell culture experiments (e.g., induction of dsg3‐internalization or keratinocyte dissociation using HaCaT cells or normal human epidermal keratinocytes) the HSOC model is more sophisticated and physiologically relevant. In this model, all three layers of the human skin are present, all cells are sustained in their physiological niche and orientation, and the cell‐cell‐contacts remain intact. Here we describe a protocol for HSOC, an ex vivo model of human skin, that has proved to be well‐established and informative in our laboratory. © 2019 by John Wiley & Sons, Inc.

show abstract

“…Paraneoplastic autoimmune multiorgan syndrome (PAMS) is a distinct multiorganopathy, once thought to be related to PV. Underlying malignancy or lymphoproliferative disorders are frequently found in affected patients, along with autoantibodies to numerous proteins including desmoplakin 1 or 2, envoplakin, BP180, BP230, desmoglein 1 or 3, desmocollin 1 or 2 or 3 . Pemphigoid diseases are comprised of several conditions including bullous pemphigoid (BP) and epidermolysis bullosa acquisita (EBA) .…”

Section: Introductionmentioning

confidence: 99%

“…Underlying malignancy or lymphoproliferative disorders are frequently found in affected patients, along with autoantibodies to numerous proteins including desmoplakin 1 or 2, envoplakin, BP180, BP230, desmoglein 1 or 3, desmocollin 1 or 2 or 3. 12 Pemphigoid diseases are comprised of several conditions including bullous pemphigoid (BP) and epidermolysis bullosa acquisita (EBA). 13 Targeting of distinct antigens results in immune complex (IC)mediated complement activation, granulocyte recruitment and subsequent protease and reactive oxygen species (ROS) mediated basement membrane zone (BMZ) destruction.…”

Section: Introductionmentioning

confidence: 99%

From bench to bedside: evolving therapeutic targets in autoimmune blistering disease

Kridin

Kowalski

Kneiber

et al. 2019

Acad Dermatol Venereol

View full text Add to dashboard Cite

Autoimmune blistering diseases comprise a group of heterogenous conditions characterized by the loss of tolerance and subsequent development of autoantibodies targeting epidermal and subepidermal adhesion proteins. Blisters and erosions form on the skin and mucous membranes leading to significant morbidity and mortality. Traditional therapies rely on systemic immunosuppression. Advancements in our understanding of the pathophysiology of pemphigus and pemphigoid have led to the development of molecules which target specific pathways involved in induction and perpetuation of disease. In this review, we outline the novel therapeutic strategies including B‐cell depletion, T‐regulatory cell repletion, cell signalling inhibitors and small molecular inhibitors, inhibitory monoclonal antibodies, as well as complement inhibition. We additionally review their current level of clinical evidence. We lastly review therapeutics targets gleaned from the experimental epidermolysis bullosa acquisita mouse model. These emerging treatments offer an exciting progression from basic science discoveries that have the potential to transform the treatment paradigm in autoimmune blistering diseases.

show abstract

Paraneoplastic autoimmune multiorgan syndrome (PAMS): Beyond the single phenotype of paraneoplastic pemphigus

Cited by 62 publications

References 133 publications

Persistence of antienvoplakin and antiperiplakin antibodies in a patient with paraneoplastic pemphigus 20 years after remission

Persistence of antienvoplakin and antiperiplakin antibodies in a patient with paraneoplastic pemphigus 20 years after remission

Translational Use of a Standardized Full Human Skin Organ Culture Model in Autoimmune Blistering Diseases

From bench to bedside: evolving therapeutic targets in autoimmune blistering disease

Contact Info

Product

Resources

About