Efficacy of intravenous immunoglobulins for laryngopharyngeal lesions and upper airway obstruction in epidermolysis bullosa acquisita

SummaryPemphigus and pemphigoid are two potentially life‐threatening groups of autoimmune diseases, characterized by autoantibodies targeting structural components of desmosomes or hemidesmosomes, respectively. Affected patients typically show itchy/painful plaques or blistering skin lesions and/or impairing mucosal blistering and erosions, which may strongly impact their quality of life. Since the milestone work of Walter Lever in 1953, who differentiated these two groups of diseases by histopathological analysis of the level of antibody‐mediated skin cleavage, enormous progresses occurred. Achievements made in laboratory diagnostics now allow to identify antigen specific structural proteins of the skin that are targeted by pathogenic autoantibodies. These progresses were accompanied by an increased understanding of the pathogenesis of these diseases thanks to the establishment of animal models reproducing disease and on studies on skin and blood of affected individuals, which have been leading to novel and disease‐specific treatments. Yet, given their phenotypical overlap with more common dermatological diseases, correct diagnosis and appropriate treatment are often delayed, in some cases leading to irreversible sequelae, including organ dysfunction (i.e., loss of vision in mucous membrane pemphigoid). Here, we provide a concise overview of the clinical appearance, diagnosis and therapeutic management of pemphigus and pemphigoid diseases.

Section: Epidermolysis Bullosa Acquisitamentioning

confidence: 99%

Pemphigus and pemphigoids: Clinical presentation, diagnosis and therapy

Didona,

Schmidt,

Maglie

et al. 2023

“…158 Insbesondere die SHP-ähnliche EBA bedarf hoher Vorsicht, da sie zu vernarbenden Läsionen, einschließlich atropher Narben, Synechien und Stenosen mit irreversiblen Funktionseinschränkungen führen kann. Folgen sind Dysphagie und Unterernährung (Pharynx-und Ösophagusstenose), 159 Symblepharon und Sehverlust, 160,161 Perforation der Nasenschei-dewand, Dysphonie (Larynxstenose) 162 und sogar Asphyxie (Tracheal-und Bronchialstenose). 145,163…”

Section: Epidermolysis Bullosa Acquisitaunclassified

Pemphigus‐ und Pemphigoid‐Erkrankungen: Klinik, Diagnostik und Therapie

Didona,

Schmidt,

Maglie

et al. 2023

ZusammenfassungPemphigus und Pemphigoid sind seltene Autoimmunkrankheiten der Haut mit potenziell lebensbedrohlichem Verlauf. Autoantikörper gegen epidermale und junktionale Strukturproteine (Desmosomen sowie Hemidesmosomen) führen bei Betroffenen typischerweise zu juckenden, schmerzhaften Plaques oder Blasen an der Haut und/oder Blasenbildung und Erosionen der Schleimhäute mit möglicher Einschränkung der Lebensqualität. Seit der bahnbrechenden Arbeit von Walter Lever im Jahr 1953, dem es gelang, mittels histopathologischer Untersuchung diese beiden Krankheitsgruppen anhand des Musters der Antikörper‐vermittelten Blasenbildung zu differenzieren, wurden enorme Fortschritte im Verständnis der Erkrankungen erzielt. Die Errungenschaften in der Labordiagnostik ermöglichten die Identifikation von Zielstrukturen zur präzisen Unterscheidung verschiedener Varianten der bullösen Autoimmunerkrankungen. Diese Fortschritte gingen dank der Entwicklung von Tiermodellen mit einem besseren Verständnis der Pathogenese einher. Außerdem haben Studien an Haut und Blut betroffener Patienten zu neuen und krankheitsspezifischen Behandlungen geführt. Aufgrund ihrer Seltenheit und der klinischen Ähnlichkeit mit anderen dermatologischen Erkrankungen verzögern sich die korrekte Diagnosestellung und die Einleitung einer entsprechenden Therapie häufig, was in einigen Fällen zu irreversiblen Folgeerscheinungen, einschließlich Funktionsstörungen von Organen (zum Beispiel Verlust des Sehvermögens beim Schleimhautpemphigoid) führt. Wir geben hier einen Überblick über das klinische Erscheinungsbild, den Diagnosealgorithmus und das therapeutische Management von Pemphigus‐ und Pemphigoid‐Erkrankungen.

“…During the past few decades, high dose intravenous immunoglobulin (IVIG) obtained from pooled donor plasma has been used to treat an increasing number of dermatological diseases, including various autoimmune and inflammatory disorders, and severe adverse drug reactions [1][2][3][4][5][6][7][8]. Guideline recommendations [9] addressing the off-label use of IVIG in dermatology are mainly based on case series and a limited number of small randomized controlled trials [6][7][8].…”

Section: Introductionmentioning

confidence: 99%

Neutropenia resulting from high‐dose intravenous immunoglobulin in dermatological patients

Stoevesandt

Heitmann

Goebeler

et al. 2020

SummaryBackground and objectivesCurrent guidelines recommend high‐dose intravenous immunoglobulin (IVIG) as a rescue therapy to treat severe cutaneous autoimmune disorders. Data on IVIG‐induced hematological adverse events are limited in dermatological patients. We assessed the incidence and clinical implications of IVIG‐induced neutropenia.Patients and methodsPatients who received one or several cycles of IVIG between 2014 and 2019 were retrospectively evaluated. IVIG was given according to standardized infusion protocols. Daily differential blood counts were performed. Information on clinical baseline data, dermatological diagnosis, immunosuppressive pre‐treatment, and IVIG‐related adverse events was retrieved from patient files.ResultsSeventeen patients received 106 IVIG treatment cycles. Neutrophil counts below 1,500/μL were documented during 36 (34.0 %) cycles, and neutrophils fell below 1,000/μL in 14 (13.2 %) cases. The average drop of neutrophils from day one (pre‐dose) to days 2 and 3 of IVIG therapy was statistically significant (p = 0.006, and p = 0.002, respectively) despite correction for hemodilution, and so was a slight decrease of thrombocytes (p = 0.029, and p = 0.011, respectively). Four patients developed seven episodes of bacterial infections during or immediately after IVIG therapy.ConclusionsIVIG‐induced neutropenia is frequent in dermatological patients. A risk of secondary bacterial infections cannot be excluded.