Pemfigus vulgaris – Rokowania, prognozy i postęp choroby

Pemfigus vulgaris
Rokowania, prognozy i postęp choroby

Pemfigus vulgaris (PV) to przewlekła choroba autoimmunologiczna pęcherzowa, której rokowanie uległo znaczącej poprawie dzięki terapii kortykosteroidowej i lekom oszczędzającym steroidy. Nieleczony PV charakteryzuje się śmiertelnością przekraczającą 75%, natomiast leczenie systemowymi sterydami redukuje ją do 5-15%. Kluczowymi czynnikami prognostycznymi są wiek zachorowania ≥65 lat, obecność choroby wieńcowej serca oraz poziom autoprzeciwciał anty-desmogleina 1 ≥100 U/ml w momencie diagnozy, które wiążą się z wyższą śmiertelnością. Wczesne rozpoznanie i szybkie wdrożenie terapii, w tym rytuksymabu jako leczenia pierwszego rzutu, poprawiają rokowanie i zmniejszają ryzyko nawrotów, które występują u ponad 50% pacjentów. Dodatkowo, rewersja bezpośredniej immunofluorescencji (DIF) do negatywnej może wskazywać na trwałą remisję po odstawieniu leczenia.

Pemfigus vulgaris – ogólna prognoza

Pemfigus vulgaris (PV) to przewlekła, długotrwała choroba autoimmunologiczna pęcherzowa, która przy odpowiednim leczeniu medycznym może być skutecznie kontrolowana. Rokowanie w przypadku pemfigusa vulgaris uległo drastycznej poprawie na przestrzeni ostatnich dekad, głównie dzięki postępom w terapii kortykosteroidowej oraz lekach oszczędzających steroidy.¹ Niemniej jednak, bez właściwego leczenia, choroba ta może mieć przebieg śmiertelny – śmiertelność nieleczonego pemfigusa vulgaris przekracza 75%.²³

Przed wprowadzeniem kortykosteroidów większość pacjentów z pemfigusem vulgaris umierała. Obecnie, dzięki terapii systemowymi sterydami, śmiertelność została znacząco zredukowana – do około 5-15%. Nieleczony lub niewłaściwie leczony pemfigus vulgaris nadal może prowadzić do zgonu, głównie z powodu podatności na infekcje oraz zaburzeń gospodarki wodno-elektrolitowej.⁴ Głównymi przyczynami zgonów pozostają infekcje bakteryjne i wirusowe oraz zaburzenia elektrolitowe.⁵

Większość zgonów następuje w ciągu pierwszych kilku lat trwania choroby. Jeśli pacjent przeżyje 5 lat, rokowanie jest dobre. Wczesna choroba jest prawdopodobnie łatwiejsza do kontrolowania niż rozległa, a śmiertelność może być wyższa, jeśli terapia jest opóźniona.⁶ Bez leczenia, pemfigus vulgaris jest często śmiertelny, zazwyczaj w ciągu 5 lat od początku choroby.⁷

Czynniki prognostyczne w pemfigus vulgaris

Chorobowość i śmiertelność w pemfigusie vulgaris zależą od kilku kluczowych czynników. W analizie jednowymiarowej wykazano istotny statystycznie związek między wiekiem zachorowania ≥65 lat, obecnością choroby wieńcowej serca, obecnością arytmii serca, poziomem autoprzeciwciał anty-desmogleina 1 ≥100 U/ml w momencie diagnozy a przeżywalnością pacjentów.⁸

Niezależne czynniki ryzyka zwiększonej śmiertelności

Analiza wielowymiarowa zidentyfikowała dwa główne niezależne czynniki ryzyka związane z ogólną śmiertelnością:⁹

Wiek zachorowania ≥65 lat¹⁰
Obecność choroby wieńcowej serca w momencie diagnozy¹¹

Śmiertelność u pacjentów z pemfigusem vulgaris jest trzy razy wyższa niż w populacji ogólnej.¹² Rokowanie jest gorsze u starszych pacjentów i osób z rozległą chorobą. Pacjenci, u których zdiagnozowano chorobę przed 65. rokiem życia, mają zazwyczaj lepsze rokowanie niż ci zdiagnozowani w wieku ≥65 lat.¹³ Rokowanie jest zwykle lepsze w dzieciństwie niż w wieku dorosłym.¹⁴

Znaczenie poziomu autoprzeciwciał

Oprócz typowych czynników prognostycznych, takich jak starszy wiek i choroby współistniejące układu sercowo-naczyniowego, poziom autoprzeciwciał okazał się być specyficznym dla choroby czynnikiem związanym z ogólną śmiertelnością w pemfigus vulgaris.¹⁵ Poziom autoprzeciwciał anty-desmogleina 1 ≥100 U/ml w momencie diagnozy wiąże się z wyższym ryzykiem ogólnej śmiertelności u pacjentów z pemfigus vulgaris.¹⁶

Dodatkowo, rewersja wyników bezpośredniej immunofluorescencji (DIF) do negatywnych może być użyteczna w przewidywaniu trwałej remisji po odstawieniu leczenia. Wyrwane włosy stanowią alternatywę dla biopsji skóry jako materiał do badania immunofluorescencyjnego; nabłonek osłonki włosa włosa w fazie anagenu zwykle wykazuje immunofluorescencję porównywalną ze skórą.¹⁷

Czynniki wpływające na przebieg choroby i odpowiedź na leczenie

Chorobowość i śmiertelność są związane z rozległością choroby, maksymalną dawką prednizolonu wymaganą do indukcji remisji oraz obecnością innych chorób.¹⁸ Powikłania wtórne związane ze stosowaniem wysokich dawek kortykosteroidów również przyczyniają się do śmiertelności.¹⁹²⁰

Czynniki ryzyka nawrotów

Nawroty mogą wystąpić u ponad 50% pacjentów z pemfigusem. W jednym z badań zidentyfikowano następujące czynniki ryzyka nawrotu:²¹

Rozległa powierzchnia skóry objęta chorobą
Wysoki wskaźnik masy ciała (BMI) pacjenta
Wysoki stopień nasilenia choroby na początku, określony przez wskaźnik PDAI (Pemphigus Disease Area Index)
Opóźnione rozpoczęcie leczenia
Wysokie miana przeciwciał anty-DSG1 i anty-DSG3 po leczeniu

Czynniki prognostyczne w leczeniu rytuksymabem

Rytuksymab, zatwierdzony jako leczenie pierwszego rzutu w pemfigusie vulgaris, skutecznie indukuje remisję. W badaniach analizujących czynniki prognostyczne dla lepszych wyników leczenia rytuksymabem wykazano:²²

61,9% pacjentów wcześniej leczonych mykofenolanu mofetylem osiągnęło częściową remisję, podczas gdy tylko 34,7% osiągnęło całkowitą remisję
Pacjenci z cukrzycą wykazywali znacznie krótszy średni czas do nawrotu w porównaniu z osobami bez cukrzycy
Pacjenci z czasem trwania choroby ≥16 miesięcy przed terapią rytuksymabem wykazywali krótszy średni czas do nawrotu
Wcześniejsze leczenie dapsonem wydłużało czas do nawrotu

Co ciekawe, płeć, wiek w momencie wystąpienia objawów i terapii rytuksymabem, pochodzenie etniczne, choroby współistniejące, zajęcie skóry, masa ciała, schemat dawkowania rytuksymabu i inne zmienne nie były statystycznie istotne pomiędzy grupami z całkowitą remisją i częściową remisją.²³

Jakość życia i chorobowość

Pemfigus vulgaris obejmuje błony śluzowe u 50-70% pacjentów. Może to ograniczać przyjmowanie pokarmów z powodu dysfagii. Pęcherze i nadżerki wtórne do pęknięcia pęcherzy mogą być bolesne i mogą ograniczać codzienne aktywności pacjenta.²⁴

Pacjenci z pemfigusem vulgaris zwykle goją się bez bliznowacenia, chyba że choroba jest powikłana ciężkim wtórnym zakażeniem.²⁵ Zmiany na błonach śluzowych i/lub skórne ewoluują od wiotkich pęcherzy (pęcherzyków i/lub pęcherzy) do głębokich nadżerek i są związane z bólem, szczególnie gdy zmiany rozwijają się w jamie ustnej.²⁶

Wyniki leczenia i remisja

Na podstawie danych z badań weterynaryjnych dotyczących leczenia pemfigusa vulgaris u psów, całkowitą remisję (CR) uzyskano u 65% psów, które otrzymały leczenie. Czas do osiągnięcia CR wahał się od 2 do 36 tygodni. Częściową remisję (PR) odnotowano u pozostałych zwierząt, choć definicja PR różniła się między przypadkami.²⁷ Te dane, choć pochodzą z medycyny weterynaryjnej, mogą dawać pewien wgląd w odpowiedź na leczenie w przypadku pemfigusa vulgaris.

Należy zaznaczyć, że rokowanie w przypadku pemfigusa paraneoplastycznego jest niekorzystne, ze śmiertelnością sięgającą 75-90%. Śmiertelność związana z PNP może być przypisana częściowo chorobie nowotworowej leżącej u podstaw jego patogenezy.²⁸

Wnioski końcowe

Podsumowując, pemfigus vulgaris stanowi istotne wyzwanie kliniczne, a jego przebieg i rokowanie zależą od wielu czynników. Najważniejsze czynniki prognostyczne obejmują wiek pacjenta w momencie zachorowania, obecność chorób współistniejących układu sercowo-naczyniowego oraz poziom autoprzeciwciał anty-desmogleina. Dzięki nowoczesnym metodom leczenia, w tym kortykosteroidom i lekom oszczędzającym steroidy, śmiertelność związana z pemfigusem vulgaris uległa znaczącej redukcji, choć wciąż pozostaje wyższa niż w populacji ogólnej.²⁹³⁰

Wczesne rozpoznanie i odpowiednie leczenie mają kluczowe znaczenie dla poprawy rokowania. Poziom autoprzeciwciał anty-desmogleina 1 jawi się jako potencjalny czynnik predykcyjny ogólnej przeżywalności, co powinno zostać potwierdzone w dalszych badaniach.³¹ Zrozumienie tych czynników może pomóc klinicystom w przewidywaniu wyników leczenia i wyborze odpowiedniej populacji pacjentów do terapii, w tym nowoczesnymi lekami biologicznymi jak rytuksymab.³²

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Materiały źródłowe

#1 An Updated Review of Pemphigus Diseases
https://pmc.ncbi.nlm.nih.gov/articles/PMC8540565/
PV represents a chronic, long-lasting condition that can be adequately controlled with medical therapy. The prognosis of PV has improved drastically over the decades as a result of recent advancements in corticosteroid and steroid-sparing treatments. Despite these advances in treatment, death still occurs in select patients with pemphigus, largely due to secondary infection. If left untreated, PV is typically fatal as a result of bacterial and viral infections and fluid and electrolyte imbalances. Most patients who are left untreated ultimately die within the first few years following diagnosis. Without adequate treatment, the reported mortality of PV is greater than 75%. […] Morbidity and mortality of PV is related to several factors, including the extent of mucocutaneous involvement, the dose of corticosteroid treatment required for treatment, and high-risk comorbidities. The prognosis of patients with PV is generally worse in elderly or immunocompromised patients and in those with extensive or severe disease. Patients who are diagnosed at 65 years of age are likely to fare better than those who are diagnosed at 65 years of age. […] The prognosis associated with PNP is largely unfavorable with a 75% to 90% mortality rate. Mortality associated with PNP can be attributed in part to the underlying malignancy that plays a central role in its pathogenesis.
#2 An Updated Review of Pemphigus Diseases
https://pmc.ncbi.nlm.nih.gov/articles/PMC8540565/
PV represents a chronic, long-lasting condition that can be adequately controlled with medical therapy. The prognosis of PV has improved drastically over the decades as a result of recent advancements in corticosteroid and steroid-sparing treatments. Despite these advances in treatment, death still occurs in select patients with pemphigus, largely due to secondary infection. If left untreated, PV is typically fatal as a result of bacterial and viral infections and fluid and electrolyte imbalances. Most patients who are left untreated ultimately die within the first few years following diagnosis. Without adequate treatment, the reported mortality of PV is greater than 75%. […] Morbidity and mortality of PV is related to several factors, including the extent of mucocutaneous involvement, the dose of corticosteroid treatment required for treatment, and high-risk comorbidities. The prognosis of patients with PV is generally worse in elderly or immunocompromised patients and in those with extensive or severe disease. Patients who are diagnosed at 65 years of age are likely to fare better than those who are diagnosed at 65 years of age. […] The prognosis associated with PNP is largely unfavorable with a 75% to 90% mortality rate. Mortality associated with PNP can be attributed in part to the underlying malignancy that plays a central role in its pathogenesis.
#3 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
The severity and natural history of pemphigus vulgaris are variable. Before the advent of steroids, most patients with pemphigus vulgaris died. Treatment with systemic steroids has reduced the mortality dramatically, to approximately 5-15%. If not properly treated, pemphigus vulgaris still is often fatal because of the susceptibility to infection and fluid and electrolyte disturbances. […] Mortality in patients with pemphigus vulgaris is three times higher than that in the general population. Most deaths occur during the first few years of disease; if the patient survives 5 years, the prognosis is good. Early disease probably is easier to control than widespread disease, and mortality may be higher if therapy is delayed. Complications secondary to the use of high-dose corticosteroids contribute to mortality as well.
#4 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
The severity and natural history of pemphigus vulgaris are variable. Before the advent of steroids, most patients with pemphigus vulgaris died. Treatment with systemic steroids has reduced the mortality dramatically, to approximately 5-15%. If not properly treated, pemphigus vulgaris still is often fatal because of the susceptibility to infection and fluid and electrolyte disturbances. […] Mortality in patients with pemphigus vulgaris is three times higher than that in the general population. Most deaths occur during the first few years of disease; if the patient survives 5 years, the prognosis is good. Early disease probably is easier to control than widespread disease, and mortality may be higher if therapy is delayed. Complications secondary to the use of high-dose corticosteroids contribute to mortality as well.
#5 An Updated Review of Pemphigus Diseases
https://pmc.ncbi.nlm.nih.gov/articles/PMC8540565/
PV represents a chronic, long-lasting condition that can be adequately controlled with medical therapy. The prognosis of PV has improved drastically over the decades as a result of recent advancements in corticosteroid and steroid-sparing treatments. Despite these advances in treatment, death still occurs in select patients with pemphigus, largely due to secondary infection. If left untreated, PV is typically fatal as a result of bacterial and viral infections and fluid and electrolyte imbalances. Most patients who are left untreated ultimately die within the first few years following diagnosis. Without adequate treatment, the reported mortality of PV is greater than 75%. […] Morbidity and mortality of PV is related to several factors, including the extent of mucocutaneous involvement, the dose of corticosteroid treatment required for treatment, and high-risk comorbidities. The prognosis of patients with PV is generally worse in elderly or immunocompromised patients and in those with extensive or severe disease. Patients who are diagnosed at 65 years of age are likely to fare better than those who are diagnosed at 65 years of age. […] The prognosis associated with PNP is largely unfavorable with a 75% to 90% mortality rate. Mortality associated with PNP can be attributed in part to the underlying malignancy that plays a central role in its pathogenesis.
#6 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
The severity and natural history of pemphigus vulgaris are variable. Before the advent of steroids, most patients with pemphigus vulgaris died. Treatment with systemic steroids has reduced the mortality dramatically, to approximately 5-15%. If not properly treated, pemphigus vulgaris still is often fatal because of the susceptibility to infection and fluid and electrolyte disturbances. […] Mortality in patients with pemphigus vulgaris is three times higher than that in the general population. Most deaths occur during the first few years of disease; if the patient survives 5 years, the prognosis is good. Early disease probably is easier to control than widespread disease, and mortality may be higher if therapy is delayed. Complications secondary to the use of high-dose corticosteroids contribute to mortality as well.
#7 Pemphigus Vulgaris – Dermatologic Disorders – Merck Manual Professional Edition
https://www.merckmanuals.com/professional/dermatologic-disorders/bullous-diseases/pemphigus-vulgaris
Without treatment, pemphigus vulgaris is often fatal, usually within 5 years of disease onset. […] Systemic corticosteroid and immunosuppressive therapy has improved prognosis, but death may still result from complications of therapy.
#8 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
Factors associated with survival in pemphigus have not yet been thoroughly addressed. Therefore, in the present study, risk factors for overall mortality in a large group of patients with pemphigus vulgaris and foliaceus were investigated. […] In pemphigus vulgaris group, univariate analysis found a statistically significant association between the age of onset 65 years, presence of coronary heart disease, presence of cardiac arrhythmia, level of anti-desmoglein1 autoantibodies 100 U/mL at diagnosis and the survival of the patients. […] Multivariate analysis identified the age of onset 65 years and the presence of coronary heart disease at diagnosis as independent risk factors associated with overall mortality. […] In addition to common prognostic factors, including older age and cardiovascular comorbidities, level of autoantibodies was found to be a disease-specific factor associated with overall mortality in pemphigus vulgaris.
#9 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
Factors associated with survival in pemphigus have not yet been thoroughly addressed. Therefore, in the present study, risk factors for overall mortality in a large group of patients with pemphigus vulgaris and foliaceus were investigated. […] In pemphigus vulgaris group, univariate analysis found a statistically significant association between the age of onset 65 years, presence of coronary heart disease, presence of cardiac arrhythmia, level of anti-desmoglein1 autoantibodies 100 U/mL at diagnosis and the survival of the patients. […] Multivariate analysis identified the age of onset 65 years and the presence of coronary heart disease at diagnosis as independent risk factors associated with overall mortality. […] In addition to common prognostic factors, including older age and cardiovascular comorbidities, level of autoantibodies was found to be a disease-specific factor associated with overall mortality in pemphigus vulgaris.
#10 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
An age of onset 65 years is associated with a higher risk of overall mortality in patients with pemphigus vulgaris and foliaceus. […] Presence of coronary heart disease and cardiac arrhythmia at diagnosis is associated with a higher risk of overall mortality in patients with pemphigus vulgaris. […] A level of anti-desmoglein 1 autoantibodies 100 U/mL at diagnosis associates with a higher risk of overall mortality in patients with pemphigus vulgaris. […] In conclusion, in addition to common prognostic factors, including older age and cardiovascular comorbidities, our study signals the level of anti-desmoglein 1 autoantibodies as a candidate prediction factor for overall survival that should be reinforced in further studies.
#11 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
An age of onset 65 years is associated with a higher risk of overall mortality in patients with pemphigus vulgaris and foliaceus. […] Presence of coronary heart disease and cardiac arrhythmia at diagnosis is associated with a higher risk of overall mortality in patients with pemphigus vulgaris. […] A level of anti-desmoglein 1 autoantibodies 100 U/mL at diagnosis associates with a higher risk of overall mortality in patients with pemphigus vulgaris. […] In conclusion, in addition to common prognostic factors, including older age and cardiovascular comorbidities, our study signals the level of anti-desmoglein 1 autoantibodies as a candidate prediction factor for overall survival that should be reinforced in further studies.
#12 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
The severity and natural history of pemphigus vulgaris are variable. Before the advent of steroids, most patients with pemphigus vulgaris died. Treatment with systemic steroids has reduced the mortality dramatically, to approximately 5-15%. If not properly treated, pemphigus vulgaris still is often fatal because of the susceptibility to infection and fluid and electrolyte disturbances. […] Mortality in patients with pemphigus vulgaris is three times higher than that in the general population. Most deaths occur during the first few years of disease; if the patient survives 5 years, the prognosis is good. Early disease probably is easier to control than widespread disease, and mortality may be higher if therapy is delayed. Complications secondary to the use of high-dose corticosteroids contribute to mortality as well.
#13 An Updated Review of Pemphigus Diseases
https://pmc.ncbi.nlm.nih.gov/articles/PMC8540565/
PV represents a chronic, long-lasting condition that can be adequately controlled with medical therapy. The prognosis of PV has improved drastically over the decades as a result of recent advancements in corticosteroid and steroid-sparing treatments. Despite these advances in treatment, death still occurs in select patients with pemphigus, largely due to secondary infection. If left untreated, PV is typically fatal as a result of bacterial and viral infections and fluid and electrolyte imbalances. Most patients who are left untreated ultimately die within the first few years following diagnosis. Without adequate treatment, the reported mortality of PV is greater than 75%. […] Morbidity and mortality of PV is related to several factors, including the extent of mucocutaneous involvement, the dose of corticosteroid treatment required for treatment, and high-risk comorbidities. The prognosis of patients with PV is generally worse in elderly or immunocompromised patients and in those with extensive or severe disease. Patients who are diagnosed at 65 years of age are likely to fare better than those who are diagnosed at 65 years of age. […] The prognosis associated with PNP is largely unfavorable with a 75% to 90% mortality rate. Mortality associated with PNP can be attributed in part to the underlying malignancy that plays a central role in its pathogenesis.
#14 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
Morbidity and mortality are related to the extent of disease, the maximum dose of prednisolone required to induce remission, and the presence of other diseases. The outlook is worse in older patients and in patients with extensive disease. Prognosis is usually better in childhood than in adulthood. […] Pemphigus vulgaris involves mucosa in 50-70% of patients. This may limit oral intake secondary to dysphagia. Blistering and erosions secondary to the rupture of blisters may be painful and may limit the patient’s daily activities. Patients with pemphigus vulgaris typically heal without scarring unless the disease is complicated by severe secondary infection. […] Reversion of DIF to negative can be useful for predicting sustained remission after withdrawal of medication. Plucked hairs are an alternative to skin biopsy in providing a specimen for immunofluorescence; the pilar sheath epithelium of the anagen hair typically demonstrates immunofluorescence comparable to skin. DIF on plucked hairs may be more acceptable to the patient than serial skin biopsies.
#15 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
Factors associated with survival in pemphigus have not yet been thoroughly addressed. Therefore, in the present study, risk factors for overall mortality in a large group of patients with pemphigus vulgaris and foliaceus were investigated. […] In pemphigus vulgaris group, univariate analysis found a statistically significant association between the age of onset 65 years, presence of coronary heart disease, presence of cardiac arrhythmia, level of anti-desmoglein1 autoantibodies 100 U/mL at diagnosis and the survival of the patients. […] Multivariate analysis identified the age of onset 65 years and the presence of coronary heart disease at diagnosis as independent risk factors associated with overall mortality. […] In addition to common prognostic factors, including older age and cardiovascular comorbidities, level of autoantibodies was found to be a disease-specific factor associated with overall mortality in pemphigus vulgaris.
#16 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
An age of onset 65 years is associated with a higher risk of overall mortality in patients with pemphigus vulgaris and foliaceus. […] Presence of coronary heart disease and cardiac arrhythmia at diagnosis is associated with a higher risk of overall mortality in patients with pemphigus vulgaris. […] A level of anti-desmoglein 1 autoantibodies 100 U/mL at diagnosis associates with a higher risk of overall mortality in patients with pemphigus vulgaris. […] In conclusion, in addition to common prognostic factors, including older age and cardiovascular comorbidities, our study signals the level of anti-desmoglein 1 autoantibodies as a candidate prediction factor for overall survival that should be reinforced in further studies.
#17 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
Morbidity and mortality are related to the extent of disease, the maximum dose of prednisolone required to induce remission, and the presence of other diseases. The outlook is worse in older patients and in patients with extensive disease. Prognosis is usually better in childhood than in adulthood. […] Pemphigus vulgaris involves mucosa in 50-70% of patients. This may limit oral intake secondary to dysphagia. Blistering and erosions secondary to the rupture of blisters may be painful and may limit the patient’s daily activities. Patients with pemphigus vulgaris typically heal without scarring unless the disease is complicated by severe secondary infection. […] Reversion of DIF to negative can be useful for predicting sustained remission after withdrawal of medication. Plucked hairs are an alternative to skin biopsy in providing a specimen for immunofluorescence; the pilar sheath epithelium of the anagen hair typically demonstrates immunofluorescence comparable to skin. DIF on plucked hairs may be more acceptable to the patient than serial skin biopsies.
#18 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
Morbidity and mortality are related to the extent of disease, the maximum dose of prednisolone required to induce remission, and the presence of other diseases. The outlook is worse in older patients and in patients with extensive disease. Prognosis is usually better in childhood than in adulthood. […] Pemphigus vulgaris involves mucosa in 50-70% of patients. This may limit oral intake secondary to dysphagia. Blistering and erosions secondary to the rupture of blisters may be painful and may limit the patient’s daily activities. Patients with pemphigus vulgaris typically heal without scarring unless the disease is complicated by severe secondary infection. […] Reversion of DIF to negative can be useful for predicting sustained remission after withdrawal of medication. Plucked hairs are an alternative to skin biopsy in providing a specimen for immunofluorescence; the pilar sheath epithelium of the anagen hair typically demonstrates immunofluorescence comparable to skin. DIF on plucked hairs may be more acceptable to the patient than serial skin biopsies.
#19 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
The severity and natural history of pemphigus vulgaris are variable. Before the advent of steroids, most patients with pemphigus vulgaris died. Treatment with systemic steroids has reduced the mortality dramatically, to approximately 5-15%. If not properly treated, pemphigus vulgaris still is often fatal because of the susceptibility to infection and fluid and electrolyte disturbances. […] Mortality in patients with pemphigus vulgaris is three times higher than that in the general population. Most deaths occur during the first few years of disease; if the patient survives 5 years, the prognosis is good. Early disease probably is easier to control than widespread disease, and mortality may be higher if therapy is delayed. Complications secondary to the use of high-dose corticosteroids contribute to mortality as well.
#20 Pemphigus Vulgaris – Dermatologic Disorders – Merck Manual Professional Edition
https://www.merckmanuals.com/professional/dermatologic-disorders/bullous-diseases/pemphigus-vulgaris
Without treatment, pemphigus vulgaris is often fatal, usually within 5 years of disease onset. […] Systemic corticosteroid and immunosuppressive therapy has improved prognosis, but death may still result from complications of therapy.
#21 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
Relapses may occur in more than 50% of patients with pemphigus (including variants other than pemphigus vulgaris). In one study, the following were found to be risk factors for relapse: extensive body surface area (BSA) involvement, high patient body mass index (BMI), high degree of severity at onset, as determined by the Pemphigus Disease Area Index (PDAI), delayed provision of treatment, high titers of anti-DSG1 and anti-DSG3 after treatment.
#22
https://medicaljournalssweden.se/actadv/article/view/27140
Pemphigus vulgaris is a chronic autoimmune blistering disease with significant morbidity. Rituximab, approved as its first-line treatment, effectively induces remission. However, few studies have analysed the prognostic factors for improved rituximab outcomes. […] Results showed that 61.9% of patients previously treated with mycophenolate mofetil achieved partial remission, whereas only 34.7% achieved complete remission. Patients with diabetes mellitus exhibited a significantly shorter median time to relapse compared with those without. Patients with a disease duration 16 months before rituximab therapy exhibited a shorter median time to relapse. Moreover, previous dapsone treatment extended time to relapse. Notably, sex, age at symptom onset and rituximab therapy, ethnicity, comorbidities, skin involvement, weight, rituximab dosing protocol, and other variables were not statistically significant between the complete remission and partial remission groups. These findings highlight the influence of specific patient characteristics and treatment histories on response to rituximab and time to relapse in pemphigus vulgaris patients. Understanding these factors can aid clinicians in predicting treatment outcomes and selecting the appropriate patient population for rituximab therapy.
#23
https://medicaljournalssweden.se/actadv/article/view/27140
Pemphigus vulgaris is a chronic autoimmune blistering disease with significant morbidity. Rituximab, approved as its first-line treatment, effectively induces remission. However, few studies have analysed the prognostic factors for improved rituximab outcomes. […] Results showed that 61.9% of patients previously treated with mycophenolate mofetil achieved partial remission, whereas only 34.7% achieved complete remission. Patients with diabetes mellitus exhibited a significantly shorter median time to relapse compared with those without. Patients with a disease duration 16 months before rituximab therapy exhibited a shorter median time to relapse. Moreover, previous dapsone treatment extended time to relapse. Notably, sex, age at symptom onset and rituximab therapy, ethnicity, comorbidities, skin involvement, weight, rituximab dosing protocol, and other variables were not statistically significant between the complete remission and partial remission groups. These findings highlight the influence of specific patient characteristics and treatment histories on response to rituximab and time to relapse in pemphigus vulgaris patients. Understanding these factors can aid clinicians in predicting treatment outcomes and selecting the appropriate patient population for rituximab therapy.
#24 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
Morbidity and mortality are related to the extent of disease, the maximum dose of prednisolone required to induce remission, and the presence of other diseases. The outlook is worse in older patients and in patients with extensive disease. Prognosis is usually better in childhood than in adulthood. […] Pemphigus vulgaris involves mucosa in 50-70% of patients. This may limit oral intake secondary to dysphagia. Blistering and erosions secondary to the rupture of blisters may be painful and may limit the patient’s daily activities. Patients with pemphigus vulgaris typically heal without scarring unless the disease is complicated by severe secondary infection. […] Reversion of DIF to negative can be useful for predicting sustained remission after withdrawal of medication. Plucked hairs are an alternative to skin biopsy in providing a specimen for immunofluorescence; the pilar sheath epithelium of the anagen hair typically demonstrates immunofluorescence comparable to skin. DIF on plucked hairs may be more acceptable to the patient than serial skin biopsies.
#25 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
Morbidity and mortality are related to the extent of disease, the maximum dose of prednisolone required to induce remission, and the presence of other diseases. The outlook is worse in older patients and in patients with extensive disease. Prognosis is usually better in childhood than in adulthood. […] Pemphigus vulgaris involves mucosa in 50-70% of patients. This may limit oral intake secondary to dysphagia. Blistering and erosions secondary to the rupture of blisters may be painful and may limit the patient’s daily activities. Patients with pemphigus vulgaris typically heal without scarring unless the disease is complicated by severe secondary infection. […] Reversion of DIF to negative can be useful for predicting sustained remission after withdrawal of medication. Plucked hairs are an alternative to skin biopsy in providing a specimen for immunofluorescence; the pilar sheath epithelium of the anagen hair typically demonstrates immunofluorescence comparable to skin. DIF on plucked hairs may be more acceptable to the patient than serial skin biopsies.
#26 Deep pemphigus (pemphigus vulgaris, pemphigus vegetans and paraneoplastic pemphigus) in dogs, cats and horses: a comprehensive review | BMC Veterinary Research | Full Text
https://bmcvetres.biomedcentral.com/articles/10.1186/s12917-020-02677-w
Pemphigus vulgaris is a chronic autoimmune blistering dermatosis that affects the mucosal, mucocutaneous junction and/or skin; the mucosal and/or skin lesions evolve from flaccid blisters (vesicles and/or bullae) to deep erosions, and are associated with pain, especially when the lesions develop in the oral cavity. […] In dogs with PV, the information on disease treatment and outcome can be inferred from 22 reports including 47 dogs. The final outcome was not stated for the last seven. Overall, a complete remission (CR) was obtained in 26/40 dogs (65%) that received treatment. The time-to-CR varied between 2 and 36 weeks. A partial remission (PR) was reported in 12 dogs, although the definition of PR varied between cases. […] The prognosis is grave for paraneoplastic pemphigus.
#27 Deep pemphigus (pemphigus vulgaris, pemphigus vegetans and paraneoplastic pemphigus) in dogs, cats and horses: a comprehensive review | BMC Veterinary Research | Full Text
https://bmcvetres.biomedcentral.com/articles/10.1186/s12917-020-02677-w
Pemphigus vulgaris is a chronic autoimmune blistering dermatosis that affects the mucosal, mucocutaneous junction and/or skin; the mucosal and/or skin lesions evolve from flaccid blisters (vesicles and/or bullae) to deep erosions, and are associated with pain, especially when the lesions develop in the oral cavity. […] In dogs with PV, the information on disease treatment and outcome can be inferred from 22 reports including 47 dogs. The final outcome was not stated for the last seven. Overall, a complete remission (CR) was obtained in 26/40 dogs (65%) that received treatment. The time-to-CR varied between 2 and 36 weeks. A partial remission (PR) was reported in 12 dogs, although the definition of PR varied between cases. […] The prognosis is grave for paraneoplastic pemphigus.
#28 An Updated Review of Pemphigus Diseases
https://pmc.ncbi.nlm.nih.gov/articles/PMC8540565/
PV represents a chronic, long-lasting condition that can be adequately controlled with medical therapy. The prognosis of PV has improved drastically over the decades as a result of recent advancements in corticosteroid and steroid-sparing treatments. Despite these advances in treatment, death still occurs in select patients with pemphigus, largely due to secondary infection. If left untreated, PV is typically fatal as a result of bacterial and viral infections and fluid and electrolyte imbalances. Most patients who are left untreated ultimately die within the first few years following diagnosis. Without adequate treatment, the reported mortality of PV is greater than 75%. […] Morbidity and mortality of PV is related to several factors, including the extent of mucocutaneous involvement, the dose of corticosteroid treatment required for treatment, and high-risk comorbidities. The prognosis of patients with PV is generally worse in elderly or immunocompromised patients and in those with extensive or severe disease. Patients who are diagnosed at 65 years of age are likely to fare better than those who are diagnosed at 65 years of age. […] The prognosis associated with PNP is largely unfavorable with a 75% to 90% mortality rate. Mortality associated with PNP can be attributed in part to the underlying malignancy that plays a central role in its pathogenesis.
#29 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
An age of onset 65 years is associated with a higher risk of overall mortality in patients with pemphigus vulgaris and foliaceus. […] Presence of coronary heart disease and cardiac arrhythmia at diagnosis is associated with a higher risk of overall mortality in patients with pemphigus vulgaris. […] A level of anti-desmoglein 1 autoantibodies 100 U/mL at diagnosis associates with a higher risk of overall mortality in patients with pemphigus vulgaris. […] In conclusion, in addition to common prognostic factors, including older age and cardiovascular comorbidities, our study signals the level of anti-desmoglein 1 autoantibodies as a candidate prediction factor for overall survival that should be reinforced in further studies.
#30 Pemphigus Vulgaris: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/1064187-overview
The severity and natural history of pemphigus vulgaris are variable. Before the advent of steroids, most patients with pemphigus vulgaris died. Treatment with systemic steroids has reduced the mortality dramatically, to approximately 5-15%. If not properly treated, pemphigus vulgaris still is often fatal because of the susceptibility to infection and fluid and electrolyte disturbances. […] Mortality in patients with pemphigus vulgaris is three times higher than that in the general population. Most deaths occur during the first few years of disease; if the patient survives 5 years, the prognosis is good. Early disease probably is easier to control than widespread disease, and mortality may be higher if therapy is delayed. Complications secondary to the use of high-dose corticosteroids contribute to mortality as well.
#31 Prediction of survival for patients with pemphigus vulgaris and pemphigus foliaceus: a retrospective cohort study | Orphanet Journal of Rare Diseases | Full Text
https://ojrd.biomedcentral.com/articles/10.1186/s13023-015-0263-4
An age of onset 65 years is associated with a higher risk of overall mortality in patients with pemphigus vulgaris and foliaceus. […] Presence of coronary heart disease and cardiac arrhythmia at diagnosis is associated with a higher risk of overall mortality in patients with pemphigus vulgaris. […] A level of anti-desmoglein 1 autoantibodies 100 U/mL at diagnosis associates with a higher risk of overall mortality in patients with pemphigus vulgaris. […] In conclusion, in addition to common prognostic factors, including older age and cardiovascular comorbidities, our study signals the level of anti-desmoglein 1 autoantibodies as a candidate prediction factor for overall survival that should be reinforced in further studies.
#32
https://medicaljournalssweden.se/actadv/article/view/27140
Pemphigus vulgaris is a chronic autoimmune blistering disease with significant morbidity. Rituximab, approved as its first-line treatment, effectively induces remission. However, few studies have analysed the prognostic factors for improved rituximab outcomes. […] Results showed that 61.9% of patients previously treated with mycophenolate mofetil achieved partial remission, whereas only 34.7% achieved complete remission. Patients with diabetes mellitus exhibited a significantly shorter median time to relapse compared with those without. Patients with a disease duration 16 months before rituximab therapy exhibited a shorter median time to relapse. Moreover, previous dapsone treatment extended time to relapse. Notably, sex, age at symptom onset and rituximab therapy, ethnicity, comorbidities, skin involvement, weight, rituximab dosing protocol, and other variables were not statistically significant between the complete remission and partial remission groups. These findings highlight the influence of specific patient characteristics and treatment histories on response to rituximab and time to relapse in pemphigus vulgaris patients. Understanding these factors can aid clinicians in predicting treatment outcomes and selecting the appropriate patient population for rituximab therapy.