From the Guidelines
Paraneoplastic pemphigus is a rare and severe autoimmune disorder associated with underlying neoplasms, characterized by the presence of autoantibodies against multiple antigens.
Key Characteristics
- It is a severe autoimmune disorder
- Associated with underlying neoplasms
- Characterized by the presence of autoantibodies against multiple antigens Treatment of paraneoplastic pemphigus typically involves a combination of immunosuppressive medications, such as prednisolone and rituximab, as seen in the treatment of pemphigus vulgaris and pemphigus foliaceus 1.
Treatment Options
- Immunosuppressive medications, such as prednisolone (0.5–1 mg kg-1 for 3–6 months)
- Rituximab (1 g on days 0 and 14 and 0.5 g at 12 and 18 months) Previous studies have shown that rituximab is of utility in the treatment of paraneoplastic pemphigus, with rates of remission in refractory disease of up to 86% following a single cycle of treatment 1.
Important Considerations
- Management of the underlying malignancy is crucial
- In some cases, plasmapheresis may be considered to reduce autoantibody levels It is essential to note that the treatment of paraneoplastic pemphigus should be individualized, taking into account the severity of the disease, the presence of underlying neoplasms, and the patient's overall health status.
From the Research
Definition and Characteristics
- Paraneoplastic pemphigus (PNP) is a rare but life-threatening mucocutaneous disease mediated by paraneoplastic autoimmunity 2.
- It is a unique autoimmune blistering condition that can affect multiple organs other than the skin 3.
- PNP is characterized by the presence of autoantibodies that react against proteins, such as desmoplakins, desmogleins, desmocollins, and others that exist in cellular junctions 4.
Clinical Features
- Intractable stomatitis and polymorphous cutaneous eruptions, including blisters and lichenoid dermatitis, are characteristic clinical features of PNP 2.
- The earliest and most consistent finding is a painful, severe, chronic, and often recalcitrant stomatitis 3, 5.
- PNP can manifest clinically in a variety of ways, ranging from mucositis to lesions involving the skin and pulmonary changes 4.
Diagnosis
- Diagnosis can be made based on clinical and histopathological features, the presence of anti-plakin autoantibodies, and underlying neoplasms 2.
- Diagnostic criteria include the presence of chronic mucositis and polymorphic cutaneous lesions with occult or confirmed neoplasia; histopathological analysis exhibiting intraepidermal acantholysis, necrotic keratinocytes, and vacuolar interface dermatitis; direct immunofluorescence with intercellular deposits (IgG and C3) and at the basement membrane zone (IgG); indirect immunofluorescence with intercellular deposition of IgG (substrates: monkey esophagus and simple, columnar, and transitional epithelium); and, autoreactivity to desmogleins 1 and 3, desmocollins 1,2, and 3, desmoplakins I and II, envoplakin, periplakin, epiplakin, plectin, BP230, and α-2-macroglobulin-like protein 1 6.
Treatment and Prognosis
- Treatment of PNP is difficult, and the best outcomes have been reported with benign neoplasms that have been surgically excised 5.
- Immunosuppressive agents and biologics, including rituximab, have been used for the treatment of PNP; however, the prognosis is poor due to underlying malignancies, severe infections during immunosuppressive treatment, and bronchiolitis obliterans mediated by autoimmunity 2, 5.
- Reported survival rates in 1,2, and 5 years are 49%, 41%, and 38%, respectively 6.