Pemphigus
Conditions
Keywords
pemphigus, rituximab, immunoadsorption, dexamethasone, treatment, management
Brief summary
Pemphigus is a severe autoimmune blistering disease mediated by circulating antibodies against certain proteins important for maintaining skin integrity. Protein A immunoadsorption is a dialysis-like technique selectively removing the antibodies from patient's blood. Rituximab is a synthetic antibody capable of destroying B cells. B cells are responsible for production of antibodies in the patients blood that, in turn, lead to clinical signs of pemphigus. Dexamethasone pulse therapy is a high-dose short-term corticosteroid therapy that may be used to suppress autoantibody production in pemphigus. While each of these three therapies had been used to treat pemphigus, none was shown effective in all cases. The hypothesis of this study is that a combination of protein A immunoadsorption, rituximab and dexamethasone is more effective that either of these treatments alone in achieving a rapid and durable improvement or cure in patients with pemphigus.
Interventions
DRUGCombination of Protein A Immunoadsorption, Rituximab, Dexamethasone plus Azathioprine
Protein A Immunoadsorption: performed on 3 consecutive days every 3 weeks Rituximab: 1000 mg i.v. given twice at a 2-week interval Dexamethasone pulse therapy: 100 mg i.v. given on 3 consecutive days every 3 weeks Azathioprine: 2.5 mg/kg body weight daily p.o.
Sponsors
University of Luebeck
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosis of pemphigus confirmed by immunofluorescence and desmoglein ELISA. * Severe disease or past treatment(s) not effective or past treatment(s) not tolerated.
Exclusion criteria
* General condition too poor to tolerate immunoadsorption treatment. * Severe dementia or psychiatric disease.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients Achieving a Short- and Long-term Remission of Pemphigus | up to 43 months | Clinical remission was graded as partial remission on therapy, complete remission on therapy and complete remission off therapy, as described by Murell et al, J Am Acad Dermatol, 2008; 58:1043-6. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients Who Experienced Side-effects of Treatment | up to 43 months | Patients who experienced side-effects were counted. In addition, the nature and severity of side-effects were recorded. |
Countries
Germany
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Immunoadsorption/Dexamethasone/Rituximab Combination of Protein A Immunoadsorption, Rituximab, Dexamethasone plus Azathioprine/Mycophenolate mofetil
Protein A Immunoadsorption: performed on 3 consecutive days every 3-4 weeks
Rituximab: 1000 mg i.v. given twice at a 2-week interval
Dexamethasone pulse therapy: 100 mg i.v. given on 3 consecutive days initially every 3 weeks
Azathioprine: 2.5 mg/kg body weight daily p.o.
Mycophenolate mofetil 2 g/d p.o. | 23 |
| Total | 23 |
Baseline characteristics
| Characteristic | Immunoadsorption/Dexamethasone/Rituximab |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 6 Participants |
| Age, Categorical Between 18 and 65 years | 17 Participants |
| Age, Continuous | 55 years |
| Region of Enrollment Germany | 23 participants |
| Sex: Female, Male Female | 11 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 23 |
| serious Total, serious adverse events | 2 / 23 |
Outcome results
Primary
Number of Patients Achieving a Short- and Long-term Remission of Pemphigus
Clinical remission was graded as partial remission on therapy, complete remission on therapy and complete remission off therapy, as described by Murell et al, J Am Acad Dermatol, 2008; 58:1043-6.
Time frame: up to 43 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Immunoadsorption/Dexamethasone/Rituximab | Number of Patients Achieving a Short- and Long-term Remission of Pemphigus | 23 Participants |
Secondary
Number of Patients Who Experienced Side-effects of Treatment
Patients who experienced side-effects were counted. In addition, the nature and severity of side-effects were recorded.
Time frame: up to 43 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Immunoadsorption/Dexamethasone/Rituximab | Number of Patients Who Experienced Side-effects of Treatment | 2 Participants |