Original Research

Rituximab Treatment and Improvement of Health-Related Quality of Life in Patients With Pemphigus

Author and Disclosure Information

Pemphigus is an autoimmune blistering disease that can negatively affect patients’ lives. Assessing the impact of treatment from a patient’s perspective using outcome assessment measures is important and relevant in trials of new pemphigus treatments including rituximab (RTX). We sought to evaluate the effect of RTX on health-related quality of life (HRQOL) in pemphigus patients and peruse the clinical relevance of the patient-reported outcomes. A retrospective cross-sectional study was designed with 96 pemphigus patients given RTX either 3 months earlier or in the last 2 weeks. The treatment was evaluated by patients using HRQOL assessment tools: 36-Item Short Form Survey (SF-36) and Dermatology Life Quality Index (DLQI). Another patient-reported assessment was the patient global assessment (PGA). We found that RTX administration in pemphigus patients led to rapid and notable improvement in HRQOL and patient-assessed measures.

PRACTICE POINTS

  • Pemphigus is an autoimmune blistering disease that can negatively affect patients’ lives.
  • Assessing the impact of treatment from a patient’s perspective using outcome assessment measures is important and relevant in trials of new pemphigus treatments including rituximab.
  • Rituximab administration in pemphigus patients led to rapid and notable improvement in health-related quality of life and patient-assessed measures.


 

References

Pemphigus is a group of autoimmune blistering diseases characterized by the development of painful and flaccid blisters on the skin and/or mucous membranes. Pemphigus vulgaris (PV) and pemphigus foliaceus (PF) are 2 major subtypes and can be distinguished by the location of blister formation or the specificity of autoantibodies directed against different desmogleins.1,2 Although rare, pemphigus is considered a serious and life-threatening condition with a great impact on quality of life (QOL) due to disease symptoms (eg, painful lesions, physical appearance of skin lesions) as well as treatment complications (eg, adverse drug effects, cost of treatment).3-6 Moreover, the physical and psychological effects can lead to marked functional morbidity and work-related disability during patients’ productive years.7 Therefore, affected individuals usually have a remarkably compromised health-related quality of life (HRQOL).8 Effective treatments may considerably improve the QOL of patients with pemphigus.6

Despite the available treatment options, finding the best regimen for pemphigus remains a challenge. Corticosteroids are assumed to be the main treatment, though they have considerable side effects.9,10 Adjuvant therapies are used to suppress or modulate immune responses, leading to remission with the least possible need for corticosteroids. Finding an optimal steroid-sparing agent has been the aim of research, and biologic agents seem to be the best option.8 Rituximab (RTX), an anti-CD20 monoclonal antibody, has shown great promise in several studies of its clinical efficacy and has become a first-line treatment in new guidelines.11-14 Rituximab treatment has been associated with notable improvement in physician-assessed outcome measures with a favorable safety profile in patients with pemphigus.11-15 However, it is important to assess response to treatment from a patient’s perspective through the use of outcome-assessment measures that encompass patient-reported outcomes to reflect the complete patient experience and establish the overall impact of RTX as well as its likelihood of acceptance by patients with pemphigus.

In our study, we compared clinical outcomes and HRQOL through the use of disease-specific measures as well as comprehensive generic health status measures among patients with PV and PF who received RTX treatment 3 months earlier and those who received RTX in the last 2 weeks. The clinical relevance of the patient-reported outcomes is discussed.

MATERIALS AND METHODS

Study Design

We conducted a single-center cross-sectional study of 96 patients with pemphigus aged 18 to 65 years of either sex who were willing to participate in this study. Patients with a confirmed diagnosis of PV or PF who received RTX 3 months earlier or in the last 2 weeks were enrolled in the study. Patients were identified using Dermatry.ir, an archiving software that contains patients’ medical data. Exclusion criteria included lack of sufficient knowledge of the concepts of the questionnaires as well as age younger than 16 years. The study was conducted from October 2019 to April 2020 by the Autoimmune Bullous Disease Research Center at Razi Hospital in Tehran, Iran, which is the main dermatology-specific center and teaching hospital of Iran. The study protocol was approved by the relevant ethics committee.

Patients were categorized into 2 groups: (1) those who received RTX 3 months earlier (3M group); and (2) those who received RTX in the last 2 weeks (R group).

After an explanation of the study to participants, informed written consent was signed by each patient, and their personal data (eg, age, sex, education, marital status, smoking status), as well as clinical data (eg, type of pemphigus, duration of disease, site of onset, prednisolone dosage, presence of Nikolsky sign, anti-DSG1 and anti-DSG3 values, Pemphigus Disease Area Index [PDAI] score, RTX treatment protocol); any known comorbidities such as hypertension, diabetes mellitus, or morbid obesity; and any chronic pulmonary, cardiac, endocrinologic, renal, or hepatic condition, were collected and recorded in a predefined Case Record.

Patient-Reported Outcome Measures

The effect of RTX on QOL in patients with pemphigus was assessed using 2 HRQOL instruments: (1) a general health status indicator, the 36-Item Short Form Survey (SF-36), and (2) a validated, Persian version of a dermatology-specific questionnaire, Dermatology Life Quality Index (DLQI). The questionnaires were completed by each patient or by an assistant if needed.

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