Clinical outcomes in patients with pemphigus vulgaris receiving a combination rituximab and intravenous immunoglobin therapy protocol

Thesis (M.A.)--Boston University PLEASE NOTE: Boston University Libraries did not receive an Authorization To Manage form for this thesis or dissertation. It is therefore not openly accessible, though it may be available by request. If you are the author or principal advisor of this work and would like to request open access for it, please contact us at open-help@bu.edu. Thank you.Pemphigus Vulgaris is a potentially fatal autoimmune blistering disease that affects the skin and mucous membranes. The conventional therapy for this disease is high dose corticosteroids and immunosuppressive agents, which have a marked side effect profile. Therefore, subjects who suffer from this disease are not only affected by the sequelae of the pathology of pemphigus, but also by the treatments given. The advent of intravenous immunoglobulin and rituximab, a monoclonal antibody targeting the CD20 B cell marker, has helped induce a complete remission in subjects off all systemic therapy and decrease the morbidity of the disease. However, a small subset of patients with pemphigus vulgaris is unresponsive to all therapies. The advent of a rituximab and IVIg combination therapy protocol has shown positive preliminary results in inducing complete remission off all systemic therapy in this subset of subjects with recalcitrant pemphigus vulgaris. Moreover, it has shown a favorable side effect profile. However, the data on this combination therapy protocol has been limited. This research paper aimed to describe the clinical outcome of twelve subjects who have received the combination therapy protocol. The main study objective was to describe the duration of clinical remission off all systemic therapy. The secondary objectives were to describe the clinical control of disease, clinical remission on IVIg and rituximab, relapse, and adverse effects on the combination therapy. A chart review was conducted and the results were tabulated and described. The combination therapy was capable of inducing complete remission off all systemic therapy in all subjects that lasted at least two years. Moreover, eight subjects did not relapse, while four subjects relapsed only once and achieved remission after retreatment. No major or unexpected adverse events were seen during the combination therapy. Therefore, the combination therapy has shown positive and promising results in terms of efficacy and safety. This study has also added more information on this protocol to the literature, allowing clinicians to conduct more elaborate studies in the future that would allow the optimization of the protocol. The advent of this new combination protocol has given new hope to a subset of patients in whom the disease and its therapy were a great cause of morbidity and mortality, and research should continue in order to continue in improving the lives of all patients suffering from recalcitrant pemphigus vulgaris