Acta Dermato-Venereologica 99-2CompleteContent | Page 23

224 SHORT COMMUNICATION Unmet Needs in Pemphigoid Diseases: An International Survey Amongst Patients, Clinicians and Researchers Aniek LAMBERTS 1 , Marc YALE 2 , Sergei A. GRANDO 3 , Barbara HORVÁTH 1 , Detlef ZILLIKENS 4 and Marcel F. JONKMAN 1 1 Center for Blistering Diseases, Department of Dermatology, University of Groningen, University Medical Center Groningen, Hanzeplein 1, 9700 RB Groningen, The Netherlands, 2 International Pemphigus and Pemphigoid Foundation, Sacramento, 3 Department of Dermatology, University of California, UC Irvine Medical Center, Irvine, USA, and 4 Department of Dermatology, University of Lübeck, University Medical Center Schleswig-Holstein, Lübeck, Germany. E-mail: [email protected] Accepted Sep 27, 2018; E-published Sep 28, 2018 Pemphigoid diseases are subepidermal autoimmune bul- lous diseases, characterized by autoantibodies against structural proteins of the dermal–epidermal junction (1). Symptoms of severe pruritus, with or without tense blistering of the skin or mucosa, cause a high disease burden (2). Basic and clinical research has led to bet- ter understanding of the mechanisms of pemphigoid diseases and novel therapies have been developed (3). Nonetheless, there are gaps in our knowledge, and seve- ral disease areas are not studied yet. These unmet needs have not been well-characterized. While available time and resources are often limited in research, it is essential to address topics relevant to both patients and healthcare professionals (4). It is widely recognized now that pa- tients play an important role in setting the research agenda (5). The aim of this study was to explore and prioritize unmet needs in pemphigoid diseases from the perspective of patients, clinicians and researchers, in order to guide future research towards important research topics. A secondary aim was to identify points of improvement in patient care. METHODS A steering group was established in February 2017, consisting of a project coordinator (AL), experts on pemphigoid diseases (SAG, BH, DZ, MFJ) and a patient representative (MY; director of the International Pemphigus and Pemphigoid Foundation (IPPF)). A preliminary list of unmet needs was drawn up and discussed by the steering group in June 2017 at the IPPF conference in Lübeck, Germany. An online anonymous survey was developed using Qualtrics survey software (Table SI 1 ), containing questions about participants’ characteristics, and unmet needs in pemphigoid diseases. Seven or eight pre-listed needs composed by the steering group were provided and participants were asked whether they recognized the needs as unmet, and to designate a top 3 of the most urgent unmet needs. Moreover, participants were asked to complement the list composed by the steering group. In addition, patients were given questions about satisfaction with patient care, and their reasons for (dis)satisfaction. The survey was distributed internationally between October 2017 and April 2018. Patients were invited by email via the IPPF, and national German and Dutch patient organizations. Clinicians and researchers were invited by email via pemphigoid research groups, and via the European Academy of Dermatology and Ve- nereology. Data was exported from Qualtrics directly into SPSS Statistics version 23 (IBM, Chicago, IL, USA). Descriptive and https://www.medicaljournals.se/acta/content/abstract/10.2340/00015555-3052 1 doi: 10.2340/00015555-3052 Acta Derm Venereol 2019; 99: 224–225 qualitative statistics were used for data analysis. An overall ranking score was calculated by awarding 3 points every time unmet needs were ranked highest, 2 points if ranked 2 nd , and 1 point if ranked 3 rd . RESULTS The inclusion and exclusion process is shown in Fig. 1. The clinicians’ and researchers’ response rate was 36/99 (36%). The patients’ response rate is unknown. Partici- pants characteristics and the top 3 most urgent needs are shown in Table I. Patients, clinicians and researchers agreed that the most urgent need was improvement in therapeutic options for pemphigoid diseases (Table I). In addition, patients frequently expressed the need for more public information (n = 9) (Table SI 1 ). Data on patient satisfaction showed that half of the patients were unsatisfied with patient care during the diagnostic process, mainly due to misdiagnosis and long diagnostic delay (mentioned by 88% of unsatisfied patients; Table SII 1 ). Six patients visited more than 5 doc- tors before a correct diagnosis was made. Patients with epidermolysis bullosa acquisita and mucous membrane pemphigoid reported a longer diagnostic delay (mean 90.3  ±  127 and 19.7  ±  23 months), compared with patients with bullous pemphigoid (9.0  ±  22 months). Most patients 175 people visited the survey 26 people discontinued before answering the first question 149 participants 42 participants were excluded 21 pemphigus patients 2 patients filled in the survey twice 19 participants ended the survey before answering an unmet need related question 107 participants completed 134 surveys 71 patients: - 52 BP - 17 MMP - 2 EBA 35 clinicians 28 researchers Fig. 1. Flowchart of inclusion and exclusion of study participants. BP: bullous pemphigoid; MMP: mucous membrane pemphigoid; EBA: epidermolysis bullosa acquisita. This is an open access article under the CC BY-NC license. www.medicaljournals.se/acta Journal Compilation © 2019 Acta Dermato-Venereologica.