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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.