TY  - JOUR
A1  - Miesbach, Wolfgang
A1  - Menne, Jan
A1  - Bommer, Martin
A1  - Schönermarck, Ulf
A1  - Feldkamp, Thorsten
A1  - Nitschke, Martin
A1  - Westhoff, Timm
A1  - Seibert, Felix Sebastian
A1  - Woitas, Rainer
A1  - Sousa, Rui
A1  - Wolf, Michael
A1  - Walzer, Stefan
A1  - Schwander, Björn
T1  - Incidence of acquired thrombotic thrombocytopenic purpura in Germany: a hospital level study
T2  - Orphanet journal of rare diseases
N2  - Background: Acquired thrombotic thrombocytopenic Purpura (aTTP) is a life-threatening ultra-orphan disease with a reported annual incidence between 1.5 and 6.0 cases per million in Europe and mainly affecting otherwise young and healthy adults aged 40 years on average. The goal of this study was to assess the incidence of aTTP in Germany.
Methods: A systematic review was performed to determine the published evidence on the aTTP epidemiology in Germany. To obtain additional evidence on the proportion of aTTP cases within the national Thrombotic Microangiopathy (TMA) population a hospital-level study was performed, using a retrospective data collection approach. Diagnosis of aTTP was confirmed if ADAMTS13 level were < 10% and/or the medical records explicitly mentioned aTTP diagnosis. The aggregated hospital data were then projected to the national level using logistic regression techniques.
Results: The systematic literature search did not provide incidence estimates of aTTP in Germany. Eight centers (≈27% of the top 30 TMA hospitals) delivered data according to a predefined data collection form. On average (year 2014–2016) a total number of 172 aTTP episodes per year was projected (95% confidence interval [95%CI]: 132–212). The majority were newly diagnosed aTTP cases (n = 121; 95%CI: 105–129), and 51 were recurrent aTTP cases (95%CI: 27–84). The average annual projected incidence (year 2014–2016) of aTTP episodes was 2.10 per million inhabitants in Germany (95%CI: 1.60–2.58).
Conclusions: The determined annual incidence of newly diagnosed aTTP cases and the overall annual incidence of aTTP episodes in Germany confirm the ultra-orphan character of aTTP. An external validation against international registries (France, UK and USA) shows that our findings are quite comparable with those international incidence rates.
KW  - Epidemiology
KW  - Germany
KW  - Incidence
KW  - Thrombotic microangiopathy
KW  - Thrombotic thrombocytopenic purpura
Y1  - 2019
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/53343
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-533434
SN  - 1750-1172
N1  - Open Access: This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
VL  - 14
IS  - 1, Art. 260
SP  - 1
EP  - 10
PB  - BioMed Central
CY  - London
ER  -