Deficits in the Management of Patients With Adrenocortical Carcinoma in Germany

Background: Adrenocortical carcinoma (ACC) is a rare tumor with a poor prognosis. Often, the physicians who first treat patients with ACC have no prior experience with the disease. The aim of our study was to evaluate the quality of medical care for patients with ACC in Germany. Methods: Data from the German ACC registry were analyzed with regard to the patients’ preoperative diagnostic evaluation, histopathological reporting, and clinical followup. The findings were compared with the recommendations of the European Network for the Study of Adrenal Tumors (ENSAT). Results: Data were analyzed from 387 patients who had been given an initial diagnosis of ACC in the years 1998 to 2009. 21% of them underwent no hormonal evaluation before surgery, and 59% underwent an inadequate hormonal evaluation. This exposed the patients to unnecessary perioperative risks and impaired their follow-up. 48% did not undergo CT scanning of the chest, even though the lungs are the most frequent site of metastases of ACC. For 13% of the patients, the diagnosis of ACC was later revised by a reference pathologist. For 11% of the patients, the histopathology report contained no information about resection status, even though this is an important determinant of further treatment and prognosis. Optimal management requires re-staging at three-month intervals, yet some patients underwent re-staging only after a longer delay, or not at all. Conclusion: We have identified significant deficits in the care of patients with ACC in Germany. We suspect that the situation is similar for other rare diseases. The prerequisite to better care is close and early cooperation of the treating physicians with specialized centers

On the Current Care Situation and Treatment of Ocular Mucous Membrane Pemphigoid in Germany

Background Ocular involvement in mucous membrane pemphigoid (MMP) is relatively rare, with a prevalence of 25 cases per million population, equating to approx. 2,100 patients throughout Germany. Diagnosis can be difficult - especially in cases of isolated ocular involvement - and treatment can be complex and lengthy. Immunosuppressants or immunomodulatory drugs are often used. Due to the complexity of diagnosis and treatment, MMP patients are usually referred to specialized centers. The aim of this project was to evaluate the current care situation of patients with ocular MMP in Germany. Methods A paper-based survey was designed and sent to all university eye clinics and other specialized centers in Germany in April 2020. The survey asked about the existence of a specialized outpatient service, the total annual number of patients with MMP, the annual number of newly diagnosed patients, any interdisciplinary collaboration for diagnostic or therapeutic purposes, as well as the local and systemic therapy used. Results Of a total of 44 clinics, 28 (64%) responded, reporting a total average of 27 +/- 42 (0 - 200) patients and 3.6 +/- 2.2 (0 - 10) new cases per year. This corresponds to a total of 741 patients. Only nine (32%) of the responding clinics offer specialized MMP clinics. 93% of the centers collaborate with the local dermatology department. 79% perform serological and histological diagnostics in-house. About half of the centers (n = 16) apply a standardized treatment regime. Systemic glucocorticoids (66.7%) are most commonly used, followed by mycophenolate mofetil and dapsone (57.1%), rituximab (33.3%), azathioprine and cyclophosphamide (28.6%), as well as methotrexate (19.0%). The least frequently used treatment is intravenous immunoglobulin (14.3%). Conclusion This survey of German ophthalmology departments obtained data from about one third of the estimated total cohort of all patients with MMP in Germany. These are presumed to be exclusively patients with at least one ocular involvement. The complex care of these patients is usually provided in collaboration with a dermatologist and with the use of systemic anti-inflammatory medication. Currently, an ophthalmological MMP register is being established to better record the epidemiology and care situation of this rare disease in Germany and to improve it in the long term. Background Ocular involvement in mucous membrane pemphigoid (MMP) is relatively rare, with a prevalence of 25 cases per million population, equating to approx. 2,100 patients throughout Germany. Diagnosis can be difficult - especially in cases of isolated ocular involvement - and treatment can be complex and lengthy. Immunosuppressants or immunomodulatory drugs are often used. Due to the complexity of diagnosis and treatment, MMP patients are usually referred to specialized centers. The aim of this project was to evaluate the current care situation of patients with ocular MMP in Germany. Methods A paper-based survey was designed and sent to all university eye clinics and other specialized centers in Germany in April 2020. The survey asked about the existence of a specialized outpatient service, the total annual number of patients with MMP, the annual number of newly diagnosed patients, any interdisciplinary collaboration for diagnostic or therapeutic purposes, as well as the local and systemic therapy used. Results Of a total of 44 clinics, 28 (64%) responded, reporting a total average of 27 +/- 42 (0 - 200) patients and 3.6 +/- 2.2 (0 - 10) new cases per year. This corresponds to a total of 741 patients. Only nine (32%) of the responding clinics offer specialized MMP clinics. 93% of the centers collaborate with the local dermatology department. 79% perform serological and histological diagnostics in-house. About half of the centers (n = 16) apply a standardized treatment regime. Systemic glucocorticoids (66.7%) are most commonly used, followed by mycophenolate mofetil and dapsone (57.1%), rituximab (33.3%), azathioprine and cyclophosphamide (28.6%), as well as methotrexate (19.0%). The least frequently used treatment is intravenous immunoglobulin (14.3%). Conclusion This survey of German ophthalmology departments obtained data from about one third of the estimated total cohort of all patients with MMP in Germany. These are presumed to be exclusively patients with at least one ocular involvement. The complex care of these patients is usually provided in collaboration with a dermatologist and with the use of systemic anti-inflammatory medication. Currently, an ophthalmological MMP register is being established to better record the epidemiology and care situation of this rare disease in Germany and to improve it in the long term

Zur Versorgungssituation und konservativen Therapie des okulären vernarbenden Schleimhautpemphigoids in Deutschland

$\bf Hintergrund:$ Die okuläre Beteiligung bei vernarbendem Schleimhautpemphigoid (SHP) ist mit einer Prävalenz von 25 Fällen je 1 Mio. Einwohner und damit ca. 2100 Patienten in ganz Deutschland selten. Die Diagnosestellung kann – besonders in Abwesenheit anderer Beteiligungen – schwierig und die Therapie komplex und langwierig sein. Nicht selten kommen Immunsuppressiva zum Einsatz. Aufgrund der Komplexität von Diagnose und Therapie sind SHP-Patienten meist an entsprechend spezialisierte Zentren angebunden. Ziel dieses Projektes war die Erfassung der aktuellen augenärztlichen Versorgungssituation von Patienten mit SHP in Deutschland. $\bf Methoden:$ Eine papierbasierte Umfrage wurde konzipiert und im April 2020 an alle Universitätsaugenkliniken und weitere potenzielle Zentren versandt. Gefragt wurde nach dem Bestehen einer spezialisierten Sprechstunde, der jährlichen Gesamtzahl der betreuten Patienten, der jährlichen Anzahl von neu diagnostizierten Patienten, den klinischen Kooperationspartnern in Diagnostik und Therapie sowie nach der angewendeten lokalen und systemischen Therapie. $\bf Ergebnisse:$ Von insgesamt 44 angeschrieben Kliniken erfolgten 28 (64%) vollständige Rückmeldungen. Im Mittel werden in den Kliniken 27 $\pm$ 42 (0–200) Patienten betreut und jährlich pro Zentrum 3,6 $\pm$ 2,2 (0–10) neue Fälle diagnostiziert. Dies entspricht einer Gesamtpatientenzahl von 741 Patienten. Lediglich 9 (32%) der antwortenden Kliniken bieten eine spezialisierte SHP-Sprechstunde an. 93% der Zentren kooperieren mit der lokalen Klinik für Dermatologie. 79% führen die serologische und histologische Diagnostik intern durch. Etwa die Hälfte der Zentren (n = 16) wendet ein standardisiertes Therapieschema an. Systemisch werden Glukokortikoide (66,7%) am häufigsten verwendet, gefolgt von Mycophenolatmofetil und Dapson (57,1 %), Rituximab (33,3%), Azathioprin und Cyclophosphamid (28,6%) sowie Methotrexat (19,0%). Am seltensten werden i.v. Immunoglobuline eingesetzt (14,3 %). $\bf Schlussfolgerung:$ Mit dieser Umfrage unter deutschen augenärztlichen Zentren wurden Daten von etwa einem Drittel der geschätzten Gesamtzahl aller in Deutschland an einem SHP erkrankten Menschen erhoben. Dabei handelt es sich vermutlich ausschließlich um Patienten mit mindestens einer okulären Beteiligung. Aktuell wird eine augenärztliche SHP-Registerstudie etabliert, um die Epidemiologie und Versorgungssituation besser zu erfassen und langfristig zu verbessern