Vanessa F Schmidt1, Max Masthoff2, Veronika Vielsmeier3, Caroline T Seebauer3, Özlem Cangir4, Lutz Meyer4, Antje Mükke5, Werner Lang5, Axel Schmid6, Peter B Sporns7, Richard Brill8, Walter A Wohlgemuth8, Natascha Platz Batista da Silva9, Max Seidensticker10, Regina Schinner10, Julia Küppers11, Beate Häberle11, Frank Haubner12, Jens Ricke10, Martin Zenker13, Melanie A Kimm10, Moritz Wildgruber10. 1. Department of Radiology, University Hospital, LMU Munich, Munich, Germany. vanessa.schmidt@med.uni-muenchen.de. 2. Clinic for Radiology, University Hospital Muenster, Muenster, Germany. 3. Department of Otorhinolaryngology, University Hospital Regensburg, Regensburg, Germany. 4. Department of Pediatric Surgery, Center for Vascular Malformations, Klinikum Barnim GmbH, Werner Forssmann Hospital, Eberswalde, Germany. 5. Department of Vascular Surgery, University Hospital Erlangen, Erlangen, Germany. 6. Department for Radiology, University Hospital Erlangen, Erlangen, Germany. 7. Department of Neuroradiology, Clinic of Radiology and Nuclear Medicine, University Hospital Basel, Basel, Switzerland. 8. Clinic and Policlinic of Diagnostic Radiology, Martin-Luther University Halle-Wittenberg, Halle (Saale), Germany. 9. Department of Radiology, University Hospital Regensburg, Regensburg, Germany. 10. Department of Radiology, University Hospital, LMU Munich, Munich, Germany. 11. Department for Pediatric Surgery, University Hospital, LMU Munich, Munich, Germany. 12. Department of Otorhinolaryngology, University Hospital, LMU Munich, Munich, Germany. 13. Institute for Human Genetics, Otto von Guericke University of Magdeburg, Magdeburg, Germany.
Abstract
PURPOSE: Arteriovenous malformations (AVMs) as rare diseases are diagnostically and therapeutically challenging. Due to the limited evidence regarding treatment outcome, prospective data are needed on how different treatment regimens affect outcome. The aims of this prospective trial are to determine effectiveness, safety, and clinical outcome of multimodal treatment in patients with extracranial AVMs. MATERIALS AND METHODS: After clinical and magnetic resonance imaging (MRI)-based diagnosis and informed consent, 146 patients (> 4 years and < 70 years) undergoing multimodal therapy in tertiary care vascular anomalies centers will be included in this prospective observational trial. Treatment options include conservative management, medical therapy, minimally invasive image-guided procedures (embolization, sclerotherapy) and surgery as well as combinations of the latter. The primary outcome is the patient-reported QoL 6 months after completion of treatment using the short form-36 health survey version 2 (SF-36v2) and the corresponding short form-10 health survey (SF-10) for children. In addition, clinical presentation (physician-reported signs), MRI imaging (radiological assessment of devascularization), recurrence rate, and therapeutic safety will be analyzed. Further follow-up will be performed after 12, 24, and 36 months. Moreover, liquid biopsies are being obtained from peripheral blood at multiple time points to investigate potential biomarkers for therapy response and disease progression. DISCUSSION: The APOLLON trial is a prospective, multicenter, observational open-label trial with unequal study groups to generate prospective evidence for multimodal treatment of AVMs. A multicenter design with the potential to assess larger populations will provide an increased understanding of multimodal therapy outcome in this orphan disease. TRIAL REGISTRATION: German Clinical Trials Register (identification number: DRKS00021019) https://www.drks.de/drks_web/navigate.do?navigationId=trial.HTML&TRIAL_ID=DRKS00021019 .
PURPOSE: Arteriovenous malformations (AVMs) as rare diseases are diagnostically and therapeutically challenging. Due to the limited evidence regarding treatment outcome, prospective data are needed on how different treatment regimens affect outcome. The aims of this prospective trial are to determine effectiveness, safety, and clinical outcome of multimodal treatment in patients with extracranial AVMs. MATERIALS AND METHODS: After clinical and magnetic resonance imaging (MRI)-based diagnosis and informed consent, 146 patients (> 4 years and < 70 years) undergoing multimodal therapy in tertiary care vascular anomalies centers will be included in this prospective observational trial. Treatment options include conservative management, medical therapy, minimally invasive image-guided procedures (embolization, sclerotherapy) and surgery as well as combinations of the latter. The primary outcome is the patient-reported QoL 6 months after completion of treatment using the short form-36 health survey version 2 (SF-36v2) and the corresponding short form-10 health survey (SF-10) for children. In addition, clinical presentation (physician-reported signs), MRI imaging (radiological assessment of devascularization), recurrence rate, and therapeutic safety will be analyzed. Further follow-up will be performed after 12, 24, and 36 months. Moreover, liquid biopsies are being obtained from peripheral blood at multiple time points to investigate potential biomarkers for therapy response and disease progression. DISCUSSION: The APOLLON trial is a prospective, multicenter, observational open-label trial with unequal study groups to generate prospective evidence for multimodal treatment of AVMs. A multicenter design with the potential to assess larger populations will provide an increased understanding of multimodal therapy outcome in this orphan disease. TRIAL REGISTRATION: German Clinical Trials Register (identification number: DRKS00021019) https://www.drks.de/drks_web/navigate.do?navigationId=trial.HTML&TRIAL_ID=DRKS00021019 .
Authors: Vanessa F Schmidt; Martin Olivieri; Beate Häberle; Max Masthoff; Sinan Deniz; Peter B Sporns; Walter A Wohlgemuth; Moritz Wildgruber Journal: Hamostaseologie Date: 2022-03-09 Impact factor: 1.778
Authors: Sung Ki Cho; Young Soo Do; Dong Ik Kim; Young Wook Kim; Sung Wook Shin; Kwang Bo Park; Justin Sang Ko; Ae Ryoung Lee; Sung Wook Choo; In Wook Choo Journal: Korean J Radiol Date: 2008 May-Jun Impact factor: 3.500
Authors: Vanessa F Schmidt; Max Masthoff; Michael Czihal; Beatrix Cucuruz; Beate Häberle; Richard Brill; Walter A Wohlgemuth; Moritz Wildgruber Journal: Mol Cell Pediatr Date: 2021-12-07