Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Ziegler Andreas, Weiß Claudia, Vill Katharina, Baumann Matthias, Bernert Günther, Blaschek Astrid, Eisenkölbl Astrid, Flotats-Bastardas Marina, Friese Johannes, Holzwarth Dorothea, Ganter Claudia, Goldhahn Klaus, Hahn Andreas, von der Hagen Maja, Hartmann Hans, Hasselmann Oswald, Horber Veronka, Husain Ralf A, Illsinger Sabine, Jacquier David, Johannsen Jessika, Köhler Cornelia, Kölbel Heike, Kolodzig Michael, Klein Andrea, Pechmann Astrid, von Moers Arpad, Müller-Felber Wolfgang, Rauscher Christian, Schreiber Gudrun, Schwartz Oliver, Sproß Joachim, Stettner Georg M, Stoltenburg Corinna, Stumpe Eva, Trollmann Regina, Wiegand Gerd, Wilichowski Ekkehard, Schara Ulrike, Kirschner Janbernd, collaborators and members of the INTEGRATE ATMP consortium
Аннотация
OBJECTIVE: Based on systematic literature review and evidence grading from part 1, this initiative aims to develop a structured treatment plan applicable across all treatment centers in Germany, Austria and Switzerland. Additionally, it seeks to establish consensus recommendations for the clinical management of safety alerts.
METHODS: Part 2 describes the methodology used to formulate Delphi consensus statements, the development of a structured treatment plan for OA treatment, and the anonymous consensus voting process with standardized follow-up in case of disagreement.
RESULTS: A total of 12 consensus statements were developed, addressing diagnostic work-up, safety evaluation, and best practice management of common adverse drug reactions associated with OA gene therapy. All statements achieved >95% consensus in the anonymous Delphi voting. Additionally, two consensus recommendations for handling of positive newborn screening result achieved consensus of 97% and 87%, respectively. A structured treatment plan for gene therapy was consented with 100% agreement, as were standardized recommendations for laboratory testing and a consensus-based algorithm for management of liver transaminase elevations.
CONCLUSIONS: Delphi-based expert recommendations, developed in co-creation with patient representatives, provide a framework to minimize complications associated with gene therapy and establish the basis for standardized post-marketing data collections. The methodology used in this Delphi-consensus-group can serve as a blueprint for future gene therapy approvals.