Document is current
Any future updates will be listed below
-
Authors
Locatelli, Franco https://orcid.org/0000-0002-7976-3654
Rizzari, Carmelo https://orcid.org/0000-0002-4828-3893
Gruhn, Bernd https://orcid.org/0000-0003-1862-0075
Klingebiel, Thomas
Parasole, Rosanna
Linderkamp, Christin
Flotho, Christian
Petit, Arnaud https://orcid.org/0000-0001-8363-1622
Micalizzi, Concetta
Eckert, Cornelia https://orcid.org/0000-0003-1039-2872
Möricke, Anja https://orcid.org/0000-0002-5439-7862
Sartor, Mary
Hrusak, Ondrej
Peters, Christina https://orcid.org/0000-0003-0369-8515
Saha, Vaskar https://orcid.org/0000-0002-2916-9649
Vinti, Luciana
Desai, Rajendra
Henry, Michael https://orcid.org/0000-0002-3624-0706
Zugmaier, Gerhard https://orcid.org/0000-0001-6879-2671
von Stackelberg, Arend https://orcid.org/0000-0002-8103-2631
-
Funding
Funding for this research was provided by:
Amgen (Not applicable)
Amgen (Not applicable)
- License Information
-
More Information
Article History
Received: 26 May 2025
Revised: 23 September 2025
Accepted: 27 October 2025
First Online: 13 November 2025
Competing interests
: FL has received honoraria from and/or has been a member of the speaker bureau for Novartis, Sobi, Miltenyi, bluebird bio, Amgen, Gilead, and BMS, and has participated in advisory board meetings for Amgen, Sobi, and Vertex. CR has received compensation as a scientific consultant and as a speaker in sponsored symposia from Jazz Pharmaceuticals, Servier, Amgen, Clinigen, and SERB Pharmaceuticals. RP has participated in advisory board meetings for Amgen, Novartis, Clinigen, and Jazz and has received honoraria from Amgen for participating in the speaker’s bureau. CP reports research funding from Amgen, Neovii, Jazz, Medac, and Riemser. LV has received research funding from Merck Sharpe and Co, travel and accommodation expenses from Amgen and Takeda, and has been a member of the speaker’s bureau for Amgen and Takeda. MH and GZ are employees of Amgen Inc. GZ has patents 20190300609 and 20130323247 licensed. AvS has received honoraria from and has been member of the speaker bureau for Amgen and Novartis and has participated in an advisory board for Amgen, Jazz-Pharmaceuticals, and Clinigen. The remaining authors have declared that they have no conflicts of interest to disclose.
: The study was performed in accordance with the Declaration of Helsinki. The trial protocol received approval from each center’s ethics committee/institutional review board (Supplementary Table ). Written informed consent was provided by patients’ parents or legal guardians.
