加速儿童癌症优先配方的开发：环磷酰胺、依托泊苷、巯基嘌呤、甲氨蝶呤、普鲁卡因、替莫唑胺等儿童配方的目标产品简介

Target product profiles for paediatric formulationsof cyclophosphamide, etoposide, mercaptopurine,methotrexate, procarbazine, temozolomide Accelerating thedevelopment ofpriority formulationsin childhood cancer Target product profiles for paediatric formulationsof cyclophosphamide, etoposide, mercaptopurine,methotrexate, procarbazine, temozolomide Accelerating the development of priority formulations in childhood cancer: target product profilesfor paediatric formulations of cyclophosphamide, etoposide, mercaptopurine, methotrexate,procarbazine, temozolomide ISBN 978-92-4-011609-2 (electronic version)ISBN 978-92-4-011610-8 (print version) © World Health Organization 2025 Some rights reserved. This work is available under the Creative Commons Attribution-NonCommercial-ShareAlike 3.0 IGO licence (CC BY-NC-SA 3.0 IGO;https://creativecommons.org/licenses/by-nc-sa/3.0/igo). Under the terms of this licence, you may copy, redistribute and adapt the work for non-commercialpurposes, provided the work is appropriately cited, as indicated below. In any use of this work, thereshould be no suggestion that WHO endorses any specific organization, products or services. The useof the WHO logo is not permitted. If you adapt the work, then you must license your work under thesame or equivalent Creative Commons licence. If you create a translation of this work, you shouldadd the following disclaimer along with the suggested citation: “This translation was not created bythe World Health Organization (WHO). WHO is not responsible for the content or accuracy of thistranslation. The original English edition shall be the binding and authentic edition”. Any mediation relating to disputes arising under the licence shall be conducted in accordance withthe mediation rules of the World Intellectual Property Organization (http://www.wipo.int/amc/en/mediation/rules/). Suggested citation.Accelerating the development of priority formulations in childhood cancer:target product profiles for paediatric formulations of cyclophosphamide, etoposide, mercaptopurine,methotrexate, procarbazine, temozolomide. Geneva: World Health Organization; 2025. Licence:CC BY-NC-SA 3.0 IGO. Cataloguing-in-Publication (CIP) data.CIP data are available athttps://iris.who.int/. Sales, rights and licensing.To purchase WHO publications, seehttps://www.who.int/publications/book-orders. To submit requests for commercial use and queries on rights and licensing, seehttps://www.who.int/copyright. Third-party materials.If you wish to reuse material from this work that is attributed to a third party,such as tables, figures or images, it is your responsibility to determine whether permission is neededfor that reuse and to obtain permission from the copyright holder. The risk of claims resulting frominfringement of any third-party-owned component in the work rests solely with the user. General disclaimers.The designations employed and the presentation of the material in thispublication do not imply the expression of any opinion whatsoever on the part of WHO concerningthe legal status of any country, territory, city or area or of its authorities, or concerning thedelimitation of its frontiers or boundaries. Dotted and dashed lines on maps represent approximateborder lines for which there may not yet be full agreement. The mention of specific companies or of certain manufacturers’ products does not imply that theyare endorsed or recommended by WHO in preference to others of a similar nature that are notmentioned. Errors and omissions excepted, the names of proprietary products are distinguished byinitial capital letters. All reasonable precautions have been taken by WHO to verify the information contained in thispublication. However, the published material is being distributed without warranty of any kind, eitherexpressed or implied. The responsibility for the interpretation and use of the material lies with thereader. In no event shall WHO be liable for damages arising from its use. Contents AcknowledgementsivAcronyms and abbreviationsv1.Expert meeting on target product profile for priority childhood cancermedicines, 9–13 December 2024, Geneva, Switzerland: meeting report1Introduction1The target product profile development process5Assessing age appropriateness of existing formulations8Summary of key findings and discussion points12Discussions on common characteristics12Six target product profiles for childhood cancer132. Target product profile for a paediatric formulation of cyclophosphamide143. Target product profile for a paediatric formulation of etoposide184. Target product profile for a paediatric formulation of mercaptopurine225. Target product profile for a paediatric formulation of methotrexate266. Target product profile for a paediatric formulation of procarbazine307. Target product profile for a paediatric formulation of temozolomide34References38Annex 1. Scientific advisory group and meeting participants41Annex 2. Meeting agenda43Annex 3: All priority