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Paediatric Strategy Forum for medicinal product development for acute myeloid leukaemia in children and adolescents
- Source :
- European journal of cancer (Oxford, England : 1990), European Journal of Cancer
- Publication Year :
- 2020
- Publisher :
- Elsevier BV, 2020.
-
Abstract
- Purpose: The current standard-of-care for front-line therapy for acute myeloid leukaemia (AML) results in short-term and long-term toxicity, but still approximately 40% of children relapse. Therefore, there is a major need to accelerate the evaluation of innovative medicines, yet drug development continues to be adult-focused. Furthermore, the large number of competing agents in rare patient populations requires coordinated prioritisation, within the global regulatory framework and cooperative group initiatives. Methods: The fourth multi-stakeholder Paediatric Strategy Forum focused on AML in children and adolescents. Results: CD123 is a high priority target and the paediatric development should be accelerated as a proof-of-concept. Efforts must be coordinated, however, as there are a limited number of studies that can be delivered. Studies of FLT3 inhibitors in agreed paediatric investigation plans present challenges to be completed because they require enrolment of a larger number of patients than actually exist. A consensus was developed by industry and academia of optimised clinical trials. For AML with rare mutations that are more frequent in adolescents than in children, adult trials should enrol adolescents and when scientifically justified, efficacy data could be extrapolated. Methodologies and definitions of minimal residual disease need to be standardised internationally and validated as a new response criterion. Industry supported, academic sponsored platform trials could identify products to be further developed. The Leukaemia and Lymphoma Society PedAL/EUpAL initiative has the potential to be a major advance in the field. Conclusion: These initiatives continue to accelerate drug development for children with AML and ultimately improve clinical outcomes.
- Subjects :
- 0301 basic medicine
Cancer Research
medicine.medical_specialty
Childhood leukemia
Drug development
Disease
Article
Acute myeloid leukaemia
law.invention
03 medical and health sciences
0302 clinical medicine
Randomized controlled trial
law
Agency (sociology)
medicine
Intensive care medicine
Paediatric oncology
business.industry
Paediatric Strategy Forum
medicine.disease
Minimal residual disease
3. Good health
Clinical trial
030104 developmental biology
Oncology
030220 oncology & carcinogenesis
New product development
Cancer therapeutics
business
Subjects
Details
- ISSN :
- 09598049
- Volume :
- 136
- Database :
- OpenAIRE
- Journal :
- European Journal of Cancer
- Accession number :
- edsair.doi.dedup.....a151be0d38eb16c4837f136811936399