David Cucchi, a PhD candidate in the Department of Hematology at Amsterdam UMC, and colleagues have now published a comprehensive overview of the efficacy of targeted therapies against acute myeloid leukemia (AML) in clinical trials over the past two decades.
The team reviewed the website ‘ClinicalTrials.Gov’ which contains information on all clinical studies performed world-wide, and selected all AML trials with precision medicines between January 2000 and September 2020. They investigated which targets and drugs were studied, how many patients were involved and which outcomes were evaluated. Since clinical trials use varying endpoints that are difficult to compare, a standardized method was employed to assess clinical benefit.
In the set timeframe, over 450 clinical investigations were identified, involving more than 150 targeted drugs and the participation of over 65,000 AML patients. Currently, 8 precision drugs have been approved for the treatment of AML patients, the majority of which prolong survival. However, it is surprising that the impact on quality of life was not assessed for any of the approved drugs and was only reported in the minority of clinical trials with targeted drugs against AML.
For selected groups of patients, precision medicines provided clear clinical benefits. Given the high number of patients that participated in these clinical studies and the relatively low number of targeted therapies that were eventually approved, clinical research should carefully select relevant drugs and not overlook non-targeted drugs for the treatment of AML. But foremost, the assessment of patient well-being should be an integral part of clinical trials involving targeted therapies in the future.
For more information, see: Cucchi, D.G.J., Polak, T.B., Ossenkoppele, G.J. et al. Two decades of targeted therapies in acute myeloid leukemia. Leukemia (2021). https://doi.org/10.1038/s41375-021-01164-x
Researchers
David G.J. Cucchi,
PhD candidate, Department of Hematology, Amsterdam UMC, Vrije Universiteit Amsterdam, Cancer Center Amsterdam, Amsterdam.
Tobias B. Polak,
Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam.
Gert J. Ossenkoppele,
Professor of Hematology, Department of Hematology, Amsterdam UMC, Vrije Universiteit Amsterdam, Cancer Center Amsterdam, Amsterdam.
Carin Uyl-De Groot,
Professor, Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam.
Jacqueline Cloos,
Professor of Hematology, Department of Hematology, Amsterdam UMC, Vrije Universiteit Amsterdam, Cancer Center Amsterdam, Amsterdam.
Sonja Zweegman,
Professor of Hematology, Department of Hematology, Amsterdam UMC, Vrije Universiteit Amsterdam, Cancer Center Amsterdam, Amsterdam.
Jeroen J.W.M. Janssen,
Principal Investigator, Department of Hematology, Amsterdam UMC, Vrije Universiteit