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© 2025. This work is published under http://creativecommons.org/licenses/by/4.0/ (the "License"). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.

Abstract

To better characterise the survival outcomes of these patients, this retrospective cohort study evaluated overall survival (OS) of patients with PK deficiency and matched non-PK deficiency controls in the United Kingdom (UK), using data from the Clinical Practice Research Datalink (CPRD). Limitations of the current study include the potential for survivor bias and inaccurate disease diagnosis (e.g., due to limited availability of data in the CPRD regarding genetic testing, medical and treatment history [treatments include only those prescribed by a general practitioner, and not ‘over-the-counter’ medications]), low patient numbers (meaning sub-analyses, such as outcomes per age categories could not be performed) and unknown heterozygous carriers and varying disease severity. Acknowledgements Medical writing support, including assisting the authors with the development of the outline and initial draft and incorporation of comments, was provided by Alex Watson, and editorial support, including figure preparation, formatting, proofreading and submission, was provided by Thea Sheridon, both of Adelphi Communications Ltd, supported by Agios Pharmaceuticals, Inc. according to Good Publication Practice guidelines. The Sponsor was involved in the study design, collection, analysis and interpretation of data, as well as data checking of information provided in the manuscript.

Details

Title
Overall Survival of Patients With Pyruvate Kinase Deficiency in the UK: A Real‐World Study
Author
Foy, Patrick 1   VIAFID ORCID Logo  ; Higa, Sara 2 ; Zhao, Jing 2 ; Keapoletswe, Karabo 3 ; Cirneanu, Lorena 3 ; Venerus, Alessandra 4 ; Raiteri, Louise 3 ; Landfeldt, Erik 5 ; Iob, Eleonora 6 ; Lombard, Louise 2 ; Li, Junlong 2 ; Zagadailov, Erin 2 

 Division of Hematology and Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin, USA 
 Agios Pharmaceuticals, Inc., Cambridge, Massachusetts, USA 
 IQVIA Limited, London, UK 
 IQVIA Solutions Italy Srl, Milan, Italy 
 IQVIA, Stockholm, Sweden 
 IQVIA Limited, London, UK, Institute of Epidemiology and Healthcare, University College London, London, UK 
Section
CORRESPONDENCE
Publication year
2025
Publication date
Apr 1, 2025
Publisher
John Wiley & Sons, Inc.
e-ISSN
26886146
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
3194094456
Copyright
© 2025. This work is published under http://creativecommons.org/licenses/by/4.0/ (the "License"). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.