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BMJ Open|March 23, 2023
The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study designRachael F Grace, Eduard J van Beers, Joan-Lluis Vives Corrons, et al.
British Journal of Haematology|May 29, 2020
The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiencyHanny Al-Samkari, Kathryn Addonizio, Bertil Glader, et al.
British Journal of Haematology|October 11, 2014
A retrospective observational study of leucoreductive strategies to manage patients with acute myeloid leukaemia presenting with hyperleucocytosisKevin H M Kuo, Jeannie L Callum, Tony Panzarella, et al.
European Journal of Haematology|December 28, 2020
Comorbidities and complications in adults with pyruvate kinase deficiencyAudra N Boscoe, Yan Yan, Elizabeth Hedgeman, et al.
Blood Advances|August 26, 2025
Improvements in Health-Related Quality of Life in Patients with Severe Sickle Cell Disease After Exagamglogene AutotemcelAkshay Sharma, Franco Locatelli, Monica Bhatia, et al.
The New England Journal of Medicine|September 5, 2019
Safety and Efficacy of Mitapivat in Pyruvate Kinase DeficiencyRachael F Grace, Christian Rose, D Mark Layton, et al.
The Lancet. Haematology|February 13, 2025
Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trialMaria Domenica Cappellini, Vip Viprakasit, Pencho Georgiev, et al.
Blood Advances|August 27, 2025
Improvements in Health-Related Quality of Life in Patients with Transfusion-Dependent β-Thalassemia After Exagamglogene AutotemcelJosu de la Fuente, Haydar Frangoul, Peter Lang, et al.
American Journal of Hematology|June 8, 2026
Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-ThalassemiaSujit Sheth, Selim Corbacioglu, Josu de la Fuente, et al.
Lancet (London, England)|June 22, 2025
Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trialAli T Taher, Hanny Al-Samkari, Yesim Aydinok, et al.
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