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Development of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment: Disease‐specific assessments

INTRODUCTION: Currently recommended patient‐reported outcome (PRO) measures for patients with pyruvate kinase (PK) deficiency are non‐disease‐specific. The PK Deficiency Diary (PKDD) and PK Deficiency Impact Assessment (PKDIA) were developed to be more targeted measures for capturing the symptoms an...

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Dettagli Bibliografici
Pubblicato in:Eur J Haematol
Autori principali: Salek, Sam, Boscoe, Audra N., Piantedosi, Sarah, Egan, Shayna, Evans, Christopher J, Wells, Ted, Cohen, Jennifer, Klaassen, Robert J., Grace, Rachael, Storm, Michael
Natura: Artigo
Lingua:Inglês
Pubblicazione: John Wiley and Sons Inc. 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7216839/
https://ncbi.nlm.nih.gov/pubmed/31880847
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/ejh.13376
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