Development of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment: Disease‐specific assessments-Reference-Cited by-同舟云学术

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

Published:2020-02-24 Issue:5 Volume:104 Page:427-434
ISSN:0902-4441
Container-title:European Journal of Haematology
language:en
Short-container-title:Eur J Haematol

Author:

Salek Sam¹,Boscoe Audra N.²,Piantedosi Sarah²,Egan Shayna³^ORCID,Evans Christopher J³,Wells Ted³,Cohen Jennifer⁴,Klaassen Robert J.⁵,Grace Rachael⁶,Storm Michael²

Affiliation:

1. School of Life and Medical Sciences University of Hertfordshire Hatfield UK

2. Agios Pharmaceuticals Inc. Cambridge MA USA

3. Endpoint Outcomes Boston MA USA

4. Patient Advocate Wrentham MA USA

5. Division of Hematology/Oncology Children's Hospital of Eastern Ontario Ottawa ON Canada

6. Dana‐Farber/Boston Children’s Cancer and Blood Disorders Program Boston MA USA

Publisher

Wiley

Subject

Hematology,General Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/ejh.13376

Reference26 articles.

1. Erythrocyte pyruvate kinase deficiency: 2015 status report

2. Estimating the prevalence of pyruvate kinase deficiency from the gene frequency in the general white population

3. Pyruvate kinase deficiency: The genotype-phenotype association

4. Prevalence of pyruvate kinase deficiency in a northern European population in the north of England

5. Hereditary Nonspherocytic Hemolytic Anemia Due to Pyruvate Kinase Deficiency: A Prevalence Study in Quebec (Canada)

Cited by 9 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias – pilot study protocol;BMJ Open;2024-07

2. Mitapivat: New dawn in pyruvate kinase deficiency and beyond;Pediatric Hematology Oncology Journal;2024-06

3. Diagnosis and management of pyruvate kinase deficiency: international expert guidelines;The Lancet Haematology;2024-03

4. Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial;Journal of Patient-Reported Outcomes;2023-11-09

5. Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial;The Lancet Haematology;2022-10