P1548: LONG-TERM TREATMENT WITH ORAL MITAPIVAT IS ASSOCIATED WITH NORMALIZATION OF HEMOGLOBIN LEVELS IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY

• W. Barcellini,
• R. F. Grace,
• H. Al-Samkari,
• A. Glenthøj,
• J. A. Rothman,
• M. Morado Arias,
• D. M. Layton,
• O. Andres,
• M. DiBacco,
• P. Hawkins,
• M. P. Judge,
• F. Tai,
• J. Morales-Arias,
• V. Beynon,
• E. J. van Beers

Affiliations

W. Barcellini

1 Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

R. F. Grace

2 Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

H. Al-Samkari

3 Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States of America

A. Glenthøj

4 Department of Haematology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark

J. A. Rothman

5 Duke University Medical Center, Durham, NC, United States of America

M. Morado Arias

6 Hematology Department, Hospital Universitario La Paz, Madrid, Spain

D. M. Layton

7 Hammersmith Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom

O. Andres

8 Department of Paediatrics, University of Würzburg, Würzburg, Germany

M. DiBacco

9 Agios Pharmaceuticals, Inc., Cambridge, MA, United States of America

P. Hawkins

9 Agios Pharmaceuticals, Inc., Cambridge, MA, United States of America

M. P. Judge

9 Agios Pharmaceuticals, Inc., Cambridge, MA, United States of America

F. Tai

9 Agios Pharmaceuticals, Inc., Cambridge, MA, United States of America

J. Morales-Arias

9 Agios Pharmaceuticals, Inc., Cambridge, MA, United States of America

V. Beynon

9 Agios Pharmaceuticals, Inc., Cambridge, MA, United States of America

E. J. van Beers

10 Van Creveldkliniek, Department of Internal Medicine, University Medical Center Utrecht, Utrecht, Netherlands