Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study

Myrthe J. van Dijk; Minke A. E. Rab; Brigitte A. van Oirschot; Jennifer Bos; Cleo Derichs; Anita W. Rijneveld; Marjon H. Cnossen; Erfan Nur; Bart J. Biemond; Marije Bartels; Judith J. M. Jans; Wouter W. van Solinge; Roger E. G. Schutgens; Richard van Wijk; Eduard J. van Beers

doi:https://doi.org/10.1002/ajh.26554

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study

Myrthe J. van Dijk, Minke A. E. Rab, Brigitte A. van Oirschot, Jennifer Bos, Cleo Derichs, Anita W. Rijneveld, Marjon H. Cnossen, Erfan Nur, Bart J. Biemond, Marije Bartels, Judith J. M. Jans, Wouter W. van Solinge, Roger E. G. Schutgens, Richard van Wijk, Eduard J. van Beers

Research output: Contribution to journal › Comment/Letter to the editor › Academic

22 Citations (Scopus)

Original language	English
Pages (from-to)	E226-E229
Journal	American journal of hematology
Volume	97
Issue number	7
Early online date	2022
DOIs	https://doi.org/10.1002/ajh.26554
Publication status	Published - 1 Jul 2022

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van Dijk, M. J., Rab, M. A. E., van Oirschot, B. A., Bos, J., Derichs, C., Rijneveld, A. W., Cnossen, M. H., Nur, E., Biemond, B. J., Bartels, M., Jans, J. J. M., van Solinge, W. W., Schutgens, R. E. G., van Wijk, R., & van Beers, E. J. (2022). Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study. American journal of hematology, 97(7), E226-E229. https://doi.org/10.1002/ajh.26554

@article{ebf42d8d8f8c45339ff587b48a679d16,

title = "Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study",

author = "{van Dijk}, {Myrthe J.} and Rab, {Minke A. E.} and {van Oirschot}, {Brigitte A.} and Jennifer Bos and Cleo Derichs and Rijneveld, {Anita W.} and Cnossen, {Marjon H.} and Erfan Nur and Biemond, {Bart J.} and Marije Bartels and Jans, {Judith J. M.} and {van Solinge}, {Wouter W.} and Schutgens, {Roger E. G.} and {van Wijk}, Richard and {van Beers}, {Eduard J.}",

note = "Funding Information: Minke A.E. Rab and Richard van Wijk receive research funding from RR Mechatronics, Axcella Health Inc. and Global Blood Therapeutics (GBT). Richard van Wijk and Eduard J. van Beers are consultants for GBT. Minke A.E. Rab, Richard van Wijk and Eduard J. van Beers receive research funding and are consultants for Agios Pharmaceuticals Inc. M.H.C's institution has received investigator‐initiated research and travel grants as well as speaker fees over the years from the Netherlands Organization for Scientific Research (NWO) and Netherlands National Research Agenda (NWA), the Netherlands Organization for Health Research and Development (ZonMw), the Dutch Innovatiefonds Zorgverzekeraars, Baxter/Baxalta/Shire/ Takeda, Pfizer, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis, and Nordic Pharma, and for serving as a steering board member for Roche, Bayer, and Novartis for which fees go to the Erasmus MC as an institution. Erfan Nur receives research funding from Novartis and Emmaus and participates in the advisory board of Novartis. Bart J. Biemond receives research funding from Sanquin, GBT, and Novartis and participated in the advisory boards of Novartis, GBT, Novo Nordisk, Celgene, Chiesi, and Bluebird Bio. The remaining authors declare no competing financial interests. Funding Information: The authors sincerely wish to thank the patients who participated in this study. The authors also thank all members of the SCORE consortium for recruitment and helpful comments on the study design, conduct, protocol, and manuscript. This study was supported in part by research funding from Agios Pharmaceuticals Inc., Cambridge, MA, USA.",

year = "2022",

month = jul,

day = "1",

doi = "https://doi.org/10.1002/ajh.26554",

language = "English",

volume = "97",

pages = "E226--E229",

journal = "American journal of hematology",

issn = "0361-8609",

publisher = "Wiley-Liss Inc.",

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van Dijk, MJ, Rab, MAE, van Oirschot, BA, Bos, J, Derichs, C, Rijneveld, AW, Cnossen, MH, Nur, E , Biemond, BJ, Bartels, M, Jans, JJM, van Solinge, WW, Schutgens, REG, van Wijk, R & van Beers, EJ 2022, 'Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study', American journal of hematology, vol. 97, no. 7, pp. E226-E229. https://doi.org/10.1002/ajh.26554

TY - JOUR

T1 - Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease

T2 - A phase 2, open-label study

AU - van Dijk, Myrthe J.

AU - Rab, Minke A. E.

AU - van Oirschot, Brigitte A.

AU - Bos, Jennifer

AU - Derichs, Cleo

AU - Rijneveld, Anita W.

AU - Cnossen, Marjon H.

AU - Nur, Erfan

AU - Biemond, Bart J.

AU - Bartels, Marije

AU - Jans, Judith J. M.

AU - van Solinge, Wouter W.

AU - Schutgens, Roger E. G.

AU - van Wijk, Richard

AU - van Beers, Eduard J.

N1 - Funding Information: Minke A.E. Rab and Richard van Wijk receive research funding from RR Mechatronics, Axcella Health Inc. and Global Blood Therapeutics (GBT). Richard van Wijk and Eduard J. van Beers are consultants for GBT. Minke A.E. Rab, Richard van Wijk and Eduard J. van Beers receive research funding and are consultants for Agios Pharmaceuticals Inc. M.H.C's institution has received investigator‐initiated research and travel grants as well as speaker fees over the years from the Netherlands Organization for Scientific Research (NWO) and Netherlands National Research Agenda (NWA), the Netherlands Organization for Health Research and Development (ZonMw), the Dutch Innovatiefonds Zorgverzekeraars, Baxter/Baxalta/Shire/ Takeda, Pfizer, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis, and Nordic Pharma, and for serving as a steering board member for Roche, Bayer, and Novartis for which fees go to the Erasmus MC as an institution. Erfan Nur receives research funding from Novartis and Emmaus and participates in the advisory board of Novartis. Bart J. Biemond receives research funding from Sanquin, GBT, and Novartis and participated in the advisory boards of Novartis, GBT, Novo Nordisk, Celgene, Chiesi, and Bluebird Bio. The remaining authors declare no competing financial interests. Funding Information: The authors sincerely wish to thank the patients who participated in this study. The authors also thank all members of the SCORE consortium for recruitment and helpful comments on the study design, conduct, protocol, and manuscript. This study was supported in part by research funding from Agios Pharmaceuticals Inc., Cambridge, MA, USA.

PY - 2022/7/1

Y1 - 2022/7/1

UR - http://www.scopus.com/inward/record.url?scp=85128243188&partnerID=8YFLogxK

U2 - https://doi.org/10.1002/ajh.26554

DO - https://doi.org/10.1002/ajh.26554

M3 - Comment/Letter to the editor

C2 - 35384026

SN - 0361-8609

VL - 97

SP - E226-E229

JO - American journal of hematology

JF - American journal of hematology

IS - 7

ER -

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study

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