A phase 1/2, open-label, dose-escalation study of midostaurin in children with relapsed or refractory acute leukaemia

C. Michel Zwaan; Stefan Söderhäll; Benoit Brethon; Matteo Luciani; Carmelo Rizzari; Ronald W. Stam; Emmanuelle Besse; Catherine Dutreix; Franca Fagioli; Phoenix A. Ho; Carlo Dufour; Rob Pieters

doi:10.1111/bjh.15593

A phase 1/2, open-label, dose-escalation study of midostaurin in children with relapsed or refractory acute leukaemia

C. Michel Zwaan, Stefan Söderhäll, Benoit Brethon, Matteo Luciani, Carmelo Rizzari, Ronald W. Stam, Emmanuelle Besse, Catherine Dutreix, Franca Fagioli, Phoenix A. Ho, Carlo Dufour, Rob Pieters

Research output: Contribution to journal › Letter › peer-review

26 Citations (Scopus)

Original language	English
Pages (from-to)	623-627
Number of pages	5
Journal	British Journal of Haematology
Volume	185
Issue number	3
DOIs	https://doi.org/10.1111/bjh.15593
Publication status	Published - May 2019

Keywords

acute leukaemia
FLT3
midostaurin
paediatric
relapsed/refractory

Access to Document

10.1111/bjh.15593

Cite this

@article{d8deea6978934d10bdb4e5aefb54b5aa,

title = "A phase 1/2, open-label, dose-escalation study of midostaurin in children with relapsed or refractory acute leukaemia",

keywords = "acute leukaemia, FLT3, midostaurin, paediatric, relapsed/refractory",

author = "Zwaan, {C. Michel} and Stefan S{\"o}derh{\"a}ll and Benoit Brethon and Matteo Luciani and Carmelo Rizzari and Stam, {Ronald W.} and Emmanuelle Besse and Catherine Dutreix and Franca Fagioli and Ho, {Phoenix A.} and Carlo Dufour and Rob Pieters",

note = "Funding Information: This study was supported by funding from Novartis Pharmaceuticals Corporation. We thank the patients enrolled in this trial and their families for their participation. We also acknowledge all of the investigators at the study sites that participated in the trial: Erasmus MC–Sophia Children's Hospital, Rotterdam, the Netherlands; Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden; H{\^o}pital Universitaire Robert-Debr{\'e}, Paris, France; IRCCS Ospedale Pediatrico Bambino Ges{\`u}, Rome, Italy; Fondazione MBBM, Azienda Ospedaliera San Gerardo, Monza, Italy; Paediatric Onco-Haematology, Stem Cell Transplantation and Cellular Therapy Division, A.O.U. Citt{\`a} della Salute e della Scienza di Torino, Ospedale Infantile Regina Margherita, Torino, Italy; Seattle Children's Hospital, Seattle, WA; Haematology Unit, IRCCS Instituto Giannina Gaslini, Genova, Italy; and Princess M{\'a}xima Centre for Paediatric Oncology, Utrecht, the Netherlands. We would also like to thank Katherine Mills-Luj{\'a}n, PhD, CMPP, Amarallys Cintron, PhD, and Pamela Tuttle, PhD, CMPP, of ArticulateScience LLC for medical editorial assistance with this manuscript, which was funded by Novartis Pharmaceuticals Corporation. Funding Information: This study was supported by funding from Novartis Pharmaceuticals Corporation. We thank the patients enrolled in this trial and their families for their participation. We also acknowledge all of the investigators at the study sites that participated in the trial: Erasmus MC–Sophia Children{\textquoteright}s Hospital, Rotterdam, the Netherlands; Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden; H{\^o}pital Universitaire Robert-Debr{\'e}, Paris, France; IRCCS Ospedale Pediatrico Bambino Ges{\`u}, Rome, Italy; Fondazione MBBM, Azienda Ospedaliera San Gerardo, Monza, Italy; Paediatric Onco-Haematology, Stem Cell Transplantation and Cellular Therapy Division, A.O.U. Citt{\`a}della Salute e della Scienza di Torino, Ospedale Infantile Regina Margherita, Torino, Italy; Seattle Children{\textquoteright}s Hospital, Seattle, WA; Haematology Unit, IRCCS Instituto Giannina Gaslini, Genova, Italy; and Princess M{\'a}xima Centre for Paediatric Oncology, Utrecht, the Netherlands. We would also like to thank Katherine Mills-Luj{\'a}n, PhD, CMPP, Amarallys Cintron, PhD, and Pamela Tuttle, PhD, CMPP, of ArticulateScience LLC for medical editorial assistance with this manuscript, which was funded by Novartis Pharmaceuticals Corporation.",

year = "2019",

month = may,

doi = "10.1111/bjh.15593",

language = "English",

volume = "185",

pages = "623--627",

journal = "British Journal of Haematology",

issn = "0007-1048",

publisher = "Wiley-Blackwell Publishing Ltd",

number = "3",

}

TY - JOUR

T1 - A phase 1/2, open-label, dose-escalation study of midostaurin in children with relapsed or refractory acute leukaemia

AU - Zwaan, C. Michel

AU - Söderhäll, Stefan

AU - Brethon, Benoit

AU - Luciani, Matteo

AU - Rizzari, Carmelo

AU - Stam, Ronald W.

AU - Besse, Emmanuelle

AU - Dutreix, Catherine

AU - Fagioli, Franca

AU - Ho, Phoenix A.

AU - Dufour, Carlo

AU - Pieters, Rob

N1 - Funding Information: This study was supported by funding from Novartis Pharmaceuticals Corporation. We thank the patients enrolled in this trial and their families for their participation. We also acknowledge all of the investigators at the study sites that participated in the trial: Erasmus MC–Sophia Children's Hospital, Rotterdam, the Netherlands; Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden; Hôpital Universitaire Robert-Debré, Paris, France; IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy; Fondazione MBBM, Azienda Ospedaliera San Gerardo, Monza, Italy; Paediatric Onco-Haematology, Stem Cell Transplantation and Cellular Therapy Division, A.O.U. Città della Salute e della Scienza di Torino, Ospedale Infantile Regina Margherita, Torino, Italy; Seattle Children's Hospital, Seattle, WA; Haematology Unit, IRCCS Instituto Giannina Gaslini, Genova, Italy; and Princess Máxima Centre for Paediatric Oncology, Utrecht, the Netherlands. We would also like to thank Katherine Mills-Luján, PhD, CMPP, Amarallys Cintron, PhD, and Pamela Tuttle, PhD, CMPP, of ArticulateScience LLC for medical editorial assistance with this manuscript, which was funded by Novartis Pharmaceuticals Corporation. Funding Information: This study was supported by funding from Novartis Pharmaceuticals Corporation. We thank the patients enrolled in this trial and their families for their participation. We also acknowledge all of the investigators at the study sites that participated in the trial: Erasmus MC–Sophia Children’s Hospital, Rotterdam, the Netherlands; Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden; Hôpital Universitaire Robert-Debré, Paris, France; IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy; Fondazione MBBM, Azienda Ospedaliera San Gerardo, Monza, Italy; Paediatric Onco-Haematology, Stem Cell Transplantation and Cellular Therapy Division, A.O.U. Cittàdella Salute e della Scienza di Torino, Ospedale Infantile Regina Margherita, Torino, Italy; Seattle Children’s Hospital, Seattle, WA; Haematology Unit, IRCCS Instituto Giannina Gaslini, Genova, Italy; and Princess Máxima Centre for Paediatric Oncology, Utrecht, the Netherlands. We would also like to thank Katherine Mills-Luján, PhD, CMPP, Amarallys Cintron, PhD, and Pamela Tuttle, PhD, CMPP, of ArticulateScience LLC for medical editorial assistance with this manuscript, which was funded by Novartis Pharmaceuticals Corporation.

PY - 2019/5

Y1 - 2019/5

KW - acute leukaemia

KW - FLT3

KW - midostaurin

KW - paediatric

KW - relapsed/refractory

UR - http://www.scopus.com/inward/record.url?scp=85053413507&partnerID=8YFLogxK

U2 - 10.1111/bjh.15593

DO - 10.1111/bjh.15593

M3 - Letter

C2 - 30203832

AN - SCOPUS:85053413507

SN - 0007-1048

VL - 185

SP - 623

EP - 627

JO - British Journal of Haematology

JF - British Journal of Haematology

IS - 3

ER -