Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA-JMML-001 trial

Franco Locatelli; Karin Belander Stralin; Irene Schmid; Julian Sevilla; Owen P. Smith; Marry M. van den Heuvel-eibrink; Marco Zecca; Christian M. Zwaan; Allison Gaudy; Meera Patturajan; Jennifer Poon; Mathew Simcock; Charlotte M. Niemeyer

doi:10.1002/pbc.30931

Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA-JMML-001 trial

Franco Locatelli^*, Karin Belander Stralin, Irene Schmid, Julian Sevilla, Owen P. Smith, Marry M. van den Heuvel-eibrink, Marco Zecca, Christian M. Zwaan, Allison Gaudy, Meera Patturajan, Jennifer Poon, Mathew Simcock, Charlotte M. Niemeyer

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › Academic › peer-review

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Abstract

Here we report efficacy, pharmacokinetics, and safety data obtained in treatment-naive, pediatric patients with newly diagnosed advanced MDS receiving azacitidine in the AZA-JMML-001 study. The primary endpoint was response rate (proportion of patients with complete response [CR], partial response [PR], or marrow CR, sustained for >= 4 weeks). Of the 10 patients enrolled, one had an unconfirmed marrow CR and none had confirmed responses after three cycles; the study was therefore closed after stage 1. Azacitidine was well tolerated. The lack of efficacy of azacitidine in pediatric patients with newly diagnosed advanced MDS highlights the need for effective new treatments in these patients.

Original language	English
Article number	e30931
Number of pages	5
Journal	Pediatric Blood & Cancer
Volume	71
Issue number	5
Early online date	3 Mar 2024
DOIs	https://doi.org/10.1002/pbc.30931
Publication status	Published - May 2024

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© 2024 Wiley Periodicals LLC.

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Locatelli, F., Stralin, K. B., Schmid, I., Sevilla, J., Smith, O. P., van den Heuvel-eibrink, M. M., Zecca, M., Zwaan, C. M., Gaudy, A., Patturajan, M., Poon, J., Simcock, M., & Niemeyer, C. M. (2024). Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA-JMML-001 trial. Pediatric Blood & Cancer, 71(5), Article e30931. https://doi.org/10.1002/pbc.30931

@article{3449a0728f1447e48dd90741d5539f7e,

title = "Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA-JMML-001 trial",

abstract = "Here we report efficacy, pharmacokinetics, and safety data obtained in treatment-naive, pediatric patients with newly diagnosed advanced MDS receiving azacitidine in the AZA-JMML-001 study. The primary endpoint was response rate (proportion of patients with complete response [CR], partial response [PR], or marrow CR, sustained for >= 4 weeks). Of the 10 patients enrolled, one had an unconfirmed marrow CR and none had confirmed responses after three cycles; the study was therefore closed after stage 1. Azacitidine was well tolerated. The lack of efficacy of azacitidine in pediatric patients with newly diagnosed advanced MDS highlights the need for effective new treatments in these patients.",

author = "Franco Locatelli and Stralin, {Karin Belander} and Irene Schmid and Julian Sevilla and Smith, {Owen P.} and {van den Heuvel-eibrink}, {Marry M.} and Marco Zecca and Zwaan, {Christian M.} and Allison Gaudy and Meera Patturajan and Jennifer Poon and Mathew Simcock and Niemeyer, {Charlotte M.}",

note = "Publisher Copyright: {\textcopyright} 2024 Wiley Periodicals LLC.",

year = "2024",

month = may,

doi = "10.1002/pbc.30931",

language = "English",

volume = "71",

journal = "Pediatric Blood & Cancer",

issn = "1545-5009",

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Locatelli, F, Stralin, KB, Schmid, I, Sevilla, J, Smith, OP, van den Heuvel-eibrink, MM, Zecca, M, Zwaan, CM, Gaudy, A, Patturajan, M, Poon, J, Simcock, M & Niemeyer, CM 2024, 'Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA-JMML-001 trial', Pediatric Blood & Cancer, vol. 71, no. 5, e30931. https://doi.org/10.1002/pbc.30931

Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA-JMML-001 trial. / Locatelli, Franco; Stralin, Karin Belander; Schmid, Irene et al.
In: Pediatric Blood & Cancer, Vol. 71, No. 5, e30931, 05.2024.

Research output: Contribution to journal › Article › Academic › peer-review

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AU - Locatelli, Franco

AU - Stralin, Karin Belander

AU - Schmid, Irene

AU - Sevilla, Julian

AU - Smith, Owen P.

AU - van den Heuvel-eibrink, Marry M.

AU - Zecca, Marco

AU - Zwaan, Christian M.

AU - Gaudy, Allison

AU - Patturajan, Meera

AU - Poon, Jennifer

AU - Simcock, Mathew

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Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA-JMML-001 trial

Abstract

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