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 Strålin; Irene Schmid; Julián 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 Strålin, Irene Schmid, Julián 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

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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
Pages (from-to)	e30931
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

Keywords

Azacitidine/adverse effects
Child
Hematopoietic Stem Cell Transplantation/adverse effects
Humans
Leukemia, Myeloid, Acute/drug therapy
Myelodysplastic Syndromes/drug therapy
Treatment Outcome

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Locatelli, F., Strålin, 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), e30931. https://doi.org/10.1002/pbc.30931

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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.",

keywords = "MDS, azacitidine, hypomethylating agents, myelodysplastic syndromes, myeloid neoplasia, pediatrics, Azacitidine/adverse effects, Child, Hematopoietic Stem Cell Transplantation/adverse effects, Humans, Leukemia, Myeloid, Acute/drug therapy, Myelodysplastic Syndromes/drug therapy, Treatment Outcome",

author = "Franco Locatelli and Str{\aa}lin, {Karin Belander} and Irene Schmid and Juli{\'a}n 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}",

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year = "2024",

month = may,

doi = "10.1002/pbc.30931",

language = "English",

volume = "71",

pages = "e30931",

journal = "Pediatric blood & cancer",

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Locatelli, F, Strålin, 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, pp. 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; Strålin, Karin Belander; Schmid, Irene et al.
In: Pediatric blood & cancer, Vol. 71, No. 5, 05.2024, p. e30931.

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

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AU - Strålin, Karin Belander

AU - Schmid, Irene

AU - Sevilla, Julián

AU - Smith, Owen P

AU - van den Heuvel-Eibrink, Marry M

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AU - Poon, Jennifer

AU - Simcock, Mathew

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AB - 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.

KW - MDS

KW - azacitidine

KW - hypomethylating agents

KW - myelodysplastic syndromes

KW - myeloid neoplasia

KW - pediatrics

KW - Azacitidine/adverse effects

KW - Child

KW - Hematopoietic Stem Cell Transplantation/adverse effects

KW - Humans

KW - Leukemia, Myeloid, Acute/drug therapy

KW - Myelodysplastic Syndromes/drug therapy

KW - Treatment Outcome

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M3 - Article

C2 - 38433307

SN - 1545-5009

VL - 71

SP - e30931

JO - Pediatric blood & cancer

JF - Pediatric blood & cancer

IS - 5

ER -

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

Keywords

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