title: Therapy with low-dose azacitidine for MDS in children and young adults: a retrospective analysis of the EWOG-MDS study group.
abstract: Low-dose azacitidine is efficient and safe in the therapy of malignant myeloid disorders in adults but data in children are lacking. We present a retrospective analysis of 24 children and young adults with myelodysplastic syndrome (MDS) who received azacitidine at the time of first diagnosis or relapse after allotransplant (2 children were treated with azacitidine both initially and for relapse). Diagnoses were refractory cytopenia of childhood (N = 4), advanced primary MDS (N = 9) and secondary MDS (N = 11). The median duration of treatment was four cycles. Azacitidine was well tolerated, but cytopenias led to dose reduction in five cases. Treatment was discontinued in one child because of impaired renal function. Sixteen MDS patients were treated with azacitidine at first diagnosis. One complete clinical remission was observed and one child showed complete marrow remission; six children experienced stable disease with haematological improvement. Ten children received azacitidine for relapsed MDS after transplant: of these, seven experienced stable disease for 2-30 cycles (median 3), including one patient with haematological improvement for seven cycles. In summary, azacitidine is effective in some children with MDS and appears to be a non-toxic option in palliative situations to prolong survival.
authors.affiliation: Division of Paediatric Haematology-Oncology, Department of Paediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors.forename: Annamaria M
authors.initials: AM
authors.lastname: Cseh
authors.affiliation: Division of Paediatric Haematology-Oncology, Department of Paediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors.forename: Charlotte M
authors.initials: CM
authors.lastname: Niemeyer
authors.affiliation: Division of Paediatric Haematology-Oncology, Department of Paediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors.forename: Ayami
authors.initials: A
authors.lastname: Yoshimi
authors.affiliation: Hospital Sant Joan de Deu, Barcelona, Spain.
authors.forename: Albert
authors.initials: A
authors.lastname: Catala
authors.affiliation: Swabian Children's Cancer Centre, Children's Hospital Augsburg, Augsburg, Germany.
authors.forename: Michael C
authors.initials: MC
authors.lastname: Frühwald
authors.affiliation: Department of Paediatrics, Aarhus University Hospital Skejby, Aarhus, Denmark.
authors.forename: Henrik
authors.initials: H
authors.lastname: Hasle
authors.affiliation: Princess Máxima Centre for Paediatric Oncology, Utrecht, the Netherlands.
authors.forename: Mary M
authors.initials: MM
authors.lastname: van den Heuvel-Eibrink
authors.affiliation: Department of Paediatrics, University Hospital Schleswig-Holstein, University of Lübeck, Lübeck, Germany.
authors.forename: Melchior
authors.initials: M
authors.lastname: Lauten
authors.affiliation: Department of Paediatric Haematology-Oncology, Ghent University Hospital, Ghent, Belgium.
authors.forename: Barbara
authors.initials: B
authors.lastname: De Moerloose
authors.affiliation: Trinity College and Department of Paediatric Haematology and Oncology, Our Lady's Children's Hospital Crumlin, Dublin, Ireland.
authors.forename: Owen P
authors.initials: OP
authors.lastname: Smith
authors.affiliation: Department of Paediatrics, Martin-Luther-University Halle-Wittenberg, Halle, Germany.
authors.forename: Toralf
authors.initials: T
authors.lastname: Bernig
authors.affiliation: Department of Paediatric Haematology and Oncology, Jena University Hospital, Children's Clinic, Jena, Germany.
authors.forename: Bernd
authors.initials: B
authors.lastname: Gruhn
authors.affiliation: Department of Paediatric Oncology, Haematology and Immunology, University of Heidelberg, Heidelberg, Germany.
authors.forename: Andreas E
authors.initials: AE
authors.lastname: Kulozik
authors.affiliation: Paediatric Haematology and Oncology, University Hospital Erlangen, Erlangen, Germany.
authors.forename: Markus
authors.initials: M
authors.lastname: Metzler
authors.affiliation: Department of Paediatric Haematology, Dr. Abdurrahman Yurtaslan Ankara Oncology Training and Research Hospital of the Ministry of Health, Ankara, Turkey.
authors.forename: Lale
authors.initials: L
authors.lastname: Olcay
authors.affiliation: Division of Paediatric Haematology and Oncology, University Children's Hospital, Dresden, Germany.
authors.forename: Meinolf
authors.initials: M
authors.lastname: Suttorp
authors.affiliation: University Children's Hospital, Ulm, Germany.
authors.forename: Ingrid
authors.initials: I
authors.lastname: Furlan
authors.affiliation: Division of Paediatric Haematology-Oncology, Department of Paediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors.forename: Brigitte
authors.initials: B
authors.lastname: Strahm
authors.affiliation: Division of Paediatric Haematology-Oncology, Department of Paediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
authors.forename: Christian
authors.initials: C
authors.lastname: Flotho
mesh_terms: D000293:Adolescent; D000328:Adult; D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001374:Azacitidine / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D004341:Drug Evaluation / Q000379:methods; D005260:Female; D006801:Humans; D008297:Male; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D009503:Neutropenia / Q000139:chemically induced; D010166:Palliative Care / Q000379:methods; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine
keywords: azacitidine; childhood; epigenetics, hypomethylating agent; myelodysplastic syndrome