Therapy with low-dose azacitidine for MDS in children and young adults: a retrospective analysis of the EWOG-MDS study group

British Journal of Haematology
Annamaria CsehChristian Flotho

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 appear...Continue Reading

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Citations

Oct 25, 2016·Biomedicine & Pharmacotherapy = Biomédecine & Pharmacothérapie·Zhaoyun LiuRong Fu
Sep 28, 2016·Expert Opinion on Drug Discovery·Francisco BautistaLucas Moreno
Sep 17, 2020·Oncoimmunology·Julie Le NaourErika Vacchelli
Jun 24, 2017·Nature Reviews. Cancer·Michael W N DeiningerEric Solary
Sep 27, 2019·Frontiers in Pediatrics·Michaela KuhlenJessica I Hoell
Jun 11, 2020·International Journal of Hematology·Akifumi NozawaToshiyuki Fukao
Sep 8, 2019·European Journal of Pharmacology·Akira Shimada
Mar 18, 2021·Current Hematologic Malignancy Reports·Kristen B McCulloughMrinal M Patnaik
Dec 7, 2021·Pediatric Blood & Cancer·Klara-Maria HanftSimone Hettmer

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