Cseh Annamaria M, Niemeyer Charlotte M, Yoshimi Ayami, Catala Albert, Frühwald Michael C, Hasle Henrik, van den Heuvel-Eibrink Mary M, Lauten Melchior, De Moerloose Barbara, Smith Owen P, Bernig Toralf, Gruhn Bernd, Kulozik Andreas E, Metzler Markus, Olcay Lale, Suttorp Meinolf, Furlan Ingrid, Strahm Brigitte, Flotho Christian
Division of Paediatric Haematology-Oncology, Department of Paediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
Hospital Sant Joan de Deu, Barcelona, Spain.
Br J Haematol. 2016 Mar;172(6):930-6. doi: 10.1111/bjh.13915. Epub 2016 Jan 13.
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.
低剂量阿扎胞苷治疗成人恶性髓系疾病有效且安全，但儿童相关数据尚缺。我们对24例初诊或异基因移植后复发时接受阿扎胞苷治疗的儿童和青年骨髓增生异常综合征（MDS）患者进行了回顾性分析（2例儿童初始及复发时均接受阿扎胞苷治疗）。诊断包括儿童难治性血细胞减少（4例）、进展期原发性MDS（9例）和继发性MDS（11例）。中位治疗周期为4个周期。阿扎胞苷耐受性良好，但5例因血细胞减少导致剂量降低。1例儿童因肾功能损害停止治疗。16例MDS患者初诊时接受阿扎胞苷治疗。观察到1例完全临床缓解，1例儿童出现完全骨髓缓解；6例儿童病情稳定且血液学改善。10例儿童移植后复发的MDS接受阿扎胞苷治疗：其中7例病情稳定2 - 30个周期（中位3个周期），包括1例血液学改善7个周期的患者。总之，阿扎胞苷对部分儿童MDS有效，在姑息治疗中似乎是延长生存期的无毒选择。
