Valera Elvis Terci, Latorre Maria do Rosário Dias, Mendes Wellington Luiz, Seber Adriana, Lee Maria Lucia Martino, de Paula Mario José Aguiar, Loggetto Sandra Regina, Velloso Elvira, Niero-Melo Ligia, Lopes Luiz Fernando
Departamento de Puericultura e Pediatria, Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto, Universidade de São Paulo, Av. Bandeirantes, 3900 Monte Alegre, CEP 14049-900, Brazil.
Leuk Res. 2004 Sep;28(9):933-9. doi: 10.1016/j.leukres.2004.01.008.
Therapy strategies for myelodysplastic syndromes (MDS) and juvenile myelomonocytic leukemia (JMML) vary considerably.
To review the treatment of Brazilian children who were diagnosed with MDS or JMML in the past decade and reported to the Brazilian Cooperative Group on Pediatric Myelodysplastic Syndromes (BCG-MDS-PED).
Of 173 children reported to the BCG-MDS-PED from January 1997 to January 2003 with a suspected diagnosis of MDS or JMML, 91 had the diagnosis confirmed after central review of the bone marrow aspirate and biopsy. Information on previous treatments was available for 78 MDS/JMML patients. Treatment varied from different schedules of low-dose (14%) and standard-dose chemotherapy (50%), granulocyte-colony-stimulating factor (G-CSF 7%), interferon (5%), steroids (2%) and erythropoietin (2%) to allogeneic stem-cell transplantation (SCT) (14%). No survival advantage could be demonstrated based on Hasle's classification or based on treatment.
This report reflects the current practice in treating Brazilian children with MDS/JMML without specific Cooperative Group guidelines. Treatment modalities were very heterogeneous. The strategies for implementing a national protocol should consider international guidelines and focus on local experience and available resources.
骨髓增生异常综合征(MDS)和青少年粒单核细胞白血病(JMML)的治疗策略差异很大。
回顾过去十年中被诊断为MDS或JMML并向巴西小儿骨髓增生异常综合征合作组(BCG-MDS-PED)报告的巴西儿童的治疗情况。
1997年1月至2003年1月间向BCG-MDS-PED报告疑似诊断为MDS或JMML的173名儿童中,91名经骨髓穿刺和活检的中央复查后确诊。78名MDS/JMML患者有既往治疗信息。治疗方法各不相同,从不同方案的低剂量(14%)和标准剂量化疗(50%)、粒细胞集落刺激因子(G-CSF,7%)、干扰素(5%)、类固醇(2%)和促红细胞生成素(2%)到异基因干细胞移植(SCT)(14%)。基于哈斯勒分类或治疗方法均未显示出生存优势。
本报告反映了在没有特定合作组指南的情况下,巴西治疗MDS/JMML儿童的当前做法。治疗方式非常多样化。实施国家方案的策略应考虑国际指南,并关注当地经验和可用资源。