Aricò M, Bossi G, Schirò R, Galimberti M, Longoni D, Macchia P, Miniero R, Natale D, Pession A, Pillon M
Clinica Pediatrica dell'Università di, Pavia IRCCS Policlinico San Matteo, Italy.
Haematologica. 1993 Sep-Oct;78(5):264-9.
Since juvenile chronic myeloid leukemia (JCML) represents no more than 2% of leukemia in children, clinical and investigative experience of this disorder has been limited. In order to evaluate the diagnostic criteria currently applied, to provide centralized facilities for blood culture and to collect data on treatment, and to propose a uniform treatment protocol in our country, a National Registry for JCML was recently established in the "Associazione Italiana di Ematologia Oncologia Pediatrica" (AIEOP).
Out of the 24 cases reported from 9/35 centres, 22 were considered sufficiently documented and were enrolled in the Registry. Clonogenic assay on marrow and peripheral blood cells was performed in all available cases.
Common features were non-specific clinical (fever, splenomegaly, hepatomegaly, lymphadenomegaly) and hematologic alterations (anemia, thrombocytopenia, leukocytosis usually < 50 x 10(9)/l, monocytosis, circulating immature granulocytes, increased HbF, normal karyotype). In 11 out of 11 cases, in vitro blood cultures showed the spontaneous growth of CFU-C in the absence of any exogenous source of colony-stimulating activity. Twelve of the 22 patients (55%) are alive (probability of survival 47.7%); most patients were treated according to an acute myeloid leukemia-directed schedule; 5/7 children treated with interferon were alive with disease after a median time of 29 months from diagnosis (range 8-95 months); 4/5 children who underwent bone marrow transplantation were alive in complete remission 10, 24, 42 and 118 months, after the diagnosis. Age < 1 year at presentation was the most significant prognostic factor in terms of probability of survival (80% vs 28%; p = 0.0024).
JCML must be considered in young children for whom acute leukemia has been suspected but ruled out; in vitro cultures should be considered mandatory to confirm the diagnosis. Age less than one year at the presentation was associated with prolonged survival. Only bone marrow transplantation was followed by prolonged disease-free survival, whereas intensive chemotherapy seemed not very effective and potentially associated with life-threatening complications. Interferon therapy appears to be a promising alternative to chemotherapy while the value of unrelated marrow donor is explored.
由于青少年慢性粒细胞白血病（JCML）在儿童白血病中所占比例不超过2%，因此对这种疾病的临床和研究经验有限。为了评估目前应用的诊断标准，提供血液培养的集中设施并收集治疗数据，并在我国提出统一的治疗方案，最近在“意大利儿科血液肿瘤学会”（AIEOP）建立了JCML国家登记处。
在9/35个中心报告的24例病例中，22例被认为记录充分并被纳入登记处。对所有可用病例进行了骨髓和外周血细胞的克隆形成试验。
常见特征为非特异性临床（发热、脾肿大、肝肿大、淋巴结肿大）和血液学改变（贫血、血小板减少、白细胞增多通常<50×10⁹/L、单核细胞增多、循环未成熟粒细胞、HbF增加、核型正常）。在11例中的11例中，体外血液培养显示在没有任何外源性集落刺激活性来源的情况下CFU-C的自发生长。22例患者中的12例（55%）存活（生存概率47.7%）；大多数患者按照急性髓细胞白血病治疗方案进行治疗；7例接受干扰素治疗的儿童中，5例在诊断后中位时间29个月（范围8-95个月）带病存活；5例接受骨髓移植的儿童中，4例在诊断后10、24、42和118个月完全缓解存活。就诊时年龄<1岁是生存概率方面最显著的预后因素（80%对28%；p=0.0024）。
对于怀疑患有急性白血病但已排除的幼儿，必须考虑JCML；应将体外培养视为确诊的必要手段。就诊时年龄小于1岁与生存期延长相关。只有骨髓移植后实现了长期无病生存，而强化化疗似乎效果不佳且可能伴有危及生命的并发症。在探索无关骨髓供体价值的同时，干扰素治疗似乎是化疗的一个有前景的替代方案。
