Myelodysplastic syndromes in childhood. Report of 21 patients from Italy and West Germany.

Nine Italian and 12 German children fulfilled the criteria of myelodysplastic syndromes (MDS), according to the French-American-British (FAB) classification. All patients belonged to the more aggressive subtypes of myelodysplastic syndromes. Four presented with refractory anemia with excess of blasts (RAEB), 16 presented with refractory anemia with excess of blasts in transformation (RAEB-T), and 1 had chronic myelomonocytic leukemia (CMML). Dyserythropoiesis and dysgranulopoiesis were seen in all patients, and dysmegakaryopoiesis was seen in most patients. Cytogenetic studies in 13 of the 21 children showed karyotype abnormalities in 8; 5 had monosomy 7. Eleven patients were treated with intensive chemotherapy soon after diagnosis; 6 achieved complete remission (CR), and 2 of them are alive and still in complete remission after 48 and 69 months. Low-dose cytosine arabinoside (Ara-C) was given in six children without improvement. Bone marrow transplantation after progression of the disease has produced complete remission lasting 28 + months now in one of two patients. Four patients received only symptomatic treatment. The rate of 5-year survival for the total group was 20% (SD 9%). We conclude that children with MDS may benefit from more aggressive treatment, but that in general the survival rate is poor and similar to that observed in adults with the same subtypes of this disease.