Summary
Seven children with a primary myelodysplastic syndrome were seen at our center over a 9-year period. Two presented with refractory anemia, three with refractory anemia with excess of blasts, and two with refractory anemia with excess of blasts in transformation. All children received supportive therapy, including blood transfusions in five of them. Three patients developed acute myeloid leukemia and were treated with intensive chemotherapy, followed by allogeneic or autologous marrow transplantation in the two responders. All three died of either infection or progressive disease. The other four patients are still alive a median of 71 months (range 38–130) after diagnosis. These results confirm the difficulties in managing patients with myelodysplastic syndromes.
Similar content being viewed by others
References
Amadori S, Arcese W, Isacchi G, Meloni G, Petti MC, Monarca B, Testi AM, Mandelli F (1991) Mitoxantrone, Etoposide, and intermediate-dose Cytarabine: an effective and tolerable regimen for the treatment of refractory acute myeloid leukemia. J Clin Oncol 9:1210–1214
Amadori S, Ceci A, Comelli A, Madon E, Masera G, Nespoli L, Paolucci G, Zanesco L, Covelli A, Mandelli F (1987) Treatment of acute myelogenous leukemia in children: Results of the Italian cooperative study AIEOP/LAM 8204. J Clin Oncol 5:1356–1363
Anderson RL, Bagby GC (1982) The prognostic value of chromosome studies in patients with preleukemic syndrome (Hemopoietic dysplasia). Leukemia Res 6:175–181
Appelbaum FR, Barrall J, Storb R, Fisher LD, Schoch G, Ramberg RE, Shulman H, Anasetti C, Bearman SI, Beatty P, Bensinger WI, Buckner CD, Clift RA, Hansen JA, Martin P, Petersen FB, Sanders JE, Singer J, Stewart P, Sullivan KM, Witherspoon RP, Thomas ED (1990) Bone marrow transplantation for patients with myelodysplasia. Pretreatment variables and outcome. Ann Intern Med 112:590–597
Bennet JM, Catovsky D, Daniel MT, Flandrin G, Galton DAG, Gralnick HR, Sultan C, The French-American-British (FAB) Co-operative Group (1982) Proposals for classification of the Myelodysplastic Syndromes. Br J Haematol 51:189–199
Blank J, Lange B (1981) Preleukemia in children. J Pediatr 98:565–568
Brandwein JM, Horsman DE, Eaves AC, Eaves CJ, Massing BG, Wadsworth LD, Rogers PCJ, Kalousek DK (1990) Childhood myelodysplasia: suggested classification as myelodysplastic syndromes based on laboratory and clinical findings. The American Journal of Pediatric Hematology/Oncology 12:63–70
Cantù-Rajnoldi A, Porcelli P, Cattoretti E, Ferrari M, Romitti L, Adamoli L, Masera G (1984) Myelodysplastic syndromes in children: observation on five cases. Eur Pediatr Haematol/ Oncol 1:71–75
Cheson BD (1990) The myelodysplastic syndrome: Current approaches to therapy. Ann Intern Med 112:932–941
Chessels JM (1991) Myelodysplasia. Baillierres Clin Haematol 4:459–482
Creutzig U, Cantù-Rajnoldi A, Ritter J, Romitti L, Odenwald E, Conter V, Riehm H, Masera G (1987) Myelodysplastic syndromes in childhood. Report of 21 patients from Italy and West Germany. Am J Pediatr Hematol/Oncol 9:324–330
De Witte T, Zwaan F, Hermans J, Vernant J, Kolb H, Vossen J, Lonnquvist B, Beelen D, Ferrant A, Gmur J, Liu Yin X, Troussard J, Cahn J, Van Lint M, Gratwohl A (1990) Allogenic bone marrow transplantation for secondary leukaemia and myelodysplastic syndrome: A survey by the Leukaemia Working Party of the European Bone Marrow Transplantation Group (EBMTG). Br J Haematol 74:151–155
Fenaux P, Morel P, Rose C, Lai JL, Jouet JP, Bauters F (1991) Prognostic factors in adult “de novo” myelodysplastic syndromes treated by intensive chemotherapy. Br J Haematol 77:497–501
Fenaux P, Preudhomme C, Estienne MH, Morel P, Lai JL, Gardin C, Jouet JP, Bauters F (1990) De novo myelodysplastic syndromes in adults aged 50 or less. A report on 37 cases. Leukemia Res 14:1053–1059
Gadner H, Haas OA (1992) Experience in pediatric myelodysplastic syndromes. Hematol/Oncol Clin North Am 6:655–672
Greenberg P (1986) In vitro culture techniques defining biological abnormalities in the myelodysplastic syndromes and myeloproliferative disorders. Clin Haematol 15:973–993
Guinan EC, Tarbell JN, Tantravahi R, Weinstein HJ (1989) Bone marrow transplantation of children with myelodysplastic syndromes. Blood 73:619–622
Hamblin TJ, Oscier DG (1987) The myelodysplastic syndrome. A practical guide. Hematol Oncol 5:19–23
ISCN (1970) An international system for human cytogenetic nomenclature. Cytogenet Cell Genet 21:309–404
Jacobs RH, Cornbleet MA, Vardiman JW, Larson RA, Le Beau MM, Rowley JD (1986) Prognostic implications of morfology and karyotype in primary myelodysplastic syndrome. Blood 6:1765–1772
Kerkhofs H, Hermans J, Haak HL, Leeksma CHW (1987) Utility of the FAB classification for myelodysplastic syndromes: Investigation of prognostic factors in 237 cases. Br J Haematol 65:73–81
Kleihauer E (1980) The preleukemic syndrome (hematopoietic dysplasia) in childhood. Eur J Pediatr 133:5–10
Kobrinsky NL, Nesbit ME, Ramsay NKC, Arthur DC, Krivit W, Brunning RD (1982) Hematopoietic dysplasia and marrow hypocellularity in children: A preleukemic condition. J Pediatr 100:907–913
Koeffler HP (1986) Myelodysplastic Syndromes (Preleukemia). Semin Hematol 23:284–299
Mufti GJ, Stevens JR, Oscier DG, Hamblin TJ, Machin D (1985) Myelodysplastic syndromes: a scoring system with prognostic significance. Br J Haematol 59:425–433
Pike BL, Robinson WA (1970) Human bone marrow colony growth in agar gel. J Cell Physiol 76:77–84
Tennat GB, Bowen DT, Jacobs A (1991) Colony-cluster ratio and cluster number in cultures of circulating myeloid progenitors as indicators of high-risk myelodysplasia. Br J Haematol 77:296–300
Tricot G, Boogaerts MA, De Wolf-Peeters C, Van Der Berghe H, Verwilghen RL (1985) The myelodysplastic syndromes: different evolution patterns based on sequential morphological and cytogenetic investigations. Br J Haematol 59:659–670
Tricot G, Vlietinck R, Boogaerts MA, Hendrickx B, De Wolf-Peeters C, Van Den Berghe H, Verwilghen RL (1985) Prognostic factors in the myelodysplastic syndromes: importance of initial data on peripheral blood counts, bone marrow cytology, trephine biopsy and chromosomal analysis. Br J Haematol 60:19–32
Tucker MA, Meadows AT, Bolce Jr JD, Stovall M, Oberlin O, Stone BJ, Birch J, Voute PA, Hoover RN, Fraumeni Jr JF (1987) Leukemia after therapy with alkylating agents for children cancer. J Natl Cancer Inst 78:459–464
Uderzo C, Locasciulli A, Cantù-Rajnoldi A, Mozzana R, Lambertenghi-Deliliers G, Masera G (1993) Allogenic bone marrow transplantation for myelodysplastic syndromes of childhood: Report of three children with refractory anemia with excess of blasts in transformation and review of the literature. Med Pediatr Oncol 21:43–48
Van Wering ER, Kamps WA, Vosse JM, Van der List-Nuver CJA, Theunissen PMV (1985) Myelodysplastic syndromes in childhood: three case reports. Br J Haematol 60:137–142
Wagelius R (1986) Preleukemic states in children. Scand J Haematol 36:133–139
Yoshida Y (1987) Biology of myelodysplastic syndrome. Int J Cell Cloning 5:356–375
Yunis JJ, Rydell RE, Oken MM, Aenesen MA, Mayer MG, Lobell M (1986) Refined chromosome analysis as an independent prognostic indicator in de novo myelodysplastic syndromes. Blood 67:1721–1730
Author information
Authors and Affiliations
Additional information
Supported by CNR — Progetto Finalizzato A.C.R.O. — Contract no. 9202125.PF39
Rights and permissions
About this article
Cite this article
Vitale, A., Testi, A.M., Moleti, M.L. et al. Myelodysplastic syndromes in childhood: description of seven cases. Ann Hematol 68, 241–245 (1994). https://doi.org/10.1007/BF01737424
Received:
Accepted:
Issue Date:
DOI: https://doi.org/10.1007/BF01737424