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Çocuklarda Miyelodisplastik Sendrom

Year 2021, Volume: 4 Issue: 2, 75 - 80, 21.06.2021

Abstract

Miyelodisplastik sendrom (MDS); sitopeni, displazi ve inefektif eritropoyez ile karakterize klonal bir kök hücre bozukluğudur. Çocuklarda MDS erişkinlere göre oldukça nadir görülür. Miyelodisplastik sendromda en sık sitopeni ile ilişkili şikayetler görülür. Kanama, enfeksiyon ve solukluk sık bulgulardır. Tanı klinik bulgular, morfoloji, immünfenotip ve sitogenetik tetkiklerle konulur. Açıklanamayan uzun süren sitopeni, en az bir seride displazi, klonal sitogenetik anomali ve artmış blast sayısı tanı kriterleridir ve en az ikisinin olması tanıda yol göstericidir. Çocukluk çağı ve erişkin MDS arasında hem sınıflama, hem altta yatan hastalıklar hem de tedavi yaklaşımları açısından önemli farklılıklar vardır. Bu yazıda erişkin dönemde ortaya çıkan MDS ile karşılaştırılarak çocukluk çağında MDS anlatılmıştır.

References

  • 1.Baumann I, Niemeyer C, Benett J. Childhood myelodysplastic syndrome. In: Swerdlow S, Campo E, Harris N, al e, eds. WHO Classification of Tumors of Haematopoietic and Lymphoid tissues. Lyon: IARC Press; 2008:104-7.
  • 2.Hasle H, Baumann I, Bergstrasser E et al. The international prognostic scoring system (IPSS) for childhood myelodysplastic syndrome (MDS) and juvenile myelomonocytic leukemia (JMML). Leukemia 2004;18:2008-14.
  • 3.Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Sole F et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood 2012;120(12):2454-65.
  • 4.Swerdlow SH, Campo E, Harris NL, Jaffe ES, Pileri SA, Stein H et al. WHO Classification of Tumours of the Haematopoietic and Lymphoid Tissues. Lyon, France: IARC Press; 2008.
  • 5.Vardiman JW, Thiele J, Arber DA, Brunning RD, Borowitz MJ, Porwit A et al. The 2008 revision of the WHO classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood 2009;114: 937-51.
  • 6.Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM et al. The 2016 revision to the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia. Blood 2016;127:2391-405.
  • 7.Hasle H, Niemeyer CM, Chessells JM, Baumann I, Bennett JM, Kerndrup G, Head DR. A pediatric approach to the WHO classification of myelodysplastic and myeloproliferative diseases.Leukemia 2003;17(2):277-82.
  • 8.Kardos G, Baumann I, Passmore SJ et al. Refractory anemia in childhood: a retrospective analysis of 67 patients with particular reference to monosomy 7. Blood 2003;102(6):1997-2003.
  • 9.Gohring G, Michalova K, Beverloo HB et al. Complex karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood 2010;116(19):3766-69.
  • 10.Wlodarski MW, Hirabayashi S, Pastor V et al. Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents. Blood 2016;127(11):1387-97.
  • 11.Schwartz JR, Wang S, Ma J et al. Germline SAMD9 mutation in siblings with monosomy 7 and myelodysplastic syndrome. Leukemia 2017;31(8):1827-30.
  • 12.Yoshimi A, Niemeyer C, Baumann I, Schwarz-Furlan S, Schindler, Detlev S, Wolfram E, Brigitte S. High incidence of Fanconi Anemia in patients with a morphological picture consistent with refractory cytopenia of childhood. Br J Haematol 2013;160(1):109-11.
  • 13.Aalbers, AM, van den Heuvel-Eibrink MM, Baumann I et al. T-cell receptor Vβ skewing frequently occurs in refractory cytopenia of childhood and is associated with an expansion of effector cytotoxic T-cells: a prospective study by EWOG-MDS. Blood Cancer J 2014;4(5):e209.
  • 14.Aalbers AM, van der Velden VH, Yoshimi A et al. The clinical relevance of minor paroxysmal nocturnal hemoglobinuria clones in refractory cytopenia of childhood: a prospective study by EWOG-MDS. Leukemia 2014;28(1):189-92.
  • 15.Strahm B, Bader P, Bergstraesser E et al. Reduced-intensity conditioning for children with refractory cytopenia: results of the EWOG-MDS study. Bone Marrow Transplant 2010;45:S49.
  • 16.Waespe N, Van Den Akker M, Klaassen RJ et al.Response to treatment with azacitidine in children with advanced myelodysplastic syndrome prior to hematopoietic stem cell transplantation. Haematologica 2016;101(12):1508-15.
  • 17.Cseh AM, Niemeyer CM, Yoshimi A et al. Therapy with low-dose azacitidine for MDS in children and young adults: a retrospective analysis of the EWOG-MDS study group Br J Haematol 2016;172(6):930-6.
  • 18.Strahm B, Nollke P, Zecca M et al. Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study. Leukemia 2011;25(3):455-62.
  • 19.Yoshimi A, MM vdH-E, Baumann I et al. Comparison of horse and rabbit antithymocyte globulin in immunosuppressive therapy for refractory cytopenia of childhood. Haematologica 2014;99(4):656-63.

Myelodysplastic Syndrome in Children

Year 2021, Volume: 4 Issue: 2, 75 - 80, 21.06.2021

Abstract

Myelodysplastic syndrome (MDS); is a clonal stem cell disorder characterized by cytopenia, dysplasia and ineffective erythropoiesis. MDS is very rare in children compared to adults. In myelodysplastic syndrome, complaints related to cytopenia such as bleeding, infection, and pallor are the most common. Diagnosis is made through clinical findings, morphology, immunophenotype and cytogenetic methods. Unexplained prolonged cytopenia, dysplasia in at least one series, clonal cytogenetic anomaly and increased blasts are diagnostic criteria, and the presence of at least two guides diagnosis. There are important differences between childhood and adult MDS in terms of classification, underlying diseases and treatment approaches. In this article, MDS in childhood is described by comparing it with adult MDS.

References

  • 1.Baumann I, Niemeyer C, Benett J. Childhood myelodysplastic syndrome. In: Swerdlow S, Campo E, Harris N, al e, eds. WHO Classification of Tumors of Haematopoietic and Lymphoid tissues. Lyon: IARC Press; 2008:104-7.
  • 2.Hasle H, Baumann I, Bergstrasser E et al. The international prognostic scoring system (IPSS) for childhood myelodysplastic syndrome (MDS) and juvenile myelomonocytic leukemia (JMML). Leukemia 2004;18:2008-14.
  • 3.Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Sole F et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood 2012;120(12):2454-65.
  • 4.Swerdlow SH, Campo E, Harris NL, Jaffe ES, Pileri SA, Stein H et al. WHO Classification of Tumours of the Haematopoietic and Lymphoid Tissues. Lyon, France: IARC Press; 2008.
  • 5.Vardiman JW, Thiele J, Arber DA, Brunning RD, Borowitz MJ, Porwit A et al. The 2008 revision of the WHO classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood 2009;114: 937-51.
  • 6.Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM et al. The 2016 revision to the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia. Blood 2016;127:2391-405.
  • 7.Hasle H, Niemeyer CM, Chessells JM, Baumann I, Bennett JM, Kerndrup G, Head DR. A pediatric approach to the WHO classification of myelodysplastic and myeloproliferative diseases.Leukemia 2003;17(2):277-82.
  • 8.Kardos G, Baumann I, Passmore SJ et al. Refractory anemia in childhood: a retrospective analysis of 67 patients with particular reference to monosomy 7. Blood 2003;102(6):1997-2003.
  • 9.Gohring G, Michalova K, Beverloo HB et al. Complex karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood 2010;116(19):3766-69.
  • 10.Wlodarski MW, Hirabayashi S, Pastor V et al. Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents. Blood 2016;127(11):1387-97.
  • 11.Schwartz JR, Wang S, Ma J et al. Germline SAMD9 mutation in siblings with monosomy 7 and myelodysplastic syndrome. Leukemia 2017;31(8):1827-30.
  • 12.Yoshimi A, Niemeyer C, Baumann I, Schwarz-Furlan S, Schindler, Detlev S, Wolfram E, Brigitte S. High incidence of Fanconi Anemia in patients with a morphological picture consistent with refractory cytopenia of childhood. Br J Haematol 2013;160(1):109-11.
  • 13.Aalbers, AM, van den Heuvel-Eibrink MM, Baumann I et al. T-cell receptor Vβ skewing frequently occurs in refractory cytopenia of childhood and is associated with an expansion of effector cytotoxic T-cells: a prospective study by EWOG-MDS. Blood Cancer J 2014;4(5):e209.
  • 14.Aalbers AM, van der Velden VH, Yoshimi A et al. The clinical relevance of minor paroxysmal nocturnal hemoglobinuria clones in refractory cytopenia of childhood: a prospective study by EWOG-MDS. Leukemia 2014;28(1):189-92.
  • 15.Strahm B, Bader P, Bergstraesser E et al. Reduced-intensity conditioning for children with refractory cytopenia: results of the EWOG-MDS study. Bone Marrow Transplant 2010;45:S49.
  • 16.Waespe N, Van Den Akker M, Klaassen RJ et al.Response to treatment with azacitidine in children with advanced myelodysplastic syndrome prior to hematopoietic stem cell transplantation. Haematologica 2016;101(12):1508-15.
  • 17.Cseh AM, Niemeyer CM, Yoshimi A et al. Therapy with low-dose azacitidine for MDS in children and young adults: a retrospective analysis of the EWOG-MDS study group Br J Haematol 2016;172(6):930-6.
  • 18.Strahm B, Nollke P, Zecca M et al. Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study. Leukemia 2011;25(3):455-62.
  • 19.Yoshimi A, MM vdH-E, Baumann I et al. Comparison of horse and rabbit antithymocyte globulin in immunosuppressive therapy for refractory cytopenia of childhood. Haematologica 2014;99(4):656-63.
There are 19 citations in total.

Details

Primary Language Turkish
Subjects Clinical Sciences
Journal Section Review
Authors

Gül Özdemir 0000-0002-3204-4353

Publication Date June 21, 2021
Submission Date March 10, 2020
Published in Issue Year 2021 Volume: 4 Issue: 2

Cite

MLA Özdemir, Gül. “Çocuklarda Miyelodisplastik Sendrom”. Sağlık Bilimlerinde İleri Araştırmalar Dergisi, vol. 4, no. 2, 2021, pp. 75-80.