Transient abnormal myelopoiesis requiring advanced neonatal intensive care treatment.
Down syndrome
GATA-1 mutations
blasts
neonate
transient abnormal myelopoiesis
trisomy 21
Journal
Acta paediatrica (Oslo, Norway : 1992)
ISSN: 1651-2227
Titre abrégé: Acta Paediatr
Pays: Norway
ID NLM: 9205968
Informations de publication
Date de publication:
08 Feb 2024
08 Feb 2024
Historique:
revised:
09
01
2024
received:
08
11
2023
accepted:
25
01
2024
medline:
8
2
2024
pubmed:
8
2
2024
entrez:
8
2
2024
Statut:
aheadofprint
Résumé
Five to thirty percent of neonates with trisomy 21 develop transient abnormal myelopoiesis (TAM) with a high mortality rate. The aim of the study was to identify contributing factors that determine mortality and need for chemotherapy in this patient group. Six-year, single-centre, retrospective study of neonatal TAM cases requiring admission to intensive care. Data were collected from electronic patient records, laboratory and genetic results. The odds ratio was calculated to assess the likelihood of neonates with certain clinical characteristics having short-term mortality and needing chemotherapy. Twenty-one neonates were studied with a mortality rate of 28%. Neonates requiring inotropic support (OR 19, 95% CI: 0.9-399, p = 0.05) and inhaled nitric oxide (iNO) (OR 13, 95% CI: 1.4-124.3, p = 0.03) were less likely to survive to discharge. Neonates needing mechanical ventilation (OR 14, 95% CI: 1.1-185.5, p = 0.04), or a white cell count >50 × 10 A high mortality rate was identified in TAM neonates with symptomatic pulmonary hypertension (PH) needing active treatment strategies, such as inotropes and iNO. The presence of PH should be considered in the clinical management, prognosis and parental counselling.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024 Foundation Acta Paediatrica. Published by John Wiley & Sons Ltd.
Références
Zipursky A. Transient leukaemia-a benign form of leukaemia in newborn infants with trisomy 21. Br J Haematol. 2003;120(6):930-938.
Pine SR, Guo Q, Yin C, Jayabose S, Druschel CM, Sandoval C. Incidence and clinical implications of GATA1 mutations in newborns with down syndrome. Blood. 2007;110(6):2128-2131.
Klusmann JH, Creutzig U, Zimmermann M, et al. Treatment and prognostic impact of transient leukemia in neonates with down syndrome. Blood. 2008;111(6):2991-2998.
Roberts I, Alford K, Hall G, et al. GATA1-mutant clones are frequent and often unsuspected in babies with down syndrome: identification of a population at risk of leukemia. Blood. 2013;122(24):3908-3917.
Kanezaki R, Toki T, Terui K, et al. Down syndrome and GATA1 mutations in transient abnormal myeloproliferative disorder: mutation classes correlate with progression to myeloid leukemia. Blood. 2010;116(22):4631-4638.
Rainis L, Bercovich D, Strehl S, et al. Mutations in exon 2 of GATA1 are early events in megakaryocytic malignancies associated with trisomy 21. Blood. 2003;102(3):981-986.
Muramatsu H, Kato K, Watanabe N, et al. Risk factors for early death in neonates with down syndrome and transient leukaemia. Br J Haematol. 2008;142(4):610-615.
Gamis AS, Alonzo TA, Gerbing RB, et al. Natural history of transient myeloproliferative disorder clinically diagnosed in down syndrome neonates: a report from the Children's oncology group study A2971. Blood. 2011;118(26):6752-6759. quiz 996.
Tamblyn JA, Norton A, Spurgeon L, et al. Prenatal therapy in transient abnormal myelopoiesis: a systematic review. Arch Dis Child Fetal Neonatal Ed. 2016;101(1):F67-F71.
Tunstall O, Bhatnagar N, James B, et al. Guidelines for the investigation and management of transient Leukaemia of down syndrome. Br J Haematol. 2018;182(2):200-211.
NICE Guidence. https://www.nice.org.uk/guidance/cg98/chapter/Recommendations#care-of-babies-with-prolonged-jaundice
Visser IH, Hazelzet JA, Albers MJ, et al. Mortality prediction models for pediatric intensive care: comparison of overall and subgroup specific performance. Intensive Care Med. 2013;39(5):942-950.
Straney L, Clements A, Parslow RC, et al. Paediatric index of mortality 3: an updated model for predicting mortality in pediatric intensive care*. Pediatr Crit Care Med. 2013;14(7):673-681.
Pagano M GK, Mattie H. Principles of Biostatistics, CRC Press. Taylor & Francis Group. 2000.
Deeks JJHJ. Statistical algorithms in review manager 5. Statistical methods group of the cochrane collaboration. 2010.
MedCalc Statistical Software Version 19.2.6. MedCalc Software Ltd, Ostend, Belgium. 2020 https://www.medcalc.org
Massey GV, Zipursky A, Chang MN, et al. A prospective study of the natural history of transient leukemia (TL) in neonates with down syndrome (DS): Children's oncology group (COG) study POG-9481. Blood. 2006;107(12):4606-4613.
Watanabe K. Recent advances in the understanding of transient abnormal myelopoiesis in down syndrome. Pediatr Int. 2019;61(3):222-229.
Hayasaka I, Cho K, Morioka K, et al. Exchange transfusion in patients with down syndrome and severe transient leukemia. Pediatr Int. 2015;57(4):620-625.
Hoskote A, Chessells J, Pierce C. Transient abnormal myelopoiesis (TAM) causing multiple organ failure. Intensive Care Med. 2002;28(6):758-762.
Bush D, Galambos C, Dunbar ID. Pulmonary hypertension in children with down syndrome. Pediatr Pulmonol. 2021;56(3):621-629.
Yamato G, Park MJ, Sotomatsu M, et al. Clinical features of 35 down syndrome patients with transient abnormal myelopoiesis at a single institution. Int J Hematol. 2021;113(5):662-667.
Park MJ, Sotomatsu M, Ohki K, et al. Liver disease is frequently observed in down syndrome patients with transient abnormal myelopoiesis. Int J Hematol. 2014;99(2):154-161.
Shimada A, Hayashi Y, Ogasawara M, et al. Pro-inflammatory cytokinemia is frequently found in down syndrome patients with hematological disorders. Leuk Res. 2007;31(9):1199-1203.
Hattori H, Matsuzaki A, Suminoe A, Ihara K, Nakayama H, Hara T. High expression of platelet-derived growth factor and transforming growth factor-beta 1 in blast cells from patients with down syndrome suffering from transient myeloproliferative disorder and organ fibrosis. Br J Haematol. 2001;115(2):472-475.
Taub JW, Huang X, Matherly LH, et al. Expression of chromosome 21-localized genes in acute myeloid leukemia: differences between down syndrome and non-down syndrome blast cells and relationship to in vitro sensitivity to cytosine arabinoside and daunorubicin. Blood. 1999;94(4):1393-1400.
Zwaan CM, Kaspers GJ, Pieters R, et al. Different drug sensitivity profiles of acute myeloid and lymphoblastic leukemia and normal peripheral blood mononuclear cells in children with and without down syndrome. Blood. 2002;99(1):245-251.