scholarly journals Low-dose cytarabine to prevent myeloid leukemia in children with Down syndrome: TMD Prevention 2007 study

2018 ◽  
Vol 2 (13) ◽  
pp. 1532-1540 ◽  
Author(s):  
Marius Flasinski ◽  
Kira Scheibke ◽  
Martin Zimmermann ◽  
Ursula Creutzig ◽  
Katarina Reinhardt ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Myeloid Leukemia ◽  
Low Dose ◽  
Historical Control ◽  
Children With Down Syndrome ◽  
Key Points ◽  
Low Dose Cytarabine ◽  
Leukemia In Children

Key Points Low-dose cytarabine treatment reduced mortality in symptomatic TMD patients compared with the historical control. An MRD monitoring–based low-dose cytarabine treatment does not prevent progression from preleukemic TMD to ML-DS.

scholarly journals Therapy reduction in patients with Down syndrome and myeloid leukemia: the international ML-DS 2006 trial

Blood ◽  
2017 ◽  
Vol 129 (25) ◽  
pp. 3314-3321 ◽  
Author(s):  
Madita Uffmann ◽  
Mareike Rasche ◽  
Martin Zimmermann ◽  
Christine von Neuhoff ◽  
Ursula Creutzig ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Prognostic Factors ◽  
Treatment Response ◽  
Early Treatment ◽  
Myeloid Leukemia ◽  
Chromosome 8 ◽  
Historical Control ◽  
Early Treatment Response ◽  
Key Points ◽  
Therapy Reduction

Key Points Reducing therapy intensity in the ML-DS 2006 trial did not impair the excellent prognosis in ML-DS compared with the historical control. Early treatment response and gain of chromosome 8 are independent prognostic factors.

Recommendations for Diagnosis and Treatment of Children with Transient Abnormal Myelopoiesis (TAM) and Myeloid Leukemia in Down Syndrome (ML-DS)

2021 ◽  
Author(s):  
Sina Al-Kershi ◽  
Richard Golnik ◽  
Marius Flasinski ◽  
Katharina Waack ◽  
Mareike Rasche ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Myeloid Leukemia ◽  
Low Dose ◽  
Early Death ◽  
Spontaneous Remission ◽  
Therapeutic Strategies ◽  
Treatment Protocols ◽  
Transient Abnormal Myelopoiesis ◽  
Cure Rates ◽  
Low Dose Cytarabine

AbstractChildren with Down syndrome are at a high risk of developing transient abnormal myelopoiesis (TAM; synonym: TMD) or myeloid leukemia (ML-DS). While most patients with TAM are asymptomatic and go into spontaneous remission without a need for therapy, around 20% of patients die within the first six months due to TAM-related complications. Another 20–30% of patients progress from TAM to ML-DS. ML-DS patients are particularly vulnerable to therapy-associated toxicity, but the prognosis of relapsed ML-DS is extremely poor – thus, ML-DS therapy schemata must strive for a balance between appropriate efficacy (to avoid relapses) and treatment-related toxicity. This guideline presents diagnostic and therapeutic strategies for TAM and ML-DS based on the experience and results of previous clinical studies from the BFM working group, which have helped reduce the risk of early death in symptomatic TAM patients using low-dose cytarabine, and which have achieved excellent cure rates for ML-DS using intensity-reduced treatment protocols.

scholarly journals Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome

Cancer Cell ◽  
2019 ◽  
Vol 36 (2) ◽  
pp. 123-138.e10 ◽  
Author(s):  
Maurice Labuhn ◽  
Kelly Perkins ◽  
Sören Matzk ◽  
Leila Varghese ◽  
Catherine Garnett ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Myeloid Leukemia ◽  
Children With Down Syndrome ◽  
Leukemia In Children

scholarly journals Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome

Cancer Cell ◽  
2019 ◽  
Vol 36 (3) ◽  
pp. 340 ◽  
Author(s):  
Maurice Labuhn ◽  
Kelly Perkins ◽  
Sören Matzk ◽  
Leila Varghese ◽  
Catherine Garnett ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Myeloid Leukemia ◽  
Children With Down Syndrome ◽  
Leukemia In Children

scholarly journals Acute lymphoblastic leukemia in children with Down syndrome: a retrospective analysis from the Ponte di Legno study group

Blood ◽  
2014 ◽  
Vol 123 (1) ◽  
pp. 70-77 ◽  
Author(s):  
Trudy D. Buitenkamp ◽  
Shai Izraeli ◽  
Martin Zimmermann ◽  
Erik Forestier ◽  
Nyla A. Heerema ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Acute Lymphoblastic Leukemia ◽  
Lymphoblastic Leukemia ◽  
Good Prognosis ◽  
Study Group ◽  
Children With Down Syndrome ◽  
Key Points ◽  
Leukemia In Children ◽  
Treatment Related Mortality ◽  
Related Mortality

Key Points Although the risk of ALL relapse is significantly higher in children with DS, good-prognosis subgroups have been identified. Patients with DS-ALL have higher treatment-related mortality throughout the treatment period independent of the therapeutic regimen.

scholarly journals Germline variants in predisposition genes in children with Down syndrome and acute lymphoblastic leukemia

2020 ◽  
Vol 4 (4) ◽  
pp. 672-675 ◽  
Author(s):  
Peleg Winer ◽  
Ivo S. Muskens ◽  
Kyle M. Walsh ◽  
Ajay Vora ◽  
Anthony V. Moorman ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Acute Lymphoblastic Leukemia ◽  
Lymphoblastic Leukemia ◽  
Germline Variants ◽  
Children With Down Syndrome ◽  
Key Points ◽  
Predisposition Genes ◽  
Leukemia In Children

Key Points Rare and pathogenic germline variants, including in IKZF1, contribute to acute lymphoblastic leukemia in children with Down syndrome.

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