Recommendations for Diagnosis and Treatment of Children with Transient Abnormal Myelopoiesis (TAM) and Myeloid Leukemia in Down Syndrome (ML-DS)
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Recommendations for Diagnosis and Treatment of Children with Transient Abnormal Myelopoiesis (TAM) and Myeloid Leukemia in Down Syndrome (ML-DS). / Al-Kershi, Sina; Golnik, Richard; Flasinski, Marius; Waack, Katharina; Rasche, Mareike; Creutzig, Ursula; Dworzak, Michael; Reinhardt, Dirk; Klusmann, Jan-Henning.
in: KLIN PADIATR, Jahrgang 233, Nr. 6, 11.2021, S. 267-277.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Recommendations for Diagnosis and Treatment of Children with Transient Abnormal Myelopoiesis (TAM) and Myeloid Leukemia in Down Syndrome (ML-DS)
AU - Al-Kershi, Sina
AU - Golnik, Richard
AU - Flasinski, Marius
AU - Waack, Katharina
AU - Rasche, Mareike
AU - Creutzig, Ursula
AU - Dworzak, Michael
AU - Reinhardt, Dirk
AU - Klusmann, Jan-Henning
N1 - Thieme. All rights reserved.
PY - 2021/11
Y1 - 2021/11
N2 - Children 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.
AB - Children 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.
KW - Child
KW - Down Syndrome/diagnosis
KW - GATA1 Transcription Factor/genetics
KW - Humans
KW - Leukemia, Myeloid
KW - Leukemoid Reaction/diagnosis
U2 - 10.1055/a-1532-2016
DO - 10.1055/a-1532-2016
M3 - SCORING: Journal article
C2 - 34407551
VL - 233
SP - 267
EP - 277
JO - KLIN PADIATR
JF - KLIN PADIATR
SN - 0300-8630
IS - 6
ER -
