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

Author:

Al-Kershi Sina12,Golnik Richard1ORCID,Flasinski Marius3,Waack Katharina4,Rasche Mareike4,Creutzig Ursula5,Dworzak Michael6,Reinhardt Dirk4,Klusmann Jan-Henning1ORCID

Affiliation:

1. Clinic for Pediatrics, University Hospital Frankfurt, Frankfurt, Germany

2. Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

3. Department of Psychiatry, Psychosomatic Medicine and Psychotherapy, Hospital Tauberbischofsheim, Tauberbischofsheim, Germany

4. Pediatrics III, Pediatric Hematology and Oncology, University Hospital Essen, Essen, Germany

5. Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany

6. Department of Pediatrics, St. Anna Children’s Hospital and Children’s Cancer Research Institute, Wien, Austria

Abstract

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.

Publisher

Georg Thieme Verlag KG

Subject

Pediatrics, Perinatology and Child Health

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