Biology Study of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)
This study will provide pre-clinical data (i.e., cytarabine drug sensitivity testing; functional polymorphisms in drug detoxification genes; biologic targets for drug therapy) to support the development of future therapeutic trials for TMD patients. Participants in this study have been diagnosed with a condition called Transient Myeloproliferative Disorder (TMD). TMD is found in young babies (less than 3 months old) with Down syndrome and is caused by abnormal blood cells that travel throughout the body. This study will utilize a mortality risk-based classification system to characterize the distinctive biological features of each TMD risk group and correlate these findings to outcome. Evidence based treatment recommendations have been included in Appendix V of this protocol to assist physicians with clinical management decisions and to encourage uniform treatment pathways for infants with TMD.
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