AAML08B1, Biology Study of Transient Myeloproliferative Disorder (TMD) in Children with Down Syndrome (DS)

Study ID
STU 102011-025

Cancer Related
Yes

Healthy Volunteers
No

Study Sites

Contact
Beverly Kleiber
214-456-6484
beverly.kleiber@childrens.com

Principal Investigator
Naomi Winick

Summary

Blood samples will be collected from eligible and consenting participants at the
following time points:
Time point and amount of blood
at the time of TMD diagnosis- 2 teaspoons (10 mL)
When subject's liver blood tests show that the liver is working normally- 1/5 of a teaspoon (1 mL)
When subject's TMD gets better- 2 teaspoons (10 mL)
if TMD comes back- 2 teaspoons (10 mL)
During Year 1 after the TMD diagnosis x every month 1 teaspoons (5 mL)
During Year 2 after the TMD diagnosis x every 6 months 1 teaspoons (5 mL)
During Year 3 after the TMD diagnosis x every 6 months 1 teaspoons (5 mL)
During Year 4 after the TMD diagnosis x once 1 teaspoons (5 mL)
During Year 5 after the TMD diagnosis x once 1 teaspoons (5 mL)

The samples will be labeled with a CoG number and sent to CoG-designated
laboratories for research tests for Dna/Rna, liver proteins, and MRD tests. For
those participants who volunteer for the optional specimen banking, the
remainder of any blood specimens will be stored in a CoG specimen bank for
future research.

Participant Eligibility

Age - Patient is < 90 days of age at diagnosis of TMD.
Diagnosis - Patient has a diagnosis of Transient Myeloproliferative Disorder (TMD) defined as:
1. A diagnosis of Down syndrome or Down syndrome mosaicism. Patients may be enrolled On Study prior to cytogenetic or FISH confirmation of the diagnosis if the typical physical characteristics of Down syndrome are present. However confirmation of diagnosis must occur within 21 days of enrollment. AND
2. Non-erythroid and non-lymphoid blasts (any amount) in the peripheral blood verified with a second sample.
-ORTrisomy 21 positive leukemic blasts documented by biopsy of any organ (including > 5% nonerythroid/ non-lymphoid blasts documented by bone marrow aspirate or biopsy). Infants with isolated trisomy 21 positivity identified only in the leukemic blasts are eligible for study.
Institutional immunophenotype characterization is required for study enrollment.
Timing - Study enrollment must take place within 21 days following the diagnosis of TMD by a pediatric hematology/oncology specialist.
Regulatory - All patients and/or their parents or legal guardians must sign a written informed consent.
All institutional, FDA, and NCI requirements for human studies must be met.