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Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)


Phase 3
N/A
N/A
Not Enrolling
Both
Leukemia

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Trial Information

Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)


OBJECTIVES:

Primary

- Determine whether very low-dose cytarabine can improve event-free survival (EFS) rates
in infants with high-risk transient myeloproliferative disorder (TMD), using high-risk
TMD patients from clinical trial COG-A2971 for historic comparison, and in infants with
intermediate-risk TMD, using intermediate-risk TMD patients from clinical trial
COG-A2971 for historic comparison.

- Maintain the current high overall EFS rate in low-risk TMD patients.

Secondary

- Assess the toxicity of this regimen in these patients.

OUTLINE: This is a nonrandomized, multicenter, crossover study. Patients are stratified
according to disease risk (high or intermediate vs low).

- Group I (patients with high- or intermediate-risk transient myeloproliferative disorder
[TMD]): Patients receive very low-dose cytarabine subcutaneously twice daily on days
1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease
progression or unacceptable toxicity. Patients achieving stable disease or complete or
hepatic clinical remission undergo observation.

- Group II (patients with low-risk TMD): Patients are observed. If symptoms of
intermediate- or high-risk disease develop, patients may crossover to group I.

After completion of study treatment, patients are followed periodically for 10 years.

PROJECTED ACCRUAL: A total of 180 patients will be accrued for this study.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of transient myeloproliferative disorder (TMD)

- Diagnosis of Down syndrome or Down syndrome mosaicism (confirmed by karyotype
analysis within the past 3 weeks) AND 1 of the following:

- Nonerythroid and nonlymphoid blasts (any amount) in the peripheral blood with
verification of a second sample

- Trisomy 21-positive leukemic blasts documented by biopsy of any organ (including
> 5% nonerythroid/nonlymphoid blasts documented by bone marrow aspirate or
biopsy)

- Immunophenotype characterization required

- High-, intermediate-, or low-risk TMD, as defined by the following:

- High-risk TMD, meeting 1 of the following criteria:

- Life-threatening cardio-respiratory compromise due to complications of TMD
(e.g., organomegaly or effusions)

- Life-threatening cardio-respiratory compromise is defined as
cardiovascular grade 4 edema, grade 4 pericardial effusions, or grade
4 pleural effusions

- Hyperleukocytosis, defined as a WBC > 100,000/mm³

- Any degree of hepatomegaly (palpable on physical exam) combined with
life-threatening hepatic dysfunction

- Life-threatening hepatic dysfunction is defined as grade 4
disseminated intravascular coagulation, grade 4 ascites, grade 4
bilirubin (> 10.0 times upper limit of normal [ULN]), or grade 4 AST
or ALT (> 20.0 times ULN)

- Intermediate-risk TMD, meeting all of the following criteria:

- Hepatomegaly (palpable on physical exam) combined with nonlife-threatening
hepatic dysfunction (i.e., grade 1-3 hepatic dysfunction [AST or ALT ≤ 2.5
times ULN] and/or a total or direct bilirubin ≤ 1.5 times ULN)

- No evidence of life-threatening cardiovascular, respiratory, or hepatic
compromise due to complications of TMD

- Low-risk TMD, meeting all of the following criteria:

- No palpable hepatomegaly on physical exam OR hepatomegaly is present
without hepatic dysfunction (i.e., grade 0 hepatic dysfunction)

- No evidence of life-threatening cardiovascular, respiratory, or hepatic
compromise due to complications of TMD

PATIENT CHARACTERISTICS:

- See Disease Characteristics

- No biliary atresia by hepatic ultrasound for patients with bilirubin 3.0-10.0 times
ULN

PRIOR CONCURRENT THERAPY:

- No prior antileukemic therapy (except for leukapheresis or exchange transfusion)

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Primary Purpose: Treatment

Outcome Measure:

Event-free survival

Safety Issue:

No

Principal Investigator

April D. Sorrell, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Cancer Institute of New Jersey

Authority:

Unspecified

Study ID:

CDR0000518352

NCT ID:

NCT00411281

Start Date:

March 2006

Completion Date:

Related Keywords:

  • Leukemia
  • acute myeloid leukemia/transient myeloproliferative disorder
  • Down Syndrome
  • Leukemia
  • Myeloproliferative Disorders

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