Trial Information

Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood

OBJECTIVES:

- Determine, by a standard approach, the frequency of different FAB subtypes in children
with primary myelodysplastic syndromes.

- Determine the frequency of cytogenetic and molecular abnormalities in these patients.

- Determine the survival of patients treated with allogeneic stem cell transplantation
with or without induction chemotherapy.

- Determine the rate of complete remission in patients treated with these regimens.

- Determine the event-free survival of patients treated with these regimens.

- Determine the relapse rate, morbidity, and mortality of patients treated with these
regimens.

- Determine different subsets of patients who benefit from these regimens.

OUTLINE: This is a multicenter study. Patients are stratified according to FAB subtype
(refractory anemia (RA) or RA with ringed sideroblasts (RARS) vs RA with excess blasts
(RAEB) vs RAEB in transformation (RAEB-t) vs juvenile myelomonocytic leukemia (JMML)).

Patients undergo complete medical and physical examination. Patients are screened for the
following aberrations: -7, +8, +21, t(8;21), t(15;17), and inv(16). Smears of peripheral
blood and bone marrow, as well as bone marrow biopsies and all cytogenetic and molecular
studies performed on blood or bone marrow, are evaluated by a panel of international
experts.

Patients with progressive RA or RARS undergo allogeneic stem cell transplantation (ASCT)
according to EWOG-MDS SCT studies. Patients with stable RA or RARS wait for an optimal donor
before undergoing ASCT. Patients with RAEB with fewer than 15% bone marrow blasts undergo
ASCT. Patients with RAEB with at least 15% bone marrow blasts and patients with RAEB-t with
fewer than 30% bone marrow blasts receive standard acute myeloid leukemia (AML) induction
therapy and then undergo ASCT. Patients with RAEB-t with at least 30% bone marrow blasts are
considered for standard AML induction therapy.

Patients with advanced JMML undergo evaluation for splenectomy and receive chemotherapy with
mercaptopurine and cytarabine every 3-4 weeks (for 1-4 doses). Patients then undergo ASCT.

Patients are followed every 6 months.

PROJECTED ACCRUAL: Not specified