Therapy plan for children with Down syndrome and acute myeloid leukemia in Russia

Prospective Non-randomized Multicenter Trial: the Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia

This trial will test a standardized four-course chemotherapy program for children (0–18) with AML and Down syndrome in Russia to try to improve survival and reduce treatment side effects.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 1 Day to 18 Years
• Sex: All
• Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology (other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Moscow)
• Trial ID: NCT07232134 on ClinicalTrials.gov

What this trial studies

This is a prospective, non-randomized, multicenter Phase 3 program based on the ML DS 2006 protocol designed to standardize diagnosis, treatment, and monitoring of children with AML and Down syndrome across Russia. All enrolled patients receive four chemotherapy blocks (Course 1 AIE: cytarabine/idarubicin/etoposide; Course 2 AI: cytarabine/idarubicin; Course 3 HAD: high-dose cytarabine (1 g)/daunorubicin; Course 4 HA: high-dose cytarabine) with safety monitored using CTCAE v5.0. Eligible patients are children aged 0–18 with constitutional trisomy 21 and a GATA1 mutation; transient abnormal myelopoiesis (TAM) cases may be enrolled but analyzed separately. The program aims to create a national network to harmonize care and reduce cumulative chemotherapy doses where safe.

Who should consider this trial

Good fit: Children aged 0–18 years with a diagnosis of AML or MDS who have constitutional trisomy 21 and a confirmed GATA1 mutation and whose guardians provide informed consent.

Not a fit: Children with Down syndrome and ALL, those with severe comorbidities that contraindicate the protocol, patients pretreated >14 days with intensive induction, those who refuse key therapy elements, and certain M7 AML cases without the Down phenotype are unlikely to benefit or are excluded.

Why it matters

Potential benefit: If successful, the program could increase long-term overall and event-free survival for children with AML and Down syndrome while reducing immediate and late chemotherapy toxicity.

How similar studies have performed: Previous pediatric protocols for Down syndrome–associated AML have shown that tailored, lower-intensity regimens can maintain or improve survival while lowering toxicity in several cohorts.

Eligibility criteria

Inclusion Criteria:

* Age 0-18 years
* Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)
* Signed informed consent

Exclusion Criteria:

* Children with Down syndrome and acute lymphoblastic leukemia (ALL)
* Severe comorbidities with contraindications to the treatment according to the protocol
* Pre-treatment \>14 days with intensive induction therapy
* Refusal of all therapy or important elements of therapy

Where this trial is running

Moscow

• National medical research center of pediatric haematology, oncology and immulogy named after Dmytriy Rogachyov, Moscow, 117198 — Moscow, Russia (RECRUITING)

Study contacts

• Study coordinator: Zalina A Abashidze, MD

Conditions: AML, Down Syndrome, Acute Myelogenous Leukemia, DS