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ATRA and or Chemo as maintenance
ATRA (all-trans retinoic acid) induces differentiation of leukemic promyelocytes and promotes their maturation, often combined with chemotherapy to achieve complete remission in acute promyelocytic leukemia.
ATRA (all-trans retinoic acid) induces differentiation of leukemic promyelocytes and promotes their maturation, often combined with chemotherapy to achieve complete remission in acute promyelocytic leukemia. Used for Acute promyelocytic leukemia (APL) — maintenance therapy.
At a glance
| Generic name | ATRA and or Chemo as maintenance |
|---|---|
| Also known as | ATRA |
| Sponsor | Groupe d'etude et de travail sur les leucemies aigues promyelocytaires |
| Drug class | Retinoid; differentiation agent |
| Target | Retinoic acid receptor alpha (RARα) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
ATRA binds to retinoic acid receptors (RARα) and causes degradation of the PML-RARA fusion protein characteristic of acute promyelocytic leukemia (APL), forcing leukemic cells to differentiate into mature granulocytes rather than proliferate. When combined with chemotherapy as maintenance therapy, this approach reduces relapse risk by eliminating residual disease and maintaining long-term disease control in APL patients.
Approved indications
- Acute promyelocytic leukemia (APL) — maintenance therapy
Common side effects
- Retinoid acid syndrome (RAS)
- Hepatotoxicity
- Hypertriglyceridemia
- Headache
- Dry skin and mucous membranes
- Bone pain
Key clinical trials
- A Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma (PHASE2)
- Chemotherapy and Radiation Therapy in Treating Young Patients With Newly Diagnosed, Previously Untreated, High-Risk Medulloblastoma/PNET (PHASE3)
- High Dose Chemotherapy and Autologous Transplant for Neuroblastoma (NA)
- S0521, Combination Chemotherapy With or Without Gemtuzumab Followed By Tretinoin, Mercaptopurine, and Methotrexate or Observation in Treating Patients With Acute Promyelocytic Leukemia (PHASE3)
- Phase III Trial in Acute Promyelocytic Leukemia Patients (PHASE3)
- Combined Retinoic Acid,Arsenic Trioxide and Chemo for Newly-diagnosed APL (PHASE4)
- ALFA-0703 Study in Older Patients With Acute Myeloblastic Leukemia (AML) (PHASE3)
- Tretinoin, Cytarabine, and Daunorubicin Hydrochloride With or Without Arsenic Trioxide Followed by Tretinoin With or Without Mercaptopurine and Methotrexate in Treating Patients With Acute Promyelocytic Leukemia (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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