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Treatment of Newly Diagnosed APL
Source: Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: March 14, 2025 · Captured 2026-09-09.
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Context: Treatment of Acute Promyelocytic Leukemia (APL)
Treatment options for newly diagnosed APL include:
All-trans retinoic acid (ATRA) plus arsenic trioxide (ATO) for low- to intermediate-risk disease.
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ATRA plus chemotherapy, followed by ATO-based consolidation therapy for high-risk disease.
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ATRA induces terminal differentiation of the leukemic cells followed by restoration of nonclonal hematopoiesis. Administration of ATRA leads to rapid resolution of coagulopathy in most patients, and heparin administration is not required in patients receiving ATRA. However, randomized trials have not shown a reduction in morbidity and mortality during ATRA induction when compared with chemotherapy. ATRA administration may result in the following conditions:
Differentiation syndrome: Administration of ATRA can lead to a syndrome of respiratory distress, known as differentiation syndrome. Prompt recognition of the syndrome and aggressive administration of steroids can prevent severe respiratory distress.[1]
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Hyperleukocytosis: The optimal management of ATRA-induced hyperleukocytosis has not been established. Hyperleukocytosis in APL is typically treated with the addition of cytotoxic chemotherapy. Leukopheresis is not recommended because of an increased risk of complications such as bleeding events and disseminated intravascular coagulation.
Studies performed in the 1990s demonstrated that overall survival (OS) rates improved in patients receiving ATRA in addition to chemotherapy.[2,3] ATO, an agent with both differentiation-inducing and apoptosis-inducing properties against APL cells, is also used in the treatment of APL. Induction remission therapy for APL is determined by disease risk. Low- to intermediate-risk APL (white blood cell [WBC] count ≤10 × 109/L) is treated without chemotherapy (ATRA and ATO), and high-risk is treated with a combination of ATRA and ATO plus chemotherapy.
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