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Phases of Therapy
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 Option Overview for AML
The treatment of patients with acute myeloid leukemia (AML) is based on whether the disease is newly diagnosed (previously untreated), in remission, or recurrent. Also, the intensity of the treatment and the patient's overall health status are considered when choosing a treatment approach. Successful treatment of AML requires the control of bone marrow and systemic disease, and specific treatment of central nervous system (CNS) disease, if present. The cornerstone of this strategy includes systemically administered combination chemotherapy. Because only 5% or fewer of patients with AML develop CNS disease, prophylactic treatment is not indicated.[1,2]
Newly diagnosed (untreated): Untreated AML is defined as newly diagnosed leukemia that has not been previously treated. The initial treatment for patients with newly diagnosed AML is often induction therapy that aims to induce a remission. In patients with AML, a complete remission (CR) is defined as a normal peripheral blood cell count (absolute neutrophil count >1,000/mm3 and platelet count >100,000/mm3) and normocellular marrow with less than 5% blasts in the marrow and no signs or symptoms of the disease. In addition, no signs or symptoms are evident of CNS leukemia or other extramedullary infiltration.[3] Modifications to the definition of CR have been proposed because some responses are deeper than a CR, and others may not meet all the criteria for a complete response. In addition, most AML patients meeting the criteria for CR have residual leukemia.[3]
| Response Category | Definition |
|---|---|
| ANC = absolute neutrophil count; CR = complete remission; CRh = CR with partial hematologic recovery; CRi = CR with incomplete hematologic recovery; CRMRD− = CR without measurable residual disease; MLFS = morphological leukemia-free state; PR = partial remission; RT–qPCR = reverse transcription–quantitative polymerase chain reaction. | |
| CRMRD− | If studied pretreatment, CR with negativity for a genetic marker by RT–qPCR, or CR with negativity by multicolor flow cytometry. |
| CR | Bone marrow blasts <5%; absence of circulating blasts and blasts with Auer rods; absence of extramedullary disease; ANC ≥1.0 × 109/L (1,000/microL); platelet count ≥100 × 109/L (100,000/microL). |
| CRh | ANC ≥0.5 x 109/L (500/microL) and platelet count ≥50 x 109/L (100,000/microL); otherwise all other CR criteria met.[4] |
| CRi | All CR criteria except for residual neutropenia (<1.0 × 109/L [1,000/microL]) or thrombocytopenia (<100 × 109/L [100,000/microL]). |
| MLFS | Bone marrow blasts <5%; absence of blasts with Auer rods; absence of extramedullary disease; no hematologic recovery required. |
| PR | All hematologic criteria of CR; decrease of bone marrow blast percentage to 5 to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50%. |
| No response | Patients evaluable for response but not meeting criteria for CR, CRh, CRi, MLFS, or PR are categorized as having no response prior to the response landmark. Patients failing to achieve response by the designated landmark are designated as having refractory disease. |
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In remission: When patients are in a remission after induction chemotherapy, consolidation chemotherapy is given, with the aim of deepening the response and consolidating the remission. Maintenance therapy is not included in most current treatment protocols and clinical trials. Consolidation therapy appears to be effective when given immediately after remission is achieved.[5]
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Persistent/recurrent disease: Despite intensive chemotherapy, some patients with newly diagnosed AML will not go into remission and have primary refractory disease. Also, some patients who are in a remission after induction and consolidation chemotherapy may have a return of their disease.[3] The rates of primary refractory disease and relapse vary with the age of the patient, genomic variants seen in the leukemia cells, and initial treatment given.
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| Response Category | Definition | |
|---|---|---|
| CR = complete remission; CRh = CR with partial hematologic recovery; CRhMRD-LL = CR with partial hematologic recovery and measurable residual disease at a low level; CRi = CR with incomplete hematologic recovery; CRiMRD-LL = CR with incomplete hematologic recovery and measurable residual disease at a low level; CRMRD- = CR without measurable residual disease; CRMRD-LL = CR with measurable residual disease detection at a low level; MRD = measurable residual disease; MRD- = absence of measurable residual disease; MRD+ = presence of measurable residual disease; MLFS = morphological leukemia-free state; PR = partial response; RT–qPCR = reverse transcription–quantitative polymerase chain reaction. | ||
| Primary refractory disease | No CR, CRh, or CRi at the response landmark (i.e., after two courses of intensive induction treatment) or a defined landmark (e.g., 180 days after commencing less-intensive therapy). | |
| Relapsed disease (after CR, CRh, or CRi) | Bone marrow blasts ≥5%; or reappearance of blasts in the blood in at least two peripheral blood samples obtained at least 1 week apart; or development of extramedullary disease. | |
| MRD relapse (after CR, CRh, or CRi without MRD-) | Defined by one of the following: | |
| Conversion from MRD- to MRD+, independent of method; or | ||
| Increase of MRD copy numbers ≥1 log10 between any two positive samples in patients with CRMRD-LL, CRhMRD-LL, or CRiMRD-LL by qPCR. | ||
| Either result should be rapidly confirmed in a second consecutive sample from the same tissue source. | ||
| Stable disease | Absence of CRMRD-, CR, CRi, PR, MLFS; and criteria for progressive disease not met. | |
| Progressive disease | Evidence for an increase in bone marrow blast percentage and/or increase of absolute blast counts in the blood: | |
| >50% increase in marrow blasts; or | ||
| >50% increase in peripheral blasts in the absence of differentiation syndrome; or | ||
| New extramedullary disease. | ||
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