What is the prognosis for Acute Myeloid Myelomonocytic Leukemia (AMML)?

From the Guidelines

The prognosis for Acute Myeloid Myelomonocytic Leukemia (AMML) is generally poor, with a five-year survival rate ranging from 20-40%, and treatment should be planned with a curative intent, including intensive chemotherapy regimens and possibly stem cell transplantation, as recommended by the most recent guidelines 1.

Key Factors Influencing Prognosis

• Age: younger patients fare better, with those under 60 years having a better prognosis than older patients 1
• Cytogenetic profile: certain chromosomal abnormalities like inv(16) have better outcomes, while complex aberrant karyotypes are adverse prognostic factors 1
• Molecular mutations: NPM1 mutations without FLT3-ITD are favorable, while alterations of the FLT3 gene are adverse prognostic factors 1
• Response to initial therapy: patients achieving complete remission have a better prognosis than those who do not respond to treatment 1

Treatment Approach

• Intensive chemotherapy regimens, typically cytarabine plus an anthracycline like daunorubicin or idarubicin, followed by consolidation therapy and possibly stem cell transplantation, are the standard treatment approach 1
• Allogeneic stem cell transplantation offers the best chance for cure in eligible patients with intermediate or poor-risk disease 1
• Supportive care, including blood product transfusions, infection prevention, and management of treatment complications, is essential throughout the treatment course 1

Risk Assessment

• Risk assessment in AMML includes the patient’s age, initial leukocyte count, AML subtype, karyotype data, and selected molecular markers 1
• Patients with favorable cytogenetics, such as t(15;17), t(8;21), and t(16;16), have a better prognosis than those with adverse cytogenetics 1

From the FDA Drug Label

The overall response rate (CR + PR) of 15.7% in azacitidine for injection-treated patients without AML The mean and median duration of clinical response of PR or better was estimated as 512 and 330 days, respectively; Response occurred in all MDS subtypes as well as in patients with adjudicated baseline diagnosis of AML. In the Intent-to-Treat analysis, patients treated with azacitidine demonstrated a statistically significant difference in overall survival as compared to patients treated with CCR (median survival of 24.5 months vs. 15.0 months;

The prognosis for Acute Myeloid Myelomonocytic Leukemia (AMML) is poor.

• Overall survival is approximately 24.5 months with azacitidine treatment.
• Response rates are around 15.7%.
• Duration of response is estimated to be around 512 days (mean) and 330 days (median). 2

From the Research

Prognosis for Acute Myeloid Myelomonocytic Leukemia (AMML)

• The prognosis for AMML is generally poor, with a 5-year overall survival rate of around 20-30% 3, 4.
• Relapse is a common scenario in AMML treatment, occurring in 40-50% of younger patients and the great majority of elderly patients 4.
• The prognosis in relapsed AMML patients depends largely on the timing of relapse (early versus late) and the possibility of allogeneic hematopoietic stem cell transplantation (HSCT) 4.
• Treatment options for relapsed or refractory AMML are still undefined and often unsatisfactory, but may include bridge therapy to HSCT, such as the FLAD regimen (fludarabine, cytarabine, and liposomal daunorubicin) 5.
• Targeted therapy for IDH1/2- and FLT3-mutated AMML may improve outcome, with response rates of 30-40% 4.
• Allogeneic stem cell transplantation (allo-HSCT) can improve survival in AMML patients, with a significant improvement in overall survival (OS) observed in patients who underwent allo-HSCT compared to those without subsequent allo-HSCT: 9 vs. 46 months, p < 0.0001 6.

Factors Affecting Prognosis

• Age: younger patients tend to have better outcomes than older patients 3, 4.
• Prior preleukemic syndrome: presence of a prior preleukemic syndrome is a poor prognostic factor 3.
• Sex: female sex is a favorable prognostic factor 3.
• Timing of relapse: early relapse is associated with a poorer prognosis than late relapse 4.
• Possibility of allogeneic HSCT: patients who are eligible for allogeneic HSCT tend to have better outcomes than those who are not 4.

Treatment Approaches

• Induction chemotherapy with intermediate-dosed cytarabine and subsequent allo-HSCT can result in satisfactory response rates and improved survival in AMML patients 6.
• Targeted therapy for IDH1/2- and FLT3-mutated AMML may improve outcome 4.
• Bridge therapy to HSCT, such as the FLAD regimen, may be effective in a subset of patients 5.
• Comprehensive supportive care measures, including blood product transfusions, antimicrobial treatment, and frequent monitoring for chemotherapy-related complications, are essential throughout therapy 7.