What is the clinical difference between cytarabine high dose days 1,2 and 3 and days 1,3, and 5 for AML consolidation?

Comment by InpharmD Researcher

The clinical difference between the HiDAC-123 and HiDAC-135 schedules appears more focused on toxicity and resource use. The regimens demonstrate equivalent anti-leukemic efficacy and survival. However, the condensed HiDAC-123 schedule is consistently associated with faster hematologic recovery and reduced transfusion requirements, leading to its recommendation as the preferred regimen for consolidation therapy.

Background

According to the latest National Comprehensive Cancer Network (NCCN) guidelines for acute myeloid leukemia (AML), either the 1, 3, and 5 day of high-dose cytarabine (HIDAC-135) or 1, 2, and 3 day of HIDAC (HIDAC-123) are recommended dosing regimens. Only one study was referenced, suggesting no difference in hematologic toxicity or survival between the two regimens in adult patients with AML (see Table 1). [1]

One review article, published in 2021, examined the optimal dosing of cytarabine during post-remission therapy for AML. Cytarabine dosing is generally categorized as standard (100–200 mg/m²), intermediate (400-1500 mg/m²), or high (>2000 mg/m²). Early trials comparing these doses were primarily conducted in the post-remission (consolidation) setting following standard 7+3 induction therapy. Notably, the Cancer and Leukemia Group B (CALGB)/Alliance trial from 1994 popularized the HiDAC-135 schedule (3 g/m² twice daily on days 1, 3, and 5); however, subsequent analyses from German and French studies demonstrated that the alternative HiDAC-123 schedule (3 g/m² twice daily on days 1, 2, and 3) provides equivalent anti-leukemia efficacy. HiDAC-123 was associated with faster hematologic recovery, fewer infections, reduced transfusion requirements, and shorter hospital stays, with no difference in relapse-free or overall survival compared with HiDAC-135. Although these results are largely retrospective, the review concluded that HiDAC-123 should be considered the preferred regimen for administering higher-dose cytarabine in post-remission therapy following standard induction. [2], [3], [4], [5]

A 2023 presentation abstract describes a study which evaluated a condensed, three-day schedule (AC123) for administering cytarabine consolidation therapy in Acute Myeloid Leukemia (AML) and compared it to the standard five-day schedule (AC135). While the condensed schedule's efficacy had been established in patients under 60, this research specifically investigated its use in older adults. The findings demonstrated that the AC123 regimen was safe and resulted in a statistically significant faster recovery of neutrophils and platelets (p<0.05) for patients both over and under 60 years of age, without an increase in complications such as hospitalization rates (p= 0.1), fever rate (p= 0.3), infection (p= 0.2), bacteremia (p= 0.4) and bleeding requiring intervention (p= 0.4). Based on these results, the authors concluded that the condensed three-day schedule should be considered the preferred method for consolidation therapy administration. [6]

A 2022 abstract described a retrospective and prospective study comparing HiDAC-135 (days 1, 3, and 5) and HiDAC-123 (days 1, 2, and 3) used as consolidation therapy for adults with acute myeloid leukemia (AML). The study aimed to evaluate whether HiDAC-123 could shorten hospitalization and reduce transfusion needs. Patients aged 18 years or older (excluding those with acute promyelocytic leukemia) were included, with historical data analyzed for HiDAC-135 and prospective data collected for HiDAC-123. Between January 2020 and May 2022, 30 HiDAC-135 and 28 HiDAC-123 cycles were administered. Baseline characteristics, including cytogenetic and molecular profiles, were similar between groups. The median hospital stay was 5 days for HiDAC-123 and 7 days for HiDAC-135, a difference that was not statistically significant. The proportion of patients hospitalized for ≤10 days was higher with HiDAC-123 (86% vs. 70%), but again not statistically significant. Readmission rates and causes, mainly thrombocytopenia, febrile neutropenia, and anemia, were comparable between groups. Notably, transfusion requirements during elective admission were significantly lower with HiDAC-123 (3.6% vs. 20%; p= 0.05), though transfusion rates during readmission did not differ. The authors concluded that HiDAC-123 may modestly reduce transfusion needs during elective admissions but does not significantly affect readmission rates, hospitalization duration, or cytopenia incidence. They also noted that the small sample size limited statistical power and that larger studies are warranted to confirm these findings. [7]

References:

[1] National Comprehensive Cancer Network (NCCN). Acute Myeloid Leukemia Version 2.2026. Updated October 2, 2025. Accessed October 27, 2025.
[2] Walter RB, Appelbaum FR, Estey EH. Optimal dosing of cytarabine in induction and post-remission therapy of acute myeloid leukemia. Leukemia. 2021;35(2):295-298. doi:10.1038/s41375-020-01110-3
[3] Mayer RJ, Davis RB, Schiffer CA, et al. Intensive postremission chemotherapy in adults with acute myeloid leukemia. Cancer and Leukemia Group B. N Engl J Med. 1994;331(14):896-903. doi:10.1056/NEJM199410063311402
[4] Jaramillo S, Benner A, Krauter J, et al. Condensed versus standard schedule of high-dose cytarabine consolidation therapy with pegfilgrastim growth factor support in acute myeloid leukemia. Blood Cancer J. 2017;7(5):e564. Published 2017 May 26. doi:10.1038/bcj.2017.45
[5] Dumas PY, Bertoli S, Bérard E, et al. Delivering HDAC over 3 or 5 days as consolidation in AML impacts health care resource consumption but not outcome. Blood Adv. 2020;4(16):3840-3849. doi:10.1182/bloodadvances.2020002511
[6] Diebold K, Bachiashvili K, Jamy OH, et al. Outcomes of 3- versus 5-day administration schedule of consolidation cytarabine in adults with acute myeloid leukemia (Aml). JCO. 2023;41(16_suppl):7014-7014. doi:10.1200/JCO.2023.41.16_suppl.7014
[7] Alrajhi AM, Howaidi J, Alnakhli A, Alnajjar FH, Alfayez M. Conventional versus condensed regimen of high dose cytarabine consolidation in acute myeloid leukemia. Blood. 2022;140(Supplement 1):11729-11730. doi:10.1182/blood-2022-166355

Literature Review

A search of the published medical literature revealed 3 studies investigating the researchable question:

What is the clinical difference between cytarabine high dose days 1,2 and 3 and days 1,3, and 5 for AML consolidation?

Level of evidence

C - Multiple studies with limitations or conflicting results  Read more→



Please see Tables 1-3 for your response.


 

Condensed versus standard schedule of high-dose cytarabine consolidation therapy with pegfilgrastim growth factor support in acute myeloid leukemia
Design

Cohort study

N= 568

Objective To compare a condensed schedule of consolidation therapy with high-dose cytarabine on days 1, 2, and 3 (HDAC-123) with the HDAC schedule given on days 1, 3, and 5 (HDAC-135) and to evaluate the prophylactic use of pegfilgrastim after chemotherapy in younger patients with acute myeloid leukemia in first complete remission
Study Groups

HDAC-135 (n= 176)

HDAC-123 (n= 392)

Inclusion Criteria Patients aged between 18 and 60 years with newly diagnosed AML, including de novo AML, secondary AML (sAML), and therapy-related AML (tAML)
Exclusion Criteria Patients with acute promyelocytic leukemia, renal, liver or cardiac dysfunction, uncontrolled infectious disease, primary coagulation disturbance, performance status >2, or active concomitant malignant disease
Methods Patients were treated with HDAC-135 and pegfilgrastim on day 10 or HDAC-123 and pegfilgrastim on day 8. Hematologic recovery was defined as WBC count ≥1.0 × 10^9/l, neutrophil count ≥0.5 × 10^9/l, and platelet count ≥20 × 10^9/l. Infection was defined as microbiologically documented infection and/or febrile neutropenia
Duration August 2004 to August 2009
Outcome Measures

Primary: Time to hematologic recovery (WBC and neutrophils)

Secondary: Rate of infections, platelet transfusions, days in hospital, overall survival (OS), relapse-free survival (RFS)

Baseline Characteristics   German AML Intergroup (n= 41) Cohort 1 HDAC-135 (n= 135) Cohort 2 HDAC-123 (n= 392)
Age (years), median (range) 41.6 (19–60) 47.6 (18–61) 47.7 (18–61)
Gender (male), no. (%) 20 (48.8%) 65 (48.2%) 207 (52.8%)
WBC (10^9/l), median (range) 20.2 (0.2–210) 19.3 (0.9–217) 13.2 (0.3–394)
Platelets (10^9/l), median (range) 55 (8–380) 53 (14–511) 52 (5–574)
Hemoglobin (g/dl), median (range) 9.4 (2.7–16.2) 9.2 (5.2–14.4) 9.3 (3.8–16.0)
Results   HDAC-135 (n= 135) HDAC-123 (n= 392) p-value
Time to WBC recovery, days (median) 19 16 0.0003
Time to neutrophil recovery, days (median) 22 17 0.002
Infection rate 42% 30% 0.0004
Days in hospital, median 23 17 0.04
Platelet transfusions, median 6 4 0.01

Overall survival (p=0.90), relapse-free survival (p=0.48), and relapse-free survival censored for allogeneic transplant (P=0.78) were similar between groups. Likewise, cumulative incidences of relapse (p=0.75) and death (p=0.10) showed no significant differences, even among patients who completed all three consolidation cycles.

Adverse Events Infections were significantly reduced in the HDAC-123 group compared to HDAC-135. No significant difference in survival outcomes was observed between the groups
Study Author Conclusions Consolidation therapy with HDAC-123 leads to faster hematologic recovery, fewer infections, platelet transfusions, and days in hospital without affecting survival. Pegfilgrastim further reduces infection rates and hospitalization duration.
Critique The study's strengths include a large sample size and a clear demonstration of the benefits of a condensed HDAC schedule. However, the sequential cohort design rather than an upfront randomized study may limit the robustness of the conclusions. Additionally, adherence to pegfilgrastim administration was not optimal, which could affect the results.
References:

Jaramillo S, Benner A, Krauter J, et al. Condensed versus standard schedule of high-dose cytarabine consolidation therapy with pegfilgrastim growth factor support in acute myeloid leukemia. Blood Cancer J. 2017;7(5):e564. Published 2017 May 26. doi:10.1038/bcj.2017.45

Consecutive day dosing of high-dose cytarabine consolidation over 3 days is resource-efficient and safe in older adult patients with acute myeloid leukemia
Design

Retrospective cohort analysis

N= 73

Objective To compare the tolerability and efficacy of HDAC-135 and HDAC-123 delivered in sequential cohorts of adult AML patients, focusing on safety, deliverability, and resource utilization of HDAC-123 in adults with AML aged 60 years and older
Study Groups

HDAC-135 (n= 36)

HDAC-123 (n= 37)

Inclusion Criteria Consecutive patients with newly diagnosed de novo or secondary AML (excluding acute promyelocytic leukemia) who received ≥1 cycle of HDAC consolidation chemotherapy at The Royal Melbourne Hospital and Peter MacCallum Cancer Center between June 2018 and June 2023
Exclusion Criteria Not explicitly stated
Methods

Patients received HDAC-135 (1.5-3 g/m2 every 12h on days 1, 3, and 5) or HDAC-123 (1.5-3 g/m2 every 12h on days 1, 2, and 3). Patients ≥60 years received dose-adjusted cytarabine. Prophylactic G-CSF was given based on physician discretion. Data were collected from pharmacy databases and patient medical records.

Duration June 2018 to June 2023
Outcome Measures Tolerability (time to neutrophil and platelet count recovery), safety (direct adverse events or early death)
Baseline Characteristics   All patients (n= 73) Patients aged ≥60 years (n= 24)
Age, years (range) 55 (18-74) 64 (60-74)
Sex - Male 47% 33%
Sex - Female 53% 67%
ECOG at diagnosis - 0 11% 8%
ECOG at diagnosis - 1 61% 58%
ECOG at diagnosis - 2 22% 25%
ECOG at diagnosis - 3 6% 8%
WCC at diagnosis, ×109/L - Median (range) 14.2 (0.9-179) 13.8 (0.9-140.3)
Hb at diagnosis, g/L - Median (range) 90 (56-132) 95 (62-132)
Platelets at diagnosis, ×109/L - Median (range) 61 (17-328) 91 (17-253)
LDH at diagnosis, U/L - Median (range) 418 (189-1533) 299 (189-646)
AML status - De novo 86% 83%
AML status - Secondary 14% 17%
Results   HDAC-135 (n= 36) HDAC-123 (n= 37) p-value
Neutrophils >0.5 ×109/L recovery, median (range), d - Cycle 1 18 (16–38) 14 (11–25) <0.001
Neutrophils >0.5 ×109/L recovery, median (range), d - Cycle 2 20 (18–30) 16 (14–21) <0.001
Neutrophils >0.5 ×109/L recovery, median (range), d - Cycle 3 21 (14–27) 15 (11–18) 0.004
Platelets >50 ×109/L recovery, median (range), d - Cycle 1 28 (20–49) 22.5 (15–41) 0.009
Platelets >50 ×109/L recovery, median (range), d - Cycle 2 30 (20–43) 24.5 (16–33) 0.002
Platelets >50 ×109/L recovery, median (range), d - Cycle 3 30 (24–50) 24 (17–45) 0.04
Adverse Events Three patients (8%) in the HDAC-123 cohort required a change in dosing schedule to HDAC-135 due to cytarabine-related side effects. Cytarabine-induced fever was most frequently seen during cycle 1. The overall incidence rates of microbiologically-proven bacteremia were 29.7%, 27.5%, and 30% during cycles 1, 2, and 3, respectively. No significant differences in the incidence of bacteremia for each HDAC-135 vs HDAC-123 cycle
Study Author Conclusions

HDAC-123 consolidation was well-tolerated by AML patients, including those ≥60 years, and associated with tangible reductions in resource utilization. The consecutive day administration of HDAC-123 was associated with faster hematological recovery times and reduced rates of bacteremia compared to HDAC-135.

Critique

The study provides valuable insights into the feasibility and safety of HDAC-123 in older AML patients, demonstrating faster hematological recovery and reduced resource utilization. However, the retrospective design and small cohort size may limit the generalizability of the findings. Larger prospective studies are needed to confirm these results and further refine post-remission therapy approaches in this population.

 

References:

Nedumannil R, Batterham E, Harding E, Ritchie D, Wei A, Bajel A. Consecutive day dosing of high-dose cytarabine consolidation over 3 days is resource-efficient and safe in older adult patients with acute myeloid leukemia. Leuk Lymphoma. 2023;64(13):2123-2132. doi:10.1080/10428194.2023.2251071

Delivering HDAC over 3 or 5 days as consolidation in AML impacts health care resource consumption but not outcome
Design

Retrospective study

N= 221

Objective To compare the safety, efficacy, and health care resource consumption associated with HDAC-135 and HDAC-123 regimens as consolidation treatment in younger AML patients in first complete response
Study Groups

HDAC-123 (n= 92)

HDAC-135 (n= 129)

Inclusion Criteria Patients aged 18-60 years with newly diagnosed de novo or secondary AML, treated with intensive chemotherapy, and in CR or CRi after 1 course of induction chemotherapy, receiving at least 1 cycle of HDAC as postremission strategy between January 2008 and June 2017
Exclusion Criteria Excluding acute promyelocytic leukemia, primary refractory AML, and those who received multiagent chemotherapy as a postremission strategy
Methods

Patients received HDAC 3 g/m2 every 12 hours for 3 days per schedule: HDAC-123 (days 1, 2, 3) or HDAC-135 (days 1, 3, 5). Prophylactic G-CSF was used, with pegfilgrastim or standard G-CSF. Patients were discharged on day 4 after HDAC-123 or day 6 after HDAC-135 and readmitted for pancytopenia management on days 10 to 12.

Duration January 2008 to June 2017
Outcome Measures Primary: Relapse-free survival, cumulative incidence of relapse, nonrelapse mortality, overall survival
Baseline Characteristics   HDAC-123 (n= 92) HDAC-135 (n= 129)
Age at diagnosis, median (IQR) 46.1 (36.2, 53.4) 47.6 (37.2, 54.9)
ECOG at diagnosis 0-1 65 (75.6%) 106 (83.5%)
WBC at diagnosis, median (IQR) 17.5 (4.1, 76.7) 13.8 (4.1, 47.7)
AML status - De novo 84 (91.3%) 121 (93.8%)
Cytogenetic risk - Favorable 33 (35.9%) 40 (31.0%)
ELN 2010 prognosis - Favorable 43 (47.8%) 61 (47.7%)
Results   HDAC-123 (n= 92) HDAC-135 (n= 129) p-value
WBC recovery, median (IQR), days 14 (13.0-15.0) 17 (16.0-19.0) <0.0001
Neutrophil recovery, median (IQR), days 15 (14.0-16.0) 18 (17.0-20.0) <0.0001
Platelet recovery, median (IQR), days 16 (15.0-26.0) 20 (18.0-23.0) 0.16
Hospitalization duration, median (IQR), days 32 (22.0-36.5) 41 (30.5-50.0) <0.0001
Adverse Events The incidence of microbiologically documented bacteremia was 35.9% in the HDAC-123 arm and 48.1% in the HDAC-135 arm. Documented Streptococcus sp. bacteremia was significantly higher in the HDAC-135 arm (9.3% vs 1.1%; p= 0.01)
Study Author Conclusions The condensed HDAC-123 regimen induced faster hematological recovery and significantly reduced the length of hospital stay without affecting treatment response or outcome in younger AML patients
Critique The study's retrospective nature may introduce selection bias, and the lack of randomization limits the ability to draw definitive conclusions. However, the findings are consistent with previous studies, suggesting that the HDAC-123 regimen is a viable option for reducing hospital stay without compromising efficacy

 

References:

Dumas PY, Bertoli S, Bérard E, et al. Delivering HDAC over 3 or 5 days as consolidation in AML impacts health care resource consumption but not outcome. Blood Adv. 2020;4(16):3840-3849. doi:10.1182/bloodadvances.2020002511