Final safety and efficacy results from the CPX-351 early access program for older patients with high-risk or secondary acute myeloid leukemia

Figures & data

Figure 1. Patient disposition.

Table 1. Baseline demographic and clinical characteristics.

Category	Characteristic, n (%)^a	N = 52
Demographic characteristics
Age	55–65 years	12 (23)
	>65–75 years	40 (77)
Sex	Male	25 (48)
	Female	27 (52)
Race	White	36 (69)
	Black/African American	7 (13)
	Asian	2 (4)
	Other	7 (13)
ECOG PS	0	9 (17)
	1	31 (60)
	2	12 (23)
Clinical characteristics
AML subtype	Therapy-related AML	12 (23)
	History of MDS with prior HMA treatment	13 (25)
	History of MDS without prior HMA treatment	11 (21)
	History of CMML	4 (8)
	de novo AML with MDS karyotype	12 (23)
Time since diagnosis^b	Median (range), months	0.3 (0.03–36.14)
Myeloid blasts	Median (range), %	30 (10–91)

ECOG PS: Eastern Cooperative Oncology Group Performance Status; AML: acute myeloid leukemia; MDS: myelodysplastic syndrome; HMA: hypomethylating agent; CMML: chronic myelomonocytic leukemia.

^aData are shown as n (%) unless otherwise noted.

^bTime from AML diagnosis to the date of informed consent.

Figure 2. Most frequently reported TEAEs by maximum severity grade. All TEAEs reported by ≥5% of patients are included and shown by maximum Common Terminology Criteria for Adverse Events severity grade. TEAE: treatment-emergent adverse event.

Table 2. Summary of TEAEs.

Safety parameter, n (%)	N = 52
TEAEs^a	50 (96)
Febrile neutropenia	40 (77)
Hypoxia	12 (23)
Hypertension	7 (13)
Pneumonia	7 (13)
Serious TEAEs^b	30 (58)
Febrile neutropenia	10 (19)
Pneumonia	5 (10)
Infection	4 (8)
Hypoxia	3 (6)
Respiratory failure	3 (6)
Treatment-related AE	23 (44)
Grade 3 or 4 TEAE	45 (87)
Grade 5 TEAE	5 (10)
Discontinuation due to a TEAE	2 (4)
Treatment interruption due to a TEAE	0

TEAE: treatment-emergent adverse event; AE: adverse event.

^aList of individual TEAEs includes those reported for ≥10% of patients.

^bList of individual serious TEAEs includes those reported for ≥5% of patients.

Table 3. Median time to neutrophil and platelet recovery in patients who achieved CR or CRi^a.

	n = 23^b
Time to ANC recovery ≥1,000/µL
n	12/23
Median (IQR), d	66 (57–114)
Time to platelet recovery ≥100,000/µL
n	5/23
Median (IQR), d	NR (58-NR)
Time to platelet recovery ≥50,000/µL
n	10/23
Median (IQR), d	98 (58–110)

CR: complete remission; CRi: complete remission with incomplete neutrophil or platelet recovery; ANC: absolute neutrophil count; IQR: interquartile range; NR: not reached.

^aMeasured from the last dose of the first induction cycle.

^bPatients may not achieve complete hematologic recovery prior to initiation of consolidation.

Table 4. Best induction response rates.

	N = 52
Best response
CR
n (%)	15 (29)
95% CI	17.1–43.1
CRi
n (%)	8 (15)
95% CI	6.9–28.1
CR + CRi
n (%)	23 (44)
95% CI	30.5–58.7
Time to CR or CRi
n	23
Median (range), d	37.0 (15–72)
Mean (SD), d	41.5 (15.24)

CR: complete remission; CI: confidence interval; CRi: complete remission with incomplete neutrophil and platelet recovery; SD: standard deviation.

Data availability

All relevant data are provided within the manuscript and supporting files.