Table 3 Best responses by evaluable disease in bone/BM compartment and disease location at enrollment in 52 patients treated with naxitamab plus GM-CSF

From: The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial

Patient population

Endpoint

Patients

n (%)

95% CI, %

Response by evaluable disease in bone/BM compartmenta

Evaluable disease in BM (n = 23)

CR

17 (74)

52–90

MD

1 (4)

0–22

SD

2 (9)

1–28

PD

1 (4)

0–22

NE

2 (9)

Response rate

CR + PR

17 (74)

52–90

Evaluable disease in bone (n= 50)

CR

20 (40)

26–55

PR

9 (18)

9–31

SD

13 (26)

15–40

PD

6 (12)

5–24

NE

2 (4)

Response rate

CR + PR

29 (58)

43–72

Response by disease location at enrollment

BM disease only (n=2)

CR

1 (50)

1–99

PD

1 (50)

1–99

ORR

CR + PR

1 (50)

1–99

Bone disease only (n=29)

CR

10 (35)

18–54

PR

5 (17)

6–36

SD

7 (24)

10–44

PD

7 (24)

10–44

NE

0

ORR

CR + PR

15 (52)

33–71

Both bone and BM disease (n=21)

CR

9 (43)

22–66

PR

1 (5)

0–24

MR

5 (24)

8–47

SD

3 (14)

3–36

NE

3 (14)

ORR

CR + PR

10 (48)

26–70

  1. Best response according to independent review, based on INRC (efficacy population).
  2. aSubjects may provide data to both categories.
  3. BM bone marrow, CR complete response, GM-CSF granulocyte-macrophage colony-stimulating factor, INRC International Neuroblastoma Response Criteria, MD minimal disease, MR minor response, NE not evaluable, ORR overall response rate, PD progressive disease, PR partial response, SD stable disease.