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ORR was defined as a CR or PR according to the revised INRC (2017) and confirmed by at least 1 subsequent assessment.
Effectiveness of DANYELZA with GM-CSF was evaluated by independent pathology and imaging review. Responses were observed in the bone, bone marrow, or both bone and bone marrow.1
CI=confidence interval; CR=complete response; DOR=duration of response; INRC=International Neuroblastoma Response Criteria; NE=not estimable; ORR=overall response rate; PR=partial response.
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ORR was defined as a CR or PR according to the revised INRC (2017) and confirmed by at least 1 subsequent assessment.
Effectiveness of DANYELZA with GM-CSF was evaluated by independent pathology and imaging review. Responses were observed in the bone, bone marrow, or both bone and bone marrow.2
*Median follow-up: 5.9 months (range: 0.6–17.8).For the primary endpoint, a sample size of at least 37 patients in the efficacy population is sufficient to ensure at least 90% power to exclude an ORR of 20% or less at the two-sided 5% level.2
Limitations: Interim analysis may not be representative of the final analysis.
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ORR was defined as a CR or PR according to the revised INRC (2017) and confirmed by at least 1 subsequent assessment.
Effectiveness of DANYELZA with GM-CSF was evaluated by independent pathology and imaging review. Responses were observed in the bone, bone marrow, or both bone and bone marrow.2
Study design: These data underwent pre-specified analyses, including subgroup analyses of the primary endpoint.2
Limitation: These subgroup results are based on small sample sizes and could represent chance findings, and they were not adjusted for multiplicity; interpret with caution.2
Study 201 pre-specified interim analysis: swimmer plot of patients with incomplete response to induction2
*Patients with a best response of minor response (MR), stable disease (SD), or progressive disease (PD) to DANYELZA with GM-CSF are excluded from the swimmer plot.
Limitation: Patient-level data are for descriptive purposes and should not be considered indicative of typical product efficacy or duration; interpret with caution.
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ORR was defined as a CR or PR according to the revised INRC (2017) and confirmed by at least 1 subsequent assessment.
Effectiveness of DANYELZA with GM-CSF was evaluated by independent pathology and imaging review. Responses were observed in the bone, bone marrow, or both bone and bone marrow.2
Study design: These data underwent pre-specified analyses, including subgroup analyses of the primary endpoint.2
Limitation: These subgroup results are based on small sample sizes and could represent chance findings, and they were not adjusted for multiplicity; interpret with caution.2
Study 201 pre-specified interim analysis: swimmer plot of patients with incomplete response to relapse therapy2
*Patients with a best response of minor response (MR), stable disease (SD), or progressive disease (PD) to DANYELZA with GM-CSF are excluded from the swimmer plot.
Limitation: Patient-level data are for descriptive purposes and should not be considered indicative of typical product efficacy or duration; interpret with caution.
-
ORR was defined as a CR or PR according to the revised INRC (2017) and confirmed by at least 1 subsequent assessment.
Effectiveness of DANYELZA with GM-CSF was evaluated by independent pathology and imaging review. Responses were observed in the bone, bone marrow, or both bone and bone marrow.2
DANYELZA is the only FDA-approved anti-GD2 immunotherapy approved for this patient population (ie, R/R high-risk neuroblastoma in the bone or bone marrow).1
Study design: These data underwent pre-specified analyses, including subgroup analyses of the primary endpoint.2
Limitation: These subgroup results are based on small sample sizes and could represent chance findings, and they were not adjusted for multiplicity; interpret with caution.2