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. 2023 Apr 28;15(9):2535.
doi: 10.3390/cancers15092535.

Naxitamab Combined with Granulocyte-Macrophage Colony-Stimulating Factor as Consolidation for High-Risk Neuroblastoma Patients in First Complete Remission under Compassionate Use-Updated Outcome Report

Jaume Mora  1 , Alicia Castañeda  1 , Maite Gorostegui  1 , Amalia Varo  1 , Sara Perez-Jaume  1 , Margarida Simao  1 , Juan Pablo Muñoz  1 , Moira Garraus  1 , Cristina Larrosa  1 , Noelia Salvador  1 , Cinzia Lavarino  1 , Lucas Krauel  1 , Salvador Mañe  1
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Naxitamab Combined with Granulocyte-Macrophage Colony-Stimulating Factor as Consolidation for High-Risk Neuroblastoma Patients in First Complete Remission under Compassionate Use-Updated Outcome Report

Jaume Mora et al. Cancers (Basel). .

Abstract

Naxitamab is an anti-GD2 antibody approved for the treatment of relapsed/refractory HR-NB. We report the survival, safety, and relapse pattern of a unique set of HR-NB patients consolidated with naxitamab after having achieved first CR. Eighty-two patients were treated with 5 cycles of GM-CSF for 5 days at 250 μg/m2/day (-4 to 0), followed by GM-CSF for 5 days at 500 μg/m2/day (1-5) and naxitamab at 3 mg/kg/day (1, 3, 5), on an outpatient basis. All patients but one were older than 18 months at diagnosis and had stage M; 21 (25.6%) pts had MYCN-amplified (A) NB; and 12 (14.6%) detectable MRD in the BM. Eleven (13.4%) pts had received high-dose chemotherapy and ASCT and 26 (31.7%) radiotherapy before immunotherapy. With a median follow-up of 37.4 months, 31 (37.8%) pts have relapsed. The pattern of relapse was predominantly (77.4%) an isolated organ. Five-year EFS and OS were 57.9% (71.4% for MYCN A) 95% CI = (47.2, 70.9%); and 78.6% (81% for MYCN A) 95% CI = (68.7%, 89.8%), respectively. EFS showed significant differences for patients having received ASCT (p = 0.037) and pre-immunotherapy MRD (p = 0.0011). Cox models showed only MRD as a predictor of EFS. In conclusion, consolidation with naxitamab resulted in reassuring survival rates for HR-NB patients after end-induction CR.

Keywords: GM-CSF; anti-GD2 immunotherapy; consolidation; high-risk; naxitamab; neuroblastoma.

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Conflict of interest statement

J.M. declares consulting fees from Ymabs Therapeutics. The rest of the authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Kaplan–Meier survival curves for the whole study population from time of immunotherapy.
Figure 2
Figure 2
Kaplan–Meier survival curves for patients with positive or negative MRD before immunotherapy showing the significant differences for EFS but not for OS.
Figure 3
Figure 3
Kaplan–Meier survival curves for patients having or not received ASCT before immunotherapy showing the significant differences for EFS but not for OS.
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