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 MoraAlicia CastañedaMaite GorosteguiAmalia VaroSara Perez-JaumeMargarida SimaoJuan Pablo Muñoz PérezMoira GarrausCristina LarrosaNoelia SalvadorCinzia LavarinoLucas KrauelSalvador MañePublished in: Cancers (2023)
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/m 2 /day (-4 to 0), followed by GM-CSF for 5 days at 500 μg/m 2 /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.
Keyphrases
- end stage renal disease
- chronic kidney disease
- ejection fraction
- newly diagnosed
- high dose
- low dose
- squamous cell carcinoma
- adipose tissue
- acute lymphoblastic leukemia
- radiation therapy
- peritoneal dialysis
- physical activity
- radiation induced
- patient reported outcomes
- early stage
- stem cell transplantation
- diffuse large b cell lymphoma