Management of High-Risk Neuroblastoma with Soft-Tissue-Only Disease in the Era of Anti-GD2 Immunotherapy
Por:
Gorostegui M, Muñoz JP, Pérez-Jaume S, Simao-Rafael M, Cristina Larrosa Espinosa, Garraus-Oneca M, Salvador-Marcos N, Lavarino C, Krauel L, Mañe S, Castañeda-Heredia A and Mora J
Publicada:
1 may 2024
Ahead of Print:
29 abr 2024
Resumen:
Simple Summary Neuroblastoma (NB) presents with two patterns of disease:
with or without metastasis. Both types of disease presentation include
tumors with high-risk (HR) features. The management of HR-NB includes
chemotherapy, surgery, radiotherapy, and anti-GD2 immunotherapy.
Anti-GD2 monoclonal antibodies (mAbs) have significantly improved the
outcome of HR-NB patients but they are mostly effective against disease
affecting the bone and/or bone marrow (known as the osteomedullary
compartment), and less so against soft tissue disease. The question
arises as to whether anti-GD2 immunotherapy might benefit HR-NB patients
with disease compounded by only soft tissue. In this retrospective
review, we found that achieving first complete remission with
chemotherapy, surgery, and radiotherapy does not prevent the risk of
relapse. However, adding anti-GD2 mAbs once the patient has achieved
complete remission significantly decreases the chances of relapse by
80%. Our study provides further support to indicate anti-GD2 mAbs in all
cases with HR-NB.Abstract Neuroblastoma presents with two patterns of
disease: locoregional or systemic. The poor prognostic risk factors of
locoregional neuroblastoma (LR-NB) include age, MYCN or MDM2-CDK4
amplification, 11q, histology, diploidy with ALK or TERT mutations, and
ATRX aberrations. Anti-GD2 immunotherapy has significantly improved the
outcome of high-risk (HR) NB and is mostly effective against
osteomedullary minimal residual disease (MRD), but less so against soft
tissue disease. The question is whether adding anti-GD2 monoclonal
antibodies (mAbs) benefits patients with HR-NB compounded by only soft
tissue. We reviewed 31 patients treated at SJD for HR-NB with no
osteomedullary involvement at diagnosis. All tumors had molecular
genetic features of HR-NB. The outcome after first-line treatment showed
25 (80.6%) patients achieving CR. Thirteen patients remain in continued
CR, median follow-up 3.9 years. We analyzed whether adding anti-GD2
immunotherapy to first-line treatment had any prognostic significance.
The EFS analysis using Cox models showed a HR of 0.20, p = 0.0054, and
an 80% decrease in the risk of relapse in patients treated with anti-GD2
immunotherapy in the first line. Neither EFS nor OS were significantly
different by CR status after first-line treatment. In conclusion, adding
treatment with anti-GD2 mAbs at the stage of MRD helps prevent relapse
that unequivocally portends poor survival.
Filiaciones:
Gorostegui M:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Pérez-Jaume S:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Cristina Larrosa Espinosa:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Garraus-Oneca M:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Salvador-Marcos N:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Lavarino C:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Krauel L:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Castañeda-Heredia A:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Mora J:
Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, 08950 Barcelona, Spain
Green Submitted, gold
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