Adult-onset neuroblastoma is extremely rare and differs biologically and clinically from the common Pediatric type, rendering difficulty in diagnosis and treatment, and thus poorer outcome, regardless of the stage of disease at diagnosis.
Immunotherapy, mainly with AntiGD2 Antibodies, as maintenance means to prevent disease relapse, is incorporated into multi-modal therapy of pediatric high-risk neuroblastoma, while there is no standard protocol for the Adult subtype.
A 21 years old male patient has been admitted to our ward in June 2017 for treatment of metastatic Neuroblastoma, presented as indolent progressive systemic symptoms over a period of one year prior to admission. Following workup, he underwent biopsy of left unresectable adrenal mass that demonstrated Neuroblastoma, with ALK over expression and no MYCN amplification.
Following first and second line High Risk protocol treatment resulting with stable disease, ICE protocol was initiated, rendering first systemic partial response and enabling Peripheral autologous stem cell collection.
Suzuki et al. reported 44 adult patients from MKSCC, mostly diagnosed with metastatic disease and harbored somatic ATRX and ALK mutations but not MYCN-amplification. The patients with refractory disease treated with immunotherapy had more than 70% response rate with similar treatment related adverse events. Complete disease control appeared to improve long-term survival.
Mazzocco et al. reported 34 AYA patients from Italian Neuroblastoma Registry, Segmental Chromosome Aberrations were demonstrated in 85%, harboring Multi Drug Resistance, while MYCN amplification in less than 10%, confirming the hypothesis of different molecular basis of this aggressive subtype. ALK was mutated in 16% , suggesting the potential of FDA approved targeted therapy.
