Neuroblastoma: clinical and biological approach to risk stratification and treatment

Abstract

Neuroblastoma is the most common extra-cranial solid tumor of childhood and the most common in the first year of life. It is a unique malignancy in that infants often present with either localized or metastatic disease that can spontaneously regress without intervention while older children can succumb to the disease after months to years of arduous therapy. Given this wide range of outcomes, the International Neuroblastoma Risk Group was created to stratify patients based on presenting characteristics and tumor biology in order to guide intensity of treatment strategies. The goal has been to decrease therapy for low-risk patients to avoid long-term complications while augmenting and targeting therapies for high-risk patients to improve overall survival. The international risk stratification depends on age, stage, histology, MYCN gene amplification status, tumor cell ploidy and segmental chromosomal abnormalities. Treatment for asymptomatic low-risk patients with an estimated survival of > 98% is often observation or surgical resection alone, whereas intermediate-risk patients with an estimated survival of > 90% require moderate doses of response-adjusted chemotherapy along with resection. High-risk patients undergo multiple cycles of combination chemotherapy before surgery, followed by consolidation with myeloablative autologous hematopoietic stem cell transplantation and local radiation and finally immunotherapy with differentiation therapy as maintenance phase. With this approach, outcome for patients with neuroblastoma has improved, as the field continues to expand efforts in more targeted therapies for high-risk patients.

Feasibility and effectiveness of treatment strategy of tandem high‐dose chemotherapy and autologous stem cell transplantation in combination with 131I‐MIBG therapy for high‐risk neuroblastoma

“…The current treatment strategy for high-risk neuroblastoma comprises induction chemotherapy, surgery, radiation, HDCT/ASCT as consolidation therapy, and various forms of post-consolidation therapy such as differentiation therapy with 13-CRA and immunotherapy. 1 Despite advances in these multimodal therapies, durable remission rates remain unsatisfactory with EFS rates less than 50%. 2,3 Dose intensification using HDCT/ASCT has contributed to the improvement of survival rates for patients with high-risk neuroblastoma.…”

Nomograms for the Prediction of Survival for Patients with Pediatric Adrenal Cancer after Surgery

“…International Publisher cell, accounting for nearly 10% of all childhood cancers [3][4][5]. The most common site of origin of NB is the adrenal medulla, accounting for 35% of cases [6], and NB with adrenal site is associated with inferior survival [7].…”

“…Adrenal cancer is usually aggressive with a poor prognosis, since it has often invaded nearby tissues or metastasized to distant organs at the time of diagnosis [3,8,9]. Complete surgical resection of the tumor is the most important and mainstay treatment for patients with adrenal cancer, which carries the best hope for prolonged survival and potential cure [10][11][12].…”

The sympathetic nervous system: malignancy, disease, and novel functions

“…Risk stratification is important to avoid overtreatment of low-risk patients resulting in late complications and to select and improve targeted therapies for high-risk patients. Clinical presentation, diagnosis, risk stratification according to the INRG staging, and therapy are reviewed by Tolbert and Matthay (2018). Notably, targeted small molecule inhibitors and immunotherapy are presently used for relapsed and refractory patients but may be included in the personalized initial therapy of newly diagnosed patients.…”