This page is dedicated to sharing the information we have found in our quest to find the best possible care of Jackson. Hopefully others will find this helpful as we were at a complete loss when we were handed the diagnosis by the doctors.
Neuroblastoma incidence is 1 per 100,000 children in the United States or approximately 700 newly diagnosed patients per year. The median age of diagnosis is approximately 20 months. It represents approximately 8% of all malignancies diagnosed in pediatric patients younger than 15 years of age and is responsible for 15% of all pediatric cancer deaths. The cause of neuroblastoma is currently unknown and no environmental factos have been convincingly linked to its development. The disease generally occurs sporadically, but familial neuroblastoma does occur in about 2% of cases.
75% of neuroblastoma cases occur in the abdominal cavity (50% in the adrenal gland and 25% elsewhere), 20% occur in the posterior mediastinum, 4% in the pelvis, and 1% in the cervical region.
Neuroblastoma cases are staged according to the International Neuroblastoma Staging System Criteria.
|I||Localized tumor with complete gross exision, with or without microscopic residual disease; representative ipsilateral lymph nodes negative for tumor microscopically (nodes attached to and removed with the primary tumor may be positive)|
|II A||Localized tumor with incomplete gross excision; representative ipsilateral nonadherent lymph nodes negative for tumor microscopically|
|II B||Localized tumor with or without complete gross excision, with ipsilateral nonadherent lymph nodes positive for tumor. Enlarged contralateral lymph nodes must be negative microscopically|
|III||Unresectable unilateral tumor infiltrating across the midline, with or without regional lymph node involvement or localized unilateral tumor with contralateral regional lymph node involvement or midline tumor with bilateral extension by infiltration (unresectable) or by lymph node involvement|
|IV||Any primary tumor with dissemination to distant lymph nodes, bone, bone marrow, liver, skin, or other organs (except as defined for stage 4S).|
|IV S||Localized primary tumor (as defined for stage 1, 2A, or 2B), with dissemination limited to skin, liver, and bone marrow (limited to infants younger than 1 yr)|
More than 40% of patients have metastatic disease at diagnosis.
|Age||Stage I||Stage II||Stage III||Stage IV||Stage IVS||Total|
Current treatment is not based only on stage but also on risk stratification (low, intermediate, and high) that takes into account both clinical (age and stage) and biologic (MYCN status, ploidy, and histopathologic classification) variables. MYCN amplification, an indicator of aggressive tumor behavior, occurs in about 25% of all tumors and in approximately 40% of patients with advanced disease.
The treatment of high-risk neuroblastoma requires a multidisciplinary approach. The therapy for patients is generally driven by clinical research protocols. Many of these protocols are sponsored by the Children’s Oncology Group (COG) or by the larger children’s hospitals, working individually or in small groups (for example, The New Approaches to Neuroblastoma Therapy (NANT) consortium funded by the National Cancer Institute). The current “standard” therapy for high-risk neuroblastoma patients consists of the following given in sequence:
Induction Chemotherapy and Surgery
Five to six rounds of chemotherapy delivered on 21 day cycles. Patients will typically undergo a stem cell harvest to remove cells for future use in a stem cell transplant after the first two cycles of chemotherapy. Patients will also typically undergo surgery to remove the tumor after three to five rounds of chemotherapy. Tumor volume reduction typically plateaus after the second or third cycle of chemotherapy.
Myeloblative chemotherapy (high dose) followed by autologous hematopoietic progenitor stem cell transplant to rescue the bone marrow destroyed by the high dose chemotherapy. Stem cell transplant is followed by local irradiation of the primary tumor site and areas prone to relapse
Antibody treatment followed by maintenance therapy (oral doses of 13-cis-retinoic acid (cisRA) for six months (twice daily for two weeks, followed by two weeks without)).