High-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in Bone Marrow: A Phase II Study
Inclusion Criteria:
- Diagnosis of NB as defined by a) histopathology (confirmed by the MSKCC Department of
Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine
levels.
- High-risk NB as defined by risk-related treatment guidelines1 and the International
NB Staging System,89 i.e., stage 4 with (any age) or without (> or = to 18 months of
age) MYCN amplification or MYCN-amplified stage 4S.
- Patients have primary refractory disease limited to BM, i.e., high-risk NB (defined
above) resistant to standard therapy, as evidenced by incomplete response in BM, but
no measurable MIBG-avid soft tissue tumor assessable for response and no progressive
disease.
- Signed informed consent indicating awareness of the investigational nature of this
program.
Exclusion Criteria:
- Creatinine > 3.0 mg/dL
- ALT, AST and Alkaline Phosphatase > 5.0 times the upper limit of normal
- Bilirubin > 3.0 mg/dL
- Patients with grade 3 or higher toxicities (using the CTCAE v 4.0) related to
cardiac, neurological, pulmonary or gastrointestinal function as determined by
physical exam. Patients must have normal blood pressure for age.
- Progressive disease
- History of allergy to mouse proteins.
- Active life-threatening infection.
- Human anti-mouse antibody (HAMA) titer >1000 Elisa units/ml.
- Inability to comply with protocol requirements.
