title: Treatment of two cases with refractory, metastatic intermediate-risk neuroblastoma with isotretenoin alone or observation.
abstract: Patients <12 months with favorable biology, metastatic neuroblastoma have >90% overall survival following treatment with chemotherapy and surgery. We report two infants with favorable biology, stage 4 neuroblastoma with refractory disease after standard intermediate-risk chemotherapy and additional retrieval chemotherapy. One patient was treated with six additional cycles of isotretinoin and the other observed. Both remain clinically well with persistent disease but no evidence of tumor progression for 28 and 13 months following completion of cytotoxic treatment. Similar to residual tumor in primary sites, refractory metastatic disease may not portend a poor outcome in patients with favorable biology, intermediate-risk neuroblastoma.
authors.affiliation: Department of Pediatrics, Section of Pediatric Hematology/Oncology, The University of Chicago, Chicago, Illinois.
authors.forename: Navin
authors.initials: N
authors.lastname: Pinto
authors.forename: Douglas A
authors.initials: DA
authors.lastname: Cipkala
authors.forename: Patricia E
authors.initials: PE
authors.lastname: Ladd
authors.forename: Yonglin
authors.initials: Y
authors.lastname: Pu
authors.forename: Susan L
authors.initials: SL
authors.lastname: Cohn
mesh_terms: D000008:Abdominal Neoplasms / Q000188:drug therapy / Q000235:genetics / Q000556:secondary / Q000601:surgery; D000310:Adrenal Gland Neoplasms / Q000235:genetics / Q000601:surgery; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D014408:Biomarkers, Tumor; D003131:Combined Modality Therapy; D018450:Disease Progression; D005260:Female; D006801:Humans; D007223:Infant; D015474:Isotretinoin / Q000627:therapeutic use*; D008207:Lymphatic Metastasis; D008297:Male; D009447:Neuroblastoma / Q000188:drug therapy / Q000235:genetics / Q000556:secondary* / Q000601:surgery; D009918:Orbital Neoplasms / Q000188:drug therapy / Q000235:genetics / Q000556:secondary; D011379:Prognosis; D012186:Retroperitoneal Neoplasms / Q000188:drug therapy / Q000235:genetics / Q000601:surgery; D012306:Risk; D016879:Salvage Therapy; D013899:Thoracic Neoplasms / Q000188:drug therapy / Q000235:genetics / Q000556:secondary; D016896:Treatment Outcome; D057832:Watchful Waiting
chemical_list: D000970:Antineoplastic Agents; D014408:Biomarkers, Tumor; D015474:Isotretinoin
keywords: intermediate-risk neuroblastoma; neuroblastoma; refractory disease