Revisions to the International Neuroblastoma Response Criteria: A Consensus Statement From the National Cancer Institute Clinical Trials Planning Meeting

Abstract

Purpose More than two decades ago, an international working group established the International Neuroblastoma Response Criteria (INRC) to assess treatment response in children with neuroblastoma. However, this system requires modification to incorporate modern imaging techniques and new methods for quantifying bone marrow disease that were not previously widely available. The National Cancer Institute sponsored a clinical trials planning meeting in 2012 to update and refine response criteria for patients with neuroblastoma. Methods Multidisciplinary investigators from 13 countries reviewed data from published trials performed through cooperative groups, consortia, and single institutions. Data from both prospective and retrospective trials were used to refine the INRC. Monthly international conference calls were held from 2011 to 2015, and consensus was reached through review by working group leadership and the National Cancer Institute Clinical Trials Planning Meeting leadership council. Results Overall response in the revised INRC will integrate tumor response in the primary tumor, soft tissue and bone metastases, and bone marrow. Primary and metastatic soft tissue sites will be assessed using Response Evaluation Criteria in Solid Tumors (RECIST) and iodine-123 (123I) –metaiodobenzylguanidine (MIBG) scans or [18F]fluorodeoxyglucose–positron emission tomography scans if the tumor is MIBG nonavid. 123I-MIBG scans, or [18F]fluorodeoxyglucose–positron emission tomography scans for MIBG-nonavid disease, replace technetium-99m diphosphonate bone scintigraphy for osteomedullary metastasis assessment. Bone marrow will be assessed by histology or immunohistochemistry and cytology or immunocytology. Bone marrow with ≤ 5% tumor involvement will be classified as minimal disease. Urinary catecholamine levels will not be included in response assessment. Overall response will be defined as complete response, partial response, minor response, stable disease, or progressive disease. Conclusion These revised criteria will provide a uniform assessment of disease response, improve the interpretability of clinical trial results, and facilitate collaborative trial designs.

Historical time to disease progression and progression‐free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children's Oncology Group early‐phase trials

“…Kushner et al demonstrated the superiority of 123 I-MIBG for detecting relapse in routine (every 2–4 months) monitoring of high-risk patients 28 . In early-phase trials, the times to relapse/progression may artificially cluster around the times that disease assessments are typically performed or dictated by the protocol.…”

Management of thoracoabdominal neuroblastoma: a 13-year experience

“…They received between 2 and 11 (median 4) cycles of chemotherapy before surgery. However, only 4 (26.7%) patients showed tumor size reduction, with Response Evaluation Criteria in Solid Tumors >30% of the longest dimension after chemotherapy 5. The rest remained as massive tumors that posed challenges to surgical resection.…”

Computer‐assisted Curie scoring for metaiodobenzylguanidine (MIBG) scans in patients with neuroblastoma

“…Several studies have demonstrated the scores assigned after induction therapy are prognostic of outcome of high‐risk patients treated with prior treatment regimens. Curie scores are included in the recently published INRC response criteria and provide important information for treatment decisions . Tracking specific MIBG‐positive lesions over time is likely to enhance accurate response assessment, provide additional prognostic information, and may ultimately lead to more informed treatment decision‐making.…”

“…More recently, in an International Society of Pediatric Oncology European Network (SIOPEN) high‐risk study, researchers were able to validate that patients with a Curie score of ≤2 postinduction have significantly better EFS . Although the prognostic value of Curie scores has not yet been validated in high‐risk patients receiving current COG standard treatment including tandem stem cell transplants and immunotherapy following induction, MIBG relative scores on bone sectors have been integrated into the recent revision of the International Neuroblastoma Response Criteria (INRC) . The relative bone score is the ratio of the Curie scores at response assessment to diagnosis (without the soft‐tissue component).…”