Early‐phase clinical trial eligibility and response evaluation criteria for refractory, relapsed, or progressive neuroblastoma: A consensus statement from the National Cancer Institute Clinical Trials Planning Meeting

International standardized criteria for eligibility, evaluable disease sites, and disease response assessment in patients with refractory, progressive, or relapsed high‐risk neuroblastoma enrolled in early‐phase clinical trials are lacking.

[1]  F. Berthold,et al.  A new risk score for patients after first recurrence of stage 4 neuroblastoma aged ≥18 months at first diagnosis , 2019, Cancer medicine.

[2]  Rachel A. Kudgus,et al.  Phase II Trial of Alisertib in Combination with Irinotecan and Temozolomide for Patients with Relapsed or Refractory Neuroblastoma , 2018, Clinical Cancer Research.

[3]  R. Hawkins,et al.  Predictors of response, progression‐free survival, and overall survival using NANT Response Criteria (v1.0) in relapsed and refractory high‐risk neuroblastoma , 2018, Pediatric blood & cancer.

[4]  S. Servaes,et al.  Irinotecan-temozolomide with temsirolimus or dinutuximab in children with refractory or relapsed neuroblastoma (COG ANBL1221): an open-label, randomised, phase 2 trial. , 2017, The Lancet. Oncology.

[5]  S. Groshen,et al.  Expression of Five Neuroblastoma Genes in Bone Marrow or Blood of Patients with Relapsed/Refractory Neuroblastoma Provides a New Biomarker for Disease and Prognosis , 2017, Clinical Cancer Research.

[6]  K. McHugh,et al.  Revisions to the International Neuroblastoma Response Criteria: A Consensus Statement From the National Cancer Institute Clinical Trials Planning Meeting. , 2017, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[7]  F. Berthold,et al.  Recommendations for the standardization of bone marrow disease assessment and reporting in children with neuroblastoma on behalf of the International Neuroblastoma Response Criteria Bone Marrow Working Group , 2017, Cancer.

[8]  Rachel A. Kudgus,et al.  Phase I Study of the Aurora A Kinase Inhibitor Alisertib in Combination With Irinotecan and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma: A NANT (New Approaches to Neuroblastoma Therapy) Trial. , 2016, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[9]  Gudrun Schleiermacher,et al.  Advances in Risk Classification and Treatment Strategies for Neuroblastoma. , 2015, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[10]  F. Berthold,et al.  Minimal residual disease detection in autologous stem cell grafts from patients with high risk neuroblastoma , 2015, Pediatric blood & cancer.

[11]  S. Groshen,et al.  Phase I Study of Vorinostat as a Radiation Sensitizer with 131I-Metaiodobenzylguanidine (131I-MIBG) for Patients with Relapsed or Refractory Neuroblastoma , 2015, Clinical Cancer Research.

[12]  Julie R. Park,et al.  Neuroblastoma: paradigm for precision medicine. , 2015, Pediatric clinics of North America.

[13]  K. Matthay,et al.  Phase I/II study of 131I-MIBG with vincristine and 5 days of irinotecan for advanced neuroblastoma , 2015, British Journal of Cancer.

[14]  M. Parmar,et al.  More multiarm randomised trials of superiority are needed , 2014, The Lancet.

[15]  S. Navarro,et al.  Neuroblastoma after Childhood: Prognostic Relevance of Segmental Chromosome Aberrations, ATRX Protein Status, and Immune Cell Infiltration1 , 2014, Neoplasia.

[16]  J. Maris,et al.  Time to disease progression in children with relapsed or refractory neuroblastoma treated with ABT‐751: A report from the Children's Oncology Group (ANBL0621) , 2014, Pediatric Blood & Cancer.

[17]  W. Gregory,et al.  Neuroblastoma mRNAs predict outcome in children with stage 4 neuroblastoma: a European HR-NBL1/SIOPEN study. , 2014, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[18]  B. Geoerger,et al.  Phase II study of temozolomide in combination with topotecan (TOTEM) in relapsed or refractory neuroblastoma: a European Innovative Therapies for Children with Cancer-SIOP-European Neuroblastoma study. , 2014, European journal of cancer.

[19]  S. Groshen,et al.  Phase I trial of fenretinide delivered orally in a novel organized lipid complex in patients with relapsed/refractory neuroblastoma: A report from the new approaches to neuroblastoma therapy (NANT) consortium , 2013, Pediatric blood & cancer.

[20]  John Anderson,et al.  New Strategies in Neuroblastoma: Therapeutic Targeting of MYCN and ALK , 2013, Clinical Cancer Research.

[21]  A. Naranjo,et al.  Semiquantitative mIBG Scoring as a Prognostic Indicator in Patients with Stage 4 Neuroblastoma: A Report from the Children’s Oncology Group , 2013, The Journal of Nuclear Medicine.

[22]  F. Berthold,et al.  Iodine-123 metaiodobenzylguanidine scintigraphy scoring allows prediction of outcome in patients with stage 4 neuroblastoma: results of the Cologne interscore comparison study. , 2013, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[23]  Thomas Jaki,et al.  Optimal design of multi‐arm multi‐stage trials , 2012, Statistics in medicine.

[24]  M. Parmar,et al.  Flexible trial design in practice - stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial , 2012, Trials.

[25]  J. Khan,et al.  Segmental chromosomal alterations have prognostic impact in neuroblastoma: a report from the INRG project , 2012, British Journal of Cancer.

[26]  I. Ostrovnaya,et al.  Murine anti-GD2 monoclonal antibody 3F8 combined with granulocyte-macrophage colony-stimulating factor and 13-cis-retinoic acid in high-risk patients with stage 4 neuroblastoma in first remission. , 2012, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[27]  Y. Mossé,et al.  Targeting ALK in neuroblastoma—preclinical and clinical advancements , 2012, Nature Reviews Clinical Oncology.

[28]  S. Groshen,et al.  Phase I Study of Vincristine, Irinotecan, and 131I-Metaiodobenzylguanidine for Patients with Relapsed or Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Trial , 2012, Clinical Cancer Research.

[29]  Jinghui Zhang,et al.  Association of age at diagnosis and genetic mutations in patients with neuroblastoma. , 2012, JAMA.

[30]  A. Naranjo,et al.  Pilot induction regimen incorporating pharmacokinetically guided topotecan for treatment of newly diagnosed high-risk neuroblastoma: a Children's Oncology Group study. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[31]  R. George,et al.  Emerging importance of ALK in neuroblastoma. , 2011, Seminars in cancer biology.

[32]  F. Berthold,et al.  Clinical and biologic features predictive of survival after relapse of neuroblastoma: a report from the International Neuroblastoma Risk Group project. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[33]  C. Baey,et al.  Effect of a misspecification of response rates on type I and type II errors, in a phase II Simon design. , 2011, European journal of cancer.

[34]  F. Berthold,et al.  Treatment and outcomes of patients with relapsed, high‐risk neuroblastoma: Results of German trials , 2011, Pediatric blood & cancer.

[35]  N. Cheung,et al.  High‐dose carboplatin–irinotecan–temozolomide: Treatment option for neuroblastoma resistant to topotecan , 2011, Pediatric blood & cancer.

[36]  J. Maris,et al.  Phase II study of irinotecan and temozolomide in children with relapsed or refractory neuroblastoma: a Children's Oncology Group study. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[37]  N. Cheung,et al.  High-dose cyclophosphamide-irinotecan-vincristine for primary refractory neuroblastoma. , 2011, European journal of cancer.

[38]  J. Maris,et al.  Antitumor activity of hu14.18-IL2 in patients with relapsed/refractory neuroblastoma: a Children's Oncology Group (COG) phase II study. , 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[39]  D. Sargent,et al.  Randomized phase II trials: inevitable or inadvisable? , 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[40]  D. Sargent,et al.  Comparison of error rates in single-arm versus randomized phase II cancer clinical trials. , 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[41]  K. Matthay,et al.  Consensus criteria for sensitive detection of minimal neuroblastoma cells in bone marrow, blood and stem cell preparations by immunocytology and QRT-PCR: recommendations by the International Neuroblastoma Risk Group Task Force , 2009, British Journal of Cancer.

[42]  S. Groshen,et al.  Phase I trial of oral irinotecan and temozolomide for children with relapsed high-risk neuroblastoma: a new approach to neuroblastoma therapy consortium study. , 2009, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[43]  S. Groshen,et al.  Iodine-131--metaiodobenzylguanidine double infusion with autologous stem-cell rescue for neuroblastoma: a new approaches to neuroblastoma therapy phase I study. , 2009, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[44]  N. Cheung,et al.  Sensitivity of surveillance studies for detecting asymptomatic and unsuspected relapse of high-risk neuroblastoma. , 2009, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[45]  L. Schwartz,et al.  New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). , 2009, European journal of cancer.

[46]  Sin-Ho Jung,et al.  Randomized phase II trials with a prospective control , 2008, Statistics in medicine.

[47]  F. Berthold,et al.  Topotecan, cyclophosphamide, and etoposide (TCE) in the treatment of high-risk neuroblastoma. Results of a phase-II trial , 2007, Journal of Cancer Research and Clinical Oncology.

[48]  N. Cheung,et al.  Irinotecan plus temozolomide for relapsed or refractory neuroblastoma. , 2006, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[49]  N. Cheung,et al.  Early molecular response of marrow disease to biologic therapy is highly prognostic in neuroblastoma. , 2003, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[50]  N. Cheung,et al.  Oral Topotecan for Refractory and Relapsed Neuroblastoma: A Retrospective Analysis , 2003, Journal of pediatric hematology/oncology.

[51]  S. Larson,et al.  Impact of metaiodobenzylguanidine scintigraphy on assessing response of high-risk neuroblastoma to dose-intensive induction chemotherapy. , 2003, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[52]  R. A’Hern Sample size tables for exact single‐stage phase II designs , 2001, Statistics in medicine.

[53]  D. Sargent,et al.  A three-outcome design for phase II clinical trials. , 2001, Controlled Clinical Trials.

[54]  K K Matthay,et al.  The International Neuroblastoma Pathology Classification (the Shimada system) , 1999, Cancer.

[55]  B. Asselain,et al.  A new 123I-MIBG whole body scan scoring method--application to the prediction of the response of metastases to induction chemotherapy in stage IV neuroblastoma. , 1995, European journal of cancer.

[56]  F. Berthold,et al.  Revisions of the international criteria for neuroblastoma diagnosis, staging and response to treatment. , 1993, Progress in clinical and biological research.

[57]  A. Rademaker,et al.  Analysis of DNA ploidy and proliferative activity in relation to histology and N-myc amplification in neuroblastoma. , 1990, The American journal of pathology.

[58]  R. Simon,et al.  Optimal two-stage designs for phase II clinical trials. , 1989, Controlled clinical trials.