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

Julie R Park^{1

2}, Judith G Villablanca^{3

4}, Barbara Hero⁵, Brian H Kushner⁶, Keith Wheatley⁷, Klaus H Beiske⁸, Ruth L Ladenstein⁹, Sylvain Baruchel¹⁰, Margaret E Macy¹¹, Lucas Moreno¹², Nita L Seibel¹³, Andrew D Pearson^{14

15

16}, Katherine K Matthay¹⁷, Dominique Valteau-Couanet¹⁸

Affiliations

¹ Seattle Children's Hospital, Seattle, Washington, USA.
² Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington, USA.
³ Children's Hospital Los Angeles, Los Angeles, California, USA.
⁴ Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California, USA.
⁵ Children's Hospital, University of Cologne, Cologne, Germany.
⁶ Memorial Sloan Kettering Cancer Center, New York, New York, USA.
⁷ University of Birmingham, Birmingham, UK.
⁸ Department of Pathology, Oslo University Hospital, Oslo, Norway.
⁹ Children's Cancer Research Institute, St Anna Children's Hospital, Vienna, Austria.
¹⁰ Hospital for Sick Children, Toronto, Ontario, Canada.
¹¹ Department of Pediatrics, University of Colorado Anschutz Medical Campus and Children's Hospital Colorado, Aurora, Colorado, USA.
¹² Division of Paediatric Haematology and Oncology, Vall d'Hebron Hospital Universitari, Barcelona, Spain.
¹³ Clinical Investigations Branch, National Cancer Institute, Bethesda, Maryland, USA.
¹⁴ Division of Cancer Therapeutics, Institute of Cancer Research, Sutton, UK.
¹⁵ Division of Clinical Studies, Institute of Cancer Research, Sutton, UK.
¹⁶ Children and Young People's Unit, Royal Marsden NHS Foundation Trust, Sutton, UK.
¹⁷ Department of Pediatrics, School of Medicine, University of California San Francisco, San Francisco, California, USA.
¹⁸ Department of Pediatric Oncology, Gustave Roussy, Villejuif, France.

PMID: 36101004
PMCID: PMC9614386
DOI: 10.1002/cncr.34445

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

Julie R Park et al. Cancer. 2022.

. 2022 Nov 1;128(21):3775-3783.

doi: 10.1002/cncr.34445. Epub 2022 Sep 13.

Authors

Affiliations

¹ Seattle Children's Hospital, Seattle, Washington, USA.
² Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington, USA.
³ Children's Hospital Los Angeles, Los Angeles, California, USA.
⁴ Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California, USA.
⁵ Children's Hospital, University of Cologne, Cologne, Germany.
⁶ Memorial Sloan Kettering Cancer Center, New York, New York, USA.
⁷ University of Birmingham, Birmingham, UK.
⁸ Department of Pathology, Oslo University Hospital, Oslo, Norway.
⁹ Children's Cancer Research Institute, St Anna Children's Hospital, Vienna, Austria.
¹⁰ Hospital for Sick Children, Toronto, Ontario, Canada.
¹¹ Department of Pediatrics, University of Colorado Anschutz Medical Campus and Children's Hospital Colorado, Aurora, Colorado, USA.
¹² Division of Paediatric Haematology and Oncology, Vall d'Hebron Hospital Universitari, Barcelona, Spain.
¹³ Clinical Investigations Branch, National Cancer Institute, Bethesda, Maryland, USA.
¹⁴ Division of Cancer Therapeutics, Institute of Cancer Research, Sutton, UK.
¹⁵ Division of Clinical Studies, Institute of Cancer Research, Sutton, UK.
¹⁶ Children and Young People's Unit, Royal Marsden NHS Foundation Trust, Sutton, UK.
¹⁷ Department of Pediatrics, School of Medicine, University of California San Francisco, San Francisco, California, USA.
¹⁸ Department of Pediatric Oncology, Gustave Roussy, Villejuif, France.

PMID: 36101004
PMCID: PMC9614386
DOI: 10.1002/cncr.34445

Abstract

Background: 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.

Methods: A National Cancer Institute-sponsored Clinical Trials Planning Meeting was convened to develop an international consensus to refine the tumor site eligibility criteria and evaluation of disease response for early-phase clinical trials in children with high-risk neuroblastoma.

Results: Standardized data collection of patient and disease characteristics (including specified genomic data), eligibility criteria, a definition of evaluable disease, and response evaluations for primary and metastatic sites of disease were developed. Eligibility included two distinct patient groups: progressive disease and refractory disease. The refractory disease group was subdivided into responding persistent disease and stable persistent disease to better capture the clinical heterogeneity of refractory neuroblastoma. Requirements for defining disease evaluable for a response assessment were provided; they included requirements for biopsy to confirm viable neuroblastoma and/or ganglioneuroblastoma in those patients with soft tissue or bone disease not avid for iodine-123 meta-iodobenzylguanidine. Standardized evaluations for response components and time intervals for response evaluations were established.

Conclusions: The use of international consensus eligibility, evaluability, and response criteria for early-phase clinical studies will facilitate the collection of comparable data across international trials and promote more rapid identification of effective treatment regimens for high-risk neuroblastoma.

Keywords: clinical trial; consensus criteria; early phase; neuroblastoma.

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References

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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

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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

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