American Statistical Association
New York City
Metropolitan Area Chapter

Memorial Sloan Kettering Cancer Center
Department of Epidemiology and Biostatistics
Biostatistics Seminar Series


Wendy B. London, Ph.D.
Associate Professor of Pediatrics
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
Harvard Medical School

THE EVOLUTION OF PROGNOSTIC FACTORS IN NEUROBLASTOMA (NB),
AND THEIR CHANGING ROLES
IN RISK STRATIFICATION TO ASSIGN INTENSITY OF THERAPY

Historically, the intensity and type of treatment for children with neuroblastoma is determined on the basis of "risk factors", i.e., clinical, genetic, or biologic factors that are prognostic of outcome. Prognostic stratification is the process of identifying which factors are the most statistically significantly associated with outcome, and using them to stratify patients into appropriate treatment groups. Prognostic factors for neuroblastoma are migrating from clinical and phenotypic measurements to increasing use of genetic markers for targeted therapy.

The evolution of neuroblastoma risk stratification (at the time of diagnosis) over the last 20 years in the Children's Oncology Group (COG) will be presented. The development of risk stratification in the International Neuroblastoma Risk Group (INRG) project will also be presented. International consensus was reached on the risk factor definitions of pre-treatment groups to be used as the building blocks for designing future clinical trials, to permit comparison of treatment results across countries regardless of the choice of study design.

Many of the same factors prognostic of outcome at diagnosis are also prognostic at the time of relapse/progression; results from COG and the INRG will be presented. There is great interest in the early identification of a subgroup of high-risk patients with the very worst outcome, sometime referred to as "ultra-high risk" (UHR), so that these patients could be offered alternatives to standard high-risk therapy. Two approaches, one using a binary endpoint and the other using a time-to-event endpoint, will be presented to identify UHR patients. A user-friendly interactive nomogram will be demonstrated.

Lastly, risk stratification in low- and middle-income countries is challenged by lack of expertise and resources to obtain data for the COG/INRG risk factors. An alternative risk stratification, the Clinical Neuroblastoma Risk Group (CNRG) stratification, is proposed, using only age, INSS stage, LDH, and serum ferritin. The CNRG is highly concordant with the INRG stratification.


Date: Wednesday, September 12, 2018
Time: 4:00 - 5:00 P.M.
Location: Memorial Sloan Kettering Cancer Center
Department of Epidemiology and Biostatistics
485 Lexington Avenue
(Between 46th & 47th Streets)
2nd Floor, Conference Room B
New York, New York

**Outside visitors please email wernery@mskcc.org for building access.
You must be on the security list to enter the floor.

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