Neuroblastoma Elizabeth Roberts Data Coordinator CIBMTR Data Managers Mentor Tandem Meeting February 18
Objectives Know what neuroblastoma is, how it is diagnosed, and how it is treated Complete form 2026: Neuroblastoma Pre-HSCT Data Complete form 2126: Neuroblastoma Post-HSCT Data
About My Center Pediatric Center, performing average of 100 transplants per year. First transplant performed in 1981 Three Data Coordinators, specializing in disease-specific areas We use Epic for our electronic records
What Is Neuroblastoma? Starts in very early forms of nerve cells (neuroblasts) of the sympathetic nervous system Neuro = nerves Blastoma = cancer
Common Neuroblastoma Sites Adrenal glands Abdomen (nonadrenal) Chest Pelvis Neck
Neuroblastoma Statistics 8-10% of all childhood cancers ~8 children per million per year under age of 15 diagnosed Median age of diagnosis is 22 month More than 95% of cases are detected by 10 years of age
How Is Neuroblastoma Diagnosed? Blood and urine catecholamine tests Imaging tests Biopsy of tumor site Bone marrow aspiration and biopsy
Catecholamine Tests Detected in more than 85% of patients with NBL Homovanillic Acid (HVA) Vanillymandelic Acid (VMA)
Imaging Tests CT scans usually abdomen, pelvis, and chest MRI scans usually brain and spine MIBG scans preferred by many doctors as the standard test PET scans
Biopsy Tissue is often needed to obtain all data required for risk-group assignment and treatment stratification in COG clinical trials
Bone Marrow Aspiration/Biopsy Determine extent of disease/staging
Neuroblastoma Staging INSS System Stage 1: Localized tumor with complete gross excision; lymph nodes negative for tumor microscopically Stage 2A: Localized tumor with incomplete resection; lymph nodes negative Stage 2B: Localized tumor with or without complete resection; ipsilateral lymph nodes positive Stage 3: Unresectable unilateral tumor infiltrating across the midline; or localized unilateral tumor with contralateral positive lymph nodes Stage 4: Disseminated disease (except 4S) Stage 4S: Localized primary tumor with dissemination limited to skin, liver and/or marrow (<10%) (infants < 1yr)
Neuroblastoma and the MYCN Oncogene Amplification of MYCN occurs in ~20% of primary untreated NB tumors, and is associated with: Advanced stage disease (Science, 1984) Rapid tumor progression and poor prognosis (N. Engl. J. Med., 1985) High levels of MYCN expression (Science, 1984)
What Therapies Do We Use to Treat Neuroblastoma? Surgery/biopsy Chemotherapy Second look surgery ASCT Radiation therapy Cis-Retinoic therapy Immunotherapy
Surgery Remove as much tumor as possible Dependent on proximity to vital structures or blood vessels Chemotherapy may be used to shrink the tumor first
Chemotherapy Commonly used when metastases involved May be before (neoadjuvant) or after (adjuvant) surgery Most common drugs: carboplatin (or cisplatin), cyclophosphamide, doxorubicin, and etoposide in cycles
Stem Cell Transplant High-risk patients High doses of chemotherapy are effective in treating NBL, but also severely damage bone marrow Need to give stem cells to replace those that were lost Use patient s own cells (autologous transplant)
Radiation Therapy External Beam Aimed at tumor only Can be used to shrink tumor before surgery Sometimes part of treatment regiment before stem cell transplant MIBG Therapy Delivers radiation directly to tumor via blood stream
Retinoid Therapy Related to Vitamin A Differentiating Agents Help cancer cells differentiate into normal cells Currently use 13-cis-retinoic acid
Immunotherapy Typically used in high-risk patients Helps immune system recognize and destroy cancer cells more effectively Monoclonal antibodies given with cytokines
Filling Out the Forms Form 2026 is for Pre-HSCT data Form 2126 is for Post-HSCT data
Form 2026: Clinical and Laboratory Characteristics at Diagnosis Diagnosis Date - Pathology
Form 2026: Clinical and Laboratory Characteristics at Diagnosis
Form 2026: Clinical and Laboratory Characteristics at Diagnosis
Form 2026: Clinical and Laboratory Characteristics at Diagnosis Use pathology report for sites of primary disease Metastases Radiographic tests in Epic, Radiology tab Biopsies Pathology tab Shimada Classification Pathology report
Form 2026: Laboratory Values at Diagnosis of Neuroblastoma HVA and VMA in Urine Chemistry. We send ours out
Form 2026: Laboratory Values at Diagnosis of Neuroblastoma N-myc amplification - Supplementary pathology report
Form 2026: Laboratory Values at Diagnosis of Neuroblastoma
Form 2026: Laboratory Values at Diagnosis of Neuroblastoma Cytogenetic Analysis Our center tends to not perform these on NBL patients INSS most centers use this, but if yours uses POG or Evans, report those. Noted on supplemental report
Form 2026: Laboratory Values at Diagnosis of Neuroblastoma
Form 2026: Laboratory Values at Diagnosis of Neuroblastoma Surgery Radiotherapy Chemotherapy Oncology road maps Best Response Pathology reports
Form 2026: Line of Therapy
Form 2026: Disease Status Immediately Prior to Preparative Regimen
Form 2126: Disease Assessment at Time of Best Response to HSCT Planned Therapy Radiotherapy End of Treatment Note Retinoids Immunotherapy Chemotherapy
Form 2126: Disease Assessment at Time of Best Response to HSCT
Form 2126: Disease Assessment at Time of Best Response to HSCT
Form 2126: Disease Assessment at Time of Best Response to HSCT
Form 2126: Disease Assessment at Time of Best Response to HSCT
Form 2126: Disease Assessment at Time of Best Response to HSCT Recurrence/Progression Pathology Treatment for persistent, progressive, or recurrent disease
References American Cancer Society. Cancer Facts and Figures 2015. Atlanta, Ga: American Cancer Society; 2015. Cheung NK, Dyer MA. Neuroblastoma: Developmental biology, cancer genomics and immunotherapy. Nat Rev Cancer. 2013; 13:397-411. Grupp SA, Dvorak CC, Nieder ML, et.al. Children s Oncology Group s 2013 blueprint for research: Stem cell transplantation. Pediatr Blood Cancer. 2013; 60:1044-1047 National Cancer Institute. Pysician Data Query. Neuroblastoma Treatment. 2013. Accessed at www.cancer.gove/cancertopics/pdq/treatment/neuroblastoma/he althprofessional on 2/4/16. Yu AL, Gilman AL, Ozkaynak MF, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010; 363:1324-1334.
Questions?