Q229 – 261: Disease Status Immediately Prior to Preparative Regimen

*Disease Assessments Prior to Preparative Regimen
All values reported below must reflect the most recent testing performed prior to the start of the preparative regimen (or infusion if no preparative regimen was given). Assessments performed during the last line of therapy or after the last lie of therapy may be reported. If multiple assessments were performed, report the most recent. If additional therapy was given after the last assessment, do not report the test.

Question 229: Were tumor marker analyses performed immediately prior to the preparative regimen?

Tumor markers, also known as biomarkers, are substances produced by cancer tissue or by the body in response to cancer at higher than normal levels. In certain situations, these substances can be used to detect and monitor disease due to their elevated presence in blood, urine, and/or tissue. The substances listed below have been identified as potential tumor markers for neuroblastoma.

Specify if tumor marker analyses were performed at the last evaluation prior to the start of the preparative regimen / infusion. If testing was not performed or unknown if performed, select No.

Questions 229 – 241: Specify the tumor marker analyses performed

For each tumor marker, indicate if it was analyzed at the last evaluation prior to the preparative regimen / infusion. If analyzed, select Known and specify the value. If the analysis was not performed or it is unknown if performed, select Not known.

• Homovanillic acid (HVA): Catecholamines (e.g. epinephrine/adrenaline) are secreted as hormones from the adrenal medulla. Neuroblastomas typically produce excessive levels of these catecholamines. After catecholamines are secreted they are broken down into metabolites including Homovanillic acid (HVA) and Vanillylmandelic acid (VMA). Both HVA and VMA are excreted in the urine. As a result, elevated levels of HVA and VMA detected by urinary analysis can be indicative of neuroblastoma.
• Neuron specific enolase (NSE): A glycolytic enzyme (enolase) specific to neuronal-type tissues. NSE may be excessively expressed by neuroblastomas and indicative of disease.
• Other tumor marker analysis: If testing for a tumor marker was performed at diagnosis and is not listed above, select Known, specify the tumor marker, value and units of measurements. Examples of other testing can include Chromogranin A (CgA) or Neuropeptide Y (NpY).

Question 242: Specify the total number of complete remissions

Report the total number of complete remissions (CR) achieved, including the most recent CR from diagnosis until the start of the preparative regimen / infusion.

For subsequent infusions, include all CRs achieved from the original diagnosis to the start of the preparative regimen for the current infusion.

Questions 243 – 260: Specify any known sites of disease immediately prior to the preparative regimen

For each site, indicate whether disease was identified at the last evaluation prior to the start of the preparative regimen / infusion.

If disease was present in the recipient’s bone marrow immediately, indicate if bone marrow morphology, flow cytometry, and / or immunofluorescence was performed to evaluate the bone marrow at the last evaluation prior to the start of the preparative regimen / infusion.

If a site was known to have disease at the time immediately prior to the start of the preparative regimen and is not listed as an option, select Yes, for Other site and specify the site.

Question 261: Specify the percent of cells positive for neuroblastoma

Report the percent of cells positive for neuroblastoma from the site(s) reported above at the last evaluation prior to the start of the preparative regimen / infusion. If multiple sites were selected, the percentage of positive cells identified in the bone marrow should be reported.

Section Updates

Question Number	Date of Change	Add/Remove/Modify	Description	Reasoning (if applicable)
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