Neuroblastoma Response Criteria

!Neuroblastoma Disease Status Response Options
With the Fall 2025 Release, the neuroblastoma response criteria and the Disease Classification (2402) Form were updated to reflect the current International Neuroblastoma Response Criteria. The Neuroblastoma Pre-Infusion (2026) and Neuroblastoma Post-Infusion (2126) Forms have not yet been updated and therefore, the response options on the neuroblastoma forms do not match the updated response criteria. These forms will be updated when they are next revised. In the interim when reporting disease status on the neuroblastoma forms, review the Neuroblastoma Disease Status Response Options (Table 2) below to determine which options should be selected.

*Relative MIBG Score¹
Relative MIBG Score is the absolute score for bone lesions at time of response assessment divided by the absolute score for bone lesions at baseline (before therapeutic interventions). The same scoring method (e.g., Curie or International Society of Pediatric Oncology European Neuroblastoma) must be used at all assessment timepoints. MIBG single-photon emission computed tomography (SPECT) or MIBG-SPECT/CT may be used for scoring purposes, but the same imaging methodology should be used for all evaluations.

!Overall Disease Response Criteria and Disease Components
Each disease response criteria listed below is based off components, which include primary soft tissue, metastatic soft tissue and bone, and metastatic bone marrow. The overall disease response criteria listed below includes the individual disease components which determine the overall disease response. The Primary Soft Tissue, Metastatic Soft Tissue and Bone, and Metastatic Bone Marrow Components Response Criteria (Table 1) list the disease component specific response criteria. It may be helpful to review Table 1 and determine the response for each component. From there, review the overall response criteria to determine the disease status.

*Bone Marrow Biopsy and Complete Remission
In order to report CR, there must be a negative bone marrow biopsy prior to infusion. If multiple bone marrow biopsies were completed prior to infusion, the most recent assessment must be negative. If all CR criteria are met but there has never been a negative bone marrow biopsy, report the disease status as PR.

Neuroblastoma Response Criteria¹

Complete Response (CR)

• Requires all the following:
• Primary soft tissue
  • < 10 mm residual soft tissue at primary site
  • Complete resolution of MIBG or FDG-PET uptake (for MIBG-nonavid tumors at primary site)
• Metastatic soft tissue and bone
  • Nonprimary target and nontarget lesions decrease to a short axis < 10 mm
  • Lymph nodes identified as target lesions decrease to a short axis < 10 mm
  • MIBG uptake or FDG-PET uptake (for MIBG-nonavid tumors) resolution of nonprimary lesions
• Metastatic bone marrow
  • No tumor infiltration of the bone marrow on reassessment, independent of baseline tumor involvement

Partial Response (PR)

Partial response requires all of the following:

• Requires all the following:
  • PR in at least one component
  • CR, PR (soft tissue or bone), or no involvement of all other components
  • Minimal disease (MD) of the bone marrow

*Minor Response and Partial Response
The International Neuroblastoma Response Criteria notes Partial Response (PR) and Minor Response (MR) are options for the overall response. However, MR is not an option on the CIBMTR Forms. If MR criteria are met, report the overall response as PR.

Minor Response (MR)

Minor response requires all of the following:

• PR or CR in at least one component
  • At least one other component with SD
  • No component with PD
• If this criterion is met, report the status as Partial response
  

Stable Disease (SD)

• Requires all the following:
  • SD in one component with no better than SD or no involvement in any other components
  • No component with PD

Progressive Disease (PD)

• Any component with PD

¹ Park, J. R., Bagatell, R., Cohn, S. L., Pearson, A. D., Villablanca, J. G., Berthold, F., Burchill, S., Boubaker, A., McHugh, K., Nuchtern, J. G., London, W. B., Seibel, N. L., Lindwasser, O. W., Maris, J. M., Brock, P., Schleiermacher, G., Ladenstein, R., Matthay, K. K., & Valteau-Couanet, D. (2017). Revisions to the International Neuroblastoma Response Criteria: A consensus statement from the National Cancer Institute Clinical Trials Planning Meeting. Journal of Clinical Oncology, 35(22), 2580–2587. https://doi.org/10.1200/JCO.2016.72.7217

Table 1. Primary Soft Tissue, Metastatic Soft Tissue and Bone, and Metastatic Bone Marrow Components Response Criteria

	CR	PR	SD	PD
Primary Soft Tissue	<10 mm residual soft tissue at primary site Complete resolution of MIBG or FDG-PET uptake (for MIBG-nonavid tumors at primary site)	> 30% decrease in the longest diameter of primary site MIBG or FDG-PET uptake at primary site is stable, improved, or resolved	Primary site not meeting the PR nor PD criteria	> 20% increase in longest diameter taking as reference the smallest sum on study (includes the baseline sum if that is the smallest) and Minimum absolute increase of 5 mm in longest dimension
Metastatic Soft Tissue and Bone	Nonprimary target and nontarget lesions decrease to a short axis < 10 mm Lymph nodes identified as target lesions decrease to a short axis < 10 mm MIBG uptake or FDG-PET uptake (for MIBG-nonavid tumors) resolution of nonprimary lesions	> 30% decrease in sum of diameters of nonprimary target lesions compared to baseline Nontarget lesions stable or smaller No new lesions > 50% reduction in MIBG absolute bone score (relative MIBG bone score > 0.1 to < 0.5) or > 50% reduction in number of FDG-PET avid bone lesions (resolution of MIBG / FDG-PET uptake of soft tissue not required)	Nonprimary lesions not meeting the PR nor PD criteria	Any new MIBG-avid or FDG-PET avid soft tissue lesion detected by CT/MRI; or Any new soft tissue lesions identified on imaging and confirmed neuroblastoma or ganglioneuroblastoma via biopsy Any new MIBG-avid bone site A new FDG-PET avid bone site (for MIBG-nonavid tumors) and with CT / MRI findings consistent with tumor or confirm neuroblastoma or ganglioneruoblastoma via histology > 20% increase in longest diameter taking as reference the smallest sum on study (includes baseline sum if that is the smallest) and minimum absolute increase of 5 mm in sum of diameters of targe soft tissue lesions Relative MIBG score > 1.2
Metastatic Bone Marrow	No tumor infiltration of the bone marrow on reassessment, independent of baseline tumor involvement	Bone marrow with < 5% tumor infiltration and remains > 0 to < 5% tumor infiltration on reassessment; or Bone marrow with no tumor infiltration that has < 5% tumor infiltration on reassessment; or Bone marrow with > 20% tumor infiltration that has > 0 to < 5% tumor infiltration on reassessment	Bone marrow tumor infiltration remaining positive with > 5% tumor infiltration on reassessment but does not meet CR, MD, or PD bone marrow criteria	Bone marrow without tumor infiltration that now has > 5% tumor infiltration on reassessment; or Bone marrow with tumor infiltration that increased by > two-fold and has > 20% tumor infiltration on reassessment

Table 2. Neuroblastoma Disease Status Response Options

2026 / 2126 Disease Status Response Option	Neuroblastoma Response Criteria
Complete response	Complete response
Very good partial response	Partial response
Partial response	Partial response
Minimal response	Partial response
No response	Stable disease
Progressive disease	Progressive disease
Not evaluable	Not an applicable response, select CR, PR, SD, or PD
Not tested / unknown	Not an applicable response, select CR, PR, SD, or PD

Date	Manual Section	Add/ Remove/Modify	Description
10/24/2025	Neuroblastoma Response Criteria	Modify	Version 2 of the Neuroblastoma Response Criteria section of the Forms Instructions Manual released.

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