Prospective Study Registry of Peripheral Neuroblastic Tumours Presenting With Spinal Canal Involvement (SCI) (NB-SCI)

To describe the natural history of peripheral neuroblastic tumour presenting with SCI and evaluate the combined effects of different risk factors on the eventual neurologic and orthopaedic outcomes

Study Overview

• Status: Recruiting
• Conditions: Neuroblastic Tumors
• Intervention / Treatment: Other: survival and late effects

Detailed Description

Multi-centre, observational, prospective study registry. About 15% of patients with peripheral neuroblastic tumour present with extradural SCI, of whom approximately 60% are symptomatic. Since SCI may progress to irreversible paraplegia, early diagnosis and prompt treatment is of critical importance.1-3 The treatment options include chemotherapy, neurosurgical decompression and radiation therapy. All may relieve epidural compression, but there is no consensus on which to use first in the individual patient. However, the choice of the treatment could be relevant in the perspective of reducing to the minimum the risk of long-term sequelae.4 Guidelines for the diagnostic work-up and treatment of SCI for International Society of Paediatric Oncology European Neuroblastoma (SIOPEN) neuroblastoma patients were already available in the "guidelines for the treatment of patients with localized resectable neuroblastoma and analysis of prognostic factors" (LNESG1 Protocol), back in 1994,5 although it is unknown how they were applied in the SIOPEN Centres. These guidelines have been reformulated in occasion of the recently activated "European Low and Intermediate risk Neuroblastoma" (LINES Protocol).

There are few publications addressing the diagnosis and treatment of SCI; most are retrospective studies, case reports, or reviews that may be affected by reporting bias.1-9 Therefore new guidelines could be designed based on the information derived from a prospective data collection of newly diagnosed patients.

For this study Spinal Canal Involvement (SCI) is defined when, referring to an axial plane of the spinal cord MRI scan (Figure 1), the tumour extends into the vertebral canal and goes beyond a mentally drown ellipsoid (red circle) passing through the cortical bone of both anterior and posterior arches of the vertebra. This involvement is called "intraspinal" or, better, "intracanal".

Patient data to be collected and included in the corresponding Forms

1. work-up at diagnosis, including radiology report (Computed Tomography or Magnetic Resonance Imaging)
2. treatment administered
3. response to treatment, including radiology report
4. follow-up
5. outcome

• Study Type: Observational
• Enrollment (Estimated): 150

Contacts and Locations

• Study Contact: Name: Riccardo Haupt, MD; Phone Number: +39 01056363462; Email: riccardohaupt@ospedale-gaslini.ge.it
• Study Contact Backup: Name: tiziana landi; Phone Number: +39 051/2144667; Email: segreteria@aieop.org

Study Locations

• Italy
  • Genova, Italy, 16147
    • Recruiting
    • Istituto Giannina Gaslini
    • Contact: Riccardo Haupt, MD; Phone Number: 01056363462; Email: riccardohaupt@ospedale-gaslini.ge.it
    • Contact: Stefania Sorrentino; Email: stefaniasorrentino@ospedale-gaslini.ge.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patients in the age range 0-18 years diagnosed with peripheral neuroblastic tumour and SCI in the period from May 2014 to April 2017

Description

Inclusion Criteria:

• Diagnosis of peripheral neuroblastic tumour - peripheral neuroblastic tumour (neuroblastoma, ganglioneuroblastoma, ganglioneuroma) presenting with SCI, symptomatic or asymptomatic, independent of disease extension (stage), and clinical course (first diagnosis or relapse/progression)
• No previous chemotherapy, except steroids, in the last 6 months
• Age <18 years
• Minimal planned follow-up of 5 years
• Parent/patient written informed consent (Appendix 1(A,B,C))

Exclusion Criteria:

• Invasion of intervertebral foramina only

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
patients NB with SCI; Survival and late effects. Diagnosis of peripheral neuroblastic tumour - peripheral neuroblastic tumour (neuroblastoma, ganglioneuroblastoma, ganglioneuroma) presenting with SCI, symptomatic or asymptomatic, independent of disease extension (stage), and clinical course (first diagnosis or relapse/progression).	Other: survival and late effects; survival and late effects

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
prevalence of neurologic consequences	American Spinal Injury Association ASIA impairment scale	for 5 years
prevalence of orthopedic consequences	using Common Terminology Criteria for Adverse Events v. 4.0	for 5 years
prevalence of pain	using Face, Legs, Activity, Cry, Consolability scale (FLACC scale)	for 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
relapse or second tumour	event free survival	5 years
survival	overall survival	5 years

Collaborators and Investigators

• Sponsor: Associazione Italiana Ematologia Oncologia Pediatrica
• Collaborators: Istituto Giannina Gaslini
• Investigators: Principal Investigator: Riccardo Haupt, MD, Istituto Giannina Gaslini Genova

Study record dates

Study Major Dates

• Study Start: May 1, 2014
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): May 1, 2029

Study Registration Dates

• First Submitted: September 11, 2015
• First Submitted That Met QC Criteria: September 23, 2015
• First Posted (Estimated): September 24, 2015

Study Record Updates

• Last Update Posted (Estimated): April 15, 2024
• Last Update Submitted That Met QC Criteria: April 12, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Other Study ID Numbers: NB with SCI