Value of Right-sided Hemicolectomy for Chldren With High-risk Neuroendocrine Tumors of the Appendix (NETkids)

The Value of Complementary Right-sided Hemicolectomy for Pediatric Patients With High-risk Neuroendocrine Tumors (NETs) of the Appendix; Towards the Development of an (Inter-) National Consensus Guideline for the Pediatric Population.

The goal of this observational study is to investigate the beneficial value of complementary surgery for appendiceal neuro-endocrine tumours in children.

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Study Overview

• Status: Recruiting
• Conditions: Neoplasms; Appendix Cancer

Detailed Description

Aim Current guidelines recommend complementary right-sided hemicolectomy for high-risk (pT2(with risk factors)/pT3) neuro-endocrine tumors (NET) of the appendix (based on adult studies). In contrast to adults, high-risk NET of the appendix in children seems to be a relatively benign disease with high disease-free survival (100% versus 70-80% in adults), but high quality data are lacking. Therefore these recommendations are now being questioned. We aim to investigate the value of complementary right-sided hemicolectomy for children with high-risk NET of the appendix. Ultimately leading to the development of a consensus guideline and solid information for patients/parents.

Plan of investigation In order to generate big data, an international historical cohort study is planned to compare complementary right-sided hemicolectomy with appendectomy alone for children with high-risk NET of the appendix. Results will be CONFIDENTIAL used by an international expert group to formulate treatment recommendations. Subsequently, these recommendations will be tested in an international Delphi study in order to develop a consensus guideline on the treatment of pediatric high-risk NET of the appendix.

Expected results The cohort study will generate high quality information on overall/disease-free survival, recurrence, complications, costs, and hr-QoL. Recommendations made will be tested in a Delphi study; not only on the beneficial value of complementary right-sided hemicolectomy, but also on follow-up protocols and preoperative work-up. Ultimately, an international consensus guideline that redefines low-risk and high-risk NET of the appendix will be developed, leading to global de-escalation and uniformity of treatment.

Relevance for childhood cancer Results are relevant for pediatric oncologists/surgeons/gastro-enterologists across the world, as redefining low-risk and high-risk patient groups, will lead to de-escalation of treatment. Furthermore, QoL of child and parents can be improved by reducing exposure to complications after complementary right-sided hemicolectomy, and by reducing the fear of recurrence by obtaining high-quality data to accurately inform patients and parents.

• Study Type: Observational
• Enrollment (Estimated): 1000

Contacts and Locations

• Study Contact: Name: Ramon R Gorter, MD PhD; Phone Number: 0205669111; Email: rr.gorter@amsterdamumc.nl
• Study Contact Backup: Name: Martine F Raphael, MD PhD; Phone Number: 0205669111; Email: m.raphael@amsterdamumc.nl

Study Locations

• Netherlands
  • Amsterdam-Zuidoost, Netherlands
    • Recruiting
    • Amsterdam UMC
    • Contact: Ramon R Gorter; Email: rr.gorter@amsterdamumc.nl

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: N/A

• Sampling Method: Probability Sample

Study Population

All patients treated for an appendiceal neuro endocrine tumour before the age of 18 during 1990 and 2020

Description

Inclusion Criteria:

• All patients that were treated for an appendiceal NET before the age of 18 years old
• Time period: 1990-2020

Exclusion Criteria:

• Other appendiceal malignancies/tumours, for example:
• goblet cell carcinoma
• adenocarcinoma
• neuroendocrine carcinoma

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease free survival rate	defined as alive and free of recurrence of NET at telephone follow-up performed for this study purpose	cross-sectional design. follow-up will be done in 2023/2024
Recurrence rate	defined as histopathologically proven metastasis/residual tumor at appendiceal stump of NET after a disease free period	cross-sectional design. follow-up will be done in 2023/2024

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival rate	defined as alive at telephone follow-up performed for this study purpose	cross-sectional design. follow-up will be done in 2023/2024
Complications directly related to primary and secondary treatment divided into major and minor complications according to Clavien-dindo.	Complications include, but are not limited to: Intra-abdominal abscess, defined as a radiologically confirmed accumulation of purulent fluid in a walled-off space within the abdominal cavity.; (Adhesive) bowel obstruction requiring readmission (diagnosis based on clinical signs and symptoms such as a history of constipation, nausea, vomiting and distended abdomen); Superficial Surgical Site Infection, as defined by the CDC criteria. (see table 1.); Deep Surgical Site Infection, as defined by the CDC criteria.	cross-sectional design. follow-up will be done in 2023/2024
Number of hospital readmission for complications related to treatment of NET	see title	cross-sectional design. follow-up will be done in 2023/2024
Length of hospital stay	initial and total length of stay	cross-sectional design. follow-up will be done in 2023/2024
number of imaging studies performed for follow-up of NET	ultrasound, computed tomography (CT), magnetic resonance imaging (MRI), octreotide scintigraphy, PET-CT	cross-sectional design. follow-up will be done in 2023/2024
Number of outpatient check-ups(regular visits / telephone call) for follow-up of NET	see title	cross-sectional design. follow-up will be done in 2023/2024
Health related Quality of Life at follow-up moment for this study	measured by the PedsQL(generic)	cross-sectional design. follow-up will be done in 2023/2024
Health related Quality of Life at follow-up moment for this study	measured by the QLQ-C30(generic)	cross-sectional design. follow-up will be done in 2023/2024
Health related Quality of Life at follow-up moment for this study	measured by the QLQ-GINET-21 questionnaire(disease specific)	cross-sectional design. follow-up will be done in 2023/2024

Collaborators and Investigators

• Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
• Investigators: Study Chair: Ramon R Gorter, MD PhD, Amsterdam UMC, department of pediatric surgery

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2023
• Primary Completion (Estimated): June 1, 2024
• Study Completion (Estimated): June 1, 2025

Study Registration Dates

• First Submitted: May 8, 2023
• First Submitted That Met QC Criteria: June 23, 2023
• First Posted (Actual): June 26, 2023

Study Record Updates

• Last Update Posted (Actual): June 26, 2023
• Last Update Submitted That Met QC Criteria: June 23, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Gastrointestinal Neoplasms; Digestive System Neoplasms; Gastrointestinal Diseases; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Intestinal Diseases; Intestinal Neoplasms; Cecal Diseases; Cecal Neoplasms; Neuroendocrine Tumors; Appendiceal Neoplasms
• Other Study ID Numbers: W21_169#21.184

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: individual participant data will not be shared with other researchers. Collaborating institution will however enter participant data in a coded/pseudonimyzed manner in Castor EDC database.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No