Malignant transformation and tumour recurrence in sacrococcygeal teratoma, a congenital disorder in newborns

Plain English Summary

Background and study aims
Sacrococcygeal teratoma (SCT) is a rare congenital disorder with an estimated incidence of 1:15,000 to 1:30,000. The treatment is complete resection, but in a substantial proportion of patients, the tumour recurs, often as malignancy. There is scant data on the true recurrence rate of SCT, the timing of recurrence, how recurrence is diagnosed and risk factors for recurrence including histology of the primary tumour. This study aims to assess the SCT recurrence rate, the time after which SCT recurrence occurs, pathology, whether recurrence is a primary or recurrent tumour, and how recurrent SCT is diagnosed.

Who can participate?
All patients with SCT treated by each participating centre during a set period of time will be included. Patients with Currarino triad-associated SCT (arising from defects that occur during embryonic development) are specifically included.

What does the study involve?
Participating centers confirm their participation, the inclusion of consecutive patients in a set period of time and sign a data transfer agreement form which defines data ownership, data use, protection and storage of data and authorship.

What are the possible benefits and risks of participating?
There is no benefit for the individual included patient in this study. However, The United Nations encourages nations to create networks of experts for rare diseases and to increase research support, by strengthening international collaboration and coordination of research efforts and the sharing of data, while respecting its protection and privacy. This collaborative study enables the identification of risk factors for recurrent SCT and essential information regarding malignant SCT transformation. With large, shared patient data sets, it is possible to answer important clinical questions that cannot be answered otherwise to improve the quality of care in every part of the world.

Due to the design of the study including only retrospective patient data, there are no risks for individual patients.

Where is the study run from?
Amsterdam University Medical Centre in the Netherlands

When is the study starting and how long is it expected to run for?
December 2020 to January 2022

Who is funding the study?
KiKa Children's Cancer Free Foundation

Who is the main contact?
Prof. Dr. L.W.E. van Heurn, e.vanheurn@amsterdamumc.nl

Study website

Type

Principal Investigator

Contact name

Prof Ernst van Heurn

ORCID ID

Contact details

Meibergdreef 9
Amsterdam
1105 AZ
Netherlands
+31 20 566 5693
e.vanheurn@amsterdamumc.nl

Type

Public

Contact name

Dr Lieke van Heurn

ORCID ID

Contact details

Meibergdreef 9
Amsterdam
1105 AZ
Netherlands
+31 20 566 5693
l.j.vanheurn@amsterdamumc.nl

Type

Scientific

Contact name

Dr Joep Derik

ORCID ID

Contact details

Meibergdreeg 9
Amsterdam
1105 AZ
Netherlands
+31 20 566 5693
j.derikx@amsterdamumc.nl

EudraCT/CTIS number

Nil known

IRAS number

ClinicalTrials.gov number

Nil known

Protocol/serial number

KiKa project 440

Scientific title

Malignant transformation and tumour recurrence in patients with SacroCoccygeal Teratoma: a global, retrospective cohort study

Acronym

SCT-study

Study hypothesis

Sacrococcygeal teratoma (SCT) is a rare congenital disorder with an estimated incidence of 1:15,000 to 1:30,000. The treatment is complete resection, but in a substantial proportion of patients, the tumour recurs, often as malignancy. There is limited data on the true recurrence rate of SCT, the timing of recurrence, how recurrence is diagnosed and risk factors for recurrence including histology of the primary tumour.

Ethics approval(s)

Ethics approval not required

Ethics approval additional information

The Medical Ethical Board of Amsterdam University Medical Centre (Amsterdam UMC), determined that the Medical Research Involving Human Subject Act (WMO) does not apply to the study and that official approval of the committee was not required (reference number W19_329 # 19.388).

Study design

Multicentre observational retrospective cohort study

Primary study design

Observational

Secondary study design

Cohort study

Study setting(s)

Medical and other records

Study type

Diagnostic, Treatment

Patient information sheet

No participant information sheet available

Condition

Sacrococcygeal teratoma

Intervention

This observational study looks at tumour histology at initial resection and the time between birth and surgery. Furthermore, risk factors associated with recurrence will be analysed.

Collected data included generic and condition-specific variables. Generic variables included: country, gender (male/female/unknown), age at diagnosis (days), pre-operative imaging modalities (none/ultrasound/computed tomography/magnetic resonance imaging/unknown), initial tumour resection at the participating centre (yes/no/unknown), age at initial resection (days), outcome (survival/deceased/unknown), age at follow-up (days), age at death (days), and cause of death. Condition-specific variables were Altman classification (I/II/III/IV/unknown), CS (yes/no/unknown), initial SCT treatment (chemotherapy/surgery/no treatment/unknown), pathology (mature/immature/malignant/unknown), recurrence (yes/no/unknown), the period between birth and recurrence (days), detection of recurrence (clinical examination/imaging/AFP/unknown), serum AFP-level at recurrence (μg/L), recurrent SCT pathology (mature/immature/malignant/unknown) and treatment of recurrent SCT (chemotherapy/surgery/no treatment/unknown). These data were used to calculate the risk of malignant transformation and risk factors associated with sacrococcygeal teratoma recurrence.

Intervention type

Procedure/Surgery

Primary outcome measure

The following primary outcomes are measured using patient medical records at a one-time point:
1. Malignant transformation of the initial tumour confirmed by pathology.
2. Risk factors associated with sacrococcygeal teratoma recurrence defined as relapse of the tumour at least three months after initial resection.

Secondary outcome measures

The following secondary outcomes are measured using patient medical records at a one-time point:
1. Treatment modality for sacrococcygeal teratoma patients from different income countries
2. Outcomes of treatment for sacrococcygeal teratoma patients from different income countries

Overall study start date

01/01/2019

Overall study end date

01/01/2022

Reason abandoned (if study stopped)

Participant inclusion criteria

All patients with SCT treated by each participating centre during a set time

Participant type(s)

Patient

Age group

All

Sex

Both

Target number of participants

1000

Total final enrolment

3612

Participant exclusion criteria

Patients treated for sacrococcygeal teratoma before 1980

Recruitment start date

01/12/2020

Recruitment end date

01/01/2022

Countries of recruitment

Afghanistan, Argentina, Austria, Bangladesh, Belarus, Belgium, Brazil, Bulgaria, Cameroon, Canada, Chile, China, Cote d'Ivoire, Croatia, Czech Republic, Denmark, Egypt, Estonia, Ethiopia, Finland, France, Germany, Ghana, Greece, Hong Kong, Hungary, India, Indonesia, Iran, Iraq, Ireland, Israel, Italy, Japan, Korea, South, Latvia, Lithuania, Macedonia, Malaysia, Mexico, Montenegro, Morocco, Netherlands, Nigeria, Norway, Pakistan, Philippines, Poland, Portugal, Romania, Russian Federation, Serbia, Singapore, Slovakia, Slovenia, Spain, Sri Lanka, Sweden, Syria, Taiwan, Thailand, Tunisia, Turkey, Ukraine, United Kingdom, United States of America, Zambia

Study participating centre

Hospital Garrahan Buenos Aires
-
Argentina

Study participating centre

Hospital Italiano de Buenos Aires
-
Argentina

Study participating centre

Medical University of Graz
-
Austria

Study participating centre

Bangladesh Shishu Hospital & Institute
-
Bangladesh

Study participating centre

Center of Pediatric Surgery of Belarusb
-
Belarus

Study participating centre

Universitair Ziekenhuis Brussel
-
Belgium

Study participating centre

Hôpital des Enfants Reine Fabiola, Université Libre de Bruxelles
-
Belgium

Study participating centre

Federal University of São Paulo
-
Brazil

Study participating centre

University Hospital "St. George"
-
Bulgaria

Study participating centre

UMHATEM "N. I. Pirogov" Sofia
-
Bulgaria

Study participating centre

Yaounde Gynaeco-Obstetric and Pediatric Hospital-faculty of medecine
-
Cameroon

Study participating centre

McGill University, Montreal Children’s Hospital
-
Canada

Study participating centre

Shriners Hospital for Children
-
Canada

Study participating centre

The Hospital for Sick Children
-
Canada

Study participating centre

Hospital de Niños Dr. Roberto del Rio
-
Chile

Study participating centre

Guangzhou Women and Children’s Medical Center
510623
China

Study participating centre

University Hospital centre Zagreb
-
Croatia

Study participating centre

University Hospital of Split and Department of Surgery, University of Split
-
Croatia

Study participating centre

Charles University and University Hospital Motol
150 06
Czech Republic

Study participating centre

Rigshospitalet Copenhagen University Hospital
-
Denmark

Study participating centre

Ain Shams University
-
Egypt

Study participating centre

Kasr Al Ainy Faculty of Medicine
-
Egypt

Study participating centre

Pediatric Surgery Cairo University
-
Egypt

Study participating centre

Tallinn Children's Hospital
-
Estonia

Study participating centre

St.Peter Specialized Hospital
-
Ethiopia

Study participating centre

New Children´s Hospital
-
Finland

Study participating centre

Hôpital des Enfants de Toulouse
-
France

Study participating centre

CHU Rennes, Univ Rennes
-
France

Study participating centre

Limoges University Hospital Center
-
France

Study participating centre

University Hospital Angers
Angers
-
France

Study participating centre

Hospital for sick children La Timone, Assistance publique des hôpitaux de Marseille
Marseille
-
France

Study participating centre

orbonne Université, Armand Trousseau Hopsital –Assistance Publique Hôpitaux de Paris,
Paris
-
France

Study participating centre

Hopitaux Pédiatriques de Nice CHU-Lenval
Nice
-
France

Study participating centre

University Hospital, Poitiers
Poitiers
-
France

Study participating centre

Hôpital Necker-Enfants Malades - APHP GH Centre and Université de Paris Cité
Paris
-
France

Study participating centre

University Hospital of Lille Jeanne de Flandre
Lille
-
France

Study participating centre

Dr. von Hauner Children's Hospital, University Hospital
Munich
-
Germany

Study participating centre

University Medical Center Mannheim, Heidelberg University
Mannheim
-
Germany

Study participating centre

University of Leipzig
Leipzig
-
Germany

Study participating centre

Charité Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt Universität zu Berlin
Berlin
-
Germany

Study participating centre

University Hospital Frankfurt/M.
Frankfurt
-
Germany

Study participating centre

Accra College of Medicine
Accra
-
Ghana

Study participating centre

P. & A. Kyriakou Children’s Hospital
Athens
-
Greece

Study participating centre

Semmelweis University
Budapest
-
Hungary

Study participating centre

Medical School, University of Pécs
Pécs
-
Hungary

Study participating centre

Li Ka Shing Faculty of Medicine, University of Hong Kong
-
Hong Kong

Study participating centre

Amrita Institute Of medical sciences
Kerala
-
India

Study participating centre

All India Institute of Medical Sciences
Jodhpur
-
India

Study participating centre

All India Institute of Medical Sciences
New Dehli
-
India

Study participating centre

Faculty of Medicine, Public Health and Nursing, Universitas Gadjah Mada, Dr. Sardjito Hospital
Yogyakarta
-
Indonesia

Study participating centre

Research Institute for Children’s Health, Shahid Beheshti University of Medical Sciences
Teheran
-
Iran

Study participating centre

Alkhansaa teaching hospital
Mosul
-
Iraq

Study participating centre

Children's Health Ireland at Crumlin
Dublin
-
Ireland

Study participating centre

Tel Aviv Sourasky Medical Center
Tel Aviv
-
Israel

Study participating centre

IRCCS Istituto Giannina Gaslini
Genoa
-
Italy

Study participating centre

Children's Hospital Bambino Gesù-Research Institute
Rome
-
Italy

Study participating centre

University of Padua
Padua
-
Italy

Study participating centre

Regina Margherita Children’s Hospital
Turin
-
Italy

Study participating centre

Bambino Gesu' Pediatric Hospital
Rome
-
Italy

Study participating centre

Sant'Orsola Hospital, IRCSS, University of Bologna
Bologna
-
Italy

Study participating centre

Meyer Children's Hospital IRCCS Florence
Florence
-
Italy

Study participating centre

Fondazione IRCCS Policlinico San Matteo
Pavia
-
Italy

Study participating centre

Cocody Teaching Hospital at Abidjan
Abidjan
-
Cote d'Ivoire

Study participating centre

Kyoto Prefectural University of Medicine
Kyoto
-
Japan

Study participating centre

Saitama Children's Medical Center
Saitama
-
Japan

Study participating centre

National Center for Child Health and Development
Tokyo
-
Japan

Study participating centre

Graduate School of Medical Sciences, Kyushu University
Fukuoka
-
Japan

Study participating centre

Osaka City General Hospital
Osaka
-
Japan

Study participating centre

Graduate School of Medical Sciences, Kyushu University,
Fukuoka
-
Japan

Study participating centre

Tokyo Metropolitan Children's Medical Center
Tokyo
-
Japan

Study participating centre

Riga Stradins University & Children's Clinical University Hospital
Riga
-
Latvia

Study participating centre

Vaikų chirurgijos klinika, Lietuvos sveikatos mokslų universiteto ligoninė Kauno klinikos
Kaunsas
LT-50161
Lithuania

Study participating centre

University Clinic for Pediatric Surgery, Faculty of Medicine, Ss. Cyril and Methodius
Skopje
-
Macedonia

Study participating centre

Faculty of Medicine, Universiti Kebangsaan Malaysia
Kuala Lumpur
-
Malaysia

Study participating centre

Hospital Tunku Azizah, Kuala Lumpur Women's and Children's Hospital
Kuala Lumpur
-
Malaysia

Study participating centre

Instituto Nacional de Pediatría
Mexico City
-
Mexico

Study participating centre

Institute for children's diseases, Clinical Centre of Montenegro
Podgorica
-
Montenegro

Study participating centre

University hospital Mohamed VI, Cadi Ayyad University,
Marrakesh
-
Morocco

Study participating centre

University Medical Center Groningen
Groningen
-
Netherlands

Study participating centre

Radboud University Medical Center, Amalia Children's Hospital
Nijmegen
-
Netherlands

Study participating centre

Emma Children’s Hospital, Amsterdam UMC, location University of Amsterdam,
Amsterdam
-
Netherlands

Study participating centre

University Medical Centre Maastricht
Maastricht
-
Netherlands

Study participating centre

Erasmus MC Sophia Children’s Hospital
Rotterdam
-
Netherlands

Study participating centre

Amsterdam Public Health Research Institute, Amsterdam UMC, Vrije Universiteit Amsterdam
Amsterdam
1081 HV
Netherlands

Study participating centre

University of Utrecht, Wilhelmina Children's Hospital, UMC Utrecht
Utrecht
-
Netherlands

Study participating centre

Lagos University Teaching Hospital
Lagos
-
Nigeria

Study participating centre

Jos University Teaching Hospital
Jos
PMB 2076
Nigeria

Study participating centre

Nnamdi Azikiwe University Teaching Hospital
Nnewi
-
Nigeria

Study participating centre

Abubakar Tafawa Balewa University Teaching Hospital
Bauchi
-
Nigeria

Study participating centre

Oslo University Hospital
Oslo
4950
Norway

Study participating centre

Liaquat National Hospital and Aga khan university
Karachi
-
Pakistan

Study participating centre

Children Hospital, Shaheed Zulfiqar Ali Bhutto Medical University
Islamabad
-
Pakistan

Study participating centre

University of Child Health Sciences
Lahore
-
Pakistan

Study participating centre

Philippine Children's Medical Center
Quezon City
-
Philippines

Study participating centre

Philippine General Hospital
Manila
-
Philippines

Study participating centre

The Children's Memorial Health Institute
Warshaw
-
Poland

Study participating centre

Medical University of Gdansk
Gdansk
-
Poland

Study participating centre

Hospital Dona Estefania
Lisboa
-
Portugal

Study participating centre

Hospital Dona Estefânia
Lisboa
-
Portugal

Study participating centre

"Victor Babes" University of Medicine and Pharmacy Timisoara
Timisoara
-
Romania

Study participating centre

FSAI "NMRC of Сhildren Health" MHRF
Moscow
-
Russian Federation

Study participating centre

Volgograd State Medical University
Volgograd
-
Russian Federation

Study participating centre

Irkutsk Regional Children’s Hospital
Irkutsk
-
Russian Federation

Study participating centre

Stavropol State Medical University
Stavropol
-
Russian Federation

Study participating centre

Institute for Mother and Child Healthcare of Serbia "Dr Vukan Cupic"
Belgrade
-
Serbia

Study participating centre

University Children’s Hospital, Center for Pediatric Surgery and Medical Faculty University of Belgrade
Belgrade
-
Serbia

Study participating centre

KK Women’s and Children’s Hospital
-
Singapore

Study participating centre

National Institute of Children´s Diseases
Bratislava
-
Slovakia

Study participating centre

University Medical centre Ljubljana
Ljubljana
-
Slovenia

Study participating centre

Seoul National University Hospital
Seoul
03080
Korea, South

Study participating centre

Asan Medical Center
Seoul
-
Korea, South

Study participating centre

Inje University Busan Paik hospital
Busan
-
Korea, South

Study participating centre

University Hospital Vall d´Hebron
Barcelona
-
Spain

Study participating centre

Children's Hospital La Paz
La Paz
-
Spain

Study participating centre

Virgen del Rocio Children's Hospital
Sevilla
-
Spain

Study participating centre

Hospital Universitario Reina Sofía
Córdoba
-
Spain

Study participating centre

Virgen de la Arrixaca University Clinical Hospital
Murcia
-
Spain

Study participating centre

La Fe University and Polytechnic Hospital
Valencia
-
Spain

Study participating centre

Lady Ridgeway Hospital
Colombo
-
Sri Lanka

Study participating centre

Skane University Hospital Lund
Lund
-
Sweden

Study participating centre

Karolinska University Hospital
Stockholm
-
Sweden

Study participating centre

Astrid Lindgren´s Childrens Hospital
Stockholm
-
Sweden

Study participating centre

Children Hospital
Damascus
-
Syria

Study participating centre

Chang Gung University College of Medicine
Taoyuan
-
Taiwan

Study participating centre

Queen Sirikit National Institute of Child Health
Bangkok
-
Thailand

Study participating centre

Hedi Chaker Hospital University of medecine of Sfax
Sfax
-
Tunisia

Study participating centre

Fattouma Bourguiba Hospital
Monastir
-
Tunisia

Study participating centre

Ege University
Izmir
-
Turkey

Study participating centre

Istanbul Medeniyet University Faculty of Medicine)
Istanbul
-
Turkey

Study participating centre

Dr Sami Ulus Maternity and Children Health and Research Application Center
Ankara
-
Turkey

Study participating centre

Faculty of Medicine & Cancer Institute Ankara
Ankara
-
Turkey

Study participating centre

Hacettepe University, Faculty of Medicine
Ankara
-
Turkey

Study participating centre

National Specialized Children Hospital “Ohmatdyt“
Kyiv
-
Ukraine

Study participating centre

Leeds Teaching Hospitals
Leeds
-
United Kingdom

Study participating centre

Cambridge University Hospitals NHS Foundation Trust
Cambridge
-
United Kingdom

Study participating centre

Great North Children's Hospital
Newcastle
-
United Kingdom

Study participating centre

Birmingham Children hospital UK
Birmingham
-
United Kingdom

Study participating centre

Royal Manchester Children's Hospital
Manchester
-
United Kingdom

Study participating centre

The Royal London Hospital
London
-
United Kingdom

Study participating centre

Bristol Royal Children's Hospital
Bristol
-
United Kingdom

Study participating centre

UCL Great Ormond Street Institute of Child Health
London
-
United Kingdom

Study participating centre

Faculty of Medicine, University of Southampton
Southampton
-
United Kingdom

Study participating centre

Institute Of Life Course And Medical Sciences
Liverpool
-
United Kingdom

Study participating centre

Royal Hospital for Sick Children
Edinburgh
-
United Kingdom

Study participating centre

Nationwide Children’s Hospital
Columbus
-
United States of America

Study participating centre

University of Utah School of Medicine
Salt Lake City
-
United States of America

Study participating centre

University of Tennessee Health Science Center
Tennessee
-
United States of America

Study participating centre

University of Chicago
Chicago
-
United States of America

Study participating centre

Children’s Mercy Kansas City
Kansas City
-
United States of America

Study participating centre

University Teaching Hospital of Lusaka
Lusaka
-
United States of America

Organisation

Foundation KiKa

Sponsor details

Olympisch Stadion 11
Amsterdam
1076 DE
Netherlands
+31203458535
research@kika.nl

Sponsor type

Charity

Website

https://www.kika.nl/

ROR

https://ror.org/05gxzef39

Funder type

Other

Funder name

Investigator initiated and funded

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Publication and dissemination plan

Planned publication in a high-impact peer-reviewed journal.

Intention to publish date

01/05/2024

Individual participant data (IPD) sharing plan

Following publication of the study results, the full, anonymous de-identified patient dataset will be made available. Proposals should be directed to sct-study@amsterdamumc.nl to gain access. Data requestors will need to sign a data access agreement.

IPD sharing plan summary

Available on request

Study outputs

• 45299_Protocol.pdf