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

ISRCTN	ISRCTN17493302
DOI	https://doi.org/10.1186/ISRCTN17493302
Secondary identifying numbers	KiKa project 440

Submission date: 10/04/2024
Registration date: 28/04/2024
Last edited: 28/11/2024

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Cancer

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

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

Contact information

Prof Ernst van Heurn
Principal Investigator

Meibergdreef 9
Amsterdam
1105 AZ
Netherlands

Phone	+31 20 566 5693
Email	e.vanheurn@amsterdamumc.nl

Dr Lieke van Heurn
Public

Meibergdreef 9
Amsterdam
1105 AZ
Netherlands

Phone	+31 20 566 5693
Email	l.j.vanheurn@amsterdamumc.nl

Dr Joep Derik
Scientific

Meibergdreeg 9
Amsterdam
1105 AZ
Netherlands

Phone	+31 20 566 5693
Email	j.derikx@amsterdamumc.nl

Study information

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
Participant information sheet	No participant information sheet available
Scientific title	Malignant transformation and tumour recurrence in patients with SacroCoccygeal Teratoma: a global, retrospective cohort study
Study acronym	SCT-study
Study objectives	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).
Health condition(s) or problem(s) studied	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
Completion date	01/01/2022

Eligibility

Participant type(s)	Patient
Age group	All
Sex	Both
Target number of participants	1000
Total final enrolment	3612
Key inclusion criteria	All patients with SCT treated by each participating centre during a set time
Key exclusion criteria	Patients treated for sacrococcygeal teratoma before 1980
Date of first enrolment	01/12/2020
Date of final enrolment	01/01/2022

Locations

Countries of recruitment

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

Study participating centres

Hospital Garrahan Buenos Aires

-
Argentina

Hospital Italiano de Buenos Aires

-
Argentina

Medical University of Graz

-
Austria

Bangladesh Shishu Hospital & Institute

-
Bangladesh

Center of Pediatric Surgery of Belarusb

-
Belarus

Universitair Ziekenhuis Brussel

-
Belgium

Hôpital des Enfants Reine Fabiola, Université Libre de Bruxelles

-
Belgium

Federal University of São Paulo

-
Brazil

University Hospital "St. George"

-
Bulgaria

UMHATEM "N. I. Pirogov" Sofia

-
Bulgaria

Yaounde Gynaeco-Obstetric and Pediatric Hospital-faculty of medecine

-
Cameroon

McGill University, Montreal Children’s Hospital

-
Canada

Shriners Hospital for Children

-
Canada

The Hospital for Sick Children

-
Canada

Hospital de Niños Dr. Roberto del Rio

-
Chile

Guangzhou Women and Children’s Medical Center

510623
China

University Hospital centre Zagreb

-
Croatia

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

-
Croatia

Charles University and University Hospital Motol

150 06
Czech Republic

Rigshospitalet Copenhagen University Hospital

-
Denmark

Ain Shams University

-
Egypt

Kasr Al Ainy Faculty of Medicine

-
Egypt

Pediatric Surgery Cairo University

-
Egypt

Tallinn Children's Hospital

-
Estonia

St.Peter Specialized Hospital

-
Ethiopia

New Children´s Hospital

-
Finland

Hôpital des Enfants de Toulouse

-
France

CHU Rennes, Univ Rennes

-
France

Limoges University Hospital Center

-
France

University Hospital Angers

Angers
-
France

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

Marseille
-
France

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

Paris
-
France

Hopitaux Pédiatriques de Nice CHU-Lenval

Nice
-
France

University Hospital, Poitiers

Poitiers
-
France

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

Paris
-
France

University Hospital of Lille Jeanne de Flandre

Lille
-
France

Dr. von Hauner Children's Hospital, University Hospital

Munich
-
Germany

University Medical Center Mannheim, Heidelberg University

Mannheim
-
Germany

University of Leipzig

Leipzig
-
Germany

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

Berlin
-
Germany

University Hospital Frankfurt/M.

Frankfurt
-
Germany

Accra College of Medicine

Accra
-
Ghana

P. & A. Kyriakou Children’s Hospital

Athens
-
Greece

Semmelweis University

Budapest
-
Hungary

Medical School, University of Pécs

Pécs
-
Hungary

Li Ka Shing Faculty of Medicine, University of Hong Kong

-
Hong Kong

Amrita Institute Of medical sciences

Kerala
-
India

All India Institute of Medical Sciences

Jodhpur
-
India

All India Institute of Medical Sciences

New Dehli
-
India

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

Yogyakarta
-
Indonesia

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

Teheran
-
Iran

Alkhansaa teaching hospital

Mosul
-
Iraq

Children's Health Ireland at Crumlin

Dublin
-
Ireland

Tel Aviv Sourasky Medical Center

Tel Aviv
-
Israel

IRCCS Istituto Giannina Gaslini

Genoa
-
Italy

Children's Hospital Bambino Gesù-Research Institute

Rome
-
Italy

University of Padua

Padua
-
Italy

Regina Margherita Children’s Hospital

Turin
-
Italy

Bambino Gesu' Pediatric Hospital

Rome
-
Italy

Sant'Orsola Hospital, IRCSS, University of Bologna

Bologna
-
Italy

Meyer Children's Hospital IRCCS Florence

Florence
-
Italy

Fondazione IRCCS Policlinico San Matteo

Pavia
-
Italy

Cocody Teaching Hospital at Abidjan

Abidjan
-
Côte d'Ivoire

Kyoto Prefectural University of Medicine

Kyoto
-
Japan

Saitama Children's Medical Center

Saitama
-
Japan

National Center for Child Health and Development

Tokyo
-
Japan

Graduate School of Medical Sciences, Kyushu University

Fukuoka
-
Japan

Osaka City General Hospital

Osaka
-
Japan

Graduate School of Medical Sciences, Kyushu University,

Fukuoka
-
Japan

Tokyo Metropolitan Children's Medical Center

Tokyo
-
Japan

Riga Stradins University & Children's Clinical University Hospital

Riga
-
Latvia

Vaikų chirurgijos klinika, Lietuvos sveikatos mokslų universiteto ligoninė Kauno klinikos

Kaunsas
LT-50161
Lithuania

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

Skopje
-
North Macedonia

Faculty of Medicine, Universiti Kebangsaan Malaysia

Kuala Lumpur
-
Malaysia

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

Kuala Lumpur
-
Malaysia

Instituto Nacional de Pediatría

Mexico City
-
Mexico

Institute for children's diseases, Clinical Centre of Montenegro

Podgorica
-
Montenegro

University hospital Mohamed VI, Cadi Ayyad University,

Marrakesh
-
Morocco

University Medical Center Groningen

Groningen
-
Netherlands

Radboud University Medical Center, Amalia Children's Hospital

Nijmegen
-
Netherlands

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

Amsterdam
-
Netherlands

University Medical Centre Maastricht

Maastricht
-
Netherlands

Erasmus MC Sophia Children’s Hospital

Rotterdam
-
Netherlands

Amsterdam Public Health Research Institute, Amsterdam UMC, Vrije Universiteit Amsterdam

Amsterdam
1081 HV
Netherlands

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

Utrecht
-
Netherlands

Lagos University Teaching Hospital

Lagos
-
Nigeria

Jos University Teaching Hospital

Jos
PMB 2076
Nigeria

Nnamdi Azikiwe University Teaching Hospital

Nnewi
-
Nigeria

Abubakar Tafawa Balewa University Teaching Hospital

Bauchi
-
Nigeria

Oslo University Hospital

Oslo
4950
Norway

Liaquat National Hospital and Aga khan university

Karachi
-
Pakistan

Children Hospital, Shaheed Zulfiqar Ali Bhutto Medical University

Islamabad
-
Pakistan

University of Child Health Sciences

Lahore
-
Pakistan

Philippine Children's Medical Center

Quezon City
-
Philippines

Philippine General Hospital

Manila
-
Philippines

The Children's Memorial Health Institute

Warshaw
-
Poland

Medical University of Gdansk

Gdansk
-
Poland

Hospital Dona Estefania

Lisboa
-
Portugal

Hospital Dona Estefânia

Lisboa
-
Portugal

"Victor Babes" University of Medicine and Pharmacy Timisoara

Timisoara
-
Romania

FSAI "NMRC of Сhildren Health" MHRF

Moscow
-
Russian Federation

Volgograd State Medical University

Volgograd
-
Russian Federation

Irkutsk Regional Children’s Hospital

Irkutsk
-
Russian Federation

Stavropol State Medical University

Stavropol
-
Russian Federation

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

Belgrade
-
Serbia

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

Belgrade
-
Serbia

KK Women’s and Children’s Hospital

-
Singapore

National Institute of Children´s Diseases

Bratislava
-
Slovakia

University Medical centre Ljubljana

Ljubljana
-
Slovenia

Seoul National University Hospital

Seoul
03080
Korea, South

Asan Medical Center

Seoul
-
Korea, South

Inje University Busan Paik hospital

Busan
-
Korea, South

University Hospital Vall d´Hebron

Barcelona
-
Spain

Children's Hospital La Paz

La Paz
-
Spain

Virgen del Rocio Children's Hospital

Sevilla
-
Spain

Hospital Universitario Reina Sofía

Córdoba
-
Spain

Virgen de la Arrixaca University Clinical Hospital

Murcia
-
Spain

La Fe University and Polytechnic Hospital

Valencia
-
Spain

Lady Ridgeway Hospital

Colombo
-
Sri Lanka

Skane University Hospital Lund

Lund
-
Sweden

Karolinska University Hospital

Stockholm
-
Sweden

Astrid Lindgren´s Childrens Hospital

Stockholm
-
Sweden

Children Hospital

Damascus
-
Syria

Chang Gung University College of Medicine

Taoyuan
-
Taiwan

Queen Sirikit National Institute of Child Health

Bangkok
-
Thailand

Hedi Chaker Hospital University of medecine of Sfax

Sfax
-
Tunisia

Fattouma Bourguiba Hospital

Monastir
-
Tunisia

Ege University

Izmir
-
Türkiye

Istanbul Medeniyet University Faculty of Medicine)

Istanbul
-
Türkiye

Dr Sami Ulus Maternity and Children Health and Research Application Center

Ankara
-
Türkiye

Faculty of Medicine & Cancer Institute Ankara

Ankara
-
Türkiye

Hacettepe University, Faculty of Medicine

Ankara
-
Türkiye

National Specialized Children Hospital “Ohmatdyt“

Kyiv
-
Ukraine

Leeds Teaching Hospitals

Leeds
-
United Kingdom

Cambridge University Hospitals NHS Foundation Trust

Cambridge
-
United Kingdom

Great North Children's Hospital

Newcastle
-
United Kingdom

Birmingham Children hospital UK

Birmingham
-
United Kingdom

Royal Manchester Children's Hospital

Manchester
-
United Kingdom

The Royal London Hospital

London
-
United Kingdom

Bristol Royal Children's Hospital

Bristol
-
United Kingdom

UCL Great Ormond Street Institute of Child Health

London
-
United Kingdom

Faculty of Medicine, University of Southampton

Southampton
-
United Kingdom

Institute Of Life Course And Medical Sciences

Liverpool
-
United Kingdom

Royal Hospital for Sick Children

Edinburgh
-
United Kingdom

Nationwide Children’s Hospital

Columbus
-
United States of America

University of Utah School of Medicine

Salt Lake City
-
United States of America

University of Tennessee Health Science Center

Tennessee
-
United States of America

University of Chicago

Chicago
-
United States of America

Children’s Mercy Kansas City

Kansas City
-
United States of America

University Teaching Hospital of Lusaka

Lusaka
-
United States of America

Sponsor information

Foundation KiKa
Charity

Olympisch Stadion 11
Amsterdam
1076 DE
Netherlands

Phone	+31203458535
Email	research@kika.nl
Website	https://www.kika.nl/
"ROR"	https://ror.org/05gxzef39

Funders

Funder type

Other

Investigator initiated and funded

No information available

Results and Publications

Intention to publish date	01/05/2024
Individual participant data (IPD) Intention to share	Yes
IPD sharing plan summary	Available on request
Publication and dissemination plan	Planned publication in a high-impact peer-reviewed journal.
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.

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Protocol file			17/04/2024	No	No
Results article		06/09/2024	28/11/2024	Yes	No

Additional files

45299_Protocol.pdf

Editorial Notes

28/11/2024: Publication reference added.
10/04/2024: Study's existence confirmed by the Medical Ethical Board of Amsterdam University Medical Centre.