The observational clinical registry (cohort design) of the
European Reference Network on Rare Adult Solid Cancers: The
protocol for the rare head and neck cancers

J 2023

The observational clinical registry (cohort design) of the European Reference Network on Rare Adult Solid Cancers: The protocol for the rare head and neck cancers

TRAMA, Annalisa, Lisa LICITRA, Stefano CAVALIERI, Simone BONFARNUZZO, Paolo BAILI et. al.

Basic information

Original name

The observational clinical registry (cohort design) of the European Reference Network on Rare Adult Solid Cancers: The protocol for the rare head and neck cancers

Authors

TRAMA, Annalisa, Lisa LICITRA, Stefano CAVALIERI, Simone BONFARNUZZO, Paolo BAILI, Antonio CIARFELLA, Pablo PARENTE, Giovanni ALMADORI, Mohssen ANSARIN, Almalina BACIGALUPO, Philipp BAUMEISTER, Bertrand BAUJAT, Paolo BOSSI, Elisa CAVALERA, Maria Cecilia CERCATO, Francois DIELEMAN, Nicolas FAKHRY, Virginia FERRARESI, Francesca GAINO, Danilo GALIZIA, Jana HALÁMKOVÁ (203 Czech Republic, belonging to the institution), Elina HALME, Jose HARDILLO, Benedikt HOFAUER, Emma KINLOCH, Lorenzo LIVI, Laura Deborah LOCATI, Stefan MATTHEIS, Giuseppe MERCANTE, Aurora MIRABILE, Gabriele MOLTENI, Ester ORLANDI, Roberto PERSIO, Stefania SCIALLERO, Ludi SMEELE, Marta TAGLIABUE, Valentino VALENTINI, Van Harpen CARLA, Christoph Benedikt WESTPHALEN and Laura BOTTA

Edition

Plos one, San Francisco, Public Library of Science, 2023, 1932-6203

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30204 Oncology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 3.700 in 2022

RIV identification code

RIV/00216224:14110/23:00133907

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1371/journal.pone.0283071

UT WoS

000985134400045

Keywords in English

Rare Adult Solid Cancers

Abstract

V originále

IntroductionCare for head and neck cancers is complex in particular for the rare ones. Knowledge is limited and histological heterogeneity adds complexity to the rarity. There is a wide consensus that to support clinical research on rare cancer, clinical registries should be developed within networks specializing in rare cancers. In the EU, a unique opportunity is provided by the European Reference Networks (ERN). The ERN EURACAN is dedicated to rare adults solid cancers, here we present the protocol of the EURACAN registry on rare head and neck cancers (ClinicalTrials.gov Identifier: NCT05483374). Study designRegistry-based cohort study including only people with rare head and neck cancers. Objectives to help describe the natural history of rare head and neck cancers;to evaluate factors that influence prognosis;to assess treatment effectiveness;to measure indicators of quality of care. MethodsSettings and participantsIt is an hospital based registry established in hospitals with expertise in head and neck cancers. Only adult patients with epithelial tumours of nasopharynx; nasal cavity and paranasal sinuses; salivary gland cancer in large and small salivary glands; and middle ear will be included in the registry. This registry won't select a sample of patients. Each patient in the facility who meets the above mentioned inclusion criteria will be followed prospectively and longitudinally with follow-up at cancer progression and / or cancer relapse or patient death. It is a secondary use of data which will be collected from the clinical records. The data collected for the registry will not entail further examinations or admissions to the facility and/or additional appointments to those normally provided for the patient follow-up.VariablesData will be collected on patient characteristics (eg. patient demographics, lifestyle, medical history, health status); exposure data (eg. disease, procedures, treatments of interest) and outcomes (e.g. survival, progression, progression-free survival, etc.). In addition, data on potential confounders (e.g. comorbidity; functional status etc.) will be also collected.Statistical methodsThe data analyses will include descriptive statistics showing patterns of patients' and cancers' variables and indicators describing the quality of care. Multivariable Cox's proportional hazards model and Hazard ratios (HR) for all-cause or cause specific mortality will be used to determine independent predictors of overall survival, recurrence etc. Variables to include in the multivariable regression model will be selected based on the results of univariable analysis. The role of confounding or effect modifiers will be evaluated using stratified analysis or sensitivity analysis. To assess treatment effectiveness, multivariable models with propensity score adjustment and progression-free survival will be performed. Adequate statistical (eg. marginal structural model) methods will be used if time-varying treatments/confounders and confounding by indication (selective prescribing) will be present. ResultsThe registry initiated recruiting in May 2022. The estimated completion date is December 2030 upon agreement on the achievement of all the registry objectives. As of October 2022, the registry is recruiting. There will be a risk of limited representativeness due to the hospital-based nature of the registry and to the fact that hospital contributing to the registry are expert centres for these rare cancers.

Citovat

TRAMA, Annalisa, Lisa LICITRA, Stefano CAVALIERI, Simone BONFARNUZZO, Paolo BAILI, Antonio CIARFELLA, Pablo PARENTE, Giovanni ALMADORI, Mohssen ANSARIN, Almalina BACIGALUPO, Philipp BAUMEISTER, Bertrand BAUJAT, Paolo BOSSI, Elisa CAVALERA, Maria Cecilia CERCATO, Francois DIELEMAN, Nicolas FAKHRY, Virginia FERRARESI, Francesca GAINO, Danilo GALIZIA, Jana HALÁMKOVÁ, Elina HALME, Jose HARDILLO, Benedikt HOFAUER, Emma KINLOCH, Lorenzo LIVI, Laura Deborah LOCATI, Stefan MATTHEIS, Giuseppe MERCANTE, Aurora MIRABILE, Gabriele MOLTENI, Ester ORLANDI, Roberto PERSIO, Stefania SCIALLERO, Ludi SMEELE, Marta TAGLIABUE, Valentino VALENTINI, Van Harpen CARLA, Christoph Benedikt WESTPHALEN and Laura BOTTA. The observational clinical registry (cohort design) of the European Reference Network on Rare Adult Solid Cancers: The protocol for the rare head and neck cancers. Plos one. San Francisco: Public Library of Science, 2023, vol. 18, No 3, p. 1-13. ISSN 1932-6203. Available from: https://dx.doi.org/10.1371/journal.pone.0283071.

@article{2388217,
   author = {Trama, Annalisa and Licitra, Lisa and Cavalieri, Stefano and Bonfarnuzzo, Simone and Baili, Paolo and Ciarfella, Antonio and Parente, Pablo and Almadori, Giovanni and Ansarin, Mohssen and Bacigalupo, Almalina and Baumeister, Philipp and Baujat, Bertrand and Bossi, Paolo and Cavalera, Elisa and Cercato, Maria Cecilia and Dieleman, Francois and Fakhry, Nicolas and Ferraresi, Virginia and Gaino, Francesca and Galizia, Danilo and Halámková, Jana and Halme, Elina and Hardillo, Jose and Hofauer, Benedikt and Kinloch, Emma and Livi, Lorenzo and Locati, Laura Deborah and Mattheis, Stefan and Mercante, Giuseppe and Mirabile, Aurora and Molteni, Gabriele and Orlandi, Ester and Persio, Roberto and Sciallero, Stefania and Smeele, Ludi and Tagliabue, Marta and Valentini, Valentino and Carla, Van Harpen and Westphalen, Christoph Benedikt and Botta, Laura},
   article_location = {San Francisco},
   article_number = {3},
   doi = {http://dx.doi.org/10.1371/journal.pone.0283071},
   keywords = {Rare Adult Solid Cancers},
   language = {eng},
   issn = {1932-6203},
   journal = {Plos one},
   title = {The observational clinical registry (cohort design) of the European Reference Network on Rare Adult Solid Cancers: The protocol for the rare head and neck cancers},
   url = {https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0283071},
   volume = {18},
   year = {2023}
}

TY  - JOUR
ID  - 2388217
AU  - Trama, Annalisa - Licitra, Lisa - Cavalieri, Stefano - Bonfarnuzzo, Simone - Baili, Paolo - Ciarfella, Antonio - Parente, Pablo - Almadori, Giovanni - Ansarin, Mohssen - Bacigalupo, Almalina - Baumeister, Philipp - Baujat, Bertrand - Bossi, Paolo - Cavalera, Elisa - Cercato, Maria Cecilia - Dieleman, Francois - Fakhry, Nicolas - Ferraresi, Virginia - Gaino, Francesca - Galizia, Danilo - Halámková, Jana - Halme, Elina - Hardillo, Jose - Hofauer, Benedikt - Kinloch, Emma - Livi, Lorenzo - Locati, Laura Deborah - Mattheis, Stefan - Mercante, Giuseppe - Mirabile, Aurora - Molteni, Gabriele - Orlandi, Ester - Persio, Roberto - Sciallero, Stefania - Smeele, Ludi - Tagliabue, Marta - Valentini, Valentino - Carla, Van Harpen - Westphalen, Christoph Benedikt - Botta, Laura
PY  - 2023
TI  - The observational clinical registry (cohort design) of the European Reference Network on Rare Adult Solid Cancers: The protocol for the rare head and neck cancers
JF  - Plos one
VL  - 18
IS  - 3
SP  - 1-13
EP  - 1-13
PB  - Public Library of Science
SN  - 19326203
KW  - Rare Adult Solid Cancers
UR  - https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0283071
N2  - IntroductionCare for head and neck cancers is complex in particular for the rare ones. Knowledge is limited and histological heterogeneity adds complexity to the rarity. There is a wide consensus that to support clinical research on rare cancer, clinical registries should be developed within networks specializing in rare cancers. In the EU, a unique opportunity is provided by the European Reference Networks (ERN). The ERN EURACAN is dedicated to rare adults solid cancers, here we present the protocol of the EURACAN registry on rare head and neck cancers (ClinicalTrials.gov Identifier: NCT05483374). Study designRegistry-based cohort study including only people with rare head and neck cancers. Objectives to help describe the natural history of rare head and neck cancers;to evaluate factors that influence prognosis;to assess treatment effectiveness;to measure indicators of quality of care. MethodsSettings and participantsIt is an hospital based registry established in hospitals with expertise in head and neck cancers. Only adult patients with epithelial tumours of nasopharynx; nasal cavity and paranasal sinuses; salivary gland cancer in large and small salivary glands; and middle ear will be included in the registry. This registry won't select a sample of patients. Each patient in the facility who meets the above mentioned inclusion criteria will be followed prospectively and longitudinally with follow-up at cancer progression and / or cancer relapse or patient death. It is a secondary use of data which will be collected from the clinical records. The data collected for the registry will not entail further examinations or admissions to the facility and/or additional appointments to those normally provided for the patient follow-up.VariablesData will be collected on patient characteristics (eg. patient demographics, lifestyle, medical history, health status); exposure data (eg. disease, procedures, treatments of interest) and outcomes (e.g. survival, progression, progression-free survival, etc.). In addition, data on potential confounders (e.g. comorbidity; functional status etc.) will be also collected.Statistical methodsThe data analyses will include descriptive statistics showing patterns of patients' and cancers' variables and indicators describing the quality of care. Multivariable Cox's proportional hazards model and Hazard ratios (HR) for all-cause or cause specific mortality will be used to determine independent predictors of overall survival, recurrence etc. Variables to include in the multivariable regression model will be selected based on the results of univariable analysis. The role of confounding or effect modifiers will be evaluated using stratified analysis or sensitivity analysis. To assess treatment effectiveness, multivariable models with propensity score adjustment and progression-free survival will be performed. Adequate statistical (eg. marginal structural model) methods will be used if time-varying treatments/confounders and confounding by indication (selective prescribing) will be present. ResultsThe registry initiated recruiting in May 2022. The estimated completion date is December 2030 upon agreement on the achievement of all the registry objectives. As of October 2022, the registry is recruiting. There will be a risk of limited representativeness due to the hospital-based nature of the registry and to the fact that hospital contributing to the registry are expert centres for these rare cancers.
ER  -

TRAMA, Annalisa, Lisa LICITRA, Stefano CAVALIERI, Simone BONFARNUZZO, Paolo BAILI, Antonio CIARFELLA, Pablo PARENTE, Giovanni ALMADORI, Mohssen ANSARIN, Almalina BACIGALUPO, Philipp BAUMEISTER, Bertrand BAUJAT, Paolo BOSSI, Elisa CAVALERA, Maria Cecilia CERCATO, Francois DIELEMAN, Nicolas FAKHRY, Virginia FERRARESI, Francesca GAINO, Danilo GALIZIA, Jana HALÁMKOVÁ, Elina HALME, Jose HARDILLO, Benedikt HOFAUER, Emma KINLOCH, Lorenzo LIVI, Laura Deborah LOCATI, Stefan MATTHEIS, Giuseppe MERCANTE, Aurora MIRABILE, Gabriele MOLTENI, Ester ORLANDI, Roberto PERSIO, Stefania SCIALLERO, Ludi SMEELE, Marta TAGLIABUE, Valentino VALENTINI, Van Harpen CARLA, Christoph Benedikt WESTPHALEN and Laura BOTTA. The observational clinical registry (cohort design) of the European Reference Network on Rare Adult Solid Cancers: The protocol for the rare head and neck cancers. \textit{Plos one}. San Francisco: Public Library of Science, 2023, vol.~18, No~3, p.~1-13. ISSN~1932-6203. Available from: https://dx.doi.org/10.1371/journal.pone.0283071.

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