Clinical trial NCT04599660: Study in Low Risk Gastrointestinal Stromal Tumor (GISTs)
Official title: Follow-up in Low Risk Gastrointestinal Stromal Tumors (GISTs) - Retrospective Analysis of Clinical Features and Outcomes
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: GIST
Keywords: GIST
Gastro Intestinal Stromal Tumor
Lead sponsor: Italian Sarcoma Group
Sponsor class: NETWORK
First posted: 2020-10-23
Enrollment: 700
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: patients affected by primary GIST at very-low and low risk of recurrence/progression, referred to participating Institutions between January 2000 and February 2020.

Brief summary: This is a multi-institutional retrospective study in order to identify the most relevant and advisable features of follow-up, and to explore its impact on principal clinical outcomes. Moreover, a dedicated effort will be pursued to identify the peculiar characteristics (if any) of patients that experienced recurrence of the disease.

The study will collect data about patients affected by primary GIST at very-low and low risk of recurrence/progression, referred to participating Institutions between January 2000 and February 2020

Detailed description: In the field of soft tissue sarcomas, Gastrointestinal Stromal Tumors (GIST) represents a really peculiar neoplasm for its biological and clinical properties. Surgery (if feasible) is the main therapeutic approach for all the patients with localized disease, while a pharmacological adjuvant treatment is reserved to those with a relevant risk of recurrence/progression.

After tumor removal, clinical and radiological follow-up is of central importance to early intercept recurrence and to evaluate the most correct subsequent therapeutic approach. In particular, for the group of patients with GIST at very-low and low risk of recurrence/progression, the evidences to support a specific follow-up program and its features are poor.

On the basis of the aforementioned considerations, we propose a multi-institutional retrospective study in order to identify the most relevant and advisable features of follow-up, and to explore its impact on principal clinical outcomes. Moreover, a dedicated effort will be pursued to identify the peculiar characteristics (if any) of patients that experienced recurrence of the disease.

The study will collect data about patients affected by primary GIST at very-low and low risk of recurrence/progression, referred to participating Institutions between January 2000 and February 2020

Eligibility criteria: Inclusion Criteria:

* \>18 years at diagnosis
* primary GIST removed by surgery or endoscopic procedures
* availability of medical data needed for the study
* very-low and low risk GIST defined as:

  * largest size of \< 3 cm (for all sites of origin)
  * gastric GIST with ≤ 5/50 High Power Fields (HPF) mitoses and ≤ 10 cm in the largest size
  * gastric GIST with \> 5/50 HPF mitoses and ≤ 5 cm in the largest size
  * intestinal GIST with ≤ 5/50 HPF mitoses and ≤ 5 cm in the largest size

Exclusion Criteria:

* Metastases at diagnosis.
* Previous treatment with imatinib

Primary outcomes:
measure: To describe the most relevant features of follow-up in very-low and low risk GIST patients
timeFrame: Change from diagnosis (baseline)
measure: To describe the most relevant features of follow-up in very-low and low risk GIST patients
timeFrame: At 1 year
measure: To describe the most relevant features of follow-up in very-low and low risk GIST patients
timeFrame: At 2 years
measure: To describe the most relevant features of follow-up in very-low and low risk GIST patients
timeFrame: At 3 years
measure: To describe the most relevant features of follow-up in very-low and low risk GIST patients
timeFrame: At 5 years
measure: To evaluate the onset of other neoplasms in very-low and low risk GIST patients
timeFrame: Chage from baseline (time of diagnosis) at 5 years
Secondary outcomes:
measure: To assess baseline clinical and disease-specific factors with possible impact on survival analyses.
timeFrame: Every 3 months (Month 3, Month 6, Month 9...) up to 5 years
measure: Recurrence-free survival (RFS)
timeFrame: Every 3 months (Month 3, Month 6, Month 9...) up to 5 years
measure: Post-recurrence progression-free survival (PR-PFS)
timeFrame: Every 3 months (Month 3, Month 6, Month 9...) up to 5 years
measure: Disease-Specific survival (DSS)
timeFrame: at 5 years
measure: Overall survival (OS).
timeFrame: at 5 years
Other outcomes:
Locations:
city: Bologna
country: Italy
facility: Azienda Ospedaliero-Universitaria Di Bologna
state: BO
city: Prato
country: Italy
facility: Nuovo Ospedale di Prato
state: Firenze
city: Milano
country: Italy
facility: Istituto Europeo di Oncologia
state: MI
city: Rozzano
country: Italy
facility: Istituto Clinico Humanitas
state: MI
city: Palermo
country: Italy
facility: Azienda Ospedaliera Universitaria Paolo Giaccone
state: PA
city: Aviano
country: Italy
facility: Centro di Riferimento Oncologico - Unit of Medical Oncology
state: Pordenone
city: Roma
country: Italy
facility: Policlinico Universitario Campus Biomedico
state: RM
city: Candiolo
country: Italy
facility: Fondazione del Piemonte per l'Oncologia IRCC Candiolo
state: Torino
city: Torino
country: Italy
facility: Ospedale San Giovanni Bosco
state: TO
city: Bologna
country: Italy
facility: Policlinico S.Orsola Malpighi - Unit of Medical Oncology
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
city: Milano
country: Italy
facility: Fondazione IRCCS INT Milano
city: Roma
country: Italy
facility: Istituto Nazionale Tumori Regina Elena - Unit of Medical Oncology I
Study officials:
affiliation: giovanni.grignani@ircc.it
name: Giovanni Grignani
role: STUDY_CHAIR
References:
citation: Joensuu H, Hohenberger P, Corless CL. Gastrointestinal stromal tumour. Lancet. 2013 Sep 14;382(9896):973-83. doi: 10.1016/S0140-6736(13)60106-3. Epub 2013 Apr 24.
citation: Gold JS, Gonen M, Gutierrez A, Broto JM, Garcia-del-Muro X, Smyrk TC, Maki RG, Singer S, Brennan MF, Antonescu CR, Donohue JH, DeMatteo RP. Development and validation of a prognostic nomogram for recurrence-free survival after complete surgical resection of localised primary gastrointestinal stromal tumour: a retrospective analysis. Lancet Oncol. 2009 Nov;10(11):1045-52. doi: 10.1016/S1470-2045(09)70242-6. Epub 2009 Sep 28.
citation: D'Ambrosio L, Palesandro E, Boccone P, Tolomeo F, Miano S, Galizia D, Manca A, Chiara G, Bertotto I, Russo F, Campanella D, Venesio T, Sangiolo D, Pignochino Y, Siatis D, De Simone M, Ferrero A, Pisacane A, Dei Tos AP, Aliberti S, Aglietta M, Grignani G. Impact of a risk-based follow-up in patients affected by gastrointestinal stromal tumour. Eur J Cancer. 2017 Jun;78:122-132. doi: 10.1016/j.ejca.2017.03.025. Epub 2017 Apr 24.
citation: Joensuu H, Reichardt P, Eriksson M, Sundby Hall K, Vehtari A. Gastrointestinal stromal tumor: a method for optimizing the timing of CT scans in the follow-up of cancer patients. Radiology. 2014 Apr;271(1):96-103. doi: 10.1148/radiol.13131040. Epub 2013 Nov 18.
citation: Rossi S, Miceli R, Messerini L, Bearzi I, Mazzoleni G, Capella C, Arrigoni G, Sonzogni A, Sidoni A, Toffolatti L, Laurino L, Mariani L, Vinaccia V, Gnocchi C, Gronchi A, Casali PG, Dei Tos AP. Natural history of imatinib-naive GISTs: a retrospective analysis of 929 cases with long-term follow-up and development of a survival nomogram based on mitotic index and size as continuous variables. Am J Surg Pathol. 2011 Nov;35(11):1646-56. doi: 10.1097/PAS.0b013e31822d63a7.
citation: Casali PG, Abecassis N, Aro HT, Bauer S, Biagini R, Bielack S, Bonvalot S, Boukovinas I, Bovee JVMG, Brodowicz T, Broto JM, Buonadonna A, De Alava E, Dei Tos AP, Del Muro XG, Dileo P, Eriksson M, Fedenko A, Ferraresi V, Ferrari A, Ferrari S, Frezza AM, Gasperoni S, Gelderblom H, Gil T, Grignani G, Gronchi A, Haas RL, Hassan B, Hohenberger P, Issels R, Joensuu H, Jones RL, Judson I, Jutte P, Kaal S, Kasper B, Kopeckova K, Krakorova DA, Le Cesne A, Lugowska I, Merimsky O, Montemurro M, Pantaleo MA, Piana R, Picci P, Piperno-Neumann S, Pousa AL, Reichardt P, Robinson MH, Rutkowski P, Safwat AA, Schoffski P, Sleijfer S, Stacchiotti S, Sundby Hall K, Unk M, Van Coevorden F, van der Graaf WTA, Whelan J, Wardelmann E, Zaikova O, Blay JY; ESMO Guidelines Committee and EURACAN. Gastrointestinal stromal tumours: ESMO-EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2018 Oct 1;29(Suppl 4):iv267. doi: 10.1093/annonc/mdy320. No abstract available.