Clinical trial NCT04460950: Observational Study for the Evaluation of Incidence of Familial AML/MDSs in Patients With Myeloid Neoplasms
Official title: A Retrospective and Prospective Multicentre Observational Study for the Evaluation of Incidence of Familial AML/MDSs in Patients With Myeloid Neoplasms (AML/MDS)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Acute Myeloid Leukemia
Familial Acute Myeloid Leukemia
Myelodysplastic Syndromes
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2020-07-08
Enrollment: 237
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients diagnoses with AML and MDSs from January 2014 to December 2022 will be registered. Study population will include patients with MDSs or AML with at least one family member of first or second degree affected by myeloid neoplasm or other hematological malignancy, or by solid tumors with onset at age \< 40 years.

Brief summary: Observational study aimed at evaluating the incidence of familial AML/MDSs in patients with de novo MDSs or AML with almost one relative affected by hematologic neoplasms and/or other cancers at young age (\< 40 years)

Detailed description: This is a multicenter, retrospective and prospective, observational study aims to collect clinical information on patients with familial AML/MDSs from January 2014 to December 2022. No intervention is expected.

The purpose of this study is to identify and characterize the patients with familial MDSs or AML (i.e. with relatives affected by hematologic neoplasms and/or other cancers at young age (\< 40 years)) or presence of signs, symptoms or laboratory tests compatible with one of the known syndromes with germinal susceptibility to AML/MDSs.

In order to estimate the incidence of familial AML/MDSs, a survey will be sent every year to all participating sites to collect only the number of the all diagnosis of AML/MDS. All patients will be followed until December 2023 in order to have at least 1 year of observation.

Eligibility criteria: Inclusion Criteria:

* Patient age \> 18 years old
* Diagnosis of AML or MDSs from January 2014 to December 2022
* Suspected familiarity for myeloid neoplasm: at least one family member of first or second degree affected by myeloid neoplasm or other hematological malignancy, or by solid tumors with onset at age \< 40 years or presence of signs, symptoms or laboratory tests compatible with one of the known syndromes with germinal susceptibility to AML/MDSs
* Signed written informed consent according to ICH/EU/GCP and national local laws.

Exclusion Criteria:

* none

Primary outcomes:
measure: Incidence of familial AML/MDSs
timeFrame: at 1 year
Secondary outcomes:
measure: Hematological characteristics of familial AML/MDSs
timeFrame: at 1 year
measure: Distribution of familial hematologic neoplasms and/or other cancers
timeFrame: at 1 year
measure: Incidence of transplantation
timeFrame: at 1 year
measure: Survival Distribution
timeFrame: at 1 year
measure: Standardized Mortality Ratio (SMR)
timeFrame: at 1 year
Other outcomes:
Locations:
city: Alessandria
country: Italy
facility: Aon Ss. Antonio E Biagio E C. Arrigo - Alessandria - Soc Ematologia
city: Ancona
country: Italy
facility: Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
city: Avellino
country: Italy
facility: Ao Di Rilievo Nazionale E Di Alta Specialità "San Giuseppe Moscati" - Avellino - Uoc Ematologia Con Unità Di Trapianto
city: Aversa
country: Italy
facility: Asl Caserta, Presidio Ospedaliero S.G.Moscati - Aversa - Uo Oncoematologia
city: Bologna
country: Italy
facility: Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
city: Brescia
country: Italy
facility: Asst Degli Spedali Civili Di Brescia - Ssvd Centro Trapianti Midollo Per Adulti - Cattedra Di Ematologia
city: Brescia
country: Italy
facility: Asst Degli Spedali Civili Di Brescia - Uo Ematologia
city: Cagliari
country: Italy
facility: Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo
city: Foggia
country: Italy
facility: Aou Ospedali Riuniti - Foggia - Uoc Ematologia
city: Lecce
country: Italy
facility: Asl Lecce, Ospedale 'V. Fazzi' - Uo Ematologia
city: Messina
country: Italy
facility: Ao Ospedali Riuniti "Papardo Piemonte" - Po Papardo - Messina - Sc Ematologia
city: Milano
country: Italy
facility: Asst Grande Ospedale Metropolitano Niguarda - Milano - Sc Ematologia
city: Milano
country: Italy
facility: Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
city: Orbassano
country: Italy
facility: Aou San Luigi Gonzaga - Orbassano - Scdu Ematologia Generale E Oncoematologia
city: Pagani
country: Italy
facility: Asl Salerno, Presidio Ospedaliero Tortora Pagani - Ematologia
city: Palermo
country: Italy
facility: Aou Policlinico P. Giaccone - Palermo - Uo Ematologia
city: Parma
country: Italy
facility: Aou Di Parma - Sc Ematologia E Centro Trapianti Midollo Osseo
city: Perugia
country: Italy
facility: Ao Di Perugia, Ospedale S. Maria Della Misericordia - Ematologia E Trapianto Midollo Osseo
city: Pisa
country: Italy
facility: Aou Pisana - Uo Ematologia Universitaria
city: Reggio Emilia
country: Italy
facility: Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia
city: Rimini
country: Italy
facility: Ausl Della Romagna, Ospedale "Infermi" - Rimini - Uo Ematologia
city: Roma
country: Italy
facility: Asl Roma 2, Ospedale S. Eugenio- Ospedale S.Eugenio - Uoc Ematologia
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli Irccs - Roma - Area Ematologica
city: San Giovanni Rotondo
country: Italy
facility: Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia
city: Sassari
country: Italy
facility: Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia
city: Torino
country: Italy
facility: Aou Città Della Salute E Della Scienza, Ospedale S. Giovanni Battista Molinette - Torino - Sc Ematologia - Università Degli Studi Di Torino
city: Udine
country: Italy
facility: Asui Di Udine - Presidio Ospedaliero "Santa Maria Della Misericordia" - Clinica Ematologica
Study officials:
References: