Clinical trial NCT02709681: Hydroxyurea in the Treatment of Sickle Cell Disease
Official title: Hydroxyurea in Sickle Cell Disease: a Large Nation-wide Cohort Study From Italy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Keywords: Sickle Cell Disease
Hydroxyurea
Lead sponsor: Società Italiana Talassemie ed Emoglobinopatie
Sponsor class: OTHER
First posted: 2016-03-16
Enrollment: 628
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: A population of SCD (Homozigous HbS and Beta Thal /HbS) patients followed in 32 Italian Centers was included in this analysis.

Brief summary: This is a retrospective cohort study of Sickle Cell Disease (SCD) patients attending 32 treatment centers across Italy. The aim of this study will be to report the Italian experience with the use of hydroxyurea in a large cohort of SCD patients and to evaluate the benefits and safety of this intervention for the prevention and management of a wide range of clinical morbidities

Detailed description: The indication for hydroxyurea initiation was 2-3 vaso-occlusive crisis and/or hospitalizations in the last year.

The study will analyze demographics (age and gender), origin, genotype, clinical phenotype (vaso-occlusive or hemolytic), transfusion history (including exchange), and folic acid use, average laboratory values up to three years pre-hydroxyurea and for the period post-hydroxyurea therapy including total hemoglobin level, fetal hemoglobin level, hemoglobin S level, white blood count, platelet count, lactate dehydrogenase level, total and direct bilirubin levels, aspartate and alanine aminotransferase levels, and serum creatinine level.

The incidence of complications pre- and post-hydroxyurea therapy will be also analyzed including: stroke, silent cerebral infraction, acute chest syndrome, vaso-occlusive crisis, hospitalization, pulmonary hypertension, leg ulcers, bone necrosis, and kidney injury. Safety data included adverse events as reported by the treating physician and the incidence of malignancy or death as well as pregnancy incidents and their outcomes will be also pointed out.

Eligibility criteria: Inclusion Criteria:

* Sickle Cell Disease affected patients
* 2-3 vaso-occlusive crisis and/or hospitalizations in the last year

Exclusion Criteria

* none

Primary outcomes:
measure: Changes in laboratory parameters is being assessed
timeFrame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
measure: Changes in complication rates is being assessed
timeFrame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
measure: Rate of hospitalizations
timeFrame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Secondary outcomes:
measure: Changing in the incidence of complications according to specific subgroups
timeFrame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Other outcomes:
Locations:
city: Sciacca
country: Italy
facility: Ospedali Civili Riuniti di Sciacca
state: Agrigento
city: Gela
country: Italy
facility: Ospedale Vittorio Emanuele III
state: Caltanisetta
city: Bari
country: Italy
facility: Azienda Ospedaliero-Universitaria
city: Brescia
country: Italy
facility: U.O. Oncoematologia Pediatrica Ospedali Civili
city: Brindisi
country: Italy
facility: Ospedale "A. Perrino"
city: Cagliari
country: Italy
facility: Azienda Ospedaliera Universitaria Di Cagliari
city: Catania
country: Italy
facility: Ospedale Vittorio Emanuele
city: Catania
country: Italy
facility: ARNAS "Garibaldi"
city: Catania
country: Italy
facility: University of Catania
city: Catanzaro
country: Italy
facility: A.O. "Pugliese-Ciaccio"
city: Crotone
country: Italy
facility: Osp.San Giovanni Di Dio
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Meyer
city: Genova
country: Italy
facility: E.O. Ospedali Galliera
city: Milano
country: Italy
facility: Università degli Studi di Milano
city: Modena
country: Italy
facility: Azienda Ospedaliero-Universitaria di Modena - Policlinico
city: Monza
country: Italy
facility: Clinica pediatrica Monza S. Gerardo
city: Monza
country: Italy
facility: Azienda Ospedaliera San Gerardo di Monza
city: Napoli
country: Italy
facility: Aorn A. Cardarelli
city: Padova
country: Italy
facility: Clinica di Onco-Ematologia Pediatrica, Università di Padova
city: Palermo
country: Italy
facility: A.R.N.A.S. "Civico"
city: Palermo
country: Italy
facility: Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
city: Parma
country: Italy
facility: Azienda Ospedaliero-Universitaria di Parma
city: Pavia
country: Italy
facility: Policlinico San Matteo
city: Ravenna
country: Italy
facility: Centro Emofilia e Medicina Trasfusionale - Pres. Ospedaliero
city: Reggio Calabria
country: Italy
facility: A. O. Bianchi Melacrino Morelli
city: Roma
country: Italy
facility: Ospedale S. Eugenio - FF UOSD DH Talassemici
city: Roma
country: Italy
facility: Università Cattolica del Sacro Cuore - Policlinico A.Gemelli
city: Siracusa
country: Italy
facility: U.O.S. Talassemia P.O. Umberto I°
city: Torino
country: Italy
facility: Università degli Suidi di Torino
city: Verona
country: Italy
facility: Policlinico G.B. Rossi
Study officials:
affiliation: Servico Integrado de Tecnicas Endovasculares
name: Paolo Rigano, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Rigano P, De Franceschi L, Sainati L, Piga A, Piel FB, Cappellini MD, Fidone C, Masera N, Palazzi G, Gianesin B, Forni GL; Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators. Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent. Blood Cells Mol Dis. 2018 Mar;69:82-89. doi: 10.1016/j.bcmd.2017.08.017. Epub 2017 Oct 9.