Hydroxyurea in the Treatment of Sickle Cell Disease

NCT ID: NCT02709681

Last Updated: 2017-10-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

628 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-11-30

Study Completion Date

2017-07-31

Brief Summary

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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

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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.

Conditions

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Sickle Cell Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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SCD patients

Patients followed in 32 Italian Centers.

Physician standard-of-care in SCD patients

Intervention Type OTHER

Interventions

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Physician standard-of-care in SCD patients

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* none
Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Società Italiana Talassemie ed Emoglobinopatie

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Paolo Rigano, MD

Role: PRINCIPAL_INVESTIGATOR

Servico Integrado de Tecnicas Endovasculares

Locations

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Ospedali Civili Riuniti di Sciacca

Sciacca, Agrigento, Italy

Site Status

Ospedale Vittorio Emanuele III

Gela, Caltanisetta, Italy

Site Status

Azienda Ospedaliero-Universitaria

Bari, , Italy

Site Status

U.O. Oncoematologia Pediatrica Ospedali Civili

Brescia, , Italy

Site Status

Ospedale "A. Perrino"

Brindisi, , Italy

Site Status

Azienda Ospedaliera Universitaria Di Cagliari

Cagliari, , Italy

Site Status

Ospedale Vittorio Emanuele

Catania, , Italy

Site Status

ARNAS "Garibaldi"

Catania, , Italy

Site Status

University of Catania

Catania, , Italy

Site Status

A.O. "Pugliese-Ciaccio"

Catanzaro, , Italy

Site Status

Osp.San Giovanni Di Dio

Crotone, , Italy

Site Status

Azienda Ospedaliero Universitaria Meyer

Florence, , Italy

Site Status

E.O. Ospedali Galliera

Genova, , Italy

Site Status

Università degli Studi di Milano

Milan, , Italy

Site Status

Azienda Ospedaliero-Universitaria di Modena - Policlinico

Modena, , Italy

Site Status

Clinica pediatrica Monza S. Gerardo

Monza, , Italy

Site Status

Azienda Ospedaliera San Gerardo di Monza

Monza, , Italy

Site Status

Aorn A. Cardarelli

Napoli, , Italy

Site Status

Clinica di Onco-Ematologia Pediatrica, Università di Padova

Padua, , Italy

Site Status

A.R.N.A.S. "Civico"

Palermo, , Italy

Site Status

Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello

Palermo, , Italy

Site Status

Azienda Ospedaliero-Universitaria di Parma

Parma, , Italy

Site Status

Policlinico San Matteo

Pavia, , Italy

Site Status

Centro Emofilia e Medicina Trasfusionale - Pres. Ospedaliero

Ravenna, , Italy

Site Status

A. O. Bianchi Melacrino Morelli

Reggio Calabria, , Italy

Site Status

Ospedale S. Eugenio - FF UOSD DH Talassemici

Roma, , Italy

Site Status

Università Cattolica del Sacro Cuore - Policlinico A.Gemelli

Roma, , Italy

Site Status

U.O.S. Talassemia P.O. Umberto I°

Syracuse, , Italy

Site Status

Università degli Suidi di Torino

Torino, , Italy

Site Status

Policlinico G.B. Rossi

Verona, , Italy

Site Status

Countries

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Italy

References

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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.

Reference Type DERIVED
PMID: 29107441 (View on PubMed)

Other Identifiers

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SocietaITE

Identifier Type: -

Identifier Source: org_study_id