Hydroxyurea in the Treatment of Sickle Cell Disease

October 5, 2017 updated by: Società Italiana Talassemie ed Emoglobinopatie

Hydroxyurea in Sickle Cell Disease: a Large Nation-wide Cohort Study From Italy

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

628

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Bari, Italy
        • Azienda Ospedaliero-Universitaria
      • Brescia, Italy, 25100
        • U.O. Oncoematologia Pediatrica Ospedali Civili
      • Brindisi, Italy, 72100
        • Ospedale "A. Perrino"
      • Cagliari, Italy, 09121
        • Azienda Ospedaliera Universitaria di Cagliari
      • Catania, Italy, 95123
        • Ospedale Vittorio Emanuele
      • Catania, Italy, 95124
        • ARNAS "Garibaldi"
      • Catania, Italy, 95124
        • University of Catania
      • Catanzaro, Italy, 88100
        • A.O. "Pugliese-Ciaccio"
      • Crotone, Italy
        • Osp.San Giovanni Di Dio
      • Firenze, Italy, 50139
        • Azienda Ospedaliero Universitaria Meyer
      • Genova, Italy, 16128
        • E.O. Ospedali Galliera
      • Milano, Italy, 20122
        • Universita Degli Studi Di Milano
      • Modena, Italy, 41124
        • Azienda Ospedaliero-Universitaria di Modena - Policlinico
      • Monza, Italy, 20900
        • Azienda Ospedaliera San Gerardo di Monza
      • Monza, Italy, 20052
        • Clinica pediatrica Monza S. Gerardo
      • Napoli, Italy
        • AORN A. Cardarelli
      • Padova, Italy, 35128
        • Clinica di Onco-Ematologia Pediatrica, Università di Padova
      • Palermo, Italy, 90146
        • Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
      • Palermo, Italy, 90100
        • A.R.N.A.S. "Civico"
      • Parma, Italy, 43126
        • Azienda Ospedaliero-Universitaria di Parma
      • Pavia, Italy, 27100
        • Policlinico San Matteo
      • Ravenna, Italy, 48121
        • Centro Emofilia e Medicina Trasfusionale - Pres. Ospedaliero
      • Reggio Calabria, Italy, 89100
        • A. O. Bianchi Melacrino Morelli
      • Roma, Italy, 00144
        • Ospedale S. Eugenio - FF UOSD DH Talassemici
      • Roma, Italy, 00168
        • Università Cattolica del Sacro Cuore - Policlinico A.Gemelli
      • Siracusa, Italy, 96100
        • U.O.S. Talassemia P.O. Umberto I°
      • Torino, Italy, 90146
        • Università degli Suidi di Torino
      • Verona, Italy, 37122
        • Policlinico G.B. Rossi
    • Agrigento
      • Sciacca, Agrigento, Italy, 92019
        • Ospedali Civili Riuniti di Sciacca
    • Caltanisetta
      • Gela, Caltanisetta, Italy, 93012
        • Ospedale Vittorio Emanuele III

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

Inclusion Criteria:

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

Exclusion Criteria

  • none

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
SCD patients
Patients followed in 32 Italian Centers.
Other: Physician standard-of-care in SCD patients

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in laboratory parameters is being assessed
Time Frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Increases or decreases in percentage of total hemoglobin, fetal hemoglobin and hemoglobin S level will be assessed. Changing of white blood cells and platelets counts, lactate dehydrogenase, bilirubin, aspartate aminotransferase and serum creatinine level will be also evaluated.
An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Changes in complication rates is being assessed
Time Frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Changing in the incidence of stroke, silent cerebral infraction, acute chest syndrome, vaso-occlusive crisis, pulmonary hypertension, leg ulcers, bone necrosis and kidney injury will be evaluated.
An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Rate of hospitalizations
Time Frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Changing in rate of hospitalizations before and after start hydroxyurea therapy
An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changing in the incidence of complications according to specific subgroups
Time Frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
We also stratified the analysis according to age (≥18 years), origin (Italian and African), genotype (βS/β0, βS/β+ and βS/βS) duration of hydroxyurea treatment (≥10 years) and hydroxyurea dose(≥15 mg/kg/day).
An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Società Italiana Talassemie ed Emoglobinopatie

Investigators

  • Principal Investigator: Paolo Rigano, MD, Servico Integrado de Tecnicas Endovasculares

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2015

Primary Completion (Actual)

November 1, 2016

Study Completion (Actual)

July 1, 2017

Study Registration Dates

First Submitted

March 1, 2016

First Submitted That Met QC Criteria

March 10, 2016

First Posted (Estimate)

March 16, 2016

Study Record Updates

Last Update Posted (Actual)

October 6, 2017

Last Update Submitted That Met QC Criteria

October 5, 2017

Last Verified

March 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SocietaITE

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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