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Hydroxyurea in the Treatment of Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT02709681
Recruitment Status : Completed
First Posted : March 16, 2016
Last Update Posted : October 6, 2017
Sponsor:
Information provided by (Responsible Party):
Società Italiana Talassemie ed Emoglobinopatie

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

Condition or disease Intervention/treatment
Sickle Cell Disease Other: Physician standard-of-care in SCD patients

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.

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Study Type : Observational
Actual Enrollment : 628 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Hydroxyurea in Sickle Cell Disease: a Large Nation-wide Cohort Study From Italy
Study Start Date : November 2015
Actual Primary Completion Date : November 2016
Actual Study Completion Date : July 2017

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

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



Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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


Secondary Outcome Measures :
  1. 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).



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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Criteria

Inclusion Criteria:

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

Exclusion Criteria

  • none

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02709681


Locations
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Sponsors and Collaborators
Società Italiana Talassemie ed Emoglobinopatie
Investigators
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Principal Investigator: Paolo Rigano, MD Servico Integrado de Tecnicas Endovasculares
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Società Italiana Talassemie ed Emoglobinopatie
ClinicalTrials.gov Identifier: NCT02709681    
Other Study ID Numbers: SocietaITE
First Posted: March 16, 2016    Key Record Dates
Last Update Posted: October 6, 2017
Last Verified: March 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Società Italiana Talassemie ed Emoglobinopatie:
Sickle Cell Disease
Hydroxyurea
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn