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NCT Number: NCT02709681

Hydroxyurea in the Treatment of Sickle Cell Disease

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

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

1 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Ospedali Civili Riuniti di Sciacca, Sciacca, Agrigento, Italy

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • none

Treatment and study plan

Physician standard-of-care in SCD patients

Other

Primary outcomes

  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 outcomes

  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).

Sponsors and collaborators

Lead sponsor

Società Italiana Talassemie ed Emoglobinopatie

Other

Registry information

Official study title

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

Important dates

Study start
2015
Primary completion
2016
Study completion
2017
First posted
Mar 16, 2016
Registry last updated
Oct 6, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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