Hydroxyurea in Sickle Cell Disease: a Large Nation-wide Cohort Study From Italy
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Sickle Cell Disease
- Sponsor
- Società Italiana Talassemie ed Emoglobinopatie
- Enrollment
- 628
- Locations
- 30
- Primary Endpoint
- Changes in laboratory parameters is being assessed
- Status
- Completed
- Last Updated
- 8 years ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Sickle Cell Disease affected patients
- •2-3 vaso-occlusive crisis and/or hospitalizations in the last year
- •Exclusion Criteria
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
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.
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.
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
- Changing in the incidence of complications according to specific subgroups(An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy)