Hydroxyurea in the Treatment of Sickle Cell Disease

Study Overview
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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.

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

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Other: Physician standard-of-care in SCD patients


Participation Criteria
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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

Ages Eligible for Study
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Minimum Age: 1 Year

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| SCD patients<br>Patients followed in 32 Italian Centers. | Other: Physician standard-of-care in SCD patients<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in laboratory parameters is being assessed | 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 | 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 | 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 | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Sickle Cell Disease, Hydroxyurea