NCT02709681

Hydroxyurea in the Treatment of Sickle Cell Disease

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 628 (actual)
• Sponsor: Società Italiana Talassemie ed Emoglobinopatie · Academic / Other
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

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.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
OTHER	Physician standard-of-care in SCD patients

Timeline

• Start date: 2015-11-01
• Primary completion: 2016-11-01
• Completion: 2017-07-01
• First posted: 2016-03-16
• Last updated: 2017-10-06

Locations

30 sites across 1 country: Italy