Sickle Cell Disease
Conditions
Keywords
Sickle Cell Disease, Hydroxyurea
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.
Interventions
OTHERPhysician standard-of-care in SCD patients
Sponsors
Società Italiana Talassemie ed Emoglobinopatie
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Sickle Cell Disease affected patients * 2-3 vaso-occlusive crisis and/or hospitalizations in the last year
Exclusion criteria
* none
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Changes in laboratory parameters is being assessed | 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 | 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 | 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | 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). |
Countries
Italy
Outcome results
None listed