Hydroxyurea in the Treatment of Sickle Cell Disease
- Conditions
- Sickle Cell Disease
- Interventions
- Other: Physician standard-of-care in SCD patients
- Registration Number
- NCT02709681
- Lead Sponsor
- Società Italiana Talassemie ed Emoglobinopatie
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 628
- Sickle Cell Disease affected patients
- 2-3 vaso-occlusive crisis and/or hospitalizations in the last year
Exclusion Criteria
- none
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description SCD patients Physician standard-of-care in SCD patients Patients followed in 32 Italian Centers.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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).
Trial Locations
- Locations (30)
Azienda Ospedaliero Universitaria Meyer
🇮🇹Firenze, Italy
Policlinico San Matteo
🇮🇹Pavia, Italy
Centro Emofilia e Medicina Trasfusionale - Pres. Ospedaliero
🇮🇹Ravenna, Italy
Azienda Ospedaliero-Universitaria
🇮🇹Bari, Italy
Ospedale "A. Perrino"
🇮🇹Brindisi, Italy
Ospedale Vittorio Emanuele
🇮🇹Catania, Italy
A.O. "Pugliese-Ciaccio"
🇮🇹Catanzaro, Italy
Clinica pediatrica Monza S. Gerardo
🇮🇹Monza, Italy
Ospedale Vittorio Emanuele III
🇮🇹Gela, Caltanisetta, Italy
Osp.San Giovanni Di Dio
🇮🇹Crotone, Italy
Clinica di Onco-Ematologia Pediatrica, Università di Padova
🇮🇹Padova, Italy
Ospedali Civili Riuniti di Sciacca
🇮🇹Sciacca, Agrigento, Italy
U.O. Oncoematologia Pediatrica Ospedali Civili
🇮🇹Brescia, Italy
Azienda Ospedaliera Universitaria Di Cagliari
🇮🇹Cagliari, Italy
University of Catania
🇮🇹Catania, Italy
E.O. Ospedali Galliera
🇮🇹Genova, Italy
Università degli Studi di Milano
🇮🇹Milano, Italy
Ospedale S. Eugenio - FF UOSD DH Talassemici
🇮🇹Roma, Italy
ARNAS "Garibaldi"
🇮🇹Catania, Italy
Azienda Ospedaliera San Gerardo di Monza
🇮🇹Monza, Italy
Aorn A. Cardarelli
🇮🇹Napoli, Italy
U.O.S. Talassemia P.O. Umberto I°
🇮🇹Siracusa, Italy
Policlinico G.B. Rossi
🇮🇹Verona, Italy
Azienda Ospedaliero-Universitaria di Modena - Policlinico
🇮🇹Modena, Italy
A.R.N.A.S. "Civico"
🇮🇹Palermo, Italy
A. O. Bianchi Melacrino Morelli
🇮🇹Reggio Calabria, Italy
Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
🇮🇹Palermo, Italy
Università Cattolica del Sacro Cuore - Policlinico A.Gemelli
🇮🇹Roma, Italy
Università degli Suidi di Torino
🇮🇹Torino, Italy
Azienda Ospedaliero-Universitaria di Parma
🇮🇹Parma, Italy