Hydroxyurea in the Treatment of Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02709681
• Other study ID #: SocietaITE
• Status: Completed
• Phase: N/A
• First received: March 1, 2016
• Last updated: October 5, 2017
• Start date: November 2015
• Est. completion date: July 2017

Study information

• Verified date: March 2016
• Source: Società Italiana Talassemie ed Emoglobinopatie
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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 information / eligibility

• Status: Completed
• Enrollment: 628
• Est. completion date: July 2017
• Est. primary completion date: November 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

Inclusion Criteria:

• Sickle Cell Disease affected patients

• 2-3 vaso-occlusive crisis and/or hospitalizations in the last year

Exclusion Criteria

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Other:
• Physician standard-of-care in SCD patients

Locations

Country	Name	City	State
Italy	Azienda Ospedaliero-Universitaria	Bari
Italy	U.O. Oncoematologia Pediatrica Ospedali Civili	Brescia
Italy	Ospedale "A. Perrino"	Brindisi
Italy	Azienda Ospedaliera Universitaria Di Cagliari	Cagliari
Italy	ARNAS "Garibaldi"	Catania
Italy	Ospedale Vittorio Emanuele	Catania
Italy	University of Catania	Catania
Italy	A.O. "Pugliese-Ciaccio"	Catanzaro
Italy	Osp.San Giovanni Di Dio	Crotone
Italy	Azienda Ospedaliero Universitaria Meyer	Firenze
Italy	Ospedale Vittorio Emanuele III	Gela	Caltanisetta
Italy	E.O. Ospedali Galliera	Genova
Italy	Università degli Studi di Milano	Milano
Italy	Azienda Ospedaliero-Universitaria di Modena - Policlinico	Modena
Italy	Azienda Ospedaliera San Gerardo di Monza	Monza
Italy	Clinica pediatrica Monza S. Gerardo	Monza
Italy	Aorn A. Cardarelli	Napoli
Italy	Clinica di Onco-Ematologia Pediatrica, Università di Padova	Padova
Italy	A.R.N.A.S. "Civico"	Palermo
Italy	Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello	Palermo
Italy	Azienda Ospedaliero-Universitaria di Parma	Parma
Italy	Policlinico San Matteo	Pavia
Italy	Centro Emofilia e Medicina Trasfusionale - Pres. Ospedaliero	Ravenna
Italy	A. O. Bianchi Melacrino Morelli	Reggio Calabria
Italy	Ospedale S. Eugenio - FF UOSD DH Talassemici	Roma
Italy	Università Cattolica del Sacro Cuore - Policlinico A.Gemelli	Roma
Italy	Ospedali Civili Riuniti di Sciacca	Sciacca	Agrigento
Italy	U.O.S. Talassemia P.O. Umberto I°	Siracusa
Italy	Università degli Suidi di Torino	Torino
Italy	Policlinico G.B. Rossi	Verona

Sponsors (1)

Lead Sponsor	Collaborator
Società Italiana Talassemie ed Emoglobinopatie

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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	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 (ßS/ß0, ßS/ß+ and ßS/ßS) 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