ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study

Study Overview
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Brief Summary
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As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed. ESCORT-HU Extension study aims at evaluating the long-term safety of Siklos® focusing on some questions regarding its safety when used in current practice in adults and paediatric patients treated with Siklos® and followed for up to 5 years. The study will focus on the following concerns : occurrence and incidence of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®.

Official Title
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ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: Hydroxycarbamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female patients with symptomatic SCD, ≥ 2 years old, Treated with Siklos®, Having been informed of the study by the investigator and consenting to participate, or whose parents or legal guardians are consenting for their child to participate. To allow risk evaluation, participants must belong at least to one of the subpopulations defined below: Participants previously enrolled in ESCORT-HU who agree to participate in ESCORT-HU Extension study, New participants with any of the following criteria: history of HU treatment for more than 5 years or prepubescent over 10 years of age for girls and 13 years of age for boys at enrolment, or with history of leg ulcer, or pregnant women without interruption of Siklos® 3 months before the beginning of the pregnancy or, males treated with Siklos® whose partner is pregnant and without discontinuation of Siklos® 3 months before the beginning of the pregnancy.

Ages Eligible for Study
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Minimum Age: 2 Years

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 |
| --- | --- |
| Sickle cell disease patients treated with Siklos<br> | Drug: Hydroxycarbamide<br>* Patients, aged ≥ 2 years old, with symptomatic SCD, treated with Siklos®.<br>* Other names: Hydroxyurea;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos® |  | 5 years | 
| Occurrence and incidence of the number of leg ulcers |  | 5 years | 
| Occurrence and incidence of male fertility impairment |  | 5 years | 
| Occurrence and incidence of serious unexpected AEs causally related to Siklos® |  | 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean age of puberty | Document changes in fertility status in males and females | 5 years | 
| Mean number of patients with the use of a contraception, incidence of the use of semen cryopreservation and of semen analysis performed | Document changes in fertility status in males and females | 5 years | 
| Incidence of the use of semen cryopreservation and of semen analysis performed | Document changes in fertility status in males and females | 5 years | 
| Incidence of semen analysis performed | Document changes in fertility status in males and females | 5 years | 
| Management of Siklos® during pregnancy and outcome of pregnancy following exposure at the time of conception or at any time during the pregnancy | Outcome of pregnancies Following exposure to Siklos® at the time of conception (in participant and participant's partner) or at any time during the pregnancy | 5 years | 
| Frequency and reason for temporary or permanent discontinuations of Siklos® | Potential barriers to the compliance to the prescription of Siklos | 5 years | 
| Number of switch and combination with alternative treatment |  | 5 years | 
| Measure of quality of life with SF12 questionnaire | Quality of life for adults SCD patients | 5 years |