ESCORT – HU is a cohort follow-up study of pediatric and adult patients, suffering from Sickle Cell Disease, and who are treated by Siklos.

ESCORT – HU is the contraction of European Sickle cell disease COhoRT – HydroxyUrea.

This study has been implemented following a request of the EMEA which has granted the European Marketing Authorization.

ESCORT – HU is part of a Risk Management Plan (RMP) including a specific survey of patients taking Siklos.

Siklos contains hydroxycarbamide also named as hydroxyurea. This molecule is well known and is also indicated in the treatment of myelodysplasia.

The sickle cell disease indication is new, and Siklos is intended to be taken for all life. It is the main reason why EMEA asks for this specific type of survey.

It is the first major prospective cohort in the Sickle Cell Syndrome, either in term of size than in term of duration.

ESCORT-HU is planned to include 2000 patients treated by Siklos and to follow them for ten years. The objective is to collect long-term safety and efficacy information on Siklos® in current practice :

• Effect on growth
• Effect during pregnancy and lactation
• Risk on leg ulcers
• Risk in patients with renal or hepatic impairment
• Effect on stroke
• Effect on fertility
• Effect in elderly patients
• Effect when concomitant use of HIV drugs, myelosuppressive agent and radiotherapy
• Effect with concomitant use of live vaccines
• Occurrence of carcinomas
• Occurrence of vaso-occlusive crises
• Overall mortality – Survival rates

The eCRF has been specifically designed to guarantee a quick and easy data capture of the events occurring in the life of the patients while taking Siklos.

The data are centralized in an European data base in order to be able to merge data from all countries and to provide safety information to the authorities as requested.

Safety of siklos is generally good and occurrence of the items which are followed is anticipated to be very low, so an extensive number of patients is necessary.

Patients who are in compliance with Siklos Summary Product Characteristics (SmPC) would be included in the cohort follow-up.

Siklos is indicated for the prevention of recurrent painful vaso-occlusive crises including acute chest syndrome in paediatric and adult patients suffering from symptomatic Sickel Cell Syndrome.

Only physician experienced in the management of sickle cell syndrome can include patients in the cohort and initiate Siklos treatment.

This study is drawn to be pan European, and patient follow-up can be capture through an electronic Case Report Form accessible on www.escort-hu.com

We have set-up the eCRF in order to collect data to be able to answer the authorities. We think that electronic support could help health professionals to follow their own patients in their clinic all along the next ten years and even more if possible. Data will always be accessible. We think that this data base can constitute a useful tool.