Sickle Cell Disease
Conditions
Brief summary
In the context of the Risk Management Plan (RMP), as requested from Addmedica by the EMEA, to collect information about long-term safety of Siklos® (hydroxycarbamide) when used in patients with Sickle Cell Disease.
Interventions
DRUGSiklos
Sponsors
Theravia
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female ambulatory patients, aged 2 years and more (children, adolescents or adults) * With symptomatic sickle cell syndrome * Treated with Siklos® * Having been informed of the study by the initiating physician and consenting to participate to the cohort.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| % of Patient-years With Malignancies | During the follow-up of participant, up to 10 years | — |
| % of Patient-years With Skin Ulcerations | During the follow-up of participant, up to 10 years | Patients with at least one skin ulceration |
| % of Patient-years With Myelosuppressions | During the follow-up of participant, up to 10 years | Patients with at least one myelosuppression |
Countries
France, French Guiana, Germany, Greece, Guadeloupe, Italy, Martinique
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Patients With Sickle Cell Disease Treated With Siklos® The follow-up of the patients was strictly observational and fitted within their usual clinical monitoring without any controlled treatment, specific exam or modification of their follow-up, in compliance with the Summary of Product Characteristics of the product. | 1,903 |
| Total | 1,903 |
Baseline characteristics
| Characteristic | Patients With Sickle Cell Disease Treated With Siklos® | — |
|---|---|---|
| Age, Continuous | 21.4 years | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Region of Enrollment France | 1575 participants | — |
| Region of Enrollment Germany | 145 participants | — |
| Region of Enrollment Greece | 173 participants | — |
| Region of Enrollment Italy | 10 participants | — |
| Sex: Female, Male Female | 1051 Participants | — |
| Sex: Female, Male Male | 852 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 33 / 1,903 |
| other Total, other adverse events | 1,308 / 1,903 |
| serious Total, serious adverse events | 705 / 1,903 |
Outcome results
Primary
% of Patient-years With Malignancies
Time frame: During the follow-up of participant, up to 10 years
Population: Patients with follow-up visit
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Patients With Sickle Cell Disease Treated With Siklos® | % of Patient-years With Malignancies | 0.001 % patient-years |
Primary
% of Patient-years With Myelosuppressions
Patients with at least one myelosuppression
Time frame: During the follow-up of participant, up to 10 years
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Patients With Sickle Cell Disease Treated With Siklos® | % of Patient-years With Myelosuppressions | 5.99 % patient-years |
Primary
% of Patient-years With Skin Ulcerations
Patients with at least one skin ulceration
Time frame: During the follow-up of participant, up to 10 years
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Patients With Sickle Cell Disease Treated With Siklos® | % of Patient-years With Skin Ulcerations | 1.13 % patient-years |