Trial Outcomes & Findings for European Sickle Cell Disease Cohort - Hydroxyurea (NCT NCT02516579)

NCT ID: NCT02516579

Last Updated: 2020-03-19

Results Overview

• Recruitment status: COMPLETED
• Target enrollment: 1906 participants
• Primary outcome timeframe: During the follow-up of participant, up to 10 years
• Results posted on: 2020-03-19

Participant Flow

Participant milestones

Measure	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.
Overall Study STARTED	1906
Overall Study COMPLETED	1906
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

1906 patients were included but 1903 patients took at least one dose of Siklos and were included in the analysis

Baseline characteristics by cohort

Measure	Patients With Sickle Cell Disease Treated With Siklos® n=1903 Participants 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.
Age, Continuous	21.4 years n=1903 Participants • 1906 patients were included but 1903 patients took at least one dose of Siklos and were included in the analysis
Sex: Female, Male Female	1051 Participants n=1903 Participants • 1906 patients were included but 1903 patient took at least one dose of Siklos and were included in the analysis
Sex: Female, Male Male	852 Participants n=1903 Participants • 1906 patients were included but 1903 patient took at least one dose of Siklos and were included in the analysis
Region of Enrollment Greece	173 participants n=1903 Participants • 1906 patients were included but 1903 took at least one dose of Siklos and were included in the analysis
Region of Enrollment Italy	10 participants n=1903 Participants • 1906 patients were included but 1903 took at least one dose of Siklos and were included in the analysis
Region of Enrollment France	1575 participants n=1903 Participants • 1906 patients were included but 1903 took at least one dose of Siklos and were included in the analysis
Region of Enrollment Germany	145 participants n=1903 Participants • 1906 patients were included but 1903 took at least one dose of Siklos and were included in the analysis

PRIMARY outcome

Timeframe: During the follow-up of participant, up to 10 years

Population: Patients with follow-up visit

Outcome measures

Measure	Patients With Sickle Cell Disease Treated With Siklos® n=1854 Participants 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.
% of Patient-years With Malignancies	0.001 % patient-years

PRIMARY outcome

Timeframe: During the follow-up of participant, up to 10 years

Patients with at least one skin ulceration

Outcome measures

Measure	Patients With Sickle Cell Disease Treated With Siklos® n=1854 Participants 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.
% of Patient-years With Skin Ulcerations	1.13 % patient-years

PRIMARY outcome

Timeframe: During the follow-up of participant, up to 10 years

Patients with at least one myelosuppression

Outcome measures

Measure	Patients With Sickle Cell Disease Treated With Siklos® n=1854 Participants 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.
% of Patient-years With Myelosuppressions	5.99 % patient-years

Adverse Events

Patients With Sickle Cell Disease Treated With Siklos®

Serious events: 705 serious events

Other events: 1308 other events

Deaths: 33 deaths

Serious adverse events

Measure	Patients With Sickle Cell Disease Treated With Siklos® n=1903 participants at risk 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.
Infections and infestations infections and infestations	18.2% 346/1903 • Adverse events were collected during the follow-up of participant, up to 10 years
Blood and lymphatic system disorders Blood and lymphatic system disorders	8.1% 155/1903 • Adverse events were collected during the follow-up of participant, up to 10 years
Respiratory, thoracic and mediastinal disorders respiratory, thoracic and mediastinal disorders	7.9% 151/1903 • Adverse events were collected during the follow-up of participant, up to 10 years
General disorders general disorders and administration site conditions	5.1% 97/1903 • Adverse events were collected during the follow-up of participant, up to 10 years

Other adverse events

Measure	Patients With Sickle Cell Disease Treated With Siklos® n=1903 participants at risk 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.
Infections and infestations infections and infestations	33.9% 645/1903 • Adverse events were collected during the follow-up of participant, up to 10 years
General disorders General disorders and administration site conditions	20.3% 386/1903 • Adverse events were collected during the follow-up of participant, up to 10 years
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	19.4% 370/1903 • Adverse events were collected during the follow-up of participant, up to 10 years

Additional Information

Medical director

Addmedica

Phone: +33172690186

Email: corinne.duguet@addmedica.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place