This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD). The objective is to assess the safety profile and clinical effectiveness of Xromi® under routine clinical conditions. The study includes a prospective cohort of Xromi®-treated patients and a matched retrospective comparator cohort of untreated patients. Participants will be followed for 24 months from treatment initiation or matched index date.
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
AESI - Myelosuppression (Neutropenia)
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Myelosuppression (Reticulocytopenia)
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Myelosuppression (Thrombocytopenia)
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Myelosuppression (Anaemia)
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Abnormal Weight Gain
Timeframe: Baseline to 24 months
AESI - Abnormal Weight Loss
Timeframe: Baseline to 24 months
AESI - Increase in Hepatic Enzyme (ALT)
Timeframe: Baseline to 24 months
AESI - Increase in Hepatic Enzyme (AST)
Timeframe: Baseline to 24 months
AESI - Alopecia
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Other Hair Loss
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Skin Hyperpigmentation
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Rash
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Skin Ulcers
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Growth Retardation
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Bacterial Infection
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Viral Infection
Timeframe: Pre-baseline, Baseline to 24 months
AESI - Fungal Infection
Timeframe: Pre-baseline, Baseline to 24 months