PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]

Inclusion criteria

• Aged from 9 months to under 2 years at the index date.
• Diagnosis of SCD.
• Known β-globin genotype at the index date.
• Prescribed Xromi® for the prevention of complications of SCD.
• Parent(s) (or a legal representative(s)) provides written informed consent to participate in the study, unless there is a waiver, non-opposition, or blanket written informed consent by the parent for research studies.

Exclusion criteria

• Previous use of hydroxycarbamide of any formulation before the index date.
• Receiving regular blood transfusions (occurring every 8 weeks or more frequently) at the index date.
• Known hypersensitivity to any of the excipients of Xromi® at the index date.
• Contraindications to the drug at the index date: severe hepatic impairment (Child-Pugh classification C); severe renal impairment (creatinine clearance: CrCl \<30 ml/min); presence of at least one of the following: Absolute neutrophil count (ANC) \< 1.0 x 10\^9/L, absolute reticulocyte count (ARC) \<80 x 10\^9/L, platelets \<80 x 10\^9/L.
• Participating in another clinical study of an investigational medicinal product (IMP) at the index date.
• Anti-retroviral medicinal products for human immunodeficiency virus (HIV) at the index date.
• Active malignancy at the index date.
• Participants in the prospective exposure cohort who are prescribed Xromi® but do not initiate treatment will be excluded from the dataset.
• Retrospective Comparator cohort
• Inclusion criteria:
• Aged from 9 months to under 2 years at the index date.
• Diagnosis of SCD.
• Known β-globin genotype.
• Matched to an exposed participant.
• Parent(s) (or a legal representative(s)) provides written informed consent to participate in the study, unless there is a waiver, non-opposition, or blanket written informed consent by the parent for research studies.
• Exclusion criteria:
• Use of hydroxycarbamide of any formulation before or at the index date.
• Receiving regular blood transfusions (occurring every 8 weeks or more frequently) at the index date.
• Presence at the index date of any of the following: severe hepatic impairment (Child-Pugh classification C); severe renal impairment (CrCl \<30 ml/min); presence of at least one of the following: ANC \< 1.0 x 10\^9/L, ARC \< 80 x 10\^9/L, platelets \< 80 x 10\^9/L).
• Participating in another clinical study of an IMP at the index date.
• Anti-retroviral medicinal products for HIV at the index date.
• Active malignancy at the index date.