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

A regiszterben nem jelölt feltételek

• Prospective Exposure Cohort
• 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.