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

To be considered for the group taking Xromi, your child must be between 9 months and just under 2 years old, have sickle cell disease, and have been prescribed Xromi to help manage their condition. It's also important that they haven't taken any form of hydroxycarbamide before and aren't getting regular blood transfusions every 8 weeks or more often. Your child also shouldn't have specific serious liver or kidney problems, very low blood cell counts, other active serious illnesses, or be on certain other medications like those for HIV. You, as the parent or legal guardian, would need to give your permission for your child to be part of the study.

For the comparison group, children need to be between 9 months and just under 2 years old with sickle cell disease and haven't had Xromi or similar medicines. These children will be chosen to closely match the children taking Xromi in terms of age and their specific type of sickle cell disease. Again, parent or guardian permission is needed.

If your child takes part, they will either be in the group already prescribed Xromi or a comparison group that hasn't taken it. No new treatments or extra tests will be given specifically for this study. The researchers will simply collect information from your child's regular medical notes and clinic visits over a period of 24 months, starting from when they began Xromi or a similar 'start date' for the comparison group. You won't need to attend any extra appointments or do anything special just for the study; all information comes from your child's usual care. The study is planned to take place in specialised hospitals in the UK and Germany, and possibly other European countries.