XROMI (hydroxycarbamide)

Key points

Favourable opinion for reimbursement in the prevention of vaso-occlusive complications of Sickle Cell Disease in patients over 2 years of age.

What therapeutic improvement?

No therapeutic improvement compared to SIKLOS (hydroxycarbamide).

Role in the care pathway?

Acute complications of sickle cell disease, such as vaso-occlusive crises, acute chest syndromes, priapism and stroke are therapeutic emergencies.

The disease-modifying therapies currently available treat the anaemia (blood transfusions) and reduce HbS levels (hydroxycarbamide and exchange transfusions). These treatments are reserved for certain patients following the opinion of a physician specialising in the management of sickle cell disease. Haematopoietic stem cell transplantation is the only curative treatment approach. Reserved for severe forms, it is generally performed using an HLA-identical sibling donor.

Hydroxycarbamide is effective for reducing the frequency and severity of painful episodes in children and adults, as well as relapses of acute chest syndrome in adults.

Role of the medicinal product in the care pathway

In the same way as SIKLOS scored film-coated tablets (hydroxycarbamide), XROMI oral solution (hydroxycarbamide) has a role in the care pathway in the prevention of vaso-occlusive complications of Sickle Cell Disease in patients over 2 years of age, as recommended in the current guidelines. It is necessary to ensure good treatment compliance.

Special recommendations

The presence of two dosing syringes (a red syringe graduated to 3 mL and a white syringe graduated to 12 mL) for accurate measurement of the prescribed dose of the oral solution requires vigilance on the part of healthcare professionals and patients. The healthcare professional must advise the patient or carer which syringe to use to ensure that the correct volume is administered in order to minimise the risk of error.