Reason for request

First assessment

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.


Clinical Benefit


The Committee deems that the clinical benefit of XROMI (hydroxycarbamide) is substantial in the MA indication.

Clinical Added Value

no clinical added value


  • the well-established efficacy of hydroxycarbamide in the prevention of vaso-occlusive complications of Sickle Cell Disease, with a known safety profile,

but taking into account:

  • the efficacy and safety data provided in support of the application for inclusion of XROMI (hydroxycarbamide) based solely on bibliographic studies,
  • the absence of clinical studies conducted with XROMI (hydroxycarbamide) oral solution and, in particular, the absence of any data provided supporting a potential advantage, in terms of acceptability, treatment compliance or quality of life, of XROMI (hydroxycarbamide) oral solution compared to SIKLOS (hydroxycarbamide) tablets, which can be disintegrated in a small quantity water and mixed with a little syrup or food to mask the bitter taste in children or patients with swallowing difficulties,

the Transparency Committee considers that XROMI (hydroxycarbamide) provides no clinical added value (CAV V) compared to SIKLOS (hydroxycarbamide).

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