TRECONDI

Key points

Favourable opinion for reimbursement in combination with fludarabine as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients and in children older than one month and adolescents with malignant diseases.

Unfavourable opinion for reimbursement in combination with fludarabine as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with non-malignant diseases.

What therapeutic improvement?

In adult patients with malignant diseases: No clinical added value compared to busulfan, in combination with fludarabine as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT).

In paediatric patients older than one month with malignant diseases: No clinical added value as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT).

Role in the care pathway?

The choice of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) depends on numerous criteria, in particular, general health status, the patient’s age, comorbidities and the type of blood disease.

There are two types of conditioning treatment, depending on their intensity.

Myeloablative conditioning, which is reserved for patients in good general health, uses various chemotherapy regimens that may include alkylating agents or antimetabolites (cyclophosphamide, busulfan, melphalan, thiotepa, cytarabine), potentially combined with total body irradiation (in the event of a malignant blood disease, in particular). The protocols used are, in particular:

-    total body irradiation (TBI) + cyclophosphamide;

-    TBI + cytarabine +/- melphalan;

-    busulfan + cyclophosphamide, etc.

Reduced intensity conditioning is preferred in adult patients who are elderly, have comorbidities or an impaired general health status insofar as myeloablative conditioning is more toxic. It is based on the use of potent immunosuppressant agents and, in particular, fludarabine with variable combination with other immunosuppressant agents (anti-thymocyte globulin, mycophenolate mofetil), and low doses of chemotherapy (cyclophosphamide, busulfan, aracytine) or low doses of TBI.

Role of the medicinal product in the care pathway

In adults, adolescents and children older than one month with malignant diseases: TRECONDI (treosulfan) in combination with fludarabine constitutes a new treatment option, as a reduced-intensity conditioning regimen prior to allogeneic haematopoietic stem cell transplantation (alloHSCT). It should be noted that in adults, a study demonstrated the non-inferiority of the treosulfan-fludarabine combination versus busulfan-fludarabine in terms of event-free survival after 2 years (primary endpoint). In children, no comparative data versus another conditioning regimen are available.

In adult patients with non-malignant diseases: In the absence of data, TRECONDI (treosulfan) has no role in the therapeutic strategy for this population.