Libmeldy must be administered in a qualified treatment centre by a physician with experience in Haematopoietic Stem Cell Transplantation (HSCT) and trained for administration and management of patients treated with the medicinal product.
4) and should only be administered once. The dose of Libmeldy must be determined based on the patient’s body weight at the time of infusion. Treatment consists of a single dose for infusion containing a dispersion of viable CD34+ cells in one or more infusion bags.
The minimum recommended dose of Libmeldy is 3 × 106 CD34+ cells/kg of body weight. In clinical studies, doses up to 30 × 106 CD34+ cells/kg have been administered. 6). See the accompanying Lot Information Sheet (LIS) for additional information pertaining to dose.
Peripheral blood mobilisation and apheresis The autologous CD34+ cells are isolated from mobilised peripheral blood (mPB). This is achieved by apheresis procedure(s) following peripheral blood mobilisation. For manufacture of Libmeldy, the patient must be able to donate a minimum of 8-10 ×106 CD34+ cells/kg, considering that the optimal range is between 20- 30 × 106 CD34+ cells/kg.
The minimum CD34+ cell quantity may be achieved using one or more cycles of apheresis. 1). 4). These cells must be collected from the patient and be cryopreserved according to institutional procedures prior to myeloablative conditioning.
The back-up cells may be harvested either through mPB apheresis or bone marrow harvest. 1 for a description of the mobilisation regimen used in clinical studies). 4). 1 for a description of the myeloablative regimen used in clinical studies).
Busulfan is the recommended conditioning medicinal product. Myeloablative conditioning should not begin until the complete set of infusion bag(s) constituting the dose of Libmeldy has been received and stored at the qualified treatment centre, and the availability of the back-up collection is confirmed.
e. transplant-associated thrombotic microangiopathy (TA-TMA) or atypical haemolytic uremic syndrome (aHUS), in line with local guidelines. Depending on the myeloablative conditioning regimen administered, prophylaxis for seizures should also be considered.
Phenytoin is not recommended as it may increase busulfan clearance. Prophylactic and empiric use of anti-infectives (bacterial, fungal, viral) should be considered for the prevention and management of infections especially during the neutropenic period following conditioning.
Routine monitoring of most common viruses subject to re-activation is recommended as per local guidelines. Infection control measures and isolation procedures should be employed during the hospitalization according to local standards.
25 mg/kg, max. dose 10 mg), or equivalent medicinal products, be administered 15- 30 minutes before the infusion of Libmeldy to reduce the possibility of an infusion reaction. Special populations Elderly Libmeldy has not been studied in patients >65 years of age.
Renal impairment Libmeldy has not been studied in patients with renal impairment. Patients should be assessed for renal impairment to ensure autologous HSPC gene therapy administration is appropriate. No dose adjustment is required. Hepatic impairment Libmeldy has not been studied in patients with hepatic impairment.
Patients should be assessed for hepatic impairment to ensure autologous HSPC gene therapy administration is appropriate. No dose adjustment is required. e. with a typical onset after 7 years of age). No data are available. Method of administration Libmeldy is for intravenous infusion only.
Precautions to be taken before handling or administering the medicinal product This medicinal product contains genetically modified human cells. Healthcare professionals should therefore take appropriate precautions (wearing gloves and glasses) to avoid potential transmission of infectious diseases when handling the product.
6. Preparation for infusion Before […]