Skysona

Risk for hematologic malignancy (including myelodysplastic syndrome). Prior to initiation, consider other alternative therapies or consult with hematology experts. Monitor lifelong for hematologic malignancies with CBCs (with differential) at least every 6 months, via integration site analysis or other testing for evidence of clonal expansion and predominance at least twice in the first year, and then annually thereafter for at least the first 15 years; consider bone marrow evaluations as clinically indicated; contact bluebird if malignancy occurs. Risk for severe infections (eg, bacterial, viral, opportunistic, or unspecified). Monitor for infection; give prophylactic antimicrobials according to guidelines. Avoid in those with active infections. Risk for prolonged cytopenias. Monitor blood counts until normalization; assess for bleeding and/or infection before and after treatment. Risk for delayed platelet engraftment, increased bleeding. Monitor for thrombocytopenia and bleeding accordingly. Obtain platelet counts frequently until platelet engraftment and recovery are achieved. Perform blood cell count determination and other appropriate testing whenever bleeding symptoms occur. Risk for neutrophil engraftment failure. Monitor neutrophil counts until engraftment is achieved. Administer rescue treatment with back-up collection of CD34+ cells if neutrophil engraftment failure occurs. Patients with a full ABCD1 deletion may lead to disease progression. Patients seropositive for HIV. Renal or hepatic impairment: not studied; assess for impairment to ensure HSC transplantation is appropriate. Advise males and females of reproductive potential to use effective contraception from the start of mobilization through at least 6 months after infusion. Pregnancy, nursing mothers: no available data.