FLOCOR (purified poloxamer 188) (F) is a nonionic surfactant with anti-adhesive and Theological properties that improves microvascular blood flow. As part of a multicenter. randomized, double-blind, placebo-controlled study of F in sickle cell patients (SCD) with vaso-occlusive crisis (VOC). 73 children aged 9 to 15 yr, were enrolled (37 F; 36 P). In the trial, study medication was given as a continuous IV infusion with either F (150 mg/ ml) at 100 mg/kg x 1 hr plus 30 mg/kg/hr x 47 hr, or matching placebo (P). In the 1 endpoint of the study, length of crisis (LOC), the children showed a reduction in LOC when treated with F vs. P (127 hr vs. 148.6 h, p=0.010). In addition, the F-patients exhibited a 114% increase in the number who achieved crisis resolution within 168 hr (59.5% vs. 27.8%, p=0.009). The F-patients also showed decreases in duration of hospitalization, pain intensity and total analgesic use, but these were not statistically significant. In addition, the incidence of AEs judged by investigators as acute chest syndrome or pneumonia were reduced by 42% (4 vs. 7) and 60% (2 vs. 5), respectively, in the Fpatients. F was generally well tolerated during the study but in some patients LFTs were transiently elevated with return to baseline during the hospitalization period. F had a slightly higher incidence of LFT elevations than P (14% vs 11%). No clinically significant renal function abnormalities were observed. Mild, transient thrombocytopenia was observed in 27% of F-pediatric patients vs. 22% on P. In a related study SCD patients with VOC were enrolled in an open-label, repeat administration safety study using the same dosing regimen as described above. The study enrolled 28 patients aged 7 to 39 yr, with 11 patients aged 7 to 15 yr. These 11 patients had 26 exposures to treatment, with 4 patients having 3 or more treatments within 1 yr. Only one of the pédiatrie patients experienced an SAE, fever and sepsis, and it was judged unrelated to F. There was no obvious increase in the incidence of hepatic, renal or platelet abnormalities with repeat treatment with F. Overall, F was effective and well tolerated when administered by continuous IV infusion to pédiatrie SCD patients. We feel these data justify conduct of a definitive study to validate the benefit of F in pédiatrie SCD patients experiencing VOC.
|Original language||English (US)|
|Issue number||11 PART I|
|State||Published - 2000|
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