title: Safety of purified poloxamer 188 in sickle cell disease: phase I study of a non-ionic surfactant in the management of acute chest syndrome.
abstract: Acute chest syndrome (ACS) is the most common cause of death in patients with sickle cell anemia. Its management is primarily palliative. We performed a Phase I evaluation of purified poloxamer 188 (a non-ionic surfactant) in the management of ACS. Forty-three patients with sickle cell disease and ACS were treated with doses as high as 2960 mg/day by continuous intravenous (IV) infusion. The maximum tolerated dose has not been identified. No evidence of renal toxicity or other limiting adverse events were found. One adult patient died due to sepsis and adult respiratory distress syndrome, which were unrelated to treatment. Poloxamer 188 is safe to administer to patients with ACS, and preliminary data suggest that it may shorten its duration and the length of hospitalization in a dose related manner. Children appeared to benefit more than adults. The data and safety profile justify further studies with purified poloxamer 188 in the treatment of ACS.
authors.affiliation: Cardeza Foundation, Thomas Jefferson University, Philadelphia, Pennsylvania 19107, USA. samir.ballas@jefferson.edu
authors.forename: Samir K
authors.initials: SK
authors.lastname: Ballas
authors.forename: Beatrice
authors.initials: B
authors.lastname: Files
authors.forename: Lori
authors.initials: L
authors.lastname: Luchtman-Jones
authors.forename: Lennette
authors.initials: L
authors.lastname: Benjamin
authors.forename: Paul
authors.initials: P
authors.lastname: Swerdlow
authors.forename: Lee
authors.initials: L
authors.lastname: Hilliard
authors.forename: Thomas
authors.initials: T
authors.lastname: Coates
authors.forename: Miguel
authors.initials: M
authors.lastname: Abboud
authors.forename: Slawomir
authors.initials: S
authors.lastname: Wojtowicz-Praga
authors.forename: J Michael
authors.initials: JM
authors.lastname: Grindel
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000150:complications*; D002648:Child; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D007668:Kidney / Q000187:drug effects; D008171:Lung Diseases / Q000188:drug therapy* / Q000209:etiology / Q000401:mortality; D008297:Male; D020442:Poloxamer / Q000008:administration & dosage*; D013501:Surface-Active Agents / Q000008:administration & dosage* / Q000493:pharmacokinetics / Q000633:toxicity; D013577:Syndrome
chemical_list: D013501:Surface-Active Agents; D020442:Poloxamer