• Matthew M Heeney, Carolyn C Hoppe, Miguel R Abboud, Baba Inusa, Julie Kanter, Bernhards Ogutu, Patricia B Brown, Lori E Heath, Joseph A Jakubowski, Chunmei Zhou, Dmitry Zamoryakhin, Tsiri Agbenyega, Raffaella Colombatti, Hoda M Hassab, Videlis N Nduba, Janet N Oyieko, Nancy Robitaille, Catherine I Segbefia, David C Rees, and DOVE Investigators.
• From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston (M.M.H.); UCSF Benioff Children's Hospital Oakland, Oakland, CA (C.C.H.); the Department of Pediatrics and Adolescent Medicine, American University of Beirut, Beirut, Lebanon (M.R.A.); Evelina Children's Hospital (B.I.), Guy's and St. Thomas' Hospital (B.I.), Daiichi Sankyo Development (D.Z.), and King's College Hospital, Denmark Hill (D.C.R.) - all in London; the Department of Pediatrics, Division of Pediatric Hematology-Oncology, Medical University of South Carolina, Charleston (J.K.); U.S. Army Medical Research Unit-Kenya, Malaria Resistance (B.O.), Kenya Medical Research Institute-Walter Reed Project (J.N.O.), and the Center for Global Health Research and Public Health Collaboration (V.N.N.), Kenya Medical Research Institute - all in Kisumu, Kenya; Eli Lilly, Indianapolis (P.B.B., L.E.H., J.A.J., C.Z.); Kwame Nkrumah University of Science and Technology, Kumasi (T.A.), and the Department of Child Health, School of Medicine and Dentistry, Korle Bu Teaching Hospital, Accra (C.I.S.) - both in Ghana; the Clinic of Pediatric Hematology-Oncology, Azienda Ospedaliera-University of Padua, Padua, Italy (R.C.); Clinical Research Center, Alexandria University, Alexandria, Egypt (H.M.H.); and the Department of Pediatrics, Division of Hematology-Oncology, Centre Hospitalier Universitaire Sainte-Justine, Montreal (N.R.).
• N. Engl. J. Med. 2016 Feb 18;374(7):625-35.

BackgroundSickle cell anemia is an inherited blood disorder that is characterized by painful vaso-occlusive crises, for which there are few treatment options. Platelets mediate intercellular adhesion and thrombosis during vaso-occlusion in sickle cell anemia, which suggests a role for antiplatelet agents in modifying disease events.MethodsChildren and adolescents 2 through 17 years of age with sickle cell anemia were randomly assigned to receive oral prasugrel or placebo for 9 to 24 months. The primary end point was the rate of vaso-occlusive crisis, a composite of painful crisis or acute chest syndrome. The secondary end points were the rate of sickle cell-related pain and the intensity of pain, which were assessed daily with the use of pain diaries.ResultsA total of 341 patients underwent randomization at 51 sites in 13 countries across the Americas, Europe, Asia, and Africa. The rate of vaso-occlusive crisis events per person-year was 2.30 in the prasugrel group and 2.77 in the placebo group (rate ratio, 0.83; 95% confidence interval, 0.66 to 1.05; P=0.12). There were no significant differences between the groups in the secondary end points of diary-reported events. The safety end points, including the frequency of bleeding events requiring medical intervention, of hemorrhagic and nonhemorrhagic adverse events that occurred while patients were taking prasugrel or placebo, and of discontinuations due to prasugrel or placebo, did not differ significantly between the groups.ConclusionsAmong children and adolescents with sickle cell anemia, the rate of vaso-occlusive crisis was not significantly lower among those who received prasugrel than among those who received placebo. There were no significant between-group differences in the safety findings. (Funded by Daiichi Sankyo and Eli Lilly; ClinicalTrials.gov number, NCT01794000.).

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