Sickle Cell in the Medical Literature - December 2014

1. J Pediatr Hematol Oncol. 2015 Jan;37(1):1-9. doi: 10.1097/MPH.0000000000000291. Prasugrel in Children With Sickle Cell Disease: Pharmacokinetic and Pharmacodynamic Data From an Open-label, Adaptive-design, Dose-ranging Study. Styles L1, Heiselman D, Heath LE, Moser BA, Small DS, Jakubowski JA, Zhou C,Redding-Lallinger R, Heeney MM, Quinn CT, Rana SR, Kanter J, Winters KJ. Abstract INTRODUCTION: This phase 2 study was designed to characterize the relationship among prasugrel dose, prasugrel's active metabolite (Pras-AM), and platelet inhibition while evaluating safety in children with sickle cell disease. It was open-label, multicenter, adaptive design, dose ranging, and conducted in 2 parts. Part A: Patients received escalating single doses leading to corresponding increases in Pras-AM exposure and VerifyNow®P2Y12 (VN) platelet inhibition and decreases in VNP2Y12 reaction units and vasodilator-stimulated phosphoprotein platelet reactivity index. Part B: Patients were assigned daily doses (0.06, 0.08, and 0.12 mg/kg) based on VN pharmacodynamic measurements at the start of 2 dosing periods, each 14±4 days. Platelet inhibition was significantly higher at 0.12 mg/kg (56.3%±7.4%; least squares mean±SE) compared with 0.06 mg/kg (33.8%±7.4%) or 0.08 mg/kg (37.9%±5.6%). Patients receiving 0.12 mg/kg achieved ≥30% platelet inhibition; only 1 patient receiving 0.06 mg/kg exceeded 60% platelet inhibition. High interpatient variability in response to prasugrel and the small range of exposures precluded rigorous characterization of the relationship among dose, Pras-AM, and platelet inhibition. SAFETY: No hemorrhagic events occurred in Part A; 3 occurred in Part B, all mild and self-limited. CONCLUSIONS: Most children with sickle cell disease may achieve clinically relevant platelet inhibition with titration of daily-dose prasugrel. PMID: 25493452 [PubMed - in process] Related citations

New Autobiography “Sickle”

Dominique Friend doesn't look like she's sick. But the Lansdowne resident often deals with bouts of pain so severe she ends up in the hospital for weeks. Friend, 44, was born with sickle cell disease, an inherited blood disorder that affects an estimated 90,000 to 100,000 in the U.S., according to Centers for Disease Control and Prevention information. Her autobiography "Sickle" was released by Tate Publishing on Dec. 9 2014  in a second edition, after she self-published the book in 2009. I would take the pain of childbirth over a sickle cell crisis any day. - Dominique Friend

ASH Meeting 176 [ Abstracts about Sickle Cell Disease presented ]

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A new study that recently began enrolling patients at University Hospitals Case Medical Center is using biochip technology that one day may help physicians better manage the treatment of patients with sickle cell disease. Sickle cell disease is one of the most common genetic diseases in the United States; sickle cell anemia affects an estimated 100,000 people, mainly African Americans.

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