source: American Journal of Hematology

year: 2015

authors: Lori Luchtman‐Jones, Sara Pressel, Lee Hilliard, R. Clark Brown, Mary G. Smith, Alexis A. Thompson, Margaret T. Lee, Jennifer Rothman, Zora R. Rogers, William Owen, Hamayun Imran, Courtney Thornburg, Janet L. Kwiatkowski, Banu Aygun, Stephen Nelson, Carla Roberts, Cynthia Gauger, Connie Piccone, Theodosia Kalfa, Ofelia Alvarez, Kathryn Hassell, Barry R. Davis, Russell E. Ware

summary/abstract:

Although hemoglobin SC (HbSC) disease is usually considered less severe than sickle cell anemia (SCA), which includes HbSS and HbS/β(0) -thalassemia genotypes, many patients with HbSC experience severe disease complications, including vaso-occlusive pain, acute chest syndrome, avascular necrosis, retinopathy, and poor quality of life. Fully 20 years after the clinical and laboratory efficacy of hydroxyurea was proven in adult SCA patients, the safety and utility of hydroxyurea treatment for HbSC patients remain unclear. Recent NHLBI evidence-based guidelines highlight this as a critical knowledge gap, noting HbSC accounts for ∼30% of sickle cell patients within the United States. To date, only 5 publications have reported short-term, incomplete, or conflicting laboratory and clinical outcomes of hydroxyurea treatment in a total of 71 adults and children with HbSC. We now report on a cohort of 133 adult and pediatric HbSC patients who received hydroxyurea, typically for recurrent vaso-occlusive pain.

Hydroxyurea treatment was associated with a stable hemoglobin concentration; increased fetal hemoglobin (HbF) and mean corpuscular volume (MCV); and reduced white blood cell count (WBC), absolute neutrophil count (ANC), and absolute reticulocyte count (ARC). Reversible cytopenias occurred in 22% of patients, primarily neutropenia and thrombocytopenia. Painful events were reduced with hydroxyurea, more in patients >15 years old. These multicenter data support the safety and potentially salutary effects of hydroxyurea treatment for HbSC disease; however, a multicenter, placebo-controlled, Phase 3 clinical trial is needed to determine if hydroxyurea therapy has efficacy for patients with HbSC disease.

organization: Children's National Medical Center, USA; Cincinnati Children's Hospital Medical Center, USA; University of Texas School of Public Health, USA; University of Alabama, USA; Emory University/Children's Healthcare of Atlanta, USA; University of Mississippi Medical Center, USA; Ann and Robert H. Lurie Children's Hospital of Chicago, USA; Columbia University Medical Center, USA; Duke University Medical Center, USA; The University of Texas Southwestern Medical Center, USA; Children's Hospital of the King's Daughters, USA; University of South Alabama, USA; Rady Children's Hospital/University of California San Diego, USA; The Children's Hospital of Philadelphia, USA; Cohen Children's Medical Center, USA; Children's Hospitals and Clinics of Minnesota, USA; Nemours Children's Clinic, USA; University Hospitals/Rainbow Babies and Children's Hospital, USA; University of Miami, USA; University of Colorado, USA

DOI: 10.1002/ajh.24255

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