The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up
Creators: name: Bridges, K.
name: Telfer, M.
name: Ataga, Kenneth
name: Orringer, E.
name: Hoehner, C.
name: Hernandez, S.
name: Valdez, S.
name: Temple, D.
name: Shurin, S.
name: Johnson-Telfair, A.
name: Hagar, W.
name: Vichinsky, E.
name: Smith, Wally
name: Steinberg, Martin H.
name: Kleman, K.
name: Rosse, W.
name: Waller, L.
name: Barber, Y.
name: Chow, J.
name: Gascon, P.
name: Winograd, C.
name: Di Paolo, P.
name: Hui, M.
name: Moshang, J.
name: Olivieri, N.
name: Dorn, L.
name: Siteman, J.
name: Embury, S.
name: Eckman, J.
name: Ballas, Samir K.
name: Anderson, A.
name: White, L.
name: Harrington, T.
name: Talischy-Zahed, N.
name: Shaw, D.
name: Scott, B.
name: Waclawiw, Myron A.
name: Labrousse, W.
name: Adler, B.
name: Phillips, G.
name: Bigelow, C.
name: Armstrong, F. Danny
name: Chiarucci, K.
name: Childerie, S.
name: McLaughlin, K.
name: Mandell, E.
name: DeCastro, Laura
name: Claster, S.
name: Knors, V.
name: Gargiulo, S.
name: Jones, S.
name: Tynan, B.
name: Saunthararajah, Y.
name: Saunders, T.
name: Maddox, B.
name: Hackney-Stevens, E.
name: Keverline, L.
name: Gardner, D.
name: Carter-Randall, E.
name: Schmidt, B.
name: Tracy, A.
name: Davies, D.
name: Carlos, T.
name: Kutlar, Abdullah
name: Finke, H.
name: Nwokolo, C.
name: Perlin, E.
name: Onyekwere, O. C.
name: Okam, M.
name: Peace, D.
name: Milner, P.
name: Koshy, M.
name: Sheikhai, M.
name: Daitch, L.
name: Swerdlow, Paul
name: Pendarvis, G.
name: Harrell, R.
name: Case, E.
name: Brenner, A.
name: Bailey, J. H.
name: Strayhorn, D.
name: Prchal, J.
name: Ramirez, G.
name: Dosik, H.
name: Platt, A.
name: McGee, M.
name: Lewis, N.
name: Castro, Oswaldo
name: Palmer, A.
name: Wilkes, E.
name: Braddock, J.
name: Sabahi, V.
name: Pegelow, C.
name: Bellevue, R.
name: McArdle, T.
name: O'Brien, R.
name: Williams, K.
name: Usry, L.
name: Eskridge, L.
name: Berman, B.
name: Allen, G. E.
name: Earles, A.
name: Bryan, M.
name: Davis, A.
name: Rodriguez, E. M.
name: Souffrant, H.
name: Genther, K.
name: McCarthy, William F.
name: Roundtree-Schmotzer, A.
name: Johnson-Telfair, A.
name: Ryans, P.
Publisher: name: The University of North Carolina at Chapel Hill University Libraries
Year: 2010

description: A randomized, controlled clinical trial established the efficacy and safety of short-term use of hydroxyurea in adult sickle cell anemia. To examine the risks and benefits of long-term hydroxyurea usage, patients in this trial were followed for 17.5 years during which they could start or stop hydroxyurea. The purpose of this follow-up was to search for adverse outcomes and estimate mortality. For each outcome and for mortality, exact 95% confidence intervals were calculated, or tests were conducted at α = 0.05 level (p-value <0.05 for statistical significance). Although the death rate in the overall study cohort was high (43.1%; 4.4 per 100 person-years), mortality was reduced in individuals with long-term exposure to hydroxyurea. Survival curves demonstrated a significant reduction in deaths with long-term exposure. Twenty-four percent of deaths were due to pulmonary complications; 87.1% occurred in patients who never took hydroxyurea or took it for <5 years. Stroke, organ dysfunction, infection and malignancy were similar in all groups. Our results, while no longer the product of a randomized study because of the ethical concerns of withholding an efficacious treatment, suggest that long-term use of hydroxyurea is safe and might decrease mortality.