The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up.
Document Type
Article
Publication Date
6-1-2010
Abstract
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 alpha = 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.
Recommended Citation
Steinberg, M. H.; McCarthy, W. F.; Castro, O.; Ballas, S. K.; Armstrong, F. D.; Smith, W.; Ataga, K.; Swerdlow, P.; Kutlar, A.; DeCastro, L.; Waclawiw, M. A.; Orringer, E.; Jones, S.; Strayhorn, D.; Rosse, W.; Phillips, G.; Pearce, D.; Johnson-Telfair, A.; Daitch, L.; Milner, P.; Tracy, A.; Valdez, S.; Allen, G. E.; Moshang, J.; Scott, B.; Bigelow, C.; Anderson, A.; Sabahi, V.; Harrington, T.; Labrousse, W.; Pegelow, C.; Temple, D.; Case, E.; Harrell, R.; Childerie, S.; Embury, S.; Schmidt, B.; Davies, D.; Saunthararajah, Y.; Koshy, M.; Talischy-Zahed, N.; Dorn, L.; Pendarvis, G.; McGee, M.; Telfer, M.; Davis, A.; Onyekwere, O. C.; Nwokolo, C.; Finke, H.; Perlin, E.; Siteman, J.; Bryan, M.; Saunders, T.; Barber, Y.; Gascon, P.; Di Paolo, P.; Gargiulo, S.; Eckman, J.; Carter-Randall, E.; Bailey, J. H.; Platt, A.; Waller, L.; Ramirez, G.; Knors, V.; Hernandez, S.; Rodriguez, E. M.; Wilkes, E.; Vichinsky, E.; Hagar, W.; Hoehner, C.; Hackney-Stevens, E.; Claster, S.; Earles, A.; Kleman, K.; McLaughlin, K.; White, L.; Maddox, B.; Usry, L.; Brenner, A.; Williams, K.; O'Brien, R.; Genther, K.; Shurin, S.; Berman, B.; Chiarucci, K.; Keverline, L.; Olivieri, N.; Chow, J.; Hui, M.; Shaw, D.; Lewis, N.; Okam, M.; Mandell, E.; Palmer, A.; Bridges, K.; Tynan, B.; Winograd, C.; Bellevue, R.; Dosik, H.; Sheikhai, M.; Ryans, P.; Souffrant, H.; Adler, B.; Johnson-Telfair, A.; Eskridge, L.; Prchal, J.; Braddock, J.; McArdle, T.; Carlos, T.; Roundtree-Schmotzer, A.; and Gardner, D., "The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up." (2010). Department of Medicine Faculty Papers. Paper 45.
https://jdc.jefferson.edu/medfp/45
Figure 1.
PubMed ID
20513116
Comments
This article has been peer reviewed. It is the authors' final version prior to publication in American Journal of Hematology Volume 85, Issue 6, June 2010, Pages 403-408. The published version is available at DOI: 10.1002/ajh.21699. Copyright © Wiley InterScience