High-Dose Cyclophosphamide for Treatment of Aplastic Anemia
Authors: Neal S. Young, John F. Tisdale
Journal: Annals of Internal Medicine (2002)

Letters17 September 2002High-Dose Cyclophosphamide for Treatment of Aplastic AnemiaNeal S. Young, MD and John F. Tisdale, MDNeal S. Young, MDNational Institutes of Health; Bethesda, MD 20892Search for more papers by this author and John F. Tisdale, MDNational Institutes of Health; Bethesda, MD 20892Search for more papers by this authorAuthor, Article, and Disclosure Informationhttps://doi.org/10.7326/0003-4819-137-6-200209170-00030 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail TO THE EDITOR:Brodsky and colleagues (1) assert that high-dose cyclophosphamide provides enduring remission from and even “cures” free from long-term complications in patients with severe aplastic anemia. However, a randomized trial by the U.S. National Institutes of Health (NIH) comparing antithymocyte globulin (ATG) with cyclophosphamide was terminated early because of unacceptable toxicity in the cyclophosphamide arm (2). This early termination, along with the subsequent observation of persistent paroxysmal nocturnal hemoglobinuria (PNH) clones, relapse, and evolution to cytogenetic abnormalities typical of myelodysplasia at similar rates in both arms (3), challenges Brodsky and colleagues' assertions.Although Brodsky and colleagues report that ...References1. Brodsky RA, Sensenbrenner LL, Smith BD, Dorr D, Seaman PJ, Lee SM, et al . Durable treatment-free remission after high-dose cyclophosphamide therapy for previously untreated severe aplastic anemia. Ann Intern Med. 2001;135:477-83. [PMID: 11578150] LinkGoogle Scholar2. Tisdale JF, Dunn DE, Geller N, Plante M, Nunez O, Dunbar CE, et al . High-dose cyclophosphamide in severe aplastic anaemia: a randomised trial. Lancet. 2000;356:1554-9. [PMID: 11075769] CrossrefMedlineGoogle Scholar3. Tisdale JF, Maciejewski JP, Nunez O, Rosenfeld SJ, Young NS. A randomized trial comparing antithymocyte globulin and cyclosporin to cyclophosphamide and cyclosporine for initial treatment in severe aplastic anemia: results of long-term follow-up [Abstract]. Blood. 2001;89:223a. Google Scholar4. Brodsky RA, Mukhina GL, Li S, Nelson KL, Chiurazzi PL, Buckley JT, et al . Improved detection and characterization of paroxysmal nocturnal hemoglobinuria using fluorescent aerolysin. Am J Clin Pathol. 2000;114:459-66. [PMID: 10989647] CrossrefMedlineGoogle Scholar5. Chalmers TC. Prophylactic treatment of Wilson's disease. N Engl J Med. 1968;278:910-1. [PMID: 5641165] CrossrefMedlineGoogle Scholar Author, Article, and Disclosure InformationAffiliations: National Institutes of Health; Bethesda, MD 20892 PreviousarticleNextarticle Advertisement FiguresReferencesRelatedDetailsSee AlsoDurable Treatment-Free Remission after High-Dose Cyclophosphamide Therapy for Previously Untreated Severe Aplastic Anemia Robert A. Brodsky , Lyle L. Sensenbrenner , B. Douglas Smith , Donna Dorr , Phillip J. Seaman , Shing M. Lee , Judith E. Karp , Isadore Brodsky , and Richard J. Jones High-Dose Cyclophosphamide for Treatment of Aplastic Anemia Robert A. Brodsky and Richard J. Jones Metrics Cited byOutcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine AOutcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A 17 September 2002Volume 137, Issue 6Page: 549-550KeywordsAplastic anemiaClinical trial reportingClinical trialsCyclophosphamideCytogeneticsGlobulinsHemoglobinNeutrophilsRandomized trialsToxicity ePublished: 17 September 2002 Issue Published: 17 September 2002 Copyright & PermissionsCopyright © 2002 by American College of Physicians. All Rights Reserved.PDF downloadLoading ...