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• W1993750238 abstract "Bleeding is the major complication of patients with hemophilia. Other than life‐threatening bleeds, joint bleeds have been the major cause of morbidity, with long‐term joint damage, and restriction in activity, as well as limitation of vocational choices. The introduction of self‐infusion or family infusion of available concentrates made surgical correction and reduction in joint pathology possible. The early [1Nilsson I.M. Berntorp E. Lofqvist T. Pettersson H. Twenty‐five years’ experience of prophylactic treatment in severe haemophilia A and B.J Intern Med. 1992; 232: 25-32Crossref PubMed Google Scholar] introduction of prophylaxis initiated a therapeutic intervention that has markedly altered outcomes. These initial observations have led to a large body of literature that has included anecdotes and clinical trials. Various regimens for prophylaxis have been studied in prospective fashion, varying from initiating prophylaxis once a week in children to treatment every other day. Recently, a prospective, randomized, longitudinal study comparing prophylaxis with on‐demand treatment in children clearly showed that prophylaxis markedly reduced bleeding episodes and led to less joint damage when a highly sensitive magnetic resonance imaging technique was used [2Manco‐Johnson M.J. Abshire T.C. Shapiro A.D. Riske B. Hacker M.R. Kilcoyne R. Ingram J.D. Manco‐Johnson M.L. Funk S. Jacobson L. Valentino L.A. Hoots W.K. Buchanan G.R. DiMichele D. Recht M. Brown D. Leissinger C. Bleak S. Cohen A. Mathew P. et al.Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.N Engl J Med. 2007; 357: 535-44Crossref PubMed Scopus (0) Google Scholar]. Few prospective studies have evaluated clinical practice in adults with assessment of outcomes as they related to on‐demand treatment vs. prophylaxis. One large study [3Aledort L.M. Haschmeyer R.H. Pettersson H. the Orthopaedic Outcome Study GroupA longitudinal study of orthopaedic outcomes for severe factor‐VIII‐deficient haemophiliacs.J Intern Med. 1994; 236: 391-9Crossref PubMed Google Scholar] demonstrated that prophylaxis gave better orthopedic outcomes, involved the use of substantially more factor than on‐demand treatment, and would slow progression of arthropathy in those with previous joint disease. Patients who have been on prophylaxis from childhood not infrequently stop in adulthood, only to resume if bleeding becomes an issue. In this journal issue, an article by Collins et al. [4Collins P. Faradji A. Morfini M. Enriquez M.M. Schwartz L. Efficacy and safety of secondary prophylactic vs. on‐demand sucrose‐formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13‐month crossover study.J Thromb Haemost. 2009; 8: 83-9Abstract Full Text Full Text PDF PubMed Scopus (0) Google Scholar], entitled ‘Secondary prophylaxis versus on‐demand treatment with sucrose‐formulated recombinant factor VIII: a 13‐month crossover study of adults with severe hemophilia A’, deals with a recombinant factor VIII product in a prospective study of 19 adult patients, whose mean age was 36.4 years and who had 16 target joints among them. This was an open label study in severe hemophilia A patients with FVIII < 1%. The patients were followed for 6 months on demand, and then switched to 7 months of prophylaxis with 20–40 IU kg−1 three times a week. The change in the major outcome of bleeds was very dramatic, with the median number of joint bleeds on demand being 15 (11–26), and dropping to 0 (0–3) (P < 0.001). The decrease in all bleeds went from 20.5 (14–37) to 0 (0–3) (P < 0.001). Treatment was well tolerated. Although this article describes a small study, recruiting patients for clinical trials has become a significant challenge. These observations confirm those of the orthopedic outcome study [3Aledort L.M. Haschmeyer R.H. Pettersson H. the Orthopaedic Outcome Study GroupA longitudinal study of orthopaedic outcomes for severe factor‐VIII‐deficient haemophiliacs.J Intern Med. 1994; 236: 391-9Crossref PubMed Google Scholar], in that bleeding episodes and changes in orthopedic measurements improve on prophylaxis. Using the Gilbert score [5Gilbert M.S. Prophylaxis: musculoskeletal evaluation.Semin Hematol. 1993; 30: 3-6PubMed Google Scholar] to measure joint function, there was also significant improvement solely based on bleeding. In the long‐term study [3Aledort L.M. Haschmeyer R.H. Pettersson H. the Orthopaedic Outcome Study GroupA longitudinal study of orthopaedic outcomes for severe factor‐VIII‐deficient haemophiliacs.J Intern Med. 1994; 236: 391-9Crossref PubMed Google Scholar], the Gilbert score changed even more dramatically. In this article, the quality of life and health economy measurements were not significant, but would not be expected to be over such a short period of time. Pediatric, early primary or secondary prophylaxis has now been clearly shown to be practical, with growing numbers of patients being treated in this fashion, and with continuing investigations regarding optimal regimens. More emphasis is now being put on tailoring treatment to meet the bleeding patterns of patients. On the other hand, prophylaxis in adolescents and adults has seriously lagged behind, both from an investigative standpoint and in terms of being put into clinical practice [6Walsh C.E. Valentino L.A. Factor VIII prophylaxis for adult patients with severe haemophilia A: results of a US survey of attitudes and practices.Haemophilia. 2009; 15: 1014-21Crossref PubMed Scopus (0) Google Scholar]. The economic impact of prophylaxis has been described, and it is more costly, even when one corrects it for improvements in health‐related parameters [7Bohn R.L. Avorn J. Glynn R.J. Choodnovskiy I. Haschemeyer R. Aledort L.M. Prophylactic use of factor VIII; an economic evaluation.Thromb Haemost. 1998; 79: 932-7Crossref PubMed Scopus (0) Google Scholar]. However, in the orthopedic outcome study (OOS) programs, using equivalently high doses of factor, the on‐demand group fared worse than those on prophylaxis in both bleeding and orthopedic outcome measures. This article underscores the marked benefit of instituting a prophylaxis program in patients who have already demonstrated joint pathology. Younger adults who have never been on prophylaxis, but, because of early intervention demand therapy, have good joints, may well benefit from prophylaxis in the long term to prevent joint damage. Problems of compliance, as more frequent treatment is required, and finances may become key determinants in making therapeutic decisions in the USA. The USA has pluralistic reimbursement programs, and with many uninsured, this can be rate‐limiting in making this therapeutic modality available. This interesting study needs to be reproduced with larger, randomized designs. It is clearly better not to bleed than to bleed. The author states that he has no conflict of interest." @default.
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• W1993750238 date "2010-01-01" @default.
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• W1993750238 title "To bleed or not to bleed – is that a question?" @default.
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• W1993750238 doi "https://doi.org/10.1111/j.1538-7836.2009.03668.x" @default.
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