ASH Clinical News April 2016 | Page 33

CLINICAL NEWS TABLE 1. Disease-Related Complications Total number of patients Era A Era B Era C Era D <1958 1958–1975 1976–1982 1983–1992 Severe Mild Severe Mild Severe Mild Severe Mild 688 843 1,542 787 1,121 427 1,548 530 Employment and Disability Status Disabled 45.9% 12.9% 37.8% 14.7% 15.9% 3.5% 5.8% 1.9% Employed/ Student 31.2% 49.5% 53.1% 74.5% 68.6% 86.4% 79.6% 87.6% Retired 18.3% 31.7% 0.4% 0.5% 0% 0% 0% 0% Other 3.5% 5.9% 8.6% 10.3% 15.5% 10.1% 14.5% 10.6% Limitation to overall activity level 68.8% 21.1% 49.4% 16.3% 25.9% 6.6% 14.9% 4.3% School/work absenteeism (>10 days missed) 6.9% 2.6% 8.5% 5.2% 10% 5.6% 5.6% 3% Assistive devices: intermittent 39.8% 16% 35.1% 15.4% 28.8% 13.6% 22.9% 11.3% Assistive devices: always 18.5% 4.6% 6.6% 1.5% 1.7% 0% 1.4% 0.8% ≤2 joint bleeds in last six months 45.8% 97.2% 38.1% 92.9% 41.3% 92% 51.7% 94.7% ≥5 joint bleeds in last six months 42.6% 1.7% 48.8% 4.3% 46.6% 3.8% 35.5% 3% Target joint 32.6% 3.2% 36.2% 7.2% 35.6% 5.8% 24.9% 2.3% Physical Function Bleeding Complications TABLE 2. Treatment-Related Complications Era A Era B Era C Era D <1958 1958–1975 1976–1982 1983–1992 Severe Mild Severe Mild Severe Mild Severe Mild 688 843 1,542 787 1,121 427 1,548 530 HBV 70.5% 33% 71.7% 34.4% 38.4% 9.1% 5.2% 2.4% HCV 92.3% 54.2% 93% 60.6% 84.4% 33.3% 32.6% 6.2% HBV and HCV co-infection 69.2% 27.9% 61.2% 31.5% 34.2% 6.1% 3.7% 0.4% HIV 42.9% 4.7% 61% 11.9% 26.2% 4% 1% 0.2% HIV and HCV co-infection 42.2% 4.2% 59% 10.8% 23.3% 3.7% 0.7% 0% Not infected with HIV, HBV, or HCV 5.5% 40.7% 4.1% 35.8% 10.4% 63.7% 65.4% 91.5% 17% 3.3% 13.6% 3.6% 11.5% 2.8% 15.6% 2.8% Total number of patients Viral Infections Inhibitor Development Any inhibitor recorded HBV = hepatitis B virus; HCV = hepatitis C virus; HIV = human immunodeficiency virus recombinant coagulation factor therapy since 1992. The highest prevalence of HIV infection occurred among men born during era B, and the prevalence declined in each successive era – to 1 percent in era D. (See TABLE 1 and TABLE 2 for more details on disease- and treatmentrelated complications for each birth cohort.) A total of 551 deaths were reported during the ASHClinicalNews.org study period, with era A and era B accounting for 82 percent of deaths in the severe hemophilia population and 96 percent of deaths in the mild cohort. Liver failure was the most commonly reported cause of death overall for both severe (33%) and mild (26%) disease. “If hemophilia treatment and access to treatment for men with severe hemophilia had improved markedly over the last several decades, then we would expect health outcomes disparities to narrow over time,” said Dr. Mazepa and co-authors. “Despite improved access to comprehensive care and to pathogen-free clotting factor for those born in recent decades, the gap between severe and mild has not narrowed either for target joints or for the use of assistive devices for mobility. These “disappointing” observations in- dicate that there is a need for continued improvement in strategies for prevention and treatment of hemophilia-associated hemorrhage. One limitation of this large registry data analysis is that patients had to survive until 1998 to be included in the analysis; evaluating only survivors could underestimate the adverse outcomes in severe disease and in the older eras. Interpretation of some data is limited by the lack of historical data and treatment data, for example, whether a patient had past inhibitors or the details of secondary or primary prophylaxis. ● REFERENCE Mazepa MA, Monahan PE, Baker JR, et al. Men with severe hemophilia in the United States: Birth cohort analysis of a large national database. Blood. 2016 March 16. [Epub ahead of print] ASH Clinical News 31