CLOTTING group activities and conferences are supported by an educational grant from the Novo Nordisk Latin America Regional Office and affiliates. without inhibitors ( 0.6?BU?mL?1) and were 5?years of age. Musculoskeletal status was compared between three groups of countries, based primarily on differences in the availability of long-term prophylaxis. Overall, 143 patients (5C66?years of age) were enrolled from nine countries. In countries where long-term prophylaxis had been available for at least 10?years MS436 (Group A), patients aged 5C10?years had significantly better mean World Federation of Hemophilia clinical scores, fewer target joints and fewer affected joints than patients from countries where long-term prophylaxis has been available for about 5?years (Group B) or was not available (Group C). In Latin America, the musculoskeletal status of patients with severe haemophilia without inhibitors has improved significantly in association with the provision of long-term prophylaxis. As more countries in Latin America institute this practice, further improvements are anticipated. of patients without joint damage (0/0)* (%)6 (50)6 (50)2 (25)1 (13)1 (8)0 (0)Mean of affected joints per patient (range)1.2 (0C3)1.8 (0C6)1.6 (0C4)1.8 (0C4)2.3 (0C4)3.6 (1C6)of patients with target joints (%)1 (8)2 (17)3 (38)4 (50)6 (50)10 (63)of patients with joint procedure (of joints treated)1 (1)1 (1)1 (1)4 (6)2 (3)7 (11)Clinical score,of patients on long-term prophylaxis (%)12 (100)6 (50)6 (75)7 (88)0 (0)0 (0)Mean age at start of prophylaxis, years (range)1.7 (0.8C5)5.7 (1.1C13)3.4 (1C7)14 (10C18)NANA Open in a separate window *WFH clinical score 0/Pettersson score 0. ?Categories (in order of decreasing frequency): 1?bleed/week; 2C3?bleeds/month; 7C12?bleeds/year; 4C6?bleeds/year; 1C3?bleeds/year; 1?bleed/year. NA, not applicable; SD, standard deviation. Table 3 Musculoskeletal evaluation of patients 21?years of age with severe haemophilia A in Latin America of patients without joint damage (0/0)*000000Mean of affected joints per patient (range)4.4 (3C7)5.6 (3C6)4 (2C6)4.5 (2C6)4.7 (2C7)5.1 (4C6)of patients with target joints (%)7 (70)8 (80)7 (64)7 (64)12 (67)9 (60)of patients with joint procedure (of joints treated)7 (11)3 (4)8 (15)8 (27)9 (17)7 (9)Clinical score,of patients on long-term prophylaxis (%)2 (20)0 (0)5 (45)4 (36)0 (0)0 (0)Mean age at start of prophylaxis, years (range)21.5 (16C27)NA21.4 (19C26)45 (36C62)NANA Open in a separate window *WFH clinical score 0/Pettersson score 0. ?Categories (in order of decreasing frequency): 1?bleed/week; 2C3?bleeds/month; 7C12?bleeds/year; 4C6?bleeds/year; 1C3?bleeds/year; 1?bleed/year. NA, not applicable; SD, standard deviation. Treatment characteristics by country In countries from Group A, long-term prophylaxis was made available between 1997 and 2002; in Group B countries since 2007 or 2008; and not at all in countries from Group C (Table 1). In the 5- to 10-year-old age stratum, all 12 patients from Group A received long-term prophylaxis, beginning at a mean age of 1 1.7?years. In Group B, 6 of 8 patients received primary prophylaxis, with a mean age at initiation of 3.4?years (Table 2). The most commonly used prophylaxis regimen was a flexible protocol of 20C30?IU?kg?1 3x/week. In Panama, a fixed protocol of 25?IU?kg?1 3x/week was used. Venezuela was the only country to offer tailored prophylaxis based on the Canadian protocol (50?IU?kg?1 1x/week or 30?IU?kg?1 2x/week or 25C30?IU?kg?1 3x/week) 8. Per capita factor usage was highest in Argentina and Chile, where long-term prophylaxis has been available for the longest period of time (Table 1). Uruguay had similarly high usage despite not offering long-term prophylaxis. Mexico and Peru had the lowest usage of factor per capita, and were also the only countries without 100% access to safe treatment. The use of recombinant factor was highest in Venezuela and Colombia (about 50%) 1. In contrast, all countries provided home treatment and short-term prophylaxis for all patients (Table 1). Musculoskeletal outcomes by country group The most striking difference between country groups was with respect to the proportion of patients with no joint damage in the two younger age strata. In Group A, 12 patients (50% of the total) aged 5C21?years had no joint damage, compared with 3 (19%) in Group B and just 1 (4%) in Group C (Fig. 1a). In addition, only 2 of 24 patients had orthopaedic procedures (8%), compared with 5 of 16 (31%) from Group B and 9 of 28 (32%) from Group C (Fig. 1b). Open in a separate window Figure 1 Outcomes by country groups stratified by age. Clinical score was significantly better in Group A vs. Group C in the 5- to 10-year-old stratum ( em P? /em = em ? /em 0.04) (Fig. 1c). In contrast, in the 35-year-old stratum, Group C had a nonsignificantly lower score than Group A ( em P? /em = em ? /em 0.051) (Fig. 1c). As expected, the younger age strata showed significantly better scores for Group A compared with Groups B?+?C (5C10?years old, em P? /em = em ? /em 0.02; 11C21?years old, em P? /em = em ? /em 0.04; data not shown). Group A patients had significantly worse scores than Groups B?+?C in the 35-year-old stratum ( em P? /em = em ? /em 0.01; data not shown). In the two younger age strata, the mean number of affected joints in patients from Group C was approximately double that of patients from Group A (Table 2). The mean number.To best understand the effects of treatment on musculoskeletal outcome, we included only haemophilia A patients with severe disease and without inhibitors. from countries where long-term prophylaxis has been available for about 5?years (Group B) or was not available (Group C). In Latin America, the musculoskeletal status of patients with severe haemophilia without inhibitors has improved significantly in association with the provision of long-term prophylaxis. As more countries in Latin America institute this practice, further improvements are anticipated. of patients without joint damage (0/0)* (%)6 (50)6 (50)2 (25)1 (13)1 (8)0 (0)Mean of affected joints per patient (range)1.2 (0C3)1.8 (0C6)1.6 (0C4)1.8 (0C4)2.3 (0C4)3.6 (1C6)of patients with target joints (%)1 (8)2 (17)3 (38)4 (50)6 (50)10 (63)of patients with joint procedure (of joints treated)1 (1)1 (1)1 (1)4 (6)2 (3)7 (11)Clinical score,of patients on long-term prophylaxis (%)12 (100)6 (50)6 (75)7 (88)0 (0)0 (0)Mean age at start of prophylaxis, years (range)1.7 (0.8C5)5.7 (1.1C13)3.4 (1C7)14 (10C18)NANA Open in a separate window *WFH clinical score 0/Pettersson score 0. ?Categories (in order of decreasing frequency): 1?bleed/week; 2C3?bleeds/month; 7C12?bleeds/year; 4C6?bleeds/year; 1C3?bleeds/year; 1?bleed/year. NA, not applicable; SD, standard deviation. Table 3 Musculoskeletal evaluation of patients 21?years of age with severe haemophilia A in Latin America of patients without joint damage (0/0)*000000Mean of affected joints per patient (range)4.4 (3C7)5.6 (3C6)4 (2C6)4.5 (2C6)4.7 (2C7)5.1 (4C6)of patients with target joints (%)7 (70)8 (80)7 (64)7 (64)12 (67)9 (60)of patients with joint procedure (of joints treated)7 (11)3 (4)8 (15)8 (27)9 (17)7 (9)Clinical score,of patients on long-term prophylaxis (%)2 (20)0 (0)5 (45)4 (36)0 (0)0 (0)Mean age at start of prophylaxis, years (range)21.5 (16C27)NA21.4 (19C26)45 (36C62)NANA Open in a separate window *WFH clinical score 0/Pettersson score 0. ?Categories (in order of decreasing frequency): 1?bleed/week; 2C3?bleeds/month; 7C12?bleeds/year; 4C6?bleeds/year; 1C3?bleeds/year; 1?bleed/year. NA, not applicable; SD, standard deviation. Treatment characteristics by country In countries from Group A, long-term prophylaxis was made available between 1997 and 2002; in Group B countries since 2007 or 2008; and not at MS436 all in countries from Group C (Table 1). In the 5- to 10-year-old age stratum, all 12 patients from Group A received long-term prophylaxis, beginning at a mean age of 1 1.7?years. In Group B, 6 of 8 patients received primary prophylaxis, with a mean age at initiation of 3.4?years (Table 2). The most commonly used prophylaxis regimen was a flexible protocol of 20C30?IU?kg?1 3x/week. In Panama, a fixed protocol of 25?IU?kg?1 3x/week was used. Venezuela was the only country to offer tailored prophylaxis based on the Canadian protocol (50?IU?kg?1 1x/week or 30?IU?kg?1 2x/week or 25C30?IU?kg?1 3x/week) 8. Per capita factor usage was highest in Argentina and Chile, where long-term prophylaxis has been available for the longest period of time (Table 1). Uruguay had similarly high usage despite not offering long-term prophylaxis. Mexico and Peru had the lowest usage of factor per capita, and were also the only countries without 100% access to safe treatment. The use of recombinant factor was highest in Venezuela and Colombia (about 50%) 1. In contrast, all countries provided home treatment and short-term prophylaxis for all patients (Table 1). Musculoskeletal outcomes by country group The most striking difference between country groups was with respect to the proportion of patients with no joint damage in the two younger age strata. In Group A, 12 patients (50% of the total) aged 5C21?years had no joint damage, compared with 3 (19%) in Group B and just 1 (4%) Mouse monoclonal to PGR in Group C (Fig. 1a). In addition, only 2 of 24 patients had orthopaedic procedures (8%), compared with 5 of 16 (31%) from Group B and 9 of 28 (32%) from Group C (Fig. 1b). Open in a separate window Figure 1 Outcomes by country groups stratified by age. Clinical score was significantly better in Group A vs. Group C in the 5- to 10-year-old stratum ( em P? /em = em ? /em 0.04) (Fig. 1c). On MS436 the other hand, in the 35-year-old stratum, Group C acquired a non-significantly lower rating than Group A ( em P? /em = em ? /em 0.051) (Fig. 1c). Needlessly to say, the younger age group strata showed considerably better ratings for Group A weighed against Groupings B?+?C (5C10?years of age, em P? /em = em ? /em 0.02; 11C21?years of age, em P? /em = em ? /em 0.04; data.