RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFE
Regular prophylaxis with exogenous Factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data on bleeding, FVIII use, and quality of life (QoL) for this population in routine clinical practice are scarce. In a prospective, multinational, noninterventiona...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Elsevier
2021-10-01
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| Series: | Hematology, Transfusion and Cell Therapy |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S253113792100540X |
| _version_ | 1830200551081508864 |
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| author | GG Yamaguti-Hayakawa PR Villaça C Lorenzato MH Cerqueira LCO Oliveira ACKVD Nascimento CBF Dametto DB Reddy H Yu MC Ozelo |
| author_facet | GG Yamaguti-Hayakawa PR Villaça C Lorenzato MH Cerqueira LCO Oliveira ACKVD Nascimento CBF Dametto DB Reddy H Yu MC Ozelo |
| author_sort | GG Yamaguti-Hayakawa |
| collection | DOAJ |
| description | Regular prophylaxis with exogenous Factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data on bleeding, FVIII use, and quality of life (QoL) for this population in routine clinical practice are scarce. In a prospective, multinational, noninterventional study, male participants age ≥18 years with severe HA (FVIII ≤1 IU/dL) receiving prophylactic FVIII were followed for at least 6 months. Bleeding and FVIII use were reported weekly. Annualised bleeding rate (ABR) and FVIII infusion rate were calculated for participants with ≥6 months follow-up and combined with retrospective data from 6 months prior to baseline. Adverse events (AEs) were collected monthly. Participants completed the haemophilia-specific health-related QoL questionnaire for adults (Haemo-QoL-A) at baseline. This abstract presents a sub analysis of the study's Brazilian population. For all 54 Brazilian participants, median (range) age was 27 (18–47) years. For the 41 (76%) Brazilian participants with ≥6 months follow-up, mean (standard deviation [SD]; median) ABR for treated bleeds was 2.42 (4.05; 0.80) bleeds per year and mean (SD; median) annualised FVIII infusion rate was 167.87 (60.22; 155.17) infusions per year. Mean (SD; median) ABRs of treated traumatic, spontaneous, and joint bleeds were 0.90 (1.6; 0.0), 1.52 (3.3; 0.0), and 1.91 (3.29; 0.0) bleeds per year, respectively. Median (range) percentage of follow-up time adherent with prescribed prophylaxis frequency in Brazilian participants was 94.7%(49%-100%). Mean (SD) Haemo-QoL-A total transformed score (0–100) score was 70.3 (15.1) The lowest mean (SD) domain scores (representing lower QoL) in all Brazilian participants were for treatment concern 45.7 (30.85), physical functioning 66.9 (21.33), and consequences of bleeding 68.1 (22.92). AEs were reported in 22 (40.7%) participants, with the most common AEs being haemophilic arthropathy, nasopharyngitis, and synovitis, each reported in 2 (3.7%) Brazilian participants. Two serious AEs (oesophageal haemorrhage and haematoma) were reported, each by single participant (1.9%). Despite high prophylaxis adherence, participants reported breakthrough spontaneous and joint bleeding and impaired physical functioning. Additional haemostatic options for severe HA are needed. |
| first_indexed | 2024-12-18T02:36:09Z |
| format | Article |
| id | doaj.art-fca3a6d31ef24cbfa72733afba58ef1b |
| institution | Directory Open Access Journal |
| issn | 2531-1379 |
| language | English |
| last_indexed | 2024-12-18T02:36:09Z |
| publishDate | 2021-10-01 |
| publisher | Elsevier |
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| series | Hematology, Transfusion and Cell Therapy |
| spelling | doaj.art-fca3a6d31ef24cbfa72733afba58ef1b2022-12-21T21:23:46ZengElsevierHematology, Transfusion and Cell Therapy2531-13792021-10-0143S232S233RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFEGG Yamaguti-Hayakawa0PR Villaça1C Lorenzato2MH Cerqueira3LCO Oliveira4ACKVD Nascimento5CBF Dametto6DB Reddy7H Yu8MC Ozelo9Centro de Hematologia e Hemoterapia (Hemocentro), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, BrazilHospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (HCFMUSP), São Paulo, SP, BrazilCentro de Hematologia e Hemoterapia do Paraná (HEMEPAR), Curitiba, PR, BrazilInstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti (Hemorio), Rio de Janeiro, RJ, BrazilFundação Hemocentro de Ribeirão Preto (FUNDHERP), Faculdade de Medicina de Ribeirão Preto (FMRP), Universidade de São Paulo (USP), Ribeirão Preto, SP, BrazilServico de Hematologia, Irmandade da Santa Casa de Misericórdia de São Paulo, São Paulo, SP, BrazilHemocentro do Estado do Rio Grande do Sul (HEMORGS), Porto Alegre, RS, BrazilBioMarin Pharmaceutical Inc., Novato, United StatesBioMarin Pharmaceutical Inc., Novato, United StatesCentro de Hematologia e Hemoterapia (Hemocentro), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, BrazilRegular prophylaxis with exogenous Factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data on bleeding, FVIII use, and quality of life (QoL) for this population in routine clinical practice are scarce. In a prospective, multinational, noninterventional study, male participants age ≥18 years with severe HA (FVIII ≤1 IU/dL) receiving prophylactic FVIII were followed for at least 6 months. Bleeding and FVIII use were reported weekly. Annualised bleeding rate (ABR) and FVIII infusion rate were calculated for participants with ≥6 months follow-up and combined with retrospective data from 6 months prior to baseline. Adverse events (AEs) were collected monthly. Participants completed the haemophilia-specific health-related QoL questionnaire for adults (Haemo-QoL-A) at baseline. This abstract presents a sub analysis of the study's Brazilian population. For all 54 Brazilian participants, median (range) age was 27 (18–47) years. For the 41 (76%) Brazilian participants with ≥6 months follow-up, mean (standard deviation [SD]; median) ABR for treated bleeds was 2.42 (4.05; 0.80) bleeds per year and mean (SD; median) annualised FVIII infusion rate was 167.87 (60.22; 155.17) infusions per year. Mean (SD; median) ABRs of treated traumatic, spontaneous, and joint bleeds were 0.90 (1.6; 0.0), 1.52 (3.3; 0.0), and 1.91 (3.29; 0.0) bleeds per year, respectively. Median (range) percentage of follow-up time adherent with prescribed prophylaxis frequency in Brazilian participants was 94.7%(49%-100%). Mean (SD) Haemo-QoL-A total transformed score (0–100) score was 70.3 (15.1) The lowest mean (SD) domain scores (representing lower QoL) in all Brazilian participants were for treatment concern 45.7 (30.85), physical functioning 66.9 (21.33), and consequences of bleeding 68.1 (22.92). AEs were reported in 22 (40.7%) participants, with the most common AEs being haemophilic arthropathy, nasopharyngitis, and synovitis, each reported in 2 (3.7%) Brazilian participants. Two serious AEs (oesophageal haemorrhage and haematoma) were reported, each by single participant (1.9%). Despite high prophylaxis adherence, participants reported breakthrough spontaneous and joint bleeding and impaired physical functioning. Additional haemostatic options for severe HA are needed.http://www.sciencedirect.com/science/article/pii/S253113792100540X |
| spellingShingle | GG Yamaguti-Hayakawa PR Villaça C Lorenzato MH Cerqueira LCO Oliveira ACKVD Nascimento CBF Dametto DB Reddy H Yu MC Ozelo RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFE Hematology, Transfusion and Cell Therapy |
| title | RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFE |
| title_full | RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFE |
| title_fullStr | RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFE |
| title_full_unstemmed | RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFE |
| title_short | RESULTS FROM A 52-WEEK, NONINTERVENTIONAL STUDY OF BRAZILIAN INDIVIDUALS WITH SEVERE HAEMOPHILIA A RECEIVING PROPHYLAXIS: RATES OF BLEEDING, FVIII USE, AND QUALITY OF LIFE |
| title_sort | results from a 52 week noninterventional study of brazilian individuals with severe haemophilia a receiving prophylaxis rates of bleeding fviii use and quality of life |
| url | http://www.sciencedirect.com/science/article/pii/S253113792100540X |
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