Bleeding Disorders

Only a limited number of patients with von Willebrand disease (VWD) are likely to require treatment with von Willebrand factor (VWF) concentrates, according to results from a retrospective analysis.

The retrospective, single-center study sought to describe the need for VWF concentrates in a “real-life setting,” wrote Ana Boban, MD, PhD, of the University of Zagreb (Croatia) and colleagues in a letter to the editor published in Haemophilia. The study was conducted at the Saint‐Luc University Hospital in Brussels and included all the VWD patients listed in the hospital registry from 2000 to 2015.

The researchers evaluated the necessity for VWF concentrate therapy based on VWD severity (mild, moderate, severe) and type (1, 2A, 2B, 2M, 2N, or 3), in addition to therapeutic indication. Bleeding scores were not included in the analysis.

A total of 174 patients with VWD were included in the study, which consisted of 116 females and 61 males aged 3-81 years. The majority of study participants had type 1 VWD (n = 118, 67%).

Data collected included patient demographic information, disease type and severity, responses to specific tests, and type of treatment received.

The researchers found that just 18% (n = 32) of patients within the cohort required therapy with VWF concentrates over the study period. Additionally, the team reported that a large number of patients did not require any therapy (n = 79; 45%) or were or managed with DDAVP (desmopressin) (n = 61; 64%).

“When assessing the results according to disease severity, it clearly occurred that most patients with severe disease required VWF concentrates [100%, 75%, and 100% for type 1, type 2, and type 3 VWD, respectively],” the authors wrote.

With respect to therapeutic indication, apart from prophylaxis, contraindications to DDAVP (n = 5) and unresponsiveness to DDAVP (n = 7) were absolute indications for the use of VWF concentrates among patients within the cohort.

“Our study has confirmed that the absolute indications for using VWF concentrates in VWD patients are prophylaxis, major surgeries, and nonresponsiveness/contraindications to DDAVP,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Boban A et al. Haemophilia. 2019 May 20. doi: 10.1111/hae.13779.

Only a limited number of patients with von Willebrand disease (VWD) are likely to require treatment with von Willebrand factor (VWF) concentrates, according to results from a retrospective analysis.

The retrospective, single-center study sought to describe the need for VWF concentrates in a “real-life setting,” wrote Ana Boban, MD, PhD, of the University of Zagreb (Croatia) and colleagues in a letter to the editor published in Haemophilia. The study was conducted at the Saint‐Luc University Hospital in Brussels and included all the VWD patients listed in the hospital registry from 2000 to 2015.

The researchers evaluated the necessity for VWF concentrate therapy based on VWD severity (mild, moderate, severe) and type (1, 2A, 2B, 2M, 2N, or 3), in addition to therapeutic indication. Bleeding scores were not included in the analysis.

A total of 174 patients with VWD were included in the study, which consisted of 116 females and 61 males aged 3-81 years. The majority of study participants had type 1 VWD (n = 118, 67%).

Data collected included patient demographic information, disease type and severity, responses to specific tests, and type of treatment received.

The researchers found that just 18% (n = 32) of patients within the cohort required therapy with VWF concentrates over the study period. Additionally, the team reported that a large number of patients did not require any therapy (n = 79; 45%) or were or managed with DDAVP (desmopressin) (n = 61; 64%).

“When assessing the results according to disease severity, it clearly occurred that most patients with severe disease required VWF concentrates [100%, 75%, and 100% for type 1, type 2, and type 3 VWD, respectively],” the authors wrote.

With respect to therapeutic indication, apart from prophylaxis, contraindications to DDAVP (n = 5) and unresponsiveness to DDAVP (n = 7) were absolute indications for the use of VWF concentrates among patients within the cohort.

“Our study has confirmed that the absolute indications for using VWF concentrates in VWD patients are prophylaxis, major surgeries, and nonresponsiveness/contraindications to DDAVP,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Boban A et al. Haemophilia. 2019 May 20. doi: 10.1111/hae.13779.

Only a limited number of patients with von Willebrand disease (VWD) are likely to require treatment with von Willebrand factor (VWF) concentrates, according to results from a retrospective analysis.

The retrospective, single-center study sought to describe the need for VWF concentrates in a “real-life setting,” wrote Ana Boban, MD, PhD, of the University of Zagreb (Croatia) and colleagues in a letter to the editor published in Haemophilia. The study was conducted at the Saint‐Luc University Hospital in Brussels and included all the VWD patients listed in the hospital registry from 2000 to 2015.

The researchers evaluated the necessity for VWF concentrate therapy based on VWD severity (mild, moderate, severe) and type (1, 2A, 2B, 2M, 2N, or 3), in addition to therapeutic indication. Bleeding scores were not included in the analysis.

A total of 174 patients with VWD were included in the study, which consisted of 116 females and 61 males aged 3-81 years. The majority of study participants had type 1 VWD (n = 118, 67%).

Data collected included patient demographic information, disease type and severity, responses to specific tests, and type of treatment received.

The researchers found that just 18% (n = 32) of patients within the cohort required therapy with VWF concentrates over the study period. Additionally, the team reported that a large number of patients did not require any therapy (n = 79; 45%) or were or managed with DDAVP (desmopressin) (n = 61; 64%).

“When assessing the results according to disease severity, it clearly occurred that most patients with severe disease required VWF concentrates [100%, 75%, and 100% for type 1, type 2, and type 3 VWD, respectively],” the authors wrote.

With respect to therapeutic indication, apart from prophylaxis, contraindications to DDAVP (n = 5) and unresponsiveness to DDAVP (n = 7) were absolute indications for the use of VWF concentrates among patients within the cohort.

“Our study has confirmed that the absolute indications for using VWF concentrates in VWD patients are prophylaxis, major surgeries, and nonresponsiveness/contraindications to DDAVP,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Boban A et al. Haemophilia. 2019 May 20. doi: 10.1111/hae.13779.

Undergoing hepatectomy for the treatment of hepatocellular carcinoma in patients with von Willebrand disease or hemophilia A was found to be safe through the use of appropriate von Willebrand factor concentrate or recombinant factor VIII therapy, according to findings from a small study.

“The aim of the present study was to evaluate the safety of hepatectomy in patients with inherited blood coagulation disorders through appropriate coagulation factor replacement,” wrote Kosuke Kobayashi, MD, PhD, of the University of Tokyo and colleagues. The findings were published in Haemophilia.

The researchers retrospectively studied seven patients with hemophilia A and three patients with von Willebrand disease who underwent hepatectomy for the treatment of hepatocellular carcinoma. Specific regimens of von Willebrand factor concentrate or recombinant factor VIII therapy were administered perioperatively in these patients.

Study participants were matched in a 2:1 ratio to 20 patients without a bleeding disorder who also underwent hepatectomy. Various intraoperative and postoperative outcomes were compared between the two groups.

The researchers found no significant differences in estimated blood loss (P = .748), operative time (P = .359), or red blood cell transfusion rate (P = .605) between the bleeding disorder and nonbleeding disorder groups.

Additionally, there were no significant differences seen for mortality rate (P greater than .999) or major complication rate (P = .605).

“Even repeated hepatectomy can be safely performed in these patients, similar to patients without coagulation disorders,” the authors wrote.

Dr. Kobayashi and colleagues acknowledged two key limitations of the study were the small sample size and retrospective design.

“The administration protocol reported in the present study would certainly help surgeons when planning hepatectomy in patients with coagulation disorders,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Kobayashi K et al. Haemophilia. 2019 May 29. doi: 10.1111/hae.13759.

Undergoing hepatectomy for the treatment of hepatocellular carcinoma in patients with von Willebrand disease or hemophilia A was found to be safe through the use of appropriate von Willebrand factor concentrate or recombinant factor VIII therapy, according to findings from a small study.

“The aim of the present study was to evaluate the safety of hepatectomy in patients with inherited blood coagulation disorders through appropriate coagulation factor replacement,” wrote Kosuke Kobayashi, MD, PhD, of the University of Tokyo and colleagues. The findings were published in Haemophilia.

The researchers retrospectively studied seven patients with hemophilia A and three patients with von Willebrand disease who underwent hepatectomy for the treatment of hepatocellular carcinoma. Specific regimens of von Willebrand factor concentrate or recombinant factor VIII therapy were administered perioperatively in these patients.

Study participants were matched in a 2:1 ratio to 20 patients without a bleeding disorder who also underwent hepatectomy. Various intraoperative and postoperative outcomes were compared between the two groups.

The researchers found no significant differences in estimated blood loss (P = .748), operative time (P = .359), or red blood cell transfusion rate (P = .605) between the bleeding disorder and nonbleeding disorder groups.

Additionally, there were no significant differences seen for mortality rate (P greater than .999) or major complication rate (P = .605).

“Even repeated hepatectomy can be safely performed in these patients, similar to patients without coagulation disorders,” the authors wrote.

Dr. Kobayashi and colleagues acknowledged two key limitations of the study were the small sample size and retrospective design.

“The administration protocol reported in the present study would certainly help surgeons when planning hepatectomy in patients with coagulation disorders,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Kobayashi K et al. Haemophilia. 2019 May 29. doi: 10.1111/hae.13759.

Undergoing hepatectomy for the treatment of hepatocellular carcinoma in patients with von Willebrand disease or hemophilia A was found to be safe through the use of appropriate von Willebrand factor concentrate or recombinant factor VIII therapy, according to findings from a small study.

“The aim of the present study was to evaluate the safety of hepatectomy in patients with inherited blood coagulation disorders through appropriate coagulation factor replacement,” wrote Kosuke Kobayashi, MD, PhD, of the University of Tokyo and colleagues. The findings were published in Haemophilia.

The researchers retrospectively studied seven patients with hemophilia A and three patients with von Willebrand disease who underwent hepatectomy for the treatment of hepatocellular carcinoma. Specific regimens of von Willebrand factor concentrate or recombinant factor VIII therapy were administered perioperatively in these patients.

Study participants were matched in a 2:1 ratio to 20 patients without a bleeding disorder who also underwent hepatectomy. Various intraoperative and postoperative outcomes were compared between the two groups.

The researchers found no significant differences in estimated blood loss (P = .748), operative time (P = .359), or red blood cell transfusion rate (P = .605) between the bleeding disorder and nonbleeding disorder groups.

Additionally, there were no significant differences seen for mortality rate (P greater than .999) or major complication rate (P = .605).

“Even repeated hepatectomy can be safely performed in these patients, similar to patients without coagulation disorders,” the authors wrote.

Dr. Kobayashi and colleagues acknowledged two key limitations of the study were the small sample size and retrospective design.

“The administration protocol reported in the present study would certainly help surgeons when planning hepatectomy in patients with coagulation disorders,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Kobayashi K et al. Haemophilia. 2019 May 29. doi: 10.1111/hae.13759.

Results from a prospective study appear to confirm the utility of the Haemophilia Early Arthropathy Detection by UltraSound (HEAD‐US) scoring system to evaluate joint bleeding rates in patients with hemophilia A who are treated exclusively with secondary or tertiary prophylaxis.

HEAD-US showed a strong correlation with the joint bleeding rate over a 3 year period for ankles and knees, but not for elbows.

“Primary prophylaxis is considered the most effective regimen in preventing arthropathy formation. However, due to social and economic reasons, a considerable number of children and young adults with haemophilia worldwide are currently treated by secondary or tertiary prophylaxis,” Atanas Banchev, MD, of University Hospital Tsaritsa Giovanna – ISUL, Bulgaria, and colleagues, wrote in a letter to the editor in Haemophilia. “Due to its wide availability and low cost, the sonographic score Haemophilia Early Arthropathy Detection by UltraSound (HEAD‐US) has recently become an attractive tool for assessment of joint status.”

The researchers conducted a prospective study of 42 patients with moderate to severe hemophilia A. The patients were treated with secondary or tertiary prophylaxis for a minimum of 5 years and had no history of inhibitors or factor VIII inhibitor antibodies.

The team collected data on patient demographics, disease characteristics, and prophylactic therapy. Patients were assessed at regular intervals at various treatment facilities throughout Bulgaria.

The scoring was based on three markers: synovitis (score 0‐2), cartilage (score 0‐4) and subchondral bone (score of 0‐2) with a maximum score of eight points per joint, according to the researchers.

A total of 250 joints were evaluated via the HEAD‐US scoring system. Dr. Banchev and colleagues reported that articular damage was present in 34% (n = 85) of joints evaluated with the tool. No defects were detected in the remaining joints (n = 165).

Mean HEAD‐US scores for each specific joint were 2.2 (range 0‐8) for ankles, 0.96 (range 0‐8) for knees, and 0.73 (range 0‐6) for elbows.

The researchers reported a strong correlation between the assessed joint bleeding rates for 3 years and the corresponding HEAD-US scores in ankles and knees. For ankles, the Spearman’s correlation coefficient was 0.545 (P less than .001) and for knees it was 0.692 (P less than .001).

They found no statistically significant correlation between the HEAD-US score and the assessed joint bleeding rates for 3 years in elbows (coefficient 0.161, P = .143).

One key limitation of the study was the nonconsideration of therapeutic compliance in the outcome assessment.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Banchev A et al. Haemophilia. 2019 May 27. doi: 10.1111/hae.13771.

Results from a prospective study appear to confirm the utility of the Haemophilia Early Arthropathy Detection by UltraSound (HEAD‐US) scoring system to evaluate joint bleeding rates in patients with hemophilia A who are treated exclusively with secondary or tertiary prophylaxis.

HEAD-US showed a strong correlation with the joint bleeding rate over a 3 year period for ankles and knees, but not for elbows.

“Primary prophylaxis is considered the most effective regimen in preventing arthropathy formation. However, due to social and economic reasons, a considerable number of children and young adults with haemophilia worldwide are currently treated by secondary or tertiary prophylaxis,” Atanas Banchev, MD, of University Hospital Tsaritsa Giovanna – ISUL, Bulgaria, and colleagues, wrote in a letter to the editor in Haemophilia. “Due to its wide availability and low cost, the sonographic score Haemophilia Early Arthropathy Detection by UltraSound (HEAD‐US) has recently become an attractive tool for assessment of joint status.”

The researchers conducted a prospective study of 42 patients with moderate to severe hemophilia A. The patients were treated with secondary or tertiary prophylaxis for a minimum of 5 years and had no history of inhibitors or factor VIII inhibitor antibodies.

The team collected data on patient demographics, disease characteristics, and prophylactic therapy. Patients were assessed at regular intervals at various treatment facilities throughout Bulgaria.

The scoring was based on three markers: synovitis (score 0‐2), cartilage (score 0‐4) and subchondral bone (score of 0‐2) with a maximum score of eight points per joint, according to the researchers.

A total of 250 joints were evaluated via the HEAD‐US scoring system. Dr. Banchev and colleagues reported that articular damage was present in 34% (n = 85) of joints evaluated with the tool. No defects were detected in the remaining joints (n = 165).

Mean HEAD‐US scores for each specific joint were 2.2 (range 0‐8) for ankles, 0.96 (range 0‐8) for knees, and 0.73 (range 0‐6) for elbows.

The researchers reported a strong correlation between the assessed joint bleeding rates for 3 years and the corresponding HEAD-US scores in ankles and knees. For ankles, the Spearman’s correlation coefficient was 0.545 (P less than .001) and for knees it was 0.692 (P less than .001).

They found no statistically significant correlation between the HEAD-US score and the assessed joint bleeding rates for 3 years in elbows (coefficient 0.161, P = .143).

One key limitation of the study was the nonconsideration of therapeutic compliance in the outcome assessment.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Banchev A et al. Haemophilia. 2019 May 27. doi: 10.1111/hae.13771.

Results from a prospective study appear to confirm the utility of the Haemophilia Early Arthropathy Detection by UltraSound (HEAD‐US) scoring system to evaluate joint bleeding rates in patients with hemophilia A who are treated exclusively with secondary or tertiary prophylaxis.

HEAD-US showed a strong correlation with the joint bleeding rate over a 3 year period for ankles and knees, but not for elbows.

“Primary prophylaxis is considered the most effective regimen in preventing arthropathy formation. However, due to social and economic reasons, a considerable number of children and young adults with haemophilia worldwide are currently treated by secondary or tertiary prophylaxis,” Atanas Banchev, MD, of University Hospital Tsaritsa Giovanna – ISUL, Bulgaria, and colleagues, wrote in a letter to the editor in Haemophilia. “Due to its wide availability and low cost, the sonographic score Haemophilia Early Arthropathy Detection by UltraSound (HEAD‐US) has recently become an attractive tool for assessment of joint status.”

The researchers conducted a prospective study of 42 patients with moderate to severe hemophilia A. The patients were treated with secondary or tertiary prophylaxis for a minimum of 5 years and had no history of inhibitors or factor VIII inhibitor antibodies.

The team collected data on patient demographics, disease characteristics, and prophylactic therapy. Patients were assessed at regular intervals at various treatment facilities throughout Bulgaria.

The scoring was based on three markers: synovitis (score 0‐2), cartilage (score 0‐4) and subchondral bone (score of 0‐2) with a maximum score of eight points per joint, according to the researchers.

A total of 250 joints were evaluated via the HEAD‐US scoring system. Dr. Banchev and colleagues reported that articular damage was present in 34% (n = 85) of joints evaluated with the tool. No defects were detected in the remaining joints (n = 165).

Mean HEAD‐US scores for each specific joint were 2.2 (range 0‐8) for ankles, 0.96 (range 0‐8) for knees, and 0.73 (range 0‐6) for elbows.

The researchers reported a strong correlation between the assessed joint bleeding rates for 3 years and the corresponding HEAD-US scores in ankles and knees. For ankles, the Spearman’s correlation coefficient was 0.545 (P less than .001) and for knees it was 0.692 (P less than .001).

They found no statistically significant correlation between the HEAD-US score and the assessed joint bleeding rates for 3 years in elbows (coefficient 0.161, P = .143).

One key limitation of the study was the nonconsideration of therapeutic compliance in the outcome assessment.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Banchev A et al. Haemophilia. 2019 May 27. doi: 10.1111/hae.13771.

Combined aerobic and resistance exercise training was found to be the most effective training technique to reduce proinflammatory markers in overweight patients with moderate hemophilia A.

“Combined training has been established as the most effective type of exercise in terms of modification of cardiovascular disease risk factors,” wrote Behrouz Parhampour, of Iran University of Medical Sciences in Tehran, and colleagues. The findings of the study were published in Haemophilia.

The researchers conducted a randomized clinical study of 48 patients with moderate hemophilia A. Study patients had a body mass index of 25-30 kg/m² and were aged 35-55 years.

Study participants were randomly allocated to aerobic training (n = 12), resistance training (n = 12), combined training (n = 12), and control (n = 12) arms. The training regimens consisted of 45‐minute sessions three times per week for a total of 6 weeks.

Interleukin‐10, adiponectin, tumor necrosis factor–alpha, IL‐6, and high-sensitive C‐reactive protein were measured before and after training. Weight-related measures, including waist‐to‐hip ratio and waist circumference, were also evaluated.

The researchers found there was a significant reduction in weight, waist‐to‐hip ratio, waist circumference, and body mass index in the combined, resistance, and aerobic training arms, compared with the control arm.

Additionally, they reported a significant reduction in high-sensitive C‐reactive protein, IL‐6, and tumor necrosis factor–alpha levels in the combined training group versus the control group (P equal to or less than .02 for all three).

There were no episodes of bleeding among patients in any of the intervention groups.

“The possible mechanism for the effect of exercise training on weight loss is to increase metabolic consumption which may subsequently reduce the low‐grade inflammation commonly noted among overweight patients,” the researchers wrote.

The authors acknowledged that two key limitations of the study were the short duration of training and small sample size.

“Combined training can be used as an effective nonpharmacological strategy to improve joint function and prevent disorders associated with sedentary lifestyle like cardiovascular complications in [hemophilia patients],” they concluded.

The study was funded by the Iran University of Medical Sciences. The authors reported having no conflicts of interest.

SOURCE: Parhampour B et al. Haemophilia. 2019 May 26. doi: 10.1111/hae.13764.

Combined aerobic and resistance exercise training was found to be the most effective training technique to reduce proinflammatory markers in overweight patients with moderate hemophilia A.

“Combined training has been established as the most effective type of exercise in terms of modification of cardiovascular disease risk factors,” wrote Behrouz Parhampour, of Iran University of Medical Sciences in Tehran, and colleagues. The findings of the study were published in Haemophilia.

The researchers conducted a randomized clinical study of 48 patients with moderate hemophilia A. Study patients had a body mass index of 25-30 kg/m² and were aged 35-55 years.

Study participants were randomly allocated to aerobic training (n = 12), resistance training (n = 12), combined training (n = 12), and control (n = 12) arms. The training regimens consisted of 45‐minute sessions three times per week for a total of 6 weeks.

Interleukin‐10, adiponectin, tumor necrosis factor–alpha, IL‐6, and high-sensitive C‐reactive protein were measured before and after training. Weight-related measures, including waist‐to‐hip ratio and waist circumference, were also evaluated.

The researchers found there was a significant reduction in weight, waist‐to‐hip ratio, waist circumference, and body mass index in the combined, resistance, and aerobic training arms, compared with the control arm.

Additionally, they reported a significant reduction in high-sensitive C‐reactive protein, IL‐6, and tumor necrosis factor–alpha levels in the combined training group versus the control group (P equal to or less than .02 for all three).

There were no episodes of bleeding among patients in any of the intervention groups.

“The possible mechanism for the effect of exercise training on weight loss is to increase metabolic consumption which may subsequently reduce the low‐grade inflammation commonly noted among overweight patients,” the researchers wrote.

The authors acknowledged that two key limitations of the study were the short duration of training and small sample size.

“Combined training can be used as an effective nonpharmacological strategy to improve joint function and prevent disorders associated with sedentary lifestyle like cardiovascular complications in [hemophilia patients],” they concluded.

The study was funded by the Iran University of Medical Sciences. The authors reported having no conflicts of interest.

SOURCE: Parhampour B et al. Haemophilia. 2019 May 26. doi: 10.1111/hae.13764.

Combined aerobic and resistance exercise training was found to be the most effective training technique to reduce proinflammatory markers in overweight patients with moderate hemophilia A.

“Combined training has been established as the most effective type of exercise in terms of modification of cardiovascular disease risk factors,” wrote Behrouz Parhampour, of Iran University of Medical Sciences in Tehran, and colleagues. The findings of the study were published in Haemophilia.

The researchers conducted a randomized clinical study of 48 patients with moderate hemophilia A. Study patients had a body mass index of 25-30 kg/m² and were aged 35-55 years.

Study participants were randomly allocated to aerobic training (n = 12), resistance training (n = 12), combined training (n = 12), and control (n = 12) arms. The training regimens consisted of 45‐minute sessions three times per week for a total of 6 weeks.

Interleukin‐10, adiponectin, tumor necrosis factor–alpha, IL‐6, and high-sensitive C‐reactive protein were measured before and after training. Weight-related measures, including waist‐to‐hip ratio and waist circumference, were also evaluated.

The researchers found there was a significant reduction in weight, waist‐to‐hip ratio, waist circumference, and body mass index in the combined, resistance, and aerobic training arms, compared with the control arm.

Additionally, they reported a significant reduction in high-sensitive C‐reactive protein, IL‐6, and tumor necrosis factor–alpha levels in the combined training group versus the control group (P equal to or less than .02 for all three).

There were no episodes of bleeding among patients in any of the intervention groups.

“The possible mechanism for the effect of exercise training on weight loss is to increase metabolic consumption which may subsequently reduce the low‐grade inflammation commonly noted among overweight patients,” the researchers wrote.

The authors acknowledged that two key limitations of the study were the short duration of training and small sample size.

“Combined training can be used as an effective nonpharmacological strategy to improve joint function and prevent disorders associated with sedentary lifestyle like cardiovascular complications in [hemophilia patients],” they concluded.

The study was funded by the Iran University of Medical Sciences. The authors reported having no conflicts of interest.

SOURCE: Parhampour B et al. Haemophilia. 2019 May 26. doi: 10.1111/hae.13764.

Red blood cell (RBC) transfusions from either previously pregnant, sex-discordant, or female donors were not significantly associated with higher mortality among transfusion recipients, according to a retrospective analysis of more than 1 million donors.

“This study used data from 3 large cohorts in the United States and Scandinavia to investigate whether blood donor sex and pregnancy history were associated with mortality of transfusion recipients,” wrote Gustaf Edgren, MD, PhD, of Karolinska University Hospital, Stockholm, and colleagues. The findings were published in JAMA.

The researchers analyzed data from three separate cohorts that included a combined 1,047,382 red blood cell transfusion recipients. Data collected included donor-related information, such as sex and pregnancy history, as well as survival data of transfusion recipients. The primary outcome measured was in-hospital mortality, and the secondary outcome was long-term mortality. Data were collected until Dec. 31, 2016.

The researchers found no statistically significant associations between either sex-discordant donors (male donor to female recipient or female donor to male recipient), female donors, or previously pregnant donors and in-hospital mortality of transfusion recipients.

The hazard ratio estimates for each unit transfused from a previously pregnant donor ranged from 1.00-1.01 in the three cohorts. Similarly, the HR estimates ranged from 0.99-1.00 for female donors in the three cohorts and 0.99-1.02 for sex discordant donors.

The only significant association found was observed in the smallest cohort of 34,662 recipients. Researchers found an increased risk of death in patients who received one to two sex discordant transfusions (HR, 1.08; 95% confidence interval, 1.03-1.14) or five to six transfusions (HR, 1.14; 95%CI, 1.01-1.29), compared with recipients who received no sex-discordant transfusions.

“The results are reassuring in that the survival of patients who got transfused with red blood cells does not appear to be associated with whether the blood they received was donated by a man, by a woman who had been pregnant — or by one who had not. That’s important to know,” Simone Glynn, MD, chief of the Blood Epidemiology and Clinical Therapeutics Branch at the National Heart, Lung, and Blood Institute, as well as a study author, said in a statement.

The study was funded by the National Heart, Lung, and Blood Institute. The authors reported financial disclosures related to the National Institutes of Health, RTI International, Cerus, AABB, Creative Testing Solutions, and the Nordic Cancer Union.

SOURCE: Edgren G et al. JAMA. 2019 Jun 11. doi: 10.1001/jama.2019.7084.

Red blood cell (RBC) transfusions from either previously pregnant, sex-discordant, or female donors were not significantly associated with higher mortality among transfusion recipients, according to a retrospective analysis of more than 1 million donors.

“This study used data from 3 large cohorts in the United States and Scandinavia to investigate whether blood donor sex and pregnancy history were associated with mortality of transfusion recipients,” wrote Gustaf Edgren, MD, PhD, of Karolinska University Hospital, Stockholm, and colleagues. The findings were published in JAMA.

The researchers analyzed data from three separate cohorts that included a combined 1,047,382 red blood cell transfusion recipients. Data collected included donor-related information, such as sex and pregnancy history, as well as survival data of transfusion recipients. The primary outcome measured was in-hospital mortality, and the secondary outcome was long-term mortality. Data were collected until Dec. 31, 2016.

The researchers found no statistically significant associations between either sex-discordant donors (male donor to female recipient or female donor to male recipient), female donors, or previously pregnant donors and in-hospital mortality of transfusion recipients.

The hazard ratio estimates for each unit transfused from a previously pregnant donor ranged from 1.00-1.01 in the three cohorts. Similarly, the HR estimates ranged from 0.99-1.00 for female donors in the three cohorts and 0.99-1.02 for sex discordant donors.

The only significant association found was observed in the smallest cohort of 34,662 recipients. Researchers found an increased risk of death in patients who received one to two sex discordant transfusions (HR, 1.08; 95% confidence interval, 1.03-1.14) or five to six transfusions (HR, 1.14; 95%CI, 1.01-1.29), compared with recipients who received no sex-discordant transfusions.

“The results are reassuring in that the survival of patients who got transfused with red blood cells does not appear to be associated with whether the blood they received was donated by a man, by a woman who had been pregnant — or by one who had not. That’s important to know,” Simone Glynn, MD, chief of the Blood Epidemiology and Clinical Therapeutics Branch at the National Heart, Lung, and Blood Institute, as well as a study author, said in a statement.

The study was funded by the National Heart, Lung, and Blood Institute. The authors reported financial disclosures related to the National Institutes of Health, RTI International, Cerus, AABB, Creative Testing Solutions, and the Nordic Cancer Union.

SOURCE: Edgren G et al. JAMA. 2019 Jun 11. doi: 10.1001/jama.2019.7084.

Red blood cell (RBC) transfusions from either previously pregnant, sex-discordant, or female donors were not significantly associated with higher mortality among transfusion recipients, according to a retrospective analysis of more than 1 million donors.

“This study used data from 3 large cohorts in the United States and Scandinavia to investigate whether blood donor sex and pregnancy history were associated with mortality of transfusion recipients,” wrote Gustaf Edgren, MD, PhD, of Karolinska University Hospital, Stockholm, and colleagues. The findings were published in JAMA.

The researchers analyzed data from three separate cohorts that included a combined 1,047,382 red blood cell transfusion recipients. Data collected included donor-related information, such as sex and pregnancy history, as well as survival data of transfusion recipients. The primary outcome measured was in-hospital mortality, and the secondary outcome was long-term mortality. Data were collected until Dec. 31, 2016.

The researchers found no statistically significant associations between either sex-discordant donors (male donor to female recipient or female donor to male recipient), female donors, or previously pregnant donors and in-hospital mortality of transfusion recipients.

The hazard ratio estimates for each unit transfused from a previously pregnant donor ranged from 1.00-1.01 in the three cohorts. Similarly, the HR estimates ranged from 0.99-1.00 for female donors in the three cohorts and 0.99-1.02 for sex discordant donors.

The only significant association found was observed in the smallest cohort of 34,662 recipients. Researchers found an increased risk of death in patients who received one to two sex discordant transfusions (HR, 1.08; 95% confidence interval, 1.03-1.14) or five to six transfusions (HR, 1.14; 95%CI, 1.01-1.29), compared with recipients who received no sex-discordant transfusions.

“The results are reassuring in that the survival of patients who got transfused with red blood cells does not appear to be associated with whether the blood they received was donated by a man, by a woman who had been pregnant — or by one who had not. That’s important to know,” Simone Glynn, MD, chief of the Blood Epidemiology and Clinical Therapeutics Branch at the National Heart, Lung, and Blood Institute, as well as a study author, said in a statement.

The study was funded by the National Heart, Lung, and Blood Institute. The authors reported financial disclosures related to the National Institutes of Health, RTI International, Cerus, AABB, Creative Testing Solutions, and the Nordic Cancer Union.

SOURCE: Edgren G et al. JAMA. 2019 Jun 11. doi: 10.1001/jama.2019.7084.

The Food and Drug Administration is alerting laboratories and providers to a class I recall on Beckman Coulter hematology analyzers because of the potential for inaccurate platelet count results.

A class I recall indicates reasonable probability of serious adverse health consequences or death associated with use, according to the FDA.

The recall is related to the devices’ platelet analyzing function; among other uses, these devices help assess patients fitness for surgery, so a faulty reading on platelet counts could result in increased risk for life-threatening bleeding during a procedure in patients who have unidentified severe thrombocytopenia, according to a statement from the agency.

“Because this may cause serious injury, or even death, to a patient, we are urging health care professionals to be aware of the potential for inaccurate diagnostic results with these analyzers and to take appropriate actions including the use of alternative diagnostic testing or confirming analyzer results with manual scanning or estimate of platelets,” Tim Stenzel, MD, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health, said in the statement.

The recall applies to the UniCel DxH 800 Coulter Cellular Analysis System, UniCel DxH 600 Coulter Cellular Analysis System, and UniCel DxH 900 Coulter Cellular Analysis System. The faulty devices were first identified in 2018, and the manufacturer released an urgent medical device correction letter at that time. The company has more recently released a software patch for the devices, but the FDA has not yet assessed whether it resolves the problem. The agency has released detailed actions and recommendations related to these devices.

At this time, the FDA is unaware of any serious adverse events that have been directly linked to these devices, but the agency recommends that any events be reported through its MedWatch reporting system.

[email protected]

The Food and Drug Administration is alerting laboratories and providers to a class I recall on Beckman Coulter hematology analyzers because of the potential for inaccurate platelet count results.

A class I recall indicates reasonable probability of serious adverse health consequences or death associated with use, according to the FDA.

The recall is related to the devices’ platelet analyzing function; among other uses, these devices help assess patients fitness for surgery, so a faulty reading on platelet counts could result in increased risk for life-threatening bleeding during a procedure in patients who have unidentified severe thrombocytopenia, according to a statement from the agency.

“Because this may cause serious injury, or even death, to a patient, we are urging health care professionals to be aware of the potential for inaccurate diagnostic results with these analyzers and to take appropriate actions including the use of alternative diagnostic testing or confirming analyzer results with manual scanning or estimate of platelets,” Tim Stenzel, MD, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health, said in the statement.

The recall applies to the UniCel DxH 800 Coulter Cellular Analysis System, UniCel DxH 600 Coulter Cellular Analysis System, and UniCel DxH 900 Coulter Cellular Analysis System. The faulty devices were first identified in 2018, and the manufacturer released an urgent medical device correction letter at that time. The company has more recently released a software patch for the devices, but the FDA has not yet assessed whether it resolves the problem. The agency has released detailed actions and recommendations related to these devices.

At this time, the FDA is unaware of any serious adverse events that have been directly linked to these devices, but the agency recommends that any events be reported through its MedWatch reporting system.

[email protected]

The Food and Drug Administration is alerting laboratories and providers to a class I recall on Beckman Coulter hematology analyzers because of the potential for inaccurate platelet count results.

A class I recall indicates reasonable probability of serious adverse health consequences or death associated with use, according to the FDA.

The recall is related to the devices’ platelet analyzing function; among other uses, these devices help assess patients fitness for surgery, so a faulty reading on platelet counts could result in increased risk for life-threatening bleeding during a procedure in patients who have unidentified severe thrombocytopenia, according to a statement from the agency.

“Because this may cause serious injury, or even death, to a patient, we are urging health care professionals to be aware of the potential for inaccurate diagnostic results with these analyzers and to take appropriate actions including the use of alternative diagnostic testing or confirming analyzer results with manual scanning or estimate of platelets,” Tim Stenzel, MD, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health, said in the statement.

The recall applies to the UniCel DxH 800 Coulter Cellular Analysis System, UniCel DxH 600 Coulter Cellular Analysis System, and UniCel DxH 900 Coulter Cellular Analysis System. The faulty devices were first identified in 2018, and the manufacturer released an urgent medical device correction letter at that time. The company has more recently released a software patch for the devices, but the FDA has not yet assessed whether it resolves the problem. The agency has released detailed actions and recommendations related to these devices.

At this time, the FDA is unaware of any serious adverse events that have been directly linked to these devices, but the agency recommends that any events be reported through its MedWatch reporting system.

[email protected]

The FVIII/W (factor VIII:C/von Willebrand factor antigen) ratio appears to be a reliable biological marker to predict recovery and/or relapse in patients with acquired hemophilia A, according to a retrospective analysis.

Marc Trossaert, MD, PhD, of the CHU de Nantes, France, and colleagues conducted a retrospective analysis of 64 consecutive patients diagnosed with acquired hemophilia A over a period of 15 years (2000-2015). Data were obtained from institutional databases at the Toulouse and Nantes university hospitals in France.

Data collected included patient demographics, comorbidities, biological factors, and information related to immunosuppressive therapy. The findings of the study were published in Haemophilia.

To ascertain normal parameters and uses of the FVIII/W ratio, the team assessed FVIII:C and VWF:Ag levels of 40 healthy individuals and normal parameters of the ratio were defined using these levels.

Among the 64 patients with acquired hemophilia A who were enrolled in the study, 55 patients achieved complete remission. Of that group, 44 patients did not relapse. Researchers had follow-up data of at least 1 year for 22 of these patients. They found that the FVIII/W ratio remained within normal parameters for all 22 patients.

Researchers had follow-up data on 5 of the 11 patients who relapsed during the study period. For 4 of the patients, a decrease of FVIII/W ratio was the first indicator of relapse. In the fifth patient, an abnormal activated partial thromboplastin time (aPTT) displayed before the changes were observed in the FVIII/W ratio.

Dr. Trossaert and his colleagues acknowledged that a key limitation of the study was the retrospective design.

“We cannot eliminate the fact that in these patients less frequent testing may have influenced the chance of seeing a low FVIII/W ratio,” they wrote.

The biomarker now needs to be studied in larger cohorts, the researchers suggested.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Trossaert M et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13752.

The FVIII/W (factor VIII:C/von Willebrand factor antigen) ratio appears to be a reliable biological marker to predict recovery and/or relapse in patients with acquired hemophilia A, according to a retrospective analysis.

Marc Trossaert, MD, PhD, of the CHU de Nantes, France, and colleagues conducted a retrospective analysis of 64 consecutive patients diagnosed with acquired hemophilia A over a period of 15 years (2000-2015). Data were obtained from institutional databases at the Toulouse and Nantes university hospitals in France.

Data collected included patient demographics, comorbidities, biological factors, and information related to immunosuppressive therapy. The findings of the study were published in Haemophilia.

To ascertain normal parameters and uses of the FVIII/W ratio, the team assessed FVIII:C and VWF:Ag levels of 40 healthy individuals and normal parameters of the ratio were defined using these levels.

Among the 64 patients with acquired hemophilia A who were enrolled in the study, 55 patients achieved complete remission. Of that group, 44 patients did not relapse. Researchers had follow-up data of at least 1 year for 22 of these patients. They found that the FVIII/W ratio remained within normal parameters for all 22 patients.

Researchers had follow-up data on 5 of the 11 patients who relapsed during the study period. For 4 of the patients, a decrease of FVIII/W ratio was the first indicator of relapse. In the fifth patient, an abnormal activated partial thromboplastin time (aPTT) displayed before the changes were observed in the FVIII/W ratio.

Dr. Trossaert and his colleagues acknowledged that a key limitation of the study was the retrospective design.

“We cannot eliminate the fact that in these patients less frequent testing may have influenced the chance of seeing a low FVIII/W ratio,” they wrote.

The biomarker now needs to be studied in larger cohorts, the researchers suggested.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Trossaert M et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13752.

The FVIII/W (factor VIII:C/von Willebrand factor antigen) ratio appears to be a reliable biological marker to predict recovery and/or relapse in patients with acquired hemophilia A, according to a retrospective analysis.

Marc Trossaert, MD, PhD, of the CHU de Nantes, France, and colleagues conducted a retrospective analysis of 64 consecutive patients diagnosed with acquired hemophilia A over a period of 15 years (2000-2015). Data were obtained from institutional databases at the Toulouse and Nantes university hospitals in France.

Data collected included patient demographics, comorbidities, biological factors, and information related to immunosuppressive therapy. The findings of the study were published in Haemophilia.

To ascertain normal parameters and uses of the FVIII/W ratio, the team assessed FVIII:C and VWF:Ag levels of 40 healthy individuals and normal parameters of the ratio were defined using these levels.

Among the 64 patients with acquired hemophilia A who were enrolled in the study, 55 patients achieved complete remission. Of that group, 44 patients did not relapse. Researchers had follow-up data of at least 1 year for 22 of these patients. They found that the FVIII/W ratio remained within normal parameters for all 22 patients.

Researchers had follow-up data on 5 of the 11 patients who relapsed during the study period. For 4 of the patients, a decrease of FVIII/W ratio was the first indicator of relapse. In the fifth patient, an abnormal activated partial thromboplastin time (aPTT) displayed before the changes were observed in the FVIII/W ratio.

Dr. Trossaert and his colleagues acknowledged that a key limitation of the study was the retrospective design.

“We cannot eliminate the fact that in these patients less frequent testing may have influenced the chance of seeing a low FVIII/W ratio,” they wrote.

The biomarker now needs to be studied in larger cohorts, the researchers suggested.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Trossaert M et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13752.

A novel automated chromogenic assay demonstrated diagnostic accuracy and limited variation in patients with hemophilia A, according to recent study findings.

“The original one‐stage clotting assay is still the most widely used method for measuring FVIII activity in these patients, although the chromogenic assay is recognized to be less prone to the variability related to the use of different reagents and to the presence of interferences,” Cristina Novembrino, MD, of the Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, in Milan, and colleagues wrote in Haemophilia. “The choice of the proper assay is a crucial point in the frame of diagnosis, particularly in moderate or mild [hemophilia A] patients.”

The BIOPHEN FVIII:C assay, used on the Sysmex CS‐2400 analyzer, is a novel chromogenic diagnostic tool used to analyze FVIII clotting activity in patients with hemophilia A of all severity levels. The researchers evaluated the diagnostic and clinical capabilities of the assay in 60 patients with hemophilia A and 120 healthy controls.

Dr. Novembrino and colleagues used samples of FVIII deficient plasma and Actin FS to compare the novel tool to a one-stage assay and another chromogenic assay.

After analysis, the researchers found that the inter‐assay and intra‐assay coefficient of variation were less than 6%. The mean recovery and limit of detection were 91.7% (range, 79.8%-98.6%) and 0.2%, respectively.

The linearity test revealed positive results of up to 1/128 dilution (r = 0.99).

“The K coefficient was 0.91 when BIOPHEN FVIII:C was compared with the historical classification of the patients, demonstrating an optimal diagnostic accuracy in hemophilia A,” the researchers wrote.

The novel assay may be an appropriate laboratory tool for the diagnosis and therapeutic monitoring of patients with hemophilia A, they added.

Sysmex Corporation and Hyphen BioMed provided the instrument and reagents for the study. One of the authors is an employee of Sysmex Corporation. The authors reported having no other conflicts of interest.

SOURCE: Novembrino C et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13746.

A novel automated chromogenic assay demonstrated diagnostic accuracy and limited variation in patients with hemophilia A, according to recent study findings.

“The original one‐stage clotting assay is still the most widely used method for measuring FVIII activity in these patients, although the chromogenic assay is recognized to be less prone to the variability related to the use of different reagents and to the presence of interferences,” Cristina Novembrino, MD, of the Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, in Milan, and colleagues wrote in Haemophilia. “The choice of the proper assay is a crucial point in the frame of diagnosis, particularly in moderate or mild [hemophilia A] patients.”

The BIOPHEN FVIII:C assay, used on the Sysmex CS‐2400 analyzer, is a novel chromogenic diagnostic tool used to analyze FVIII clotting activity in patients with hemophilia A of all severity levels. The researchers evaluated the diagnostic and clinical capabilities of the assay in 60 patients with hemophilia A and 120 healthy controls.

Dr. Novembrino and colleagues used samples of FVIII deficient plasma and Actin FS to compare the novel tool to a one-stage assay and another chromogenic assay.

After analysis, the researchers found that the inter‐assay and intra‐assay coefficient of variation were less than 6%. The mean recovery and limit of detection were 91.7% (range, 79.8%-98.6%) and 0.2%, respectively.

The linearity test revealed positive results of up to 1/128 dilution (r = 0.99).

“The K coefficient was 0.91 when BIOPHEN FVIII:C was compared with the historical classification of the patients, demonstrating an optimal diagnostic accuracy in hemophilia A,” the researchers wrote.

The novel assay may be an appropriate laboratory tool for the diagnosis and therapeutic monitoring of patients with hemophilia A, they added.

Sysmex Corporation and Hyphen BioMed provided the instrument and reagents for the study. One of the authors is an employee of Sysmex Corporation. The authors reported having no other conflicts of interest.

SOURCE: Novembrino C et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13746.

A novel automated chromogenic assay demonstrated diagnostic accuracy and limited variation in patients with hemophilia A, according to recent study findings.

“The original one‐stage clotting assay is still the most widely used method for measuring FVIII activity in these patients, although the chromogenic assay is recognized to be less prone to the variability related to the use of different reagents and to the presence of interferences,” Cristina Novembrino, MD, of the Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, in Milan, and colleagues wrote in Haemophilia. “The choice of the proper assay is a crucial point in the frame of diagnosis, particularly in moderate or mild [hemophilia A] patients.”

The BIOPHEN FVIII:C assay, used on the Sysmex CS‐2400 analyzer, is a novel chromogenic diagnostic tool used to analyze FVIII clotting activity in patients with hemophilia A of all severity levels. The researchers evaluated the diagnostic and clinical capabilities of the assay in 60 patients with hemophilia A and 120 healthy controls.

Dr. Novembrino and colleagues used samples of FVIII deficient plasma and Actin FS to compare the novel tool to a one-stage assay and another chromogenic assay.

After analysis, the researchers found that the inter‐assay and intra‐assay coefficient of variation were less than 6%. The mean recovery and limit of detection were 91.7% (range, 79.8%-98.6%) and 0.2%, respectively.

The linearity test revealed positive results of up to 1/128 dilution (r = 0.99).

“The K coefficient was 0.91 when BIOPHEN FVIII:C was compared with the historical classification of the patients, demonstrating an optimal diagnostic accuracy in hemophilia A,” the researchers wrote.

The novel assay may be an appropriate laboratory tool for the diagnosis and therapeutic monitoring of patients with hemophilia A, they added.

Sysmex Corporation and Hyphen BioMed provided the instrument and reagents for the study. One of the authors is an employee of Sysmex Corporation. The authors reported having no other conflicts of interest.

SOURCE: Novembrino C et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13746.

Tertiary prophylaxis with low‐dose factor VIII (FVIII) was effective in decreasing the number of joint and total bleeding events versus on‐demand treatment in children with severe hemophilia A.

The findings have implications for low resource countries, the researchers reported.

Novie A. Chozie, MD, of Universitas Indonesia in Jakarta, along with colleagues, conducted a parallel-group, randomized controlled study of 50 children with severe hemophilia A. Study participants were randomized to receive either low‐dose FVIII prophylaxis (n = 25) or on‐demand treatment (n = 25) for a total of 12 months. The findings were published in Haemophilia.

Participants in the prophylaxis arm received FVIII at a dose of 10 IU/kg, infused twice weekly. If patients experienced an episode of acute bleeding, prophylaxis was delayed until the episode resolved.

The primary outcome was the number of total and joint bleeding events from the start of therapy to 12 months. Secondary outcomes included evidence of FVIII inhibitor, Hemophilia Joint Health Score (HJHS), and Hemophilia Early Arthropathy Detection Ultrasound (HEAD‐US) score.

After analysis, the team found that the number of joint and total bleeding events was significantly lower in the prophylaxis group. For total bleeding events, there was a median of 8 events with the prophylaxis group, compared with 25 in the on-demand treatment group (P less than .001). There was a median of three joint bleeding events in the prophylaxis group versus nine in the on-demand group (P less than .001).

Patients in the prophylaxis arm also showed improved joint function (P = .004), while those in the on‐demand arm showed evidence of deterioration (P = .001).

Two key limitations of the study were short duration of follow-up and single‐center design, which could have limited the generalizability of the results.

“In countries with limited resources, low‐dose prophylaxis is strongly recommended as a therapeutic option for severe haemophilia A [in] children,” the researchers wrote.

The study was funded by Grifols. The authors reported having no conflicts of interest.

SOURCE: Chozie NA et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13770.

Tertiary prophylaxis with low‐dose factor VIII (FVIII) was effective in decreasing the number of joint and total bleeding events versus on‐demand treatment in children with severe hemophilia A.

The findings have implications for low resource countries, the researchers reported.

Novie A. Chozie, MD, of Universitas Indonesia in Jakarta, along with colleagues, conducted a parallel-group, randomized controlled study of 50 children with severe hemophilia A. Study participants were randomized to receive either low‐dose FVIII prophylaxis (n = 25) or on‐demand treatment (n = 25) for a total of 12 months. The findings were published in Haemophilia.

Participants in the prophylaxis arm received FVIII at a dose of 10 IU/kg, infused twice weekly. If patients experienced an episode of acute bleeding, prophylaxis was delayed until the episode resolved.

The primary outcome was the number of total and joint bleeding events from the start of therapy to 12 months. Secondary outcomes included evidence of FVIII inhibitor, Hemophilia Joint Health Score (HJHS), and Hemophilia Early Arthropathy Detection Ultrasound (HEAD‐US) score.

After analysis, the team found that the number of joint and total bleeding events was significantly lower in the prophylaxis group. For total bleeding events, there was a median of 8 events with the prophylaxis group, compared with 25 in the on-demand treatment group (P less than .001). There was a median of three joint bleeding events in the prophylaxis group versus nine in the on-demand group (P less than .001).

Patients in the prophylaxis arm also showed improved joint function (P = .004), while those in the on‐demand arm showed evidence of deterioration (P = .001).

Two key limitations of the study were short duration of follow-up and single‐center design, which could have limited the generalizability of the results.

“In countries with limited resources, low‐dose prophylaxis is strongly recommended as a therapeutic option for severe haemophilia A [in] children,” the researchers wrote.

The study was funded by Grifols. The authors reported having no conflicts of interest.

SOURCE: Chozie NA et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13770.

Tertiary prophylaxis with low‐dose factor VIII (FVIII) was effective in decreasing the number of joint and total bleeding events versus on‐demand treatment in children with severe hemophilia A.

The findings have implications for low resource countries, the researchers reported.

Novie A. Chozie, MD, of Universitas Indonesia in Jakarta, along with colleagues, conducted a parallel-group, randomized controlled study of 50 children with severe hemophilia A. Study participants were randomized to receive either low‐dose FVIII prophylaxis (n = 25) or on‐demand treatment (n = 25) for a total of 12 months. The findings were published in Haemophilia.

Participants in the prophylaxis arm received FVIII at a dose of 10 IU/kg, infused twice weekly. If patients experienced an episode of acute bleeding, prophylaxis was delayed until the episode resolved.

The primary outcome was the number of total and joint bleeding events from the start of therapy to 12 months. Secondary outcomes included evidence of FVIII inhibitor, Hemophilia Joint Health Score (HJHS), and Hemophilia Early Arthropathy Detection Ultrasound (HEAD‐US) score.

After analysis, the team found that the number of joint and total bleeding events was significantly lower in the prophylaxis group. For total bleeding events, there was a median of 8 events with the prophylaxis group, compared with 25 in the on-demand treatment group (P less than .001). There was a median of three joint bleeding events in the prophylaxis group versus nine in the on-demand group (P less than .001).

Patients in the prophylaxis arm also showed improved joint function (P = .004), while those in the on‐demand arm showed evidence of deterioration (P = .001).

Two key limitations of the study were short duration of follow-up and single‐center design, which could have limited the generalizability of the results.

“In countries with limited resources, low‐dose prophylaxis is strongly recommended as a therapeutic option for severe haemophilia A [in] children,” the researchers wrote.

The study was funded by Grifols. The authors reported having no conflicts of interest.

SOURCE: Chozie NA et al. Haemophilia. 2019 May 2. doi: 10.1111/hae.13770.

Disease severity and family history appear to play a significant role in the age of diagnosis for women with congenital bleeding disorders, according to recent survey findings.

A European multinational survey has identified delays in diagnosis and other challenges faced by girls and women with congenital bleeding disorders.

“The aim of this survey, carried out by the European Haemophilia Consortium (EHC), was to provide the patient voice of their lived experiences with congenital bleeding disorders,” wrote Declan Noone of the EHC in Brussels, and colleagues. The findings were published in Haemophilia.

The researchers conducted a survey of 709 girls and women with various congenital bleeding disorders from 32 countries, primarily located in Western Europe. Most respondents were adults, with just 3.8% under age 18 years.

The questionnaire was administered to eligible patients at various hemophilia treatment centers. More than half of respondents were hemophilia carriers and nearly 28% had von Willebrand disease.

The survey explored the effects of bleeding disorders on several activities of daily life, including symptoms, physical activity, and reproductive ability.

After analysis, the researchers found that overall the median age at diagnosis of a bleeding disorder was 16 years (range, 2-28 years) among respondents. Having a family history of a bleeding disorder resulted in a significantly younger median age at diagnosis (6 years; range, 0-26 years) versus those without a family history (17 years; range 5-28 years; P less than .01).

Disease severity also appears to play a role. Women with type 3 von Willebrand disease had a median age of diagnosis of 1 year old, compared with 19.3 years old for type 2 disease (P less than .01).

Respondents reported a substantial disease burden on activities of daily life, especially for women with platelet function disorders and other factor deficiency.

Women without a known family history of a bleeding disorders reported a significantly greater impact on their physical life, social life, and romantic life (P less than .01 for all domains), compared with women with a family history of bleeding disorders.

There were no statistically significant differences across types of bleeding disorders on questions related to reproductive life. However, the researchers reported that “surprisingly,” 25% of women reported that having a bleeding disorder “has had a severe impact on their decision or has prevented them from having children.

“The bleeding symptom of biggest impact on daily life is [heavy menstrual bleeding], reported by 55% of women,” the researchers wrote.

The researchers acknowledged that a key limitation of the survey was the composition of the sample: predominantly of patients from Western Europe. As a result, the findings may not be generalizable to all patient populations.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Noone D et al. Haemophilia. 2019 Apr 29. doi: 10.1111/hae.13722.

Disease severity and family history appear to play a significant role in the age of diagnosis for women with congenital bleeding disorders, according to recent survey findings.

A European multinational survey has identified delays in diagnosis and other challenges faced by girls and women with congenital bleeding disorders.

“The aim of this survey, carried out by the European Haemophilia Consortium (EHC), was to provide the patient voice of their lived experiences with congenital bleeding disorders,” wrote Declan Noone of the EHC in Brussels, and colleagues. The findings were published in Haemophilia.

The researchers conducted a survey of 709 girls and women with various congenital bleeding disorders from 32 countries, primarily located in Western Europe. Most respondents were adults, with just 3.8% under age 18 years.

The questionnaire was administered to eligible patients at various hemophilia treatment centers. More than half of respondents were hemophilia carriers and nearly 28% had von Willebrand disease.

The survey explored the effects of bleeding disorders on several activities of daily life, including symptoms, physical activity, and reproductive ability.

After analysis, the researchers found that overall the median age at diagnosis of a bleeding disorder was 16 years (range, 2-28 years) among respondents. Having a family history of a bleeding disorder resulted in a significantly younger median age at diagnosis (6 years; range, 0-26 years) versus those without a family history (17 years; range 5-28 years; P less than .01).

Disease severity also appears to play a role. Women with type 3 von Willebrand disease had a median age of diagnosis of 1 year old, compared with 19.3 years old for type 2 disease (P less than .01).

Respondents reported a substantial disease burden on activities of daily life, especially for women with platelet function disorders and other factor deficiency.

Women without a known family history of a bleeding disorders reported a significantly greater impact on their physical life, social life, and romantic life (P less than .01 for all domains), compared with women with a family history of bleeding disorders.

There were no statistically significant differences across types of bleeding disorders on questions related to reproductive life. However, the researchers reported that “surprisingly,” 25% of women reported that having a bleeding disorder “has had a severe impact on their decision or has prevented them from having children.

“The bleeding symptom of biggest impact on daily life is [heavy menstrual bleeding], reported by 55% of women,” the researchers wrote.

The researchers acknowledged that a key limitation of the survey was the composition of the sample: predominantly of patients from Western Europe. As a result, the findings may not be generalizable to all patient populations.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Noone D et al. Haemophilia. 2019 Apr 29. doi: 10.1111/hae.13722.

Disease severity and family history appear to play a significant role in the age of diagnosis for women with congenital bleeding disorders, according to recent survey findings.

A European multinational survey has identified delays in diagnosis and other challenges faced by girls and women with congenital bleeding disorders.

“The aim of this survey, carried out by the European Haemophilia Consortium (EHC), was to provide the patient voice of their lived experiences with congenital bleeding disorders,” wrote Declan Noone of the EHC in Brussels, and colleagues. The findings were published in Haemophilia.

The researchers conducted a survey of 709 girls and women with various congenital bleeding disorders from 32 countries, primarily located in Western Europe. Most respondents were adults, with just 3.8% under age 18 years.

The questionnaire was administered to eligible patients at various hemophilia treatment centers. More than half of respondents were hemophilia carriers and nearly 28% had von Willebrand disease.

The survey explored the effects of bleeding disorders on several activities of daily life, including symptoms, physical activity, and reproductive ability.

After analysis, the researchers found that overall the median age at diagnosis of a bleeding disorder was 16 years (range, 2-28 years) among respondents. Having a family history of a bleeding disorder resulted in a significantly younger median age at diagnosis (6 years; range, 0-26 years) versus those without a family history (17 years; range 5-28 years; P less than .01).

Disease severity also appears to play a role. Women with type 3 von Willebrand disease had a median age of diagnosis of 1 year old, compared with 19.3 years old for type 2 disease (P less than .01).

Respondents reported a substantial disease burden on activities of daily life, especially for women with platelet function disorders and other factor deficiency.

Women without a known family history of a bleeding disorders reported a significantly greater impact on their physical life, social life, and romantic life (P less than .01 for all domains), compared with women with a family history of bleeding disorders.

There were no statistically significant differences across types of bleeding disorders on questions related to reproductive life. However, the researchers reported that “surprisingly,” 25% of women reported that having a bleeding disorder “has had a severe impact on their decision or has prevented them from having children.

“The bleeding symptom of biggest impact on daily life is [heavy menstrual bleeding], reported by 55% of women,” the researchers wrote.

The researchers acknowledged that a key limitation of the survey was the composition of the sample: predominantly of patients from Western Europe. As a result, the findings may not be generalizable to all patient populations.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Noone D et al. Haemophilia. 2019 Apr 29. doi: 10.1111/hae.13722.