Our study’s aim is to derive combined PMD/sgTCD

microemboli criteria to overcome this limitation. Patients with symptomatic carotid disease were prospectively enrolled within 24 h of symptom onset underwent 1 hour TCD emboli monitoring. We reviewed disparity between PMD MES criteria and sgTCD MES criteria. We compared combined PMD/sgTCD criteria to sgTCD alone criteria by measuring the intraclass correlation coefficient (ICC). Of 92 patients, 28 patients had evidence of MES on sgTCD or PMD. Total Obeticholic Acid purchase MES count was 269 based on sgTCD criteria, and 326 based on combined PMD/sgTCD criteria (P= 0.005). Combined PMD/sgTCD criteria revealed 17 MESs (4.8%) based on sgTCD criteria to represent artifacts and 57 MESs (17.5%) not to be detected by sgTCD criteria. Overall ICC based on sgTCD criteria was 0.67 [95% confidence interval (CI): 0.58–0.74]; however, introducing combined

PMD/sgTCD criteria resulted in a significant increase in the ICC, 0.91 (95% CI: 0.88–0.93). Our combined PMD/sgTCD criteria for MES appeared Dinaciclib price to improve the yield of MES detection. Reliability in MES detection interpretation was improved when combined PMD/sgTCD criteria was applied. “
“Several prospective studies have shown that carotid endarterectomy can reduce the risk for subsequent ischemic stroke in patients with 70-99% stenosis of the internal carotid artery (ICA). However, its benefits are still controversial in less than 70% stenosis of the ICA. There is increasing evidence that

medchemexpress carotid lumen irregularities may correlate with neurological symptoms. Recent development of computed tomography angiography (CTA) can provide adequate information on the carotid plaque morphology. In this study, therefore, we aimed to clarify whether carotid lumen morphology estimated by CTA correlates with neurological symptoms in patients with 30-69% ICA stenosis. This study included 67 carotid stenotic lesions with 30-69% ICA stenosis in 52 consecutive patients. These 67 lesions were examined by CTA from the viewpoints of the degree of stenosis, the prevalence of ulceration, and lumen morphology. Multivariate analysis was performed to detect significant predictors for the occurrence of ipsilateral ischemic events. Multivariate analysis showed that the irregular shape of the carotid lumen was the most powerful variable to predict symptomatic lesion in 30-69% ICA stenosis. These findings suggest that the morphology of carotid plaque may be associated with the occurrence of ipsilateral ischemic events in 30-69% ICA stenosis. J Neuroimaging 2011;21:348-354. “
“Cerebral mitochondrial dysfunction has been observed in Parkinson’s disease (PD). If mitochondrial dysfunction is an early event contributing to PD development, then noninvasive techniques that detect disturbed energy metabolism in vivo might be useful tools for early diagnosis and treatment monitoring.

Our study’s aim is to derive combined PMD/sgTCD

microemboli criteria to overcome this limitation. Patients with symptomatic carotid disease were prospectively enrolled within 24 h of symptom onset underwent 1 hour TCD emboli monitoring. We reviewed disparity between PMD MES criteria and sgTCD MES criteria. We compared combined PMD/sgTCD criteria to sgTCD alone criteria by measuring the intraclass correlation coefficient (ICC). Of 92 patients, 28 patients had evidence of MES on sgTCD or PMD. Total Ensartinib MES count was 269 based on sgTCD criteria, and 326 based on combined PMD/sgTCD criteria (P= 0.005). Combined PMD/sgTCD criteria revealed 17 MESs (4.8%) based on sgTCD criteria to represent artifacts and 57 MESs (17.5%) not to be detected by sgTCD criteria. Overall ICC based on sgTCD criteria was 0.67 [95% confidence interval (CI): 0.58–0.74]; however, introducing combined

PMD/sgTCD criteria resulted in a significant increase in the ICC, 0.91 (95% CI: 0.88–0.93). Our combined PMD/sgTCD criteria for MES appeared CH5424802 molecular weight to improve the yield of MES detection. Reliability in MES detection interpretation was improved when combined PMD/sgTCD criteria was applied. “
“Several prospective studies have shown that carotid endarterectomy can reduce the risk for subsequent ischemic stroke in patients with 70-99% stenosis of the internal carotid artery (ICA). However, its benefits are still controversial in less than 70% stenosis of the ICA. There is increasing evidence that

上海皓元 carotid lumen irregularities may correlate with neurological symptoms. Recent development of computed tomography angiography (CTA) can provide adequate information on the carotid plaque morphology. In this study, therefore, we aimed to clarify whether carotid lumen morphology estimated by CTA correlates with neurological symptoms in patients with 30-69% ICA stenosis. This study included 67 carotid stenotic lesions with 30-69% ICA stenosis in 52 consecutive patients. These 67 lesions were examined by CTA from the viewpoints of the degree of stenosis, the prevalence of ulceration, and lumen morphology. Multivariate analysis was performed to detect significant predictors for the occurrence of ipsilateral ischemic events. Multivariate analysis showed that the irregular shape of the carotid lumen was the most powerful variable to predict symptomatic lesion in 30-69% ICA stenosis. These findings suggest that the morphology of carotid plaque may be associated with the occurrence of ipsilateral ischemic events in 30-69% ICA stenosis. J Neuroimaging 2011;21:348-354. “
“Cerebral mitochondrial dysfunction has been observed in Parkinson’s disease (PD). If mitochondrial dysfunction is an early event contributing to PD development, then noninvasive techniques that detect disturbed energy metabolism in vivo might be useful tools for early diagnosis and treatment monitoring.

To understand the role of HSC in liver immunity, we investigated the effect of this transition on T cell stimulation in vitro. Unlike quiescent HSC, activated HSC did not induce proliferation of antigen-specific T cells. Phenotypic analysis of quiescent and activated HSC revealed that activated HSC expressed

the coinhibitory molecule B7-H4. Silencing B7-H4 by small interfering RNA (siRNA) in activated HSC restored the ability of T cells to proliferate, differentiate, and regain effector recall responses. Furthermore, expression of B7-H4 on HSC inhibits early T cell activation and addition of exogenous interleukin (IL)-2 reversed the T cell anergy induced by activated HSC. Conclusion: These studies reveal a novel role for activated

HSC AZD1208 in the attenuation of intrahepatic T cell responses by way of expression of the coinhibitory molecule B7-H4, and may provide fundamental insight into intrahepatic immunity during liver fibrogenesis. (HEPATOLOGY 2010) Chronic liver disease is the tenth leading cause of mortality in the United States.1 Whether a viral infection such as hepatitis C virus (HCV) or a noninfectious insult such as alcohol or a genetic disease is the inciting agent, each shares a common route to eventual liver failure characterized by inflammation, fibrosis, and cirrhosis.2 Hepatic stellate cells (HSC) are the major cell types in the liver responsible for liver fibrosis.3 These cells are nonparenchymal cells that comprise 5%-8% of the normal liver, located in the space of Disse between the endothelial layer and parenchymal hepatocytes,4 and serve as a depot of vitamin Selleckchem AZD1152HQPA A.5 In the healthy liver, HSC are present in a quiescent form and perform multiple physiologic functions including directing hepatic development and regeneration as well as producing lipoproteins, growth factors, and cytokines.5 However, during liver injury HSC become activated,6

leading to a phenotypic and functionally consequent transformation. Activated HSC (AHSC) are the major mediators 上海皓元 of liver fibrosis through extracellular matrix deposition (type I and type III collagen), secretion of the vasoconstrictor endothelin-1 which contributes to portal hypertension, and reduced production of matrix metalloprotease-1 necessary for the degradation of collagen type 1.5 In addition to their role in liver fibrosis, recent evidence has also placed HSC at the center of the intrahepatic immune response.7 HSC secrete chemokines, express various Toll-like receptors, and are phagocytic.7 HSC have been shown to prevent graft rejection in a transplantation model by inhibiting T cell responses8 and have also been shown to expand regulatory T cells in an interleukin (IL)-2-dependent manner.9 AHSC interact with, and in fact, also engulf lymphocytes through phagocytosis during liver fibrosis.

To understand the role of HSC in liver immunity, we investigated the effect of this transition on T cell stimulation in vitro. Unlike quiescent HSC, activated HSC did not induce proliferation of antigen-specific T cells. Phenotypic analysis of quiescent and activated HSC revealed that activated HSC expressed

the coinhibitory molecule B7-H4. Silencing B7-H4 by small interfering RNA (siRNA) in activated HSC restored the ability of T cells to proliferate, differentiate, and regain effector recall responses. Furthermore, expression of B7-H4 on HSC inhibits early T cell activation and addition of exogenous interleukin (IL)-2 reversed the T cell anergy induced by activated HSC. Conclusion: These studies reveal a novel role for activated

HSC GPCR & G Protein inhibitor in the attenuation of intrahepatic T cell responses by way of expression of the coinhibitory molecule B7-H4, and may provide fundamental insight into intrahepatic immunity during liver fibrogenesis. (HEPATOLOGY 2010) Chronic liver disease is the tenth leading cause of mortality in the United States.1 Whether a viral infection such as hepatitis C virus (HCV) or a noninfectious insult such as alcohol or a genetic disease is the inciting agent, each shares a common route to eventual liver failure characterized by inflammation, fibrosis, and cirrhosis.2 Hepatic stellate cells (HSC) are the major cell types in the liver responsible for liver fibrosis.3 These cells are nonparenchymal cells that comprise 5%-8% of the normal liver, located in the space of Disse between the endothelial layer and parenchymal hepatocytes,4 and serve as a depot of vitamin see more A.5 In the healthy liver, HSC are present in a quiescent form and perform multiple physiologic functions including directing hepatic development and regeneration as well as producing lipoproteins, growth factors, and cytokines.5 However, during liver injury HSC become activated,6

leading to a phenotypic and functionally consequent transformation. Activated HSC (AHSC) are the major mediators MCE of liver fibrosis through extracellular matrix deposition (type I and type III collagen), secretion of the vasoconstrictor endothelin-1 which contributes to portal hypertension, and reduced production of matrix metalloprotease-1 necessary for the degradation of collagen type 1.5 In addition to their role in liver fibrosis, recent evidence has also placed HSC at the center of the intrahepatic immune response.7 HSC secrete chemokines, express various Toll-like receptors, and are phagocytic.7 HSC have been shown to prevent graft rejection in a transplantation model by inhibiting T cell responses8 and have also been shown to expand regulatory T cells in an interleukin (IL)-2-dependent manner.9 AHSC interact with, and in fact, also engulf lymphocytes through phagocytosis during liver fibrosis.

Seventeen thousand seven hundred and thirty one H. pylori strains were collected from eight Vadimezan areas of two provinces in coastal southeast China from 2010 to

2012. The resistance of these strains to six antibiotics was tested using the agar dilution method. The resistance rates to clarithromycin, metronidazole, levofloxacin, amoxicillin, gentamicin and furazolidone were 21.5, 95.4, 20.6, 0.1, 0.1 and 0.1%, respectively. Double, triple and quadruple antibacterial resistant percentages were 25.5, 7.5 and 0.1%, respectively. A positive association between the resistance to levofloxacin and to clarithromycin was found, but there was a negative correlation in the resistances to levofloxacin and to metronidazole. The prevalence of H. pylori resistance to clarithromycin, metronidazole, levofloxacin and multiple antibiotics

in coastal southeast China is high. Choice of therapy should be individualized based on a susceptibility test in this region of the country. “
“To document the efficacy and tolerability of 14-day bismuth–lansoprazole–amoxicillin–clarithromycin (BLAC) regimen for Helicobacter pylori (H. pylori) eradication as a first-line therapy. Patients were considered eligible for the study if they underwent upper gastrointestinal endoscopy, and H. pylori infection was diagnosed through histologic examination of antral and body biopsy samples. Primary end point of this study was to evaluate the eradication rate of 14-day BLAC regimen therapies. H. pylori

eradication was assessed using the 13C urea breath test this website performed 6 weeks after the completion of treatment. All patients were asked to fill in a validated questionnaire to report therapy-related side effects. Each symptom was graded from absent or present. MCE Ninety-seven (21 men and 76 women) were enrolled. All the patients completed the study. The H. pylori eradication rate was 90.7% (88 of 97 patients). Side effects were observed in reasonable percentages, and none of the patients left the study because of drug side effect. Bismuth–lansoprazole–amoxicillin–clarithromycin regimen as a 2-week course achieved an acceptable eradication rate with relatively mild side effects. “
“This article reviews the literature published pertaining to Helicobacter pylori eradication over the last year. The general perception among clinicians and academics engaged in research on H. pylori has been that eradication rates for first-line therapies are falling, although some data published this year have cast doubt on this. The studies published this year have therefore focussed on developing alternative strategies for the first-line eradication of H. pylori. In this regard, clear evidence now exists that both levofloxacin and bismuth are viable options for first-line therapy. The sequential and “concomitant” regimes have also been studied in new settings and may have a role in future algorithms also.

Seventeen thousand seven hundred and thirty one H. pylori strains were collected from eight selleck inhibitor areas of two provinces in coastal southeast China from 2010 to

2012. The resistance of these strains to six antibiotics was tested using the agar dilution method. The resistance rates to clarithromycin, metronidazole, levofloxacin, amoxicillin, gentamicin and furazolidone were 21.5, 95.4, 20.6, 0.1, 0.1 and 0.1%, respectively. Double, triple and quadruple antibacterial resistant percentages were 25.5, 7.5 and 0.1%, respectively. A positive association between the resistance to levofloxacin and to clarithromycin was found, but there was a negative correlation in the resistances to levofloxacin and to metronidazole. The prevalence of H. pylori resistance to clarithromycin, metronidazole, levofloxacin and multiple antibiotics

in coastal southeast China is high. Choice of therapy should be individualized based on a susceptibility test in this region of the country. “
“To document the efficacy and tolerability of 14-day bismuth–lansoprazole–amoxicillin–clarithromycin (BLAC) regimen for Helicobacter pylori (H. pylori) eradication as a first-line therapy. Patients were considered eligible for the study if they underwent upper gastrointestinal endoscopy, and H. pylori infection was diagnosed through histologic examination of antral and body biopsy samples. Primary end point of this study was to evaluate the eradication rate of 14-day BLAC regimen therapies. H. pylori

eradication was assessed using the 13C urea breath test selleck chemical performed 6 weeks after the completion of treatment. All patients were asked to fill in a validated questionnaire to report therapy-related side effects. Each symptom was graded from absent or present. 上海皓元 Ninety-seven (21 men and 76 women) were enrolled. All the patients completed the study. The H. pylori eradication rate was 90.7% (88 of 97 patients). Side effects were observed in reasonable percentages, and none of the patients left the study because of drug side effect. Bismuth–lansoprazole–amoxicillin–clarithromycin regimen as a 2-week course achieved an acceptable eradication rate with relatively mild side effects. “
“This article reviews the literature published pertaining to Helicobacter pylori eradication over the last year. The general perception among clinicians and academics engaged in research on H. pylori has been that eradication rates for first-line therapies are falling, although some data published this year have cast doubt on this. The studies published this year have therefore focussed on developing alternative strategies for the first-line eradication of H. pylori. In this regard, clear evidence now exists that both levofloxacin and bismuth are viable options for first-line therapy. The sequential and “concomitant” regimes have also been studied in new settings and may have a role in future algorithms also.

3%) had a diagnosis of AKI. AKI diagnoses increased nearly 3-fold, from 5,922 in 2002 to 17,320 in 201 0, and the use of HD for AKI increased from 748 RG7420 solubility dmso to 1,441. The mean age of patients receiving HD was 57, and 65.3% were male. 21.1% of patients received HD in non-liver transplant centers. The Elixhauser comorbidity index was similar for those receiving (3.7) versus those not receiving HD (3.5). 1 1.8% of those receiving HD had decompensated

cirrhosis. Private insurance was more common among those receiving HD (30.1 vs. 24.3%). Overall inpatient mortality for those on HD decreased over time, from 50.5% in 2002 to 3 1.7% in 201 0, and was higher in transplant centers (53.4 vs. 3 1.3%). Mortality for those with decompensated cirrhosis who received HD was 42.1%. After adjusting for disease severity and other patient-level factors, HD was associated with increased mortality (odds ratio 2.15; 95% confidence interval, 2.02–2.29). Hepatic decompensation, sepsis, self-pay insurance status and hepatocellular carcinoma were also independently associated with increased mortality. Private insurance, Medicare, Medicaid, and receipt of a liver or kidney transplant were associated with decreased mortality. Median length of stay was significantly longer for

those receiving HD (1 3 versus 7 days), and median total charges were significantly higher ($92,312 versus $37,277). Conclusion: AKI appears to be increasing amongst hospitalized cirrhotics while HD utilization is increasing at a lower rate. A significant number of patients receive HD at non-transplant centers. HD in this Selleck PD332991 population is associated with substantial mortality, costs, and length of stay. More detailed information on these patients and longer-term outcomes are needed to assess the growing use of HD and its cost-effectiveness. Disclosures: Monica Schmidt – Grant/Research Support: Merck & Co.; Patent Held/Filed: HCCplex; Stock Shareholder: PleX Diagnostics Alfred 上海皓元医药股份有限公司 S. Barritt – Grant/Research Support: Salix Pharmaceuticals; Speaking and Teaching: Abbott Molecular The following people have nothing to disclose:

Paul H. Hayashi, Eric S. Orman Background: In patients with HCV, evidence of cirrhosis should trigger several therapeutic and preventive measures. One such example is hepatocellular cancer (HCC) screening. However, success of any screening program is contingent upon early identification of all at-risk patients—i.e., those with cirrhosis. The extent to which cirrhosis is under-diagnosed in HCV and the subsequent impact on HCC stage in clinical practice is unclear. Methods: We identified HCV patients from the national VA HCV Clinical Case Registry between 1995 and 2010. We determined the prevalence of cirrhosis on the basis of (a) validated ICD9 codes for cirrhosis & (b) >1 AST to platelet ratio index (APRI) score > 2.0. We calculated the incidence rate of HCC in patients with cirrhosis classified based on ICD9 codes or APRI.

3%) had a diagnosis of AKI. AKI diagnoses increased nearly 3-fold, from 5,922 in 2002 to 17,320 in 201 0, and the use of HD for AKI increased from 748 selleck products to 1,441. The mean age of patients receiving HD was 57, and 65.3% were male. 21.1% of patients received HD in non-liver transplant centers. The Elixhauser comorbidity index was similar for those receiving (3.7) versus those not receiving HD (3.5). 1 1.8% of those receiving HD had decompensated

cirrhosis. Private insurance was more common among those receiving HD (30.1 vs. 24.3%). Overall inpatient mortality for those on HD decreased over time, from 50.5% in 2002 to 3 1.7% in 201 0, and was higher in transplant centers (53.4 vs. 3 1.3%). Mortality for those with decompensated cirrhosis who received HD was 42.1%. After adjusting for disease severity and other patient-level factors, HD was associated with increased mortality (odds ratio 2.15; 95% confidence interval, 2.02–2.29). Hepatic decompensation, sepsis, self-pay insurance status and hepatocellular carcinoma were also independently associated with increased mortality. Private insurance, Medicare, Medicaid, and receipt of a liver or kidney transplant were associated with decreased mortality. Median length of stay was significantly longer for

those receiving HD (1 3 versus 7 days), and median total charges were significantly higher ($92,312 versus $37,277). Conclusion: AKI appears to be increasing amongst hospitalized cirrhotics while HD utilization is increasing at a lower rate. A significant number of patients receive HD at non-transplant centers. HD in this selleck chemicals population is associated with substantial mortality, costs, and length of stay. More detailed information on these patients and longer-term outcomes are needed to assess the growing use of HD and its cost-effectiveness. Disclosures: Monica Schmidt – Grant/Research Support: Merck & Co.; Patent Held/Filed: HCCplex; Stock Shareholder: PleX Diagnostics Alfred MCE S. Barritt – Grant/Research Support: Salix Pharmaceuticals; Speaking and Teaching: Abbott Molecular The following people have nothing to disclose:

Paul H. Hayashi, Eric S. Orman Background: In patients with HCV, evidence of cirrhosis should trigger several therapeutic and preventive measures. One such example is hepatocellular cancer (HCC) screening. However, success of any screening program is contingent upon early identification of all at-risk patients—i.e., those with cirrhosis. The extent to which cirrhosis is under-diagnosed in HCV and the subsequent impact on HCC stage in clinical practice is unclear. Methods: We identified HCV patients from the national VA HCV Clinical Case Registry between 1995 and 2010. We determined the prevalence of cirrhosis on the basis of (a) validated ICD9 codes for cirrhosis & (b) >1 AST to platelet ratio index (APRI) score > 2.0. We calculated the incidence rate of HCC in patients with cirrhosis classified based on ICD9 codes or APRI.

These children develop life-threatening complications, including refractory coagulopathies, hepatic encephalopathy, multi-organ failure and death. Therapies for patients awaiting transplant are merely supportive, including large volumes of blood products to correct coagulopathy, resulting

in volume and protein overload and citrate toxicity. Therapeutic plasma exchange (TPE) allows for a bridge to either recovery or transplant by correcting coagulopathies, PCI32765 clearing toxins, and improving cytokine balance. There are no published pediatric studies describing the safety of TPE in children with hepatic failure. Methods: Charts of ESLD patients from 2010-2013 at Texas Children’s Hospital who received TPE for hepatic encephalopathy, severe coagulopathy, selleck chemicals or ABO mismatch

liver transplant (n=20) were reviewed. TPE was performed by replacing 1.5 total plasma volume with fresh frozen plasma, and anticoagulation was regional with citrate. A protocol for TPE was used for all patients in 2013 (n = 10), and included 5 daily TPE treatments, followed by every other day treatments until recovery, transplant or death. Prior to this protocol, TPE was used randomly on a physician-directed basis. Data: Over 4 years, 20 patients with ESLD were supported with TPE for a total of 102 treatments. Patients received 5 treatments on average. No infectious complications or deaths were associated with TPE. TPE was done in tandem with CRRT in the majority of patients (85%). Citrate lock, MCE defined as a total calcium to ionized calcium ratio of ≥ 2.4, was seen in the majority of patients (85%), and improved by increasing calcium chloride. 60% of patients experienced hypotension requiring increased inotropic support, and 60% experienced complications with catheters including bleeding and/or clotting (67%). Despite these side effects, no treatment interruption was necessary, even in patients on multiple vasoactive agents. In the 10 patients subject to the 2013 TPE protocol, 4 were successfully

bridged to liver transplantation, and 1 had spontaneous resolution of disease. Prior to the 2013 protocol, 4/10 patients were successfully bridged to transplant. Conclusion: These data demonstrate the safety of TPE in children with ESLD. Despite commonly experiencing severe citrate lock, hemodynamic instability, and catheter complications, TPE was well tolerated and did not result in cessation of therapy or death. The benefits of TPE as a therapeutic bridge allows for longer survival while awaiting liver transplant. Disclosures: The following people have nothing to disclose: Amy S. Arrington, Moreshwar S. Desai, Ayse Akcan Arikan, Jun Teruya, Poyyapakkam R. Srivaths Background: Acetaminophen hepatotoxicity is the leading cause of ALF and can be fatal when liver fails to regenerate. If hepatic stem/progenitor cells were recruited in ALF efforts to amplify such cells could offer novel therapies.

This study highlights some of the challenges associated with assay of rFIX products in the laboratory and that careful consideration needs to be given to the choice of reference material used. This is especially important with the imminent arrival of new and modified rFIX products. “
“Joint bleeding is the hallmark of haemophilia. Increasingly, the pain, restricted movement

and anxiety provoked by even a single haemarthrosis are concerns for patients, families and treating physicians. Tanespimycin mw The aims of this study were to determine whether the current paradigm for prophylaxis requires a shift in focus from reducing the frequency of bleeding episodes to a goal of zero bleeding and to review and discuss the published data from in vitro and animal experiments and clinical studies in patients with haemophilia that describe the impact of joint

bleeding. More than two to three bleeding into the same joint may cause irreversible and progressive structural damage that compromise health-related quality of life (HRQoL). A goal of zero bleeding episodes – or as close to Selleck Daporinad zero as possible – is key to enhancing joint health and HRQoL in children and adults with haemophilia. Achieving this goal requires individualized, outcome-based, multidisciplinary care to maximize prophylactic efficacy without increasing overall health care costs. “
“This chapter contains sections titled: Introduction Importance of complex assembly to coagulation Extrinsic pathway to blood coagulation Attenuation of the procoagulant response Conclusion Acknowledgment References “
“It is important to assess the health-related quality of life outcomes of boys in China, but there are no tools validated for this purpose. The objective of the study was to assess the validity of the Simplified Chinese version

of the CHO-KLAT2.0. We recruited 60 boys with either haemophilia A (HA) or haemophilia B (HB) and their parents from four regions in China, and assessed the validity of CHO-KLAT compared to the PedsQL. All participants 上海皓元 complete the CHO-KLAT a second time 1–2 weeks later to assess reliability. The boys ranged in age from 7 to 18 (mean = 12.4; SD = 3.03) years. The severity distribution was: mild (9), moderate (10) and severe (41). On-demand therapy was received by 26 boys, while 18 received low-dose prophylaxis (HA: 10 IU kg−1 2–3 times week−1, and HB: 20 IU kg−1 1 time week−1). The mean CHO-KLAT scores were 63.7 (SD = 10.6) for child-report and 58.3 (SD = 11.4) for parent-report. Validity was supported by a correlation of 0.67 (P < 0.0001) with the PedsQL for child-report and 0.64 (P < 0.0001) for parent-report. The test–retest reliability was 0.88 (95% CI: 0.82–0.94) for child-report, and 0.90 (95% CI: 0.86–0.95) for parent-report. Inter-rater reliability was 0.46 (95% CI: 0.26–0.66). CHO-KLAT scores were 11 points higher among patients who had been on prophylaxis 3 times per week for ≥24 weeks.