ABSTRACT
Being a hereditary bleeding disorder, hemophilia is characterized by spontaneous bleeding, especially joint bleeding. The treatment outcome is a comprehensive evaluation system of the following three aspects: bleeding, musculoskeletal structure (imaging), and function-activity participation. Multidisciplinary testing and corresponding scales are needed in the assessment. Among them, the quality of life assessment of hemophilia patients is particularly important, through general questionnaires and hemophilia-specific questionnaires. Canadian hemophilia outcomes-Kids' life assessment tool(CHO-KLAT), a special quality of life assessment tool for children with hemophilia, is the most widely used. This paper briefly describes the development and application of comprehensive evaluation system of the children with the disease.
ABSTRACT
Objective:To understand the relationship between joint bleeding and joint disease in hemophilia children, and to provide a theoretical basis for clinical treatment and prognosis.Methods:The patients with severe hemophilia A between 1 and 7 years old and with relevant nodal bleeding records were selected.All the patients admitted in Beijing Children′s Hospital, Capital Medical University, and Chengdu New Century Women′s and Children′s Hospital since June 2016 to January 2017.All the joint bleeding of each child was taken as the study joint, and the joint bleeding was collected during the last 3 months.The joints were assessed by using ultrasound, X-ray, magnetic resonance imaging(MRI) and Hemophilia Joint Health Score (HJHS) scoring systems.The correlation analysis was conducted between the joint bleeding, ultrasound, X-ray, MRI and HJHS scores.The correlation analysis was conducted for baseline ultrasound, X-ray, MRI and HJHS scores.Results:(1) There were 18 patients enrolled.The mean age was (5.6±1.8) years old.There were 30 joints bleeding in the observation period in total, with the annul median joint bleeding times of 4 (4-16 times), and the annul median target joint bleeding times of 8 (4-16 times). (2) Joint bleeding times of was correlated with ultrasound and X-ray evaluation ( r=0.390, P=0.033; r=0.517, P=0.008), and not correlated with HJHS or MRI(all P>0.05). (3) There was significantly positive correlation among ultrasound, X-ray, HJHS and MRI [ r=0.815(ultrasound vs.X-ray), r=0.510(ultrasound vs.HJHS), r=0.812(ultrasound vs.MRI), r=0.666(X-ray vs.HJHS), r=0.911(X-ray vs.MRI), r=0.781(HJHS vs.MRI), all P<0.01]. (4) There was no correlation between times and assessment for joints whose ultrasound and /or MRI in joints with abnormal ultrasound and /or MRI evaluation( P>0.05). Conclusions:The results of joint bleeding and joint evaluation are inconsistent.Joint bleeding can not truly reflect the situation of joint diseases.The assessment of hemophilia should include comprehensive evaluation of joint structure, function, activity ability and other aspects to guide the treatment of haemophi-lia children.
ABSTRACT
Objective@#To detect the arthropathies on no bleeding history joints in pre-school hemophilia A children in order to provide evidence for further prevention and control of joint disease in children with hemophilia A.@*Methods@#This study was a cross-sectional study based on China Hemophilia Individualized prophylaxis study (CHIPS). The basic data of outpatients with hemophilia in Beijing Children's Hospital and Chengdu Women's and Children's Central Hospital between August 2016 and June 2017 were collected and a three-month follow-up was conducted. The target joints (six joints of bilateral elbows, knees and ankles) of thirty-four children aged 1-7 years old with severe hemophilia A were examined by ultrasound, X-ray and joint function examination (4-7 years old, hemophilia joint health score (HJHS)). To find out whether there are arthropathies in patient's joints with no bleeding history and analyze the relevant factors by chi-square test, rank sum test and other statistical methods.@*Results@#There were 32 analyzable cases with 112 no-bleeding history target joints, 42.9% (48/112) were elbow joints. Arthropathies were revealed in 34.8% (39/112) of them by joint structural and functional examination and 46.2% (18/39) were ankles (χ2=8.379, P=0.015) . Ultrasound showed abnormalities in 18.3% (20/109) joints, X-ray showed abnormalities in 3.8% (3/79) joints and HJHS showed abnormalities in 25.3% (20/79) joints. There was no correlation between ultrasound and HJHS (r=0.015, P=0.895), no correlation was found between X-ray and HJHS (r=-0.101, P=0.390) either, which suggested that joint structural and functional examination could not replace each other. The related risk factors of arthropathies in this group were >4.91 years old (OR=3.917, 95%CI:1.610-9.528) and combining with target joint (OR=3.530, 95%CI:1.316-9.465).@*Conclusions@#Detecting the joint structure and function on no bleeding history joints in pre-school hemophilia A children could reveal the arthropathies and majority of them were ankle arthropathies. Joint structural and functional examinations could not replace each other. For patients more than 5 years old and those with target joints, the joints with no complaint of bleeding should be examined regularly to reveal the arthropathies in time.