Functional Independence Score In Hemophilia Research Articles

Promoting pain coping skills in haemophilia: A remote intervention integrating exercise and pain education.

Chronic joint pain is a significant and widespread symptom in people with haemophilia (PWH). Despite medical advancements, effective pain management remains challenging. This study presents an innovative approach that integrates remote physical exercises, pain neuroscience education, and coping strategies to address chronic pain in PWH. The remote intervention consisted of sixteen 5-min videos encompassing physical exercises for chronic pain management and pain education strategies. These videos formed an 8-week remote intervention program. Clinical and physical assessments were conducted before and after the intervention. A total of thirty-one PWHs, with a median age of 34 years (ranging from 16 to 59years), completed the remote intervention. The study revealed significant improvements in pain intensity, disability, and physical performance among PWH with chronic pain. Enhanced functional capacity was evident in the Timed Up and Go and Single Leg Stance tests, accompanied by improved scores on the Functional Independence Score in Haemophilia (FISH). Although lacking a control group, our findings are consistent with other successful exercise and pain education programs. This innovative intervention holds promise for managing chronic pain in PWH, underscoring patient empowerment, education, and collaboration. Notably, our study stands out by uniquely combining pain education and coping strategies, bolstering evidence for effective pain management.

Effects of Plyometric-Based Hydro-Kinesiotherapy on Pain, Muscle Strength, Postural Stability, and Functional Performance in Children with Hemophilic Knee Arthropathy: A Randomized Trial

Aim To explore how plyometric-based hydro-kinesiotherapy (Plyo-HKT) would affect pain, muscle strength, postural stability, and functional performance in a convenience sample of children with hemophilic knee arthropathy (HKA). Methods Forty-eight children with HKA (age: 8–16 years) were randomly allocated to the Plyo-HKT group (n = 24; underwent the Plyo-HKT for 45 min, twice/week over 12 wk in succession) or the comparison group (n = 24; performed the standard exercise rehabilitation at an equivalent frequency and duration). Pain, peak concentric torque of quadriceps and hamstring (produced at two angular velocities: 120 and 180 o/sec), dynamic limits of postural stability (DLPS), and functional performance [Functional Independence Score in Hemophilia (FISH) and 6-Minute Walk Test (6-MWT)] were assessed pre- and post-intervention. Results In contrast with the comparison group, the Plyo-HKT group achieved more favorable pre-to-post changes in pain (p = .028, η 2 p = 0.10), peak torque of quadriceps [120°/sec (p = .007, η2 P = 0.15); 180°/sec (p = .011, η2 P = 0.13)] and hamstring [120°/sec (p = .024, η2 P = 0.11); 180°/sec (p = .036, η2 P = 0.09)], DLPSdirectional [forward (p = .007, η2 P = 0.15); backward (p = .013, η2 P = 0.12); affected side (p = .008, η2 P = 0.14); non-affected side (p = .002, η2 P = 0.20)], DLPSoverall (p < .001, η2 P = 0.32), and functional performance [FISH (p < .001, η 2 p = 0.26); 6-MWT (p = .002, η 2 p = 0.19)]. Conclusion Plyo-HKT is likely helpful for reducing pain, improving strength, enhancing postural stability, and boosting functional capabilities in children with HKA. Physical rehabilitation practitioners should, therefore, consider this intervention strategy.

Association Between Low Bone Mineral Density in Hemophilia Patients and Musculoskeletal Function Impairments

Background: Hemophilia A (HA) and B (HB) are inherited bleeding disorders due to the partial or total deficiency of coagulation factor (F) VIII or FIX, respectively. It is associated with low bone mineral density (BMD). Method: The current study was conducted on sixty-eight Iraqi patients with hemophilia to assess BMD using a DEXA scan enrolled from the National Center of Hematology / Mustansiriyah University from July 2020 to September 2021. Various medical history characteristics were recorded as (age, type of hemophilia, severity, number and site of bleeding, smoking, and alcohol intake) as well as Functional Independence Score in Haemophilia (FISH) was used to assess musculoskeletal functions and Hepatitis C virus (HCV)/Human immunodeficiency virus (HIV) seropositivity were recorded. Results: The results found that only 39.7% of patients have positive DEXA scans characterized by low BMD (abbreviated as DX+) and 60.3 % with normal BMD (abbreviated as DX0). The average Functional Independence (FISH) score in DX+ patients is significantly lower than those in the DX0 group, and about 33.3% of patients in the DX+ group have limitation of movement (LOM), which is significantly higher than of patients in DX0 group and 70.4% of patients in DX+ group are presented with seropositive viral infection, which is significantly higher than 39% of patients in DX0 group. Conclusion: It can be concluded that hemophilic patients with seropositive viral infection and severe LOM may be at higher risk of developing low BMD.

Emicizumab Prophylaxis in Patients with Severe Hemophilia A: Insights from A Resource Limited Country.

In this prospective study, severe HA patients were recruited from January 2022 to June 2023. Inhibitor positive and inhibitor negative patients with annual bleeding rate (ABR) 8 or greater and past histories of bleeding like intra-cranial, intra-abdominal, and pseudo-tumors were included. Emicizumab loading dose was 3 mg/kg in the first 4 weeks, and the maintenance dose was started at week 5 at 6 mg/kg/month. Patients' detailed bleeding history and demographics were recorded. The five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) was used to evaluate patients' HRQoL. Furthermore, Hemophilia Joint Health Score (HJHS) and Functional Independence score in Hemophilia (FISH) were applied for the assessment of joints at different time points. Results were analyzed by SPSS version 21. A total of 36 HA male patients with the mean age of 19.7 ± 14.42 years were recruited in the study; among them, 19 patients were inhibitor positive, while 17 were negative. Patients clinically presented with bleeding symptoms which included: hemarthrosis 95%, GI bleeding 13.8%, and bruises and gums bleeding 13.8%. Significant reduction was observed in the bleeding episodes after the therapeutic intervention, and joints assessment and Euro-Quality-of-life Visual Analog Scale showed a significant improvement in health after treatment. Similarly, there was a remarkable reduction in bleeding episodes and improved quality of life among HA patients. The ABR decreased from 53.6% episodes per year prior to treatment to 2.4% during Emicizumab therapy. Prior to initiating Emicizumab therapy, participants exhibited an average FISH score of 16 and HJHS score of 10, indicating moderate limitations due to joint-related issues. After treatment, the mean FISH score improved to 9 and HJHS score to 4 reflecting a substantial enhancement in participants' ability to perform daily activities (P < 0.057). Our results showed that HA patients on prophylactic treatment with Emicizumab were less restricted and had improved quality of life due to marked decrease in bleeding episodes which resulted in improved health and social lives. In addition, it was well tolerated, and no participant discontinued treatment because of adverse events.

Emicizumab Efficacy in Severe Hemophilia (A): Experience in Ecuador

Introduction: In Ecuador, there are 1708 cases of hemophilia in a population of 17,643,060 habitants; according to the World Federation of Hemophilia (WFH) Annual Global Survey Report 2020 released in October 2021. Eleven patients use emicizumab in Ecuador (ten patients under ministerial authorization, and one patient through private insurance); being the first administered in June 2021. Objectives: To describe the demographic characteristics of patients receiving emicizumab in Ecuador. To determine efficacy and response with emicizumab: based on annual bleeding rate (ABR), performance status assessment, activity, and participation. Methods: Population: Pediatric Severe Hemophilia A (PwHA) patients (&amp;lt;12 years) and adults/adolescents (&amp;gt;12 years), both from the public and private health sector. Study design: Retrospective, multicentric study, with a case-report research design. The sources of information were the medical records of the patients. Efficacy variables: ABR was calculated by: (number of bleeds/number of days times 365.25). Body structure and function was assessed with the Hemophilia Joint Health Score 2.1 (HJHS). Activity and participation were assessed with the Functional Independence Score in Hemophilia (FISH). Statistic analysis: Shapiro-Wilk test to know the distribution of the sample, t-Test paired tests (parametric), Wilcoxon statistic (non-parametric); Descriptive statistics: relative frequencies, measures of central tendency and dispersion, column graphs. Analysis and results: Ten patients with an average (Q1,Q3) age of 11.5 years (7.15) were registered. The youngest patient was one year old and the oldest, fifty years old. 90% (n:9) had hemophilia A with high-response inhibitors. Prior to starting emicizumab: 20% (n:2) presented Central Nervous System bleeding, 30% (n:3) had muscle bleeding, and 10% (n:1) developed catheter-associated infection. The mean ABR was 10 pre-emicizumab events, and 0.3 post-emicizumab events (p=0.002), with follow-ups at 22 months (n: 2), 17 months (n:2), 3 months (n:2 ), 4 months (n: 3), 5 months (n: 1). The mean HJHS (±SD, n) was 20.9 (± 20,096, 10) pre-emicizumab, and 9.7 (± 16.52, 10) (p=0.003) The mean by FISH (±SD, n) was 16.2 (± 8.59, 10) pre-emicizumab, and from 22.6 (± 11.62, 10) with the use of emicizumab. The use of the t-Test for paired samples determined that there are statistically significant differences between the pre-emicizumab and post-emicizumab groups for the variables ABR, HJHS and FISH (95% confidence level). Conclusion: The use of Emicizumab allowed better control of joint bleeding; and it contributed favorably in the evolutionary course of both the functional state and the activity. It is essential to corroborate these results with a longer follow-up and a larger sample size. Bibliography: 1. Alvarez Roman M: Spanish guidelines for the management of hemophilia. 2022, 1-190. 2. Callaghan M, Negrier C, Paz-Priel I, Chang T, Chebon S y col: Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood. 2021;137(16):2231-2242 3. Carcao M, Escurriola C, Santagostina E, Oldenburg J, Liesner R y col: The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab. Haemophilia. 2019;25:676-684. 4. Ellsworth P, Ma A: Factor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates?. Hematology Am Soc Hematol Educ Program. 2021, Dec 10;2021(1):219-225. 5. Young G: Management of children with hemophilia A: How emicizumab has changed the landscape. Journal of Thrombosis and Haemostasis. 2021 Jul;19(7):1629-1637

Effectiveness of Kinesiologic Taping on Function and Pain in Patients With Hemophilia A

ABSTRACT Background: Hemophilic arthropathy frequently results in musculoskeletal adjustments that impair functional capacity. Aim: We investigated the effects of kinesiologic taping on patients with hemophilic arthropathy in this study. Materials and Methods: Twenty-six patients were enrolled. Thirteen patients formed the Kinesiologic taping group (KT) and received the interventional protocol consisting of three kinesiologic taping sessions during three weeks; and 13 patients formed the control group (CG) and made three weeks of home exercises. All patients were evaluated using the Functional Independence Score in Hemophilia (FISH), Hemophilia Joint Health Score (HJHS), and Visual Analog Scale (VAS) at baseline, one week, and three weeks. Results: The mean scores for the VAS, HJSS, and FISH differed significantly before and after 3 weeks in both groups (P &lt; 0.001). However, the differences in medians after the treatment were significantly greater in the KT group than in the CG group (P &lt; 0.001). Conclusions: Kinesiologic taping was associated with physical improvement in hemophilia patients; however, also kinesiologic taping led to significant improvement in VAS scores.

Evaluation of Functional and Joint Health and Associated Factors in Adults With Hemophilia From a Developing Country With Government-Backed Efforts to Improve Hemophilia Care.

Introduction Hemophilic arthropathy affects people with hemophilia (PwH) and causes joint dysfunction and disability. Brazil has a unique situation and implemented policies to improve health care for PwH. The aim of this study was to evaluate the Functional Independence Score in Hemophilia (FISH), Hemophilia Joint Health Score (HJHS), and associated factors among adult PwH attending a Hemophilia Comprehensive Care Center in Brazil. Methods A post hoc analysis was conducted, including 31 patients who had submitted to a physical evaluation during a previously published cross-sectional study performed from June 2015 to May 2016 at the Brasília Blood Center Foundation, Brazil. Results The mean age was 30.8±9.4 years, and 80.6% had severe hemophilia. FISH was 27.0±3.8, and HJHS 18.0±10.8. The ankle was the most often affected joint (25/31, 80.6%). There were significant correlations between FISH and HJHS scores and the Hemophilia Quality of Life Questionnaire for Adults. Patients with severe hemophilia (P = 0.029) and PwH aged ≥ 30 years (P = 0.049) had lower FISH scores. Monthly household income > 2 Brazilian minimum wages was independently associated with improved HJHS (P = 0.033). The factors simultaneously associated with better HJHS and FISH were age < 30 years (P = 0.021) and monthly household income < 2 minimum wages (P = 0.013). Conclusion FISH and HJHS showed favorable scores despite being performed in a country with unfavorable socioeconomic conditions. In addition to hemophilia severity and age, monthly household income was independently associated with functional and articular state of PwH. The results highlight the importance of the free provision of coagulation factors in Brazil.

Factor VIII Level in the Potential Female Carrier Mothers of Patients with Hemophilia A and Its Impact on Joint Health

Background Hemophilia A is an X-linked bleeding disorder. The characteristic bleeding phenotype in patients with severe hemophilia is recurrent spontaneous hemarthrosis with development of hemophilic arthropathy. Heterozygous female carriers with factor VIII (FVIII) activity level lower than 40% may be at risk for bleeding that is usually comparable to that seen in males with mild hemophilia. Aim of the Work To assess factor VIII level in mothers of known patients of hemophilia A correlating it with the frequency of clinically and radiologically documented arthropathy. Patients and Methods A cross sectional study included 40 mothers of patient with hemophilia A during the period from September 2020 to September 2021. Studied participants were subjected to detailed analysis of bleeding history, six major joints range of motion (ROM) examination, functional independence score in hemophilia (FISH), FVIII level one stage assessment and magnetic resonant imaging (MRI) of the reported affected joints. Results Mean age of mothers is 42.25 ± 7.74 years. Eleven mothers (27.5%) had no bleeding symptoms, 16 (40%) had one bleeding symptoms and 13 (32.5%) had two or more bleeding symptoms. As regard joint pain, 13 (32.5%) had severe pain, 3 (7.5%) had ankle pain and 2 (5%) had elbow pain with a mean pain score of 2.55±3.32, and 7 (17.5%) had bilateral pain. Mean FVIII activity was 43.33 IU (± 22.79), ranging from FVIII (0 &amp;lt; 30 IU) in 13 (32.5%), FVIII (30 &amp;lt;60) in 18 (45.0%) and FVIII ≥ 60 IU in 9 (22.5%) among the studied participants. Knees were the most affected joints with decreased mean ROM (±SD) in right and left knees of (123.25 (±12.06) and 120.00 (±15.81)) respectively. The median MRI scores were studied was 5 (2.6%) with median osteochondral changes of 4 (2.5%) and median soft tissue changes of 1 (0-1), their median FISH score of 8 (8-9). By comparing groups at the level of factor VIII, there was no significant difference according to anthropometric bleeding parameters, pain, and MRI score. None of participants needed hospital admission, factor VIII replacement or packed red cells replacement. Conclusion Potential female hemophilia A carriers are at risk of spontaneous bleeding which may entail bleeding in the joints, causing early joint damage. They showed reduced overall joint ROM and structural osteochondral changes in MRI joint examination which mandates further studies about early detection of potential female hemophilia carriers for the possible need of early medical intervention.

Osteoporosis and associated risk factors in patients with severe hemophilia A: a case-control study from China

SummaryIntroductionPeople with hemophilia risk osteoporosis more than healthy people, which may be related to specific factors.MethodsThis case-control study included 53 patients with severe hemophilia type A and 49 healthy participants. Dual-energy X-ray absorptiometry (DXA) was used to determine bone mineral density (BMD). Collected information on age, body mass index (BMI), number of joint arthropathies, functional independence score in hemophilia (FISH), bone turnover markers, antibodies, treatment modalities. Identified independent risk factors for osteoporosis.ResultsThe BMD of the femoral neck (0.80 g/cm2vs.0.97 g/cm2), ward’s triangle (0.62 g/cm2vs.0.83 g/cm2), tuberosity (0.63 g/cm2vs.0.80 g/cm2) and hip (0.80 g/cm2vs.0.98 g/ cm2) in the case group were significantly lower than those in the control group, all of which were P < 0.001. However, there was no significant difference in the overall BMD of lumbar spine(L1-L4) (1.07 g / cm2vs. 1.11 g / cm2). The frequency of osteoporosis in the case group was 41.51%. BMI and FISH score were considered as independent risk factors for BMD decrease.ConclusionThe BMD of patients with severe hemophilia A is much lower than that of healthy population, and this difference is mainly reflected in the hip. The clear influencing factors were low BMI and functional independence decrease. Osteoclast was active while osteoblast activity was not enhanced synchronously, which may be the pathological mechanism of BMD decrease.

Ultrasound Evaluation of Tendinopathy in Hemophiliac Patients for the Purpose of Rehabilitation Indications.

Hemophilia is a inherited bleeding disorder that is characterized by intra-articular bleeding (hemarthrosis). The aim of the study was to evaluate the state of the satellite tendons of the target joints in the patient with hemophilic arthropathy and propose rehabilitation treatment with eccentric exercises. The tendons of the joints mainly affected by hemophilic arthropathy were evaluated by ultrasound. The ultrasound evaluation is associated with the use of evaluation clinical scales, such as the Hemophilia Joint Health Score (HJHS), the Functional Independence Score in Hemophilia (FISH), the Hemophilia Activity List (HAL), the DASH, the VISA-A, the VISA-P, and the VAS scale. In 20 patients with hemophilic arthropathy, the thickness of the tendons that were examined was normal. In six subjects with severe joint damage, echostructural alterations were present, and signs of hyperemia and neo-vascularization were detected on color Doppler, as well as the presence of intratendinous calcifications. The tendons of the target joints in patients with hemophilic arthropathy are compromised by the indirect biomechanical damage caused by the joint disease, and rehabilitation treatment with eccentric exercises can be considered safe and effective in improving the tenso-elastic properties of the tendons.

Hemophilia Joint Health Score, Functional Independence Score in Hemophilia, and Pettersson Score in Pediatric Patients With Severe Hemophilia A.

Hemophilia is an X-linked recessive disorder. Children with hemophilia go through spontaneous and trauma-provoked bleeding. Recurring joint bleeds lead to ongoing incapacity. Achieving healthy joints is the primary target of hemophilia management. The current study objective was to assess hemophilic joints in individuals with hemophilic arthropathy clinically, radiographically, and functionally. This cross-sectional study included 50 children with severe hemophilia A who were selected from the pediatric hematology clinic. All children were assessed for Hemophilia Joint Health Score (HJHS). Joint assessed functionally by Functional Independence Score in Hemophilia (FISH) and radiologically by plain radiograph and scored by the Pettersson scoring system. Data were analyzed using Statistical Package for Social Sciences. The mean age of the studied cases of hemophilia was 8.5±3.1 years. The mean FISH score among the studied patients was 26.8±4.2, the mean HJHS was 16.8±12.8, and the Pettersson score was 4.9±2.7. The number of affected joints showed a significant negative correlation to the FISH score and a significant positive correlation to HJHS. The frequency of hemarthrosis/month showed a significant positive correlation to HJHS. The number of affected joints showed a significant negative correlation to the FISH score and a significant positive correlation to HJHS. Frequency of hemarthrosis/month showed a significant positive correlation to HJHS.

Usefulness of Emicizumab Associated with Immune Tolerance Induction in Patient with Hemophilia a and Inhibitors: Experience in Cuenca-Ecuador

Background: Referring to the World Federation of Hemophilia (WFH) 2020 Annual Global Survey Report published in October 2021: In Ecuador, with a population of 17,643,060 inhabitants, there are 1,708 cases of hemophilia, 99 cases of Von Willebrand disease, and 7 cases of other coagulation disorders. Within the hemophilia A group, 21 patients have active inhibitors, and there is a record of 2 new cases of inhibitors in relation to the previous notification. Emicizumab is a bispecific, humanized monoclonal antibody that binds activated FIX and factor X (FX) resulting in the generation of activated FX (FXa) which then catalyzes the formation of thrombin from prothrombin. Based on the HAVEN 1-4 clinical trials, the FDA and EMA approved the use of Emicizumab as prophylaxis in Hemophilia A (with and without inhibitors) in both adults/adolescents (>12 years) and pediatrics (<12 years). In both the primary analyzes and long-term follow-up, emicizumab produced statistically and clinically significant reductions in annual bleeding rates compared with Factor VIII prophylaxis (in patients without inhibitors); and with bypassing agents (in patients with inhibitors). These trials excluded patients in immune tolerance (ITI). Keywords: emicizumab, immune tolerance, hemophilia. Objectives: -Assess the usefulness of Emicizumab in immune tolerance in terms of bleeding control, osteoarticular health and functional activity Materials and Methods: 1) The evaluation was carried out in a 9-year-old patient (boy) with severe Hemophilia A and high titer inhibitors who, prior to the start of ITI, had predictive factors of high risk and poor prognosis (historical peak 234 UB, 5 years since inhibitor screening) with 4-8 joint bleeds per year. The ITI scheme used was with plasmatic Factor VIII 100 IU/Kg 3 times a week with management with bypass agents on demand (ITI duration: 3 years) and inclusion of emicizumab 2 years after starting the strategy 2) The analysis was determined based on periodic clinical evaluations, osteoarticular status was assessed through the Hemophilia Joint Health Score 2.1 (HJHS), Disease Activity Score (DAS28); and functional activity through Functional Independence Score in Hemophilia (FISH), and Health Assessment Questionnaire (HAQ). An analysis was made prior to the start of immune tolerance and after reaching immunological tolerance. Results: 1) This strategy contributed to better control of joint bleeding, and therefore to the inhibitor eradication process. 2) Improved the evolutionary course of his osteoarticular state: - Prior to ITI: DAS28: 4.26 (moderate activity), HJHS 33. Target joints: left knee and ankle. - After immunological tolerance: DAS28: 1.87 (clinical remission), HJHS 12. 3) Favorable progression of his functional status, despite slight limitation of his left knee in flexion (sequel to recurrent episodes of hemarthrosis): - Prior to the start of ITI: FISH 14, HAQ: 1.50 (severely affected functional capacity), functional scale IV (use of a wheelchair). - After immunological tolerance: FISH 27, HAQ: 1.00, functional scale II (able to carry out activities of daily living with slight difficulty). Conclusions: 1) Larger-scale studies and accumulated evidence are needed that allow us to establish the usefulness of Emicizumab in ITI and therefore corroborate these results. 2) We must establish the best clinical application profile and the economic impact of these therapies; especially in health systems like ours, in which accessibility to drugs is complex and optimization of resources is the norm. Bibliography: 1. Alvarez Roman M: Guías Españolas para el manejo de la hemophilia. 2022, 1-190. 2. Callaghan M, Negrier C, Paz-Priel I, Chang T, Chebon S y col: Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. 2021;137(16):2231-2242 3. Carcao M, Escurriola C, Santagostina E, Oldenburg J, Liesner R y col: The changing face of immune tolerance induction 3. Hematology Am Soc Hematol Educ Program. 2021, Dec 10;2021(1):219-225. 4. Young G: Management of children with hemophilia A: How emicizumab has changed the landscape. Journal of Thrombosis and Haemostasis. 2021 Jul;19(7):1629-1637 Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Clinical outcome and incidence of inhibitor development in severe hemophilia patients receiving low-dose prophylaxis: a 3-year follow-up study in Senegal, West Africa

IntroductionIn Africa, where access to diagnosis and treatment of hemophilia is the lowest in the world, prophylaxis is rarely used in preference to on-demand treatment. There are limited data of prophylaxis treatment from sub-Saharan Africa. The aim of this study was to evaluate clinical outcomes and inhibitor development in people with hemophilia receiving low-dose prophylaxis (LDP) in a sub-Saharan African setting. MethodsWe conducted a three-year prospective study. A once or twice weekly prophylaxis regimen of 25 IU/kg of rFVIIIFc or 30 IU/kg of rFIXFc was given to Hemophilia A and B, respectively. We evaluated clinical outcomes and inhibitors occurrence, determined by screening and titration using the Nijmegen technique. ResultsA total of 15 patients were included in the LDP regimen. The mean age was 6.3 years (1.5 - 10). A significant reduction was noted in the annualized bleeding rate, from 7.53 to 1.33 (p = 0.0001); the annualized joint bleeding rate passed from 3.6 to 1.4 (p = 0.001) and the proportion of severe bleeding, from 86.1% to 16.7% (p = 0.0001). The Hemophilia Joint Health Score (HJHS) moved from 9.6 to 3.4 (p = 0.0001) and the Functional Independence Score in Hemophilia (FISH) improved from 25.8 to 30.9 (p = 0.0001). School absenteeism decreased from 7.33% to 2.59%. Adherence to prophylaxis was 89.5% versus 60%. Consumption was 580 IU/kg/year versus 1254.6 IU/kg/year before and after prophylaxis, respectively. Incidence of inhibitors was 23% (3 /13 HA). ConclusionThe LDP in Hemophilia improves the clinical outcome without a surplus risk of inhibitor development. Using extended half-life clotting factor concentrates (CFCs) is better for prophylaxis in resource-limited countries, as they allow better compliance in treatment.

Effectiveness of rehabilitation on pain and function in people affected by hemophilia.

Introduction:Literature provides unclear evidence of the effectiveness of many physiotherapy interventions on pain intensity, quality of life, and functional ability in hemophilic patients, and suggests that rehabilitation programs should be focused on functional goals and the disability of patients.Aim:The aim of the present study is to present the outcome of a case series of patients with hemophilia in which a rehabilitation program has been carried out on the basis of a specific individual patient's functional impairment.Methods:Fifty-one patients were enrolled: 32 patients (Rehab Group) received a rehabilitative treatment, 19 patients for different reasons (living far from the hospital, family problems) did not receive rehabilitation (Control Group). The rehabilitation program was planned with respect to the emergent problems: musculoskeletal pain, joint range of motion limitation, muscle flexibility, walking difficulties, the appearance of hematomas/hemartro, and postural problems. All the patients were assessed at baseline (T0), after 1 month (T1), and after 3 months (T2) through visual analogic scale for musculoskeletal pain, the Hemophilia Joint Health Score for joint range of motion, the Functional Independence Score in Hemophilia for disability, and postural assessment by visual inspection.Results:A significant reduction of pain and improvement of Hemophilia Joint Health Score and Functional Independence Score in Hemophilia score was found in the Rehab Group along with the follow-up. Pain in the Control Group was lower with respect to the other group at baseline and significantly decreased after 1 month.Conclusion:A rehabilitation program focused on individual impairment of patients with hemophylia determined satisfying results in terms of pain control, functional, and disability improvement in 3 months follow-up.

Effect of FVIII doses on joint structure and function in adolescents with severe hemophilia A: mid-term results of a prospective cohort study

To explore the clinical phenotype and changes in joint structure and function in adolescent patients with severe hemophilia A under different doses of FVIII. Forty- three adolescents with severe hemophilia A aged 4-18 years were divided into on-demand group (n=7), low-dose group (FVIII dose of 10-15 U/kg, 2-3 times a week, and ≤30 U/kg a week; n=17), and intermediate-dose group (FVIII dose of 15-20 U/kg, 2-3 times a week, and 45-60 U/kg a week (n=19). The 3 groups were compared for their clinical bleeding phenotype, annual bleeding rate (ABR), annual joint bleeding rate (AJBR), annual the most severe joint bleeding rate, joint imaging scores (ultrasound HEAD-US score and IPSG MRI score), Hemophilia Joint Health Score (HJHS) and Functional Independence Score in Hemophilia (FISH) within 24 months. Compared with that in on-demand group, the ABR was significantly reduced in the low- and intermediate-dose groups (P=0.004 and 0.000, respectively), and was reduced by 32.87% in the intermediate-dose group as compared with the low-dose group. The AJBR (P < 0.01) and annual the most severe joint bleeding rate (P < 0.05) also differed significantly among the 3 groups. The number of bleeding episodes increased progressively with time in the on-demand group, remained stable in the low- dose group, and tended to decrease in the intermediate-dose group. The imaging scores of the most severe joints showed lesion progression in all the 3 groups. The ultrasound scores, which increased steadily in the on-demand group, showed significantly lowered increment rates in the low- and intermediate-dose groups (P=0.002 and 0.000, respectively). The MRI scores showed also delayed increment in the low- and intermediate-dose groups as compared with the on-demand group (P=0.041 and 0.000, respectively), and the increment was accelerated in the on-demand and low-dose group but remained stable in the intermediate-dose group. The increment of the HJHS scores was significantly higher in the on-demand group than in the lowand intermediate-dose groups (P=0.003 and 0.000, respectively), and the scores increased at a steady rate in the on-demand group but tended to decrease in the latter two groups. The FISH score was decreased by 0.29±3.09 in the on-demand group but was increased significantly in the low- and intermediate-dose groups compared with the on-demand group (P=0.000). In Chinese adolescents with severe hemophilia A, low- and intermediate-dose FVIII prophylaxis, especially at the intermediate dose, is better than on- demand treatment for protecting joint structure and function.

Functional and joint evaluation in a prospective cohort of patients with severe haemophilia.

Arthropathy is the main morbidity of haemophilia. Periodic joint health assessment enables a better understanding of the limitations of these patients. To evaluate the functional and joint performance in patients with haemophilia at one-year follow-up, as well as its association with prophylactic treatment and attendance at scheduled consultations at a haemophilia treatment centre. This prospective cohort study included patients with severe haemophilia aged 7years or more and treated at Fundação Hemocentro de Brasília, Brazil, from January 2014 to December 2018. The Hemophilia Joint Health Score and Functional Independence Score in Hemophilia were assessed at the first consultation and after a one-year follow-up. The study included 69 patients. The mean age at study recruitment was 22.5±4.5years, 62.3% of patients aged 18years or older, and 29 patients were receiving primary prophylaxis (38.0%). There was a positive correlation between HJHS and age and a negative correlation between FISH and age. The worsening HJHS was associated with non-primary prophylaxis and non-attendance at scheduled multidisciplinary consultations. The worsening FISH was associated with non-primary prophylaxis. The correlation between FISH and treatment adherence was significant for the delta. The older the patient with haemophilia, the higher the probability of a worsening of the HJHS. In the presence of more arthropathies, the older the patient, the worse the FISH. Patients receiving primary prophylaxis show better results in the HJHS and FISH when compared to patients receiving secondary prophylaxis and/or on-demand treatment.

The effects of manual therapy on musculoskeletal system, functional level, joint health and kinesiophobia in young adults with severe haemophilia: A randomized pilot study.

The effects of manual therapy (MT) on joint health and functional level in haemophilic arthropathy (HA) have relatively under-explored. To investigate the effects of MT in HA of lower limb joints on musculoskeletal system, functional level, Functional Independence Score in Hemophilia (FISH), Hemophilia Joint Health Score (HJHS) and kinesiophobia. Seventeen patients were randomized to the control group (CG=9) and manual therapy group (MTG=8). The number of haemarthrosis (NoH) was recorded from the diaries. Range of motion (ROM), strength and pain intensity were measured using goniometer, digital dynamometer and visual analogue scale, respectively. Joint health was evaluated with HJHS, functional independence with FISH, and kinesiophobia with Tampa Scale. Functional Reach (FR), Timed Up-Go (TUG) and 5-Times Sit-to-Stand (5*STS) tests were used to evaluate functional level. Home exercises were given to both groups. Additionally, myofascial release techniques and mobilizations were applied to MTG. Interventions were applied 3d/weekly for 5weeks. Assessments were performed at baseline and after treatment. ROM, strength, activity pain, HJHS and FISH were improved in MTG (p˂0.05). Ankle NoH, 5*STS and FR were developed in both groups (p˂0.05), but kinesiophobia, TUG and knee NoH were not significant. Although improvements were observed in favour of MTG in HJHS, FR, activity pain and ankle strength/ROM (p˂0.05), no significant difference was found in FISH, kinesiophobia, TUG, 5*STS, resting pain and knee strength. Reduction in ankle NoH indicated that both interventions were safe. The use of MT in HA of lower limb joints was an effective physiotherapy approach to improve functional level, joint health and functional independence.

Severe haemophilia A children on low-dose tertiary prophylaxis showed less joint deterioration and better maintenance of functional independence than children on on-demand treatment: A 6-year follow-up study.

In countries with limited resource, haemophilia patients have to choose low-dose prophylaxis or on-demand treatment (ODT) because of economic constraints. Whether low-dose prophylaxis can achieve better joint function outcome than ODT over long-term remains unclear. To investigate the long-term effect of low-dose tertiary prophylaxis versus ODT on joint health in severe haemophilia A children. This was a retrospective study. We enrolled and followed 34 severe haemophilia boys in China receiving on-demand treatment (n=18) or low-dose prophylaxis (10-15IU/kg, 2-3 times per week) for a medium-term (6-18months, n=9) or longer-term (19-30months, n=7). We evaluated their haemophilia joint health score (HJHS) 2.1 and functional independence score in haemophilia (FISH) at baseline and at their 6-year follow-up visits. Their annual bleeding rate (ABR) and annual joint bleeding rate (AJBR) were also recorded. During the 6-year follow-up period, ABR and AJBR were similar between the 2 prophylaxis groups, with each of the 2 prophylaxis group rates being significantly better (lower) than the ODT group (P<.05). Compared to baseline values, evaluation at 6-year follow-up showed HJHS improvement in both prophylaxis groups, although significantly (P<.05) only in the longer-term prophylaxis group. The FISH score showed insignificant change in patients in each prophylaxis cohort, compared to significant worsening (P<.05) in the ODT group. Low-dose tertiary prophylaxis reduced ABR and AJBR of children with severe haemophilia and better maintained their functional independence by the FISH over the long term. Longer-term prophylaxis also improved their joint health status by the HJHS.

Joint scores in hemophilic arthropathy in children: Developing country perspectives.

Hemophilia is a common X-linked recessive coagulopathy causing recurrent bleeding into the synovial joints and results in articular and periarticular abnormalities. To our knowledge, this is the first comprehensive study aimed at studying the clinico-radiological joint score evaluation in hemophilic arthropathy in children from a developing country and its possible impact on the quality of life. In this hospital-based, prospective, descriptive study, all children presenting to the pediatric rheumatology clinic were studied. The joint physical examination was scored using the Hemophilia Joint Health Score 2.1 (HJHS 2.1). The patients were then subjected to imaging of the most affected joint using ultrasonography (USG) and magnetic resonance imaging (MRI). Detailed USG and MRI radiological evaluation was recorded in the predesigned proforma using the Hemophilia Early Arthropathy Detection with Ultra Sound (HEAD-US) score and MRI DENVER score. The physical quality of life as per Functional Independence Score in Hemophilia (FISH) was noted. The clinical, radiological, and functional scores were analyzed with an appropriate statistical measure. The mean age at presentation was 7.4 years (interquartile range 4.9-10), with the knee being the most common joint involved. All of the USG score, MRI score, and FISH score have a significant correlation (p<0.05), with the HJHS 2.1 score with correlation coefficients of 0.7086, -0.8916, and 0.8607, respectively. USG and MRI had a correlation coefficient of -0.7145 and -0.8326 with FISH, respectively. The degree of association between HJHS 2.1 score was found to be maximum with HEAD-US score, whereas a negative correlation was seen evaluating FISH score with both HEAD-US and MRI DENVER scores. Use of these scores, specifically HEAD-US score, will result in consistent assessment of hemophilic joints, optimizing the management of the destructive changes.

Improvement in school absence after factor replacement in students with haemophilia in Upper Assam, India

Abstract Background Patients with haemophilia who are not adequately treated experience a lifetime burden of joint complications and loss of functional ability due to repeated bleeding episodes caused by low levels of clotting factor VIII or IX in the blood. These complications can significantly impact day-to-day life, including active participation in school and academic study in children with haemophilia (CwH). Treatment with factor replacement therapy can help to prevent this, but access to factor has been challenging in low-resource settings such as Northeast India. This study shows the impact of factor replacement therapy on bleeding episodes, joint complications and school absence among CwH in this setting. Methods A retrospective observational study was undertaken to examine the impact of receiving regular factor replacement therapy (prophylaxis or on demand) on school absences among CwH registered with the haemophilia treatment centre at Assam Medical College and Hospital. Annual bleed rate (ABR), Haemophilia Joint Health Score (HJHS) and Functional Independence Score in Haemophilia (FISH) were also assessed. Results Thirty-eight CwH were eligible for the study; 26 (68.4%) were on prophylaxis therapy and 12 (31.5%) received on-demand therapy. In the year before starting regular treatment, the mean ABR was 37.8 (+20.0), HJHS was 31.1 (+18.1) and mean FISH score was 21.1 (+4.2). At the end of the study period the mean (+SD) ABR in prophylactic therapy was significantly lower at 5.8 (+4.6) (p&lt;0.001) and the HJHS was significantly lower at 4.7 (+4.6). FISH score significantly improved to 27.9 (+3.3) (p&lt;0.001). Prophylaxis showed better (but not significant) results in comparison to on-demand therapy. Conclusions Treatment with factor replacement significantly reduces school absence in CwH and correlates strongly with joint health and functional improvement, with the effect slightly better with prophylaxis than on-demand therapy. Low dose prophylaxis is a good treatment option in low-resource settings, but improvements are also needed in rates of diagnosis.