scholarly journals The Influence of Personalized 2-Year Physiotherapy on Musculoskeletal Health and Quality of Life in Severe Hemophilia Patients with Inhibitor - Interim Analysis after 1 Year of the Program

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 36-36
Author(s):  
Maria Helena Podolak-Dawidziak ◽  
Janusz Zawilski ◽  
Mariola Bober ◽  
Ewa Stefanska-Windyga ◽  
Anna Buczma ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Replacement Therapy ◽  
Hemophilia A ◽  
Conflicts Of Interest ◽  
Exercise Program ◽  
Rehabilitation Program ◽  
Severe Hemophilia ◽  
Musculoskeletal Health ◽  
Positive Effects

Development of factor VIII (FVIII) inhibitor is a serious complication of factor replacement therapy observed in about 15-30% of severe hemophilia A (HA) patients. Inhibitors interfere with the treatment by neutralizing intravenously injected FVIII. Although bleeding rate is not increased in severe HA patients who developed the inhibitor, the inhibitors reduce the efficacy of factor replacement therapy during bleeding episodes and in consequence accelerate the progression of hemophilic arthropathy. In Poland, prophylaxis in hemophilia with inhibitors has been introduced in 2016, and therefore many HA patients with inhibitor still suffer from particularly severe arthropathy, and have a reduced quality of life. The aim of the study was to evaluate the musculoskeletal health and quality of life in patients with severe hemophilia A with inhibitor before initiation of personalized physiotherapy and after 2 years of the personalized physiotherapeutic program. The study was performed in 24 persons with severe HA with inhibitor (mean age 42.1 years, median age 42 years) receiving prophylaxis regimen with aPCC 85 ± 15 IU/kg 3 times a week. At the beginning of the project, the patients were examined by hematologist and physiotherapist. Joint condition was evaluated using the Hemophilia Joint Health Score (HJHS) and magnetic resonance imaging of the most affected joint. Quality of life was evaluated using EQ-5D scale (EuroQoL). aPCC dosing schedule in context of rehabilitation program was adjusted by the hematologist. Each patient was enrolled to a personalized exercise program developed based on age, general condition, stage of arthropathy, date of the last bleeding episode, and a history of physiotherapy. The program consisted of weekly 60 to 90 minutes rehabilitation sessions at home, and participated in four 5-day rehabilitation camps with 5 hours of rehabilitation sessions a day organized in Poznan after 6, 12, 18 and 24 months of the program. All patients were examined by hematologist, and the rehabilitation was provided only to the patients on regular prophylaxis with aPCC provided that a dose of aPCC was always administered 1 hour before the initiation of rehabilitation session. HJHS varied among the participants, with mean value at baseline 34.2 (median 36.5). Baseline mean EQ-5D score was 54.2 (median 60). Evaluation at month 12 was performed in 18 participants (2 patients withdrawn from the program, and 4 patients could not participate in the evaluation due to COVID-19 pandemics). The results obtained at month 12 show a significant improving the quality of life and musculoskeletal health of patients with HA after combined prophylaxis and personalized rehabilitation. Mean HJHS decreased from 34.2 to 30.4 points (median from 36.5 to 31 points). An average 3.8 point decrease confirms positive effects of long-term aPCC prophylaxis with personalized, regular rehabilitation. Increase in mean EQ-5D score from 54.2 to 70.1 (median from 60 to 72) show enhancing patients' mood resulted from the improved health status and regained capability to conduct more normal lifestyle. After the first year of the project we can conclude that regular prophylaxis with aPCC and personalized, once-a-week rehabilitation program positively influence joint status and quality of life of adult HA patients with inhibitor complicated with significant arthropathy. Regular rehabilitation may slow down the progression of arthropathy due to the choice of the exercise program appropriate for each patient. These positive effects justify the continuation of the project in patients with hemophilia A with inhibitor. Figure Disclosures No relevant conflicts of interest to declare.

scholarly journals Health status and quality of life of elderly persons with severe hemophilia born before the advent of modern replacement therapy

2009 ◽  
Vol 7 (5) ◽  
pp. 780-786 ◽  
Author(s):  
S. M. SIBONI ◽  
P. M. MANNUCCI ◽  
A. GRINGERI ◽  
M. FRANCHINI ◽  
A. TAGLIAFERRI ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Health Status ◽  
Replacement Therapy ◽  
Elderly Persons ◽  
Severe Hemophilia

scholarly journals Problem Joints and Their Clinical and Humanistic Burden in Children and Adults with Moderate and Severe Hemophilia a: CHESS Paediatrics and CHESS II

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 33-34
Author(s):  
Paul McLaughlin ◽  
Cedric Hermans ◽  
Sohaib Asghar ◽  
Tom Burke ◽  
Francis Nissen ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Research Funding ◽  
Hemophilia A ◽  
Joint Damage ◽  
Severe Hemophilia ◽  
Publicly Traded ◽  
Hemophilic Arthropathy ◽  
Joint Health ◽  
Humanistic Burden

Introduction Severe hemophilia A (SHA) is characterized by spontaneous (non-trauma related) bleeding episodes into the joint space and muscle tissue, leading to progressive joint deterioration and chronic pain. Chronic joint damage is most often associated with severe hemophilia, however more recent research has illustrated that people with moderate hemophilia A (MHA) also experience hemophilic arthropathy and functional impairment. The need to measure joint health in children as well as adults, is underscored by findings from the Joint Outcome Continuation Study, which found that FVIII prophylaxis was insufficient to protect joints from damage, from childhood through adolescence in severe HA (Warren et al., 2020). The objective of this analysis is to gain a more patient-centric understanding of the clinical, economic and humanistic burden associated with 'Problem Joints', a measure of joint morbidity developed in consultation with an expert panel to overcome limitations with existing measures, in people with MHA and SHA. Methods A descriptive cohort analysis was conducted, utilizing retrospective, cross-sectional real-world data from the 'Cost of Haemophilia in Europe: a Socioeconomic Survey' (CHESS Paeds and CHESS II), studies of adult and pediatric persons with hemophilia. The analysis population is comprised of children (17 and below) with MHA or SHA in CHESS Paeds, and adults aged 20 and over with MHA or SHA in CHESS II. To account for the possibility that persons aged 18 or 19 in CHESS II may have participated in CHESS Paeds, these individuals were excluded from the analysis. Physician-reported clinical outcome data and patient/caregiver-reported quality of life were analyzed. A problem joint (PJ) is defined as having chronic joint pain and/or limited range of movement due to compromised joint integrity (i.e. chronic synovitis and/or hemophilic arthropathy). Analyses were stratified by number of PJs: none, 1 PJ, and 2+ PJs. We report retrospective data of the 12 months prior to study enrollment, on annualized bleeding rate (ABR), prevalence of target joints (TJ), as defined by the International Society on Thrombosis and Haemostasis, and EQ-5D-/5L/Y/Proxy score. Results are presented as mean (standard deviation) or N (%). Results Among 785 participants (N = 464 SHA; N = 321 MHA) in CHESS Paeds, mean age and BMI were 10.33 (4.63) and 22.50 (17.07), respectively. Of 493 participants (aged 20 and above) in CHESS II (N = 298 SHA; N = 195 MHA), the mean age and BMI were 38.61 (14.06) and 24.55 (2.92), respectively. Current inhibitor to FVIII replacement was more prevalent in children than in adults (10% vs. 5%). In CHESS II, approximately 40% of people with MHA and 49% with SHA had one or more PJs, respectively [1 PJ (23% vs. 28%); 2+ PJs (16% vs. 21%)]. In CHESS Paeds, approximately 14% of children with MHA and 18% with SHA had at least one PJ, respectively [1 PJ (9% vs. 14%); 2+ PJs (5% vs. 3%)]. TJs were less prevalent with MHA in comparison to SHA, in both adults (24% vs. 45%) and children (13% vs. 22%). Clinical burden was higher among both children and adults with PJs compared to those with no PJs. ABR correlates with the number of PJs, in those with MHA and SHA in CHESS II (Figure 1). Similarly, PJs were associated with higher ABR across MHA and SHA in CHESS Paeds (Figure 2). Hemophilia-related hospitalizations were higher in both adult and pediatric participants with PJs. In CHESS II, MHA with no PJs had fewer [0.73 (1.23)] hospitalizations compared to having those with 1 PJ [1.38 (1.11)] or 2+ PJs [1.28 (1.25)]. Similarly, children with MHA with 2+ PJs had 1.60 (1.92) hemophilia-related hospitalizations, compared to 1.38 (1.92) with 1 PJ and 0.71 (1.14) with no PJs. PJs were associated with impaired quality of life. In CHESS II, MHA and SHA EQ-5D-5L values in persons with no PJs were 0.81 (0.19) and 0.79 (0.18), respectively, compared to 0.65 (0.16) and 0.62 (0.23) with 1 PJ, and 0.65 (0.14) and 0.51 (0.33) in with 2+ PJs. A similar trend was observed in EQ-5D-Y and EQ-5D-proxy scores in CHESS Paeds. Conclusions Data from CHESS Paeds and CHESS II demonstrate an association between chronic joint damage, as measured by the 'problem joint' definition, and worsening clinical and quality of life outcomes, across both MHA and SHA. Further analyses will seek to expand upon the initial results presented here, to investigate the wider elements of burden associated with compromised long-term joint health. Disclosures McLaughlin: BioMarin: Consultancy; Novo Nordisk: Consultancy, Speakers Bureau; Sobi: Consultancy, Speakers Bureau; Roche/Chugai: Speakers Bureau; Takeda: Speakers Bureau. Hermans:Novo Nordisk: Consultancy, Speakers Bureau; Roche: Consultancy, Speakers Bureau; Sobi: Consultancy, Research Funding, Speakers Bureau; Biogen: Consultancy, Speakers Bureau; CAF-DCF: Consultancy, Speakers Bureau; CSL Behring: Consultancy, Speakers Bureau; Shire, a Takeda company: Consultancy, Research Funding, Speakers Bureau; Pfizer: Consultancy, Research Funding, Speakers Bureau; Bayer: Consultancy, Research Funding, Speakers Bureau; WFH: Other; EAHAD: Other; Octapharma: Consultancy, Speakers Bureau; Kedrion: Speakers Bureau; LFB: Consultancy, Speakers Bureau. Asghar:HCD Economics: Current Employment. Burke:HCD Economics: Current Employment; University of Chester: Current Employment; F. Hoffmann-La Roche Ltd: Consultancy. Nissen:GSK: Research Funding; Novartis: Research Funding; Actelion: Consultancy; F. Hoffmann-La Roche Ltd: Current Employment. Aizenas:F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company. Meier:F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company. Dhillon:HCD Economics: Current Employment; F. Hoffmann-La Roche Ltd: Other: All authors received editorial support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. . O'Hara:F. Hoffmann-La Roche Ltd: Consultancy; HCD Economics: Current Employment, Current equity holder in private company.

Factor VIII Intron 22 Inversion Screening of Newborn Males for Hemophilia A: A Cost-Effectiveness Study

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 734-734
Author(s):  
John W. Luiza ◽  
Margaret V. Ragni ◽  
Robert F. Sidonio ◽  
Kenneth J Smith
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Factor Viii ◽  
Hemophilia A ◽  
Severe Hemophilia ◽  
Pcr Screening ◽  
Intron 22 Inversion ◽  
Screening Cost ◽  
The Cost

Abstract Abstract 734 Background: Severe hemophilia A is an X-linked congenital bleeding disorder occurring in 1:5,000 male births. Among neonates with severe hemophilia A, failure to recognize hemophilia and associated bleeding may result in severe blood loss anemia from circumcision, central nervous system (CNS) bleeding during the birth process or with head trauma and associated neurologic sequelae, and unrecognized joint bleeding that, when recurrent, increases the risk of joint damage which may lead to chronic disability. In at least one-third of cases, the disease arises as a spontaneous mutation: yet, even among the two-thirds with a family history, most carriers do not undergo carrier testing or prenatal diagnosis, leaving only a minority in whom cord blood screening is performed. About half of newborns with severe hemophilia A have a factor VIII (F.VIII) intron 22 inversion mutation, readily detected by PCR screening. We, therefore, sought to determine the effects of newborn screening by F.VIII intron 22 inversion PCR on early diagnosis in children with severe hemophilia A, specifically, on prevention of early life bleeding and associated cost, morbidity, and quality of life. Methods: We constructed a decision tree model to evaluate the cost effectiveness of newborn F.VIII intron 22 screening for severe hemophilia A. We assumed all newborn males were tested as part of screening, and that treatment modifies the likelihood of bleeding but not bleeding associated morbidity. Rates of major and minor CNS, joint, and procedural/surgical bleeding, including circumcision, morbidity and mortality, cost, and quality of life utilities were obtained from the literature. We assumed the cost of intron 22 PCR testing to be $3.00 per newborn male, that test results were available within 2 days of screening, and that clotting factor was infused prior to procedures and at the first sign of joint bleeding or head trauma. The probability of severe bleeding requiring hospitalization or red blood cell transfusion was estimated to be 5% or less in children with severe hemophilia A. The cost of F.VIII concentrate was based on the average wholesale price, and transfusion and hospitalization costs were based on local data. Outcomes included medical costs for each bleeding event, effectiveness measured as quality-adjusted-life-years (QALY), and the incremental cost-effectiveness ratio (ICER) over the first two years of life. Sensitivity analysis was used to test the robustness of analysis results. Results: Compared to no screening, screening for hemophilia had an ICER of $96,918/QALY, a value considered economically reasonable. Results were sensitive to variation of screening cost and overall detection of hemophilia A by PCR screening (base case 50%). Effects of varying both these parameters in a two-way sensitivity analysis are shown in the Figure. Using a $100,000 per QALY cost-effectiveness criterion over the depicted ranges for both parameters, screening was favored if screening cost ≤$3 or if ≥56% of all newborns with hemophilia A were detected by screening. Conclusion: It is cost effective to perform factor VIII intron 22 PCR screening to identify severe hemophilia A in newborn males in order to prevent bleeding morbidity, if the cost of the test does not exceed $3.00. Disclosures: No relevant conflicts of interest to declare.

Long-Term Prophylaxis with Activated Recombinant FVII in Children with Hemophilia a and Inhibitor, Receiving Treatment with ITI Protocol

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4980-4980 ◽  
Author(s):  
Ekaterina Shiller ◽  
Victor Petrov ◽  
Pavel Svirin ◽  
Vladimir Vdovin ◽  
Igor Koltunov ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Factor Viii ◽  
Hemophilia A ◽  
Treatment Time ◽  
Conflicts Of Interest ◽  
Factor Vii ◽  
Treatment Level ◽  
Factor Viii Inhibitor ◽  
Bleeding Episodes

Abstract Background: Recent studies have shown that addition of bypassing agents to immuno-tolerance induction (ITI) protocol for patients with hemophilia A and inhibitor results in better control of bleeding episodes and improves quality of life. Few publications have addressed prophylactic usage of recombinant factors VIIa in these settings. Due to relatively low infusion volume, convenience of administration and high efficacy rFVIIa - Coagil-VII seems to be especially reasonable for ITI protocol. Aim: To assess the efficacy and safety of rFVIIa - Coagil-VII (SJC "GENERIUM", Russia) for prophylactic use during ITI protocol in patients with hemophilia A and inhibitor. Methods: Seven patients aged between 2 to 7 years with severe hemophilia A and inhibitor have been treated with ITI protocol using plasma derived factor VIII with von Willebrand factor. Seven of them simultaneously received treatment with Coagil-VII in individual doses (100-250 mkg/kg) and regimens (every 12 - 48 hours). When inhibitor reached level of 3 BU (Bethesda Unit) either dose or frequency of Coagil-VII administration were gradually reduced. After 1 BU the patients were given factor VIII only. Number and severity of bleeding events were assessed. Results: Five patients with high responding inhibitors and poor prognosis (history of high titer of factor VIII inhibitor, prolonged time between first inhibitor appearance and the beginning of ITI) received Coagil-VII in high doses 150 - 250 mkg/kg every 12-24 hours in 1-4 years. At time of booster effect titer of inhibitors reached 92 -16 000 BU. One of patients had ITI failure because of interruption of protocol, while 4 patients continue treatment. Level of 3 BU was reached by 4 patients at 40, 12, 35, and 3 months of treatment. Level of 1 BU was reached by 2 patients at 6 and 42 months of treatment. Significant clinical effect was achieved after 6 months of treatment. Time of bleeding episode was decreased from 7 (±2) to 2 (±1) days. Total number of hemorrhagic events, including hemarthrosis, hematomas and bleedings decreased by 3,7 fold (3,7 events per patient-month during first 6 months versus 1,0 events per patient-month). Only 1 hospital admission with bone fracture was recorded. All children have an active lifestyle and attend school. Two patients with low responding inhibitor and good prognosis received Coagil-VII in low doses 90 - 170 mkg/kg every 24-48 hours. Maximal titer of inhibitor was 1.3 - 1.9 BU. Both patients completed treatment with Coagil-VII in 2 and 4 months and continue ITI protocol and both achieved undetectable level of inhibitor. No bleeding episodes were recorded in these patients since the beginning of treatment. There was no clinical or laboratory evidence of thrombosis, thrombocytopenia, or disseminated intravascular coagulation. Conclusion: We report our experience of prolonged (2 months - 4 years) prophylactic treatment with recombinant activated factor VII (rFVIIa) - Coagil-VII in patients with hemophilia A and inhibitor. This prophylaxis is efficacious when doses and treatment regimens are individually determined. This approach results in reduction of bleeding episodes in all patients, as well as increase of quality of life. No any adverse events (AE and SAE) with prolonged use of Coagil-VII have been registered so far. Disclosures No relevant conflicts of interest to declare.

scholarly journals Health status and quality of life in patients with severe hemophilia A: A cross-sectional survey

2019 ◽  
Vol 11 (2) ◽  
Author(s):  
Majid Davari ◽  
Zahra Gharibnaseri ◽  
Roya Ravanbod ◽  
Abolfazl Sadeghi
Keyword(s):  
Quality Of Life ◽  
Hemophilia A ◽  
Clinical Information ◽  
Cross Sectional Study ◽  
Adult Patients ◽  
Severe Hemophilia ◽  
Cross Sectional Survey ◽  
Cross Sectional ◽  
The Mean

Among different groups of hemophiliacs, those suffering from Severe Hemophilia A (SHA) are most vulnerable to the complications of the disease. This study investigated the Health-Related Quality of Life (HR-QoL) among adult patients with SHA. A cross-sectional study was designed to gather demographic and clinical information from adult patients with SHA. Patients with inhibitors were excluded. The remaining were asked to complete the HR-QoL questionnaire after being examined for joint health using the Hemophilia Joint HealthScore (HJHS). The HR-QoL and joint conditions were measured in 38 patients. The mean EQ-5D value scores were 0.46 (SD=0.23) while the mean Visual Analogous Scale score was 50 (SD=18.7). The clinical examination of patients indicated that the HJHS were as follows: eight patients had a score of 55-75, 12 patients had a score of 40-55, 7 of them (25-40) and 11 patients had a score of 10-25. The results obtained from this study showed that HR-QoL in hemophilia patients was considerably low. Pain, anxiety/depression, and motion limitations were the main causes of the disutility for these patients respectively.

scholarly journals Impact of a cardio-oncology rehabilitation program in patients with breast cancer undergoing cardiotoxic treatment

2021 ◽  
Vol 42 (Supplement_1) ◽  
Author(s):  
C C Oliveira ◽  
M Matos ◽  
R Azevedo ◽  
R Flores ◽  
P Medeiros ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Endothelial Function ◽  
Cancer Patients ◽  
Exercise Program ◽  
Rehabilitation Program ◽  
Exercise Group ◽  
Breast Cancer Patients ◽  
The Impact

Abstract Background Cancer treatment–related cardiotoxicity (CTrCD) is commonly associated with anthracyclines and anti-HER2 agents which are widely used for the treatment of breast cancer. In order to mitgate CTrCD, exercise-based cardio-oncology rehabilitation (COR) involving a structured exercise program has been proposed. Objectives To evaluate the impact of a cardiac-rehabilitation program in breast cancer patients submitted to chemotherapy with known cardiotoxicity. Methods A systematic review was performed. Two databases were searched, PubMed and SCOPUS. All randomized or controlled trials and other prospective studies published between 2000 and March 2020 which evaluate the impact of an aerobic exercise program on cardiorespiratory fitness (CRF), health-related quality of life (QOL), vascular/endothelial function as well as cardiac assessment namely through the evaluation of left ventricular function and cardiac biomarkers in patients undergoing anthracycline and/or anti-HER2 treatments were included. Main results Fourteen studies were included enrolling 578 breast cancer patients with a mean age of 48.80 years. Regarding the impact of exercise in CRF, 5 studies (n=176) reported a significant improvement of VO2 max. and 4 studies of VO2max./kg (n=137). The peak power output was also improved in 4 studies (n=95) in the exercise-group. Considering the assessment of QOL, 3 studies (n=180) revealed significant differences favoring the exercise-group. The results regarding the evaluation of the LVEF were not clear: 2 studies (n=48) reported a significant decrease on LVEF when compared to baseline at the end of the intervention in both groups, despite the exercise program. However, 2 studies (n=97) showed a significant increase on LVEF in the exercise-group. Three studies (n=82) did not found significant differences in global longitudinal strain between groups. Limited evidence was found in vascular and endothelial functon. In 2 studies (n=50), endothelial function measured by brachial artery flow-mediated dilatation significantly improved in the exercise-group. Two studies (n=98) reported no significant impact of exercise on atenuating the increase of cTnI and BNP levels in the course of chemotherapy. On the other hand, when considering NT-proBNP, an increase in its levels was attenuated in the exercise-group. Conclusions This study confirms that exercise-based COR seems to be an effective approach to improve several cardiovascular outcomes and quality of life in breast cancer patients. FUNDunding Acknowledgement Type of funding sources: None.

scholarly journals Effect of tertiary prophylaxis with low-dose factor VIII in quality of life in adult patients with severe hemophilia A

2019 ◽  
Vol 10 (3) ◽  
pp. 88
Author(s):  
PrakasKumar Mandal ◽  
Abhijit Phukan ◽  
Amrita Bhowmik ◽  
Debasis Gantait ◽  
Prantar Chakrabarti
Keyword(s):  
Quality Of Life ◽  
Factor Viii ◽  
Low Dose ◽  
Hemophilia A ◽  
Adult Patients ◽  
Severe Hemophilia ◽  
Dose Factor
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