scholarly journals Opportunities for Improvement: The Lot of the Inhibitor Patient Is Not a Happy One

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5040-5040
Author(s):  
Frank V. McL. Booth ◽  
Johnny Mahlangu ◽  
Howard Levy
Keyword(s):  
Quality Of Life ◽  
Half Life ◽  
Research Funding ◽  
Hemophilia A ◽  
Assessment Tools ◽  
Life Assessment ◽  
Breakthrough Bleeding ◽  
Present Trial

Abstract INTRODUCTION: Hemophilia A or B carries significant morbidity - progressive throughout life. The mainstay of treatment is regular factor replacement to treat or prevent bleeding episodes. Up to 30% of Hemophilia A and up to 5% of Hemophilia B develop neutralizing antibodies (inhibitors) rendering factor replacement therapy ineffective. Hemophilia patients with inhibitors are managed with bypassing agents for treatment or prevention of bleeds. Prophylaxis is not adequate in inhibitor patients using currently available bypassing products due to their very short half-life (2-3 hours for rFVIIa; 5-6 hours for aPCC) so they are treated on an episodic basis for bleeds. On-demand treatment results in poor quality of life, significantly higher mortality and inferior musculoskeletal outcomes in inhibitor patients when compared to patients without inhibitors. The quality of life of hemophilia has been evaluated using EQ-5D and Haem-A-QOL and impaired activity has been measured with Haemophilia Activity List (HAL). The EQ-5D is a widely used internationally validated general-purpose quality of life instrument however it lacks granularity and is not specific to Hemophilia. Haem A-QOL (Mackensen et al 2004) and Hemophilia Activities List (HAL) (Van Genderen FR et al 2006.) were developed and validated specifically to address the typical disabilities and life issues faced by patients with Hemophilia. Marzeptacog alfa (activated) (MarzAA) was created using a structure-based rational protein design and has 4 amino acid substitutions to enhance the biological properties of FVIIa. This variant molecule has substantially (6-7x) greater potency than wild-type FVIIa, a greater half-life and sufficient bioavailability when given subcutaneously (SQ). There is currently a paucity of data on quality of life (QoL) of inhibitor patients. We evaluated the baseline QoL and functional activity of inhibitor patients enrolled in the MAA-201 study using the EQ-5D , Haem-A-QoL and HAL compared the results to those of reference hemophilia patients without inhibitors. METHODS A phase 2 / 3 open-label study evaluating safety and efficacy of MarzAA in hemophilia patients with inhibitors is underway MAA-201, (NCT03407651) Subject eligibility required an annualized bleeding rate of >12 and documented inhibitor to replacement factor. The primary aim of the trial was the complete prevention of breakthrough bleeding for 50 days by the daily administration of a fixed SQ dose of MarzAA. If breakthrough bleeding occurred, up to three dose escalations were permitted. At baseline and study conclusion, each subject completed quality of life assessment tools the EQ-5D, Haem-A-QOL and the Hemophilia Activities List Haem-A-QOL assesses subjects across ten domains and provides a summarized score. Subjects in the present trial (MARZAAPOP) were compared to baseline values for subjects with severe hemophilia but without inhibitors recruited into a long-term prophylaxis trial (The A-LONG trial - ALONGREFPOP). Mean baseline Haem-A-QOL summed score in the A-LONG trial was 29.3 ±15.7 contrasting sharply with a mean baseline summed score of 42 ±15.2 for subjects in the present trial (Table 1) HAL is more motor function oriented and assesses patients across ten domains of which seven assess basic functionality and three are composites intended to assess disability in performing complex tasks. HAL provides both raw and normalized scores. Normalized scores provide meaningful output in the case of missing data elements. For each domain and for the sum score, a raw score of 0 and a normalized score of 100 indicates no functional deficit. Figure 1 provides a visual comparison between recruited subjects (MARZAAPOP) in the present trial and the population used to validate the HAL tool. (HALREFPOP). CONCLUSION: In examining recruits into the present trial, it is clear that inhibitor patients have generally worse functional scores than either of two reference groups. Effective long-term prophylaxis is expected to produce measurable improvement in QOL scores in this hard-to-treat population. Disclosures Booth: Catalyst Biosciences: Consultancy. Mahlangu:Sanofi: Research Funding, Speakers Bureau; Roche: Consultancy, Research Funding, Speakers Bureau; LFB: Consultancy; NovoNordisk: Consultancy, Research Funding, Speakers Bureau; CSL Behring: Consultancy, Research Funding, Speakers Bureau; Chugai: Consultancy; Catalyst Biosciences: Consultancy, Research Funding; Biomarin: Research Funding, Speakers Bureau; Biogen: Research Funding, Speakers Bureau; Bayer: Research Funding; Amgen: Consultancy; Alnylam: Consultancy, Research Funding, Speakers Bureau; Shire: Consultancy, Research Funding, Speakers Bureau; Sobi: Research Funding, Speakers Bureau; Spark: Consultancy, Research Funding. Levy:Catalyst Biosciences: Employment, Equity Ownership.

Long-Term Effectiveness of Fingolimod for Multiple Sclerosis in a Real-World Clinical Setting

2021 ◽  
pp. 1-6
Author(s):  
Cihat Uzunköprü ◽  
Yesim Beckmann ◽  
Sabiha Türe
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Real Life ◽  
Life Assessment ◽  
Health Related Quality ◽  
Serious Adverse Events ◽  
Related Quality ◽  
Health Related

<b><i>Introduction:</i></b> The primary aim of the present study was to evaluate the long-term efficacy of fingolimod in patients with multiple sclerosis (MS); secondary aims were to describe the safety of fingolimod with the evaluation of treatment satisfaction and impact on the quality of life in real life. <b><i>Methods:</i></b> We collected clinical, demographical, neuroradiological, and treatment data, including pre- and posttreatment status health-related quality of life from 286 MS patients consecutively treated with fingolimod. Clinical assessment was based on the Expanded Disability Status Scale (EDSS), and quality of life assessment was performed with MS-related quality of life inventory (MSQOLI). The data were recorded at baseline and every 6 months for 2 years. <b><i>Results:</i></b> One hundred and fourteen males and 172 females were enrolled. The annualized relapse rate and EDSS showed a statistically significant reduction during the observation period (<i>p</i> &#x3c; 0.001). The patients also demonstrated substantial improvements in magnetic resonance imaging (MRI) outcomes (<i>p</i> &#x3c; 0.001). Health-related quality of life scores improved significantly between baseline and 24-month visit (<i>p</i> &#x3c; 0.001). No serious adverse events occurred. <b><i>Conclusion:</i></b> In our cohort, fingolimod treatment was associated with reduced relapse, MRI activity, and improved EDSS and MSQOLI scores. Additionally, fingolimod has been able to maintain its effectiveness over a considerable long period of treatment.

Liver transplantation for Wilson???s disease: long-term results and quality-of-life assessment

2003 ◽  
pp. 1003-1006 ◽  
Author(s):  
Robert P. Sutcliffe ◽  
Donal D. Maguire ◽  
Paolo Muiesan ◽  
Anil Dhawan ◽  
Giorgina Mieli-Vergani ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Liver Transplantation ◽  
Life Assessment ◽  
Long Term Results ◽  
Quality Of Life Assessment

Abstract 10394: Long-Term Functional and Quality-of-Life Outcomes of Cardiac Arrest Survivors Stratified by Shock Provider: A 10 Year Retrospective Study

Circulation ◽  
2021 ◽  
Vol 144 (Suppl_2) ◽  
Author(s):  
Brian A Haskins ◽  
Ziad Nehme ◽  
Emily Andrew ◽  
Stephen Bernard ◽  
Peter Cameron ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cardiac Arrest ◽  
Functional Recovery ◽  
Short Form ◽  
First Responders ◽  
Assessment Tools ◽  
Related Quality ◽  
Health Related

Introduction: To assess the long-term functional recovery and health related quality-of-life (HRQoL) outcomes for out-of-hospital cardiac arrest (OHCA) survivors stratified by shock provider. Methods: We included adult OHCA in initial shockable rhythms between 2010-2019. Those surviving to 12 months post arrest were invited to participate in telephone interviews to identify functional recovery and HRQoL outcomes, using the following assessment tools, Glasgow Outcome Scale-Extended (GOS-E), EuroQol-5D (EQ-5D), and 12-Item Short Form (SF-12). Results: Of the 1,581 patients surviving to 12 months, 1,325 (85.5%) responded to the interviews, of these, 227 (17.1%) and 144 (10.9%) were initially shocked by bystanders and first responders, respectively. A higher proportion of patients shocked by bystanders were located in public (p<0.001), received bystander CPR (p<0.001) and received initial defibrillation faster from time of collapse (P<0.001). Survivors receiving bystander defibrillation reported higher rates of living at home without care (p=0.004), upper good recovery (GOS-E) (p=0.008) and EQ-5D index score of 1 (perfect health) (p=0.023). After adjustment, bystander defibrillation was associated with a 64% increase in the odds of an EQ-5D current Visual Analogue Scale ≥ 80 (AOR 1.64, 95%CI: 1.17-2.31; p=0.004) and a 45% increase in the odds of a good functional recovery (GOS-E ≥ 7) (AOR 1.45, 95% CI: 1.02-2.06; p=0.037), than those initially shocked by paramedics. No improvement in adjusted outcomes were observed for survivors initially defibrillated by first responders. Conclusion: Patients receiving bystander defibrillation reported better functional recovery and HRQol outcomes at 12 months compared to those defibrillated by first responders and paramedics.

The effectiveness of various interventions versus standard stripping in patients with varicose veins in terms of quality of life

2017 ◽  
Vol 33 (7) ◽  
pp. 439-450 ◽  
Author(s):  
Christos Argyriou ◽  
Christos Papasideris ◽  
George A Antoniou ◽  
Efstratios Georgakarakos ◽  
Nikolaos Papanas ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Varicose Veins ◽  
Meta Analysis ◽  
Assessment Tools ◽  
Life Assessment ◽  
Surgical Ligation ◽  
Treatment Techniques ◽  
Meta Analyses ◽  
Q Statistic

Purpose Lower limb varicose veins have a significant effect upon the quality of life and a considerable socioeconomic impact despite their relatively benign nature. The aim of this study is to compare the effects of various therapeutic strategies among patients with varicose veins to surgical ligation and vein stripping on the basis of quality of life. Methods PubMed/Medline and Scopus databases were systemically searched from 1 January 2000 until 23 December 2015 for studies reporting outcome on the quality of life of different treatment techniques for varicose veins. We used Cohen’s d to make the outcomes of the reported scales comparable. Heterogeneity was calculated with the use of the Q statistic and the I2. Results A total of 1047 participants were randomized across all analyzed trials. The number of participants in a single trial ranged from 30 to 308. The majority of participants in any trial were C2 on the CEAP scores. Overall, the quality of evidence was low. For the meta-analysis performed at 12 months postintervention (seven studies, n = 1047 patients) and after random effects meta-analyses due to high heterogeneity, no differences are observed between intervention and surgical ligation and vein stripping. The pooled estimate is −0.001 and the 95% confidence interval is −0.069 to 0.067 with a p = 0.98. In the case of the 24 months, postintervention analysis (six studies, n = 840 patients) the inference is almost identical. The effect of various interventional modes of treatment compared to surgical ligation and vein stripping is negligible in terms of clinical outcomes and quality of life so that surgical ligation and vein stripping versus the other interventional procedures were equally effective approaches to treat great saphenous vein incompetence in terms of quality of life measurements. Conclusion The procedures were at least equally efficient in treating patients with varicose vein disease in terms of quality of life assessment tools at 12 and 24 months compared to surgical ligation and vein stripping.

scholarly journals Does long-term GH replacement therapy in hypopituitary adults with GH deficiency normalise quality of life?

2006 ◽  
Vol 155 (1) ◽  
pp. 109-119 ◽  
Author(s):  
Maria Koltowska-Häggström ◽  
Anders F Mattsson ◽  
John P Monson ◽  
Paul Kind ◽  
Xavier Badia ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Gh Deficiency ◽  
Disease Onset ◽  
Life Assessment ◽  
Hormone Deficiency ◽  
Gh Replacement ◽  
Country Specific

Objective: To determine whether impaired quality of life (QoL) in adults with GH deficiency (GHD) is reversible with long-term GH therapy and whether the responses in QoL dimensions differ from each other. Methods: QoL was measured by the Quality of Life–Assessment for Growth Hormone Deficiency in Adults (QoL-AGHDA) in general population samples in England & Wales, The Netherlands, Spain and Sweden (n = 892, 1038, 868 and 1682 respectively) and compared with corresponding patients’ data from KIMS (Pfizer International Metabolic Database) (n = 758, 247, 197 and 484 respectively) for 4–6 years a follow-up. The subsets of patients from England and Wales, and Sweden with longitudinal data for 5 years’ follow-up were also analysed. The change of the total QoL-AGHDA scores and responses within dimensions were evaluated. Subanalyses were performed to identify any specificity in response pattern for gender, age, disease-onset and aetiology. Results: Irrespective of the degree of impairment, overall QoL improved dramatically in the first 12 months, with steady progress thereafter towards the country-specific population mean. Problems with memory and tiredness were the most serious burden for untreated patients, followed by tenseness, self-confidence and problems with socialising. With treatment, these improved in the reverse order, normalising for the latter three. Conclusions: Long-term GH replacement results in sustained improvements towards the normative country-specific values in overall QoL and in most impaired dimensions. The lasting improvement and almost identical pattern of response in each patient subgroup and independent of the level of QoL impairment support the hypothesis that GHD may cause these patients’ psychological problems.

Sign in / Sign up

Export Citation Format

Share Document