Für Sie Recherchiert 1/2022
Fitness enhances psychosocial well-being and self-confidence in young men with hemophilia: Results from Project GYM
Khair K., Holland M., Dodgson S., McLaughlin P., Fletcher S., Christie D.
[In Process] Res. Pract. Thromb. Haemost. 2021 5:8
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Abstract
Introduction: Contemporary hemophilia care supports physical activity, its benefits being well recognized. Despite recognition of the psychological challenges encountered by people with hemophilia, little is known about the psychological impact of physical fitness in this population. Aim: To identify changes in psychological well-being in young men with hemophilia through participation in a gym program. Methods: This observational feasibility study of a 6-month gym participation program used validated questionnaires pre- and poststudy to evaluate motivation to exercise, physical activity levels, self-efficacy, self-esteem, and quality of life. Individual audio-recorded interviews about study participation and impact were transcribed verbatim and analyzed for recurring themes using thematic analysis. Results: Nineteen participants aged 18–25 years with hemophilia A or B (all severities ± inhibitor) consented to the study; two were lost to follow-up. There was a shift in motivation to exercise as shown by the Stages of Change grouping moving from contemplation to action and maintenance phases (p = 0.03). Self-efficacy overall scores showed a trend (p < 0.06) towards improvement. Median self-esteem scores improved from 22 (range 12–30, n = 19) to 25 (range 13–30, n = 17), a statistically significant change (p = 0.02). Three participants recorded scores below the accepted normal range before study, of whom two improved at study end. The key themes identified from the interviews were: fear, self-confidence, “being normal,” pain, weight loss, ability, getting fitter. Conclusions: The psychological wellbeing of young men with hemophilia improved during this study. This may have been related to participating in a gym-based, physical exercise program.
Estimating the risk of thrombotic events in people with congenital hemophilia A using US claims data
Faghmous I., Nissen F., Kuebler P., Flores C., Patel A.M., Pipe S.W.
J. Comp. Eff. Res. 2021 10:18 (1323-1336)
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Abstract
Aim: Compare thrombotic risk in people with congenital hemophilia A (PwcHA) to the general non-hemophilia A (HA) population. Patients & methods: US claims databases were analyzed to identify PwcHA. Incidence rates of myocardial infarction, pulmonary embolism, ischemic stroke, deep vein thrombosis and device-related thrombosis were compared with a matched cohort without HA. Results: Over 3490 PwcHA were identified and 16,380 individuals matched. PwcHA had a similar incidence of myocardial infarction and pulmonary embolism compared with the non-HA population, but a slightly higher incidence of ischemic stroke and deep vein thrombosis. The incidence of device-related thrombosis was significantly higher in PwcHA. Conclusion: This analysis suggests that PwcHA are not protected against thrombosis, and provides context to evaluate thrombotic risk of HA treatments.
Original (Non-English) Abstract
Tweetable abstract People with #hemophilia A are not protected against thrombotic events and should be monitored for thrombotic risk factors; the thrombotic risk of hemophilia A treatments should also be evaluated.
Predictors of inhibitor eradication by primary immune tolerance induction in severe haemophilia A with high responding inhibitors
Di Minno G., Coppola A., Margaglione M., Rocino A., Mancuso M.E., Tagliaferri A., Linari S., Zanon E., Santoro C., Biasoli C., Castaman G., Santagostino E., Mannucci P.M.
[Article in Press] Haemophilia 2021:
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Abstract
BACKGROUND: Immune tolerance induction (ITI) is the only proven strategy to eradicate factor VIII inhibitors in patients with haemophilia A (HA). AIM: To identify patients and treatment options with the highest chance of inhibitor eradication by primary ITI. PATIENTS AND METHODS: In the frame of the Italian ITI Registry, carried out from 1995 to 2015 (last follow-up 2018), 137 primary ITI courses in severe HA patients (90/137 with poor prognosis) were analysed for predictors of outcome (complete/partial response or failure). Sixty-six of them (48%) were prospectively evaluated. RESULTS: ITI was successful in 91/137 patients (66.4%) and 70 (51.1%) achieved complete response within 11 months (median). Historical peak titres ≤200 BU/ml (P = .033), inhibitor titres ≤5 BU/ml at ITI start (P = .001), peak titres ≤100 BU/ml during ITI (P < .001) and missense mutations and small insertions/deletions of FVIII gene (P = .027) predicted complete inhibitor eradication. A score that considers the cumulative number of these variables predicted complete response with positive predictive values up to .81 at ITI start and .91 during ITI, respectively. Patients who had no bleeding (OR, 3.45, 95% CI: 1.4-8.6) nor other adverse events (OR 2.6, 95%CI: 1.3-5.3) during ITI had higher chances of complete response. During the 120-month follow-up (median), 2/70 patients who had achieved complete response relapsed (2.9%). CONCLUSIONS: This Registry, with a centralized review of outcomes, homogeneous data collection (half of which prospective) and long-term follow-up, provides insights for optimizing ITI, with a rationale for further studies in the currently evolving scenario of inhibitor management in HA patients.
The role of physiotherapy in the new treatment landscape for haemophilia
Lobet S., Timmer M., Königs C., Stephensen D., McLaughlin P., Duport G., Hermans C., Mancuso M.E.
J. Clin. Med. 2021 10:13
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Abstract
The physiotherapist plays an essential role for people with haemophilia, an inherited bleeding disease responsible for musculoskeletal complications. Yet, with the advent of new and advanced therapies, the medical landscape is changing, and physiotherapy must adapt alongside. This paper considers whether there will still be a need for physiotherapy in the era of advanced therapies, and discusses ways in which services should evolve to complement emerging treatment paradigms for haemostasis in people with haemophilia. Ultimately, physiotherapy will remain an important element of care, even for people with little joint damage and low risks in the era of the new mild phenotype. However, competencies will need to evolve, and physiotherapists in both primary care and specialist treatment centres should work with haematology colleagues to develop more sensitive tools for detecting early joint changes. Physiotherapists will also play a crucial role in counselling and physically coaching, monitoring the musculoskeletal status of people with haemophilia who have transitioned to new treatments.
Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
Holstein K., Le Quellec S., Klamroth R., Batorova A., Holme P.A., Jiménez-Yuste V., Astermark J.
[Article in Press] Haemophilia 2021 :
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Abstract
Introduction: The development of inhibitory antibodies is a severe complication of clotting factor replacement therapy in patients with severe haemophilia A (HA). Current World Federation of Hemophilia (WFH) guidelines for haemophilia care indicate that eradication of inhibitors is best achieved through immune tolerance induction (ITI) therapy. Aim: The European Collaborative Haemophilia Network conducted a survey to determine whether ITI is still used in the routine management of patients with HA, and whether the availability of emicizumab prophylaxis has influenced treatment decisions. Methods: The survey was conducted in late 2020/early 2021 in 18 centres representing 17 countries in the Europe/Middle East region treating a total of 4955 patients, and included sections specific to patient and centre demographics, treatment protocols (both ITI and prophylactic), inhibitor development and initiation of ITI, treatment success, and the incidence of adverse events. Results: While our results indicate that ITI can still be considered a mainstay of treatment for patients with HA with inhibitors, less than daily dosing of ITI in combination with emicizumab prophylaxis is becoming commonplace across the spectrum of disease severity, with initiation being guided by bleeding patterns. The most frequently cited reasons for not initiating emicizumab prophylaxis were availability or reimbursement issues. Conclusion: ITI remains a mainstay for haemophilia treatment of patients with HA with inhibitors, but emicizumab has become a preferred first-line approach to protect against bleeds and represents an alternative to burdensome ITI in certain patient groups.
Diagnosis and treatment of chronic synovitis in patients with haemophilia: consensus statements from the Italian Association of Haemophilia Centres
Di Minno M.N.D., Napolitano M., Giuffrida A.C., Baldacci E., Carulli C., Boccalandro E., Bruno C., Forneris E., Ricca I., Passeri W., Martinelli M., Rivolta G.F., Solimeno L.P., Martinoli C., Rocino A., Pasta G., Aru B., Balestri E., Biasoli C., Cantori I., Coluccia A., Contino L., Coppola A., Cultrera D., Daniele F., Ettorre C.P., Giordano P., Iannaccaro P., Lassandro G., Linari S., Luciani M., Malcangi G., Mancuso M.E., Marchesini E., Marino R., Mazucconi M.G., Molinari A.C., Notarangelo L.D., Pasca S., Quintavalle G., Radossi P., Raso S., Sabatino V., Santagostino E., Santoro C., Scaturro D., Zanon E.
[Article in Press] Br. J. Haematol. 2021:
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Abstract
Although synovitis is recognized as a marker of joint disease activity, its periodic assessment is not included in routine clinical surveillance of patients with haemophilia (PwH). In order to evaluate the current knowledge and to identify controversial issues, a preliminary literature search by the Musculoskeletal Committee of the Italian Association of Haemophilia Centres (AICE) has been conducted. Statements have been established and sent to the Italian AICE members to collect their level of agreement or disagreement by a Delphi process. Thirty-seven consensus recommendations have been drafted. We found a general agreement on the indication to consider the presence of synovitis as a marker of joint disease activity in PwH. Accordingly, there was agreement on the indication to search for synovitis both in patients reporting joint pain and in asymptomatic ones, recognizing ultrasound as the most practical imaging technique to perform periodic joint screening. Interestingly, after detection of synovitis, there was agreement on the indication to modify the therapeutic approach, suggesting prophylaxis in patients treated on demand and tailoring treatment in patients already under prophylaxis. Whereas the need of an early consultation with a physiotherapist is recommended for PwH affected by chronic synovitis, the exact timing for an orthopaedic surgeon consultation is currently unknown.
Indirect treatment comparison of damoctocog alfa pegol versus turoctocog alfa pegol as prophylactic treatment in patients with hemophilia A
Vashi P., Batt K., Klamroth R., Mancuso M.E., Majewska R., Tiede A., Mantovani L.G.
J. Blood Med. 2021 12: (935-943)
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Abstract
Purpose: To assess the efficacy and FVIII consumption of BAY 94-9027 versus N8-GP in prophylaxis in adolescent and adult patients with severe hemophilia A (HA). Patients and Methods: A systematic literature review was conducted to identify studies on the efficacy of BAY-94-9027 and N8-GP for prophylaxis in patients with HA aged ≥12 years without a history of inhibitors. Eight studies met systematic literature review inclusion criteria, but only data from PROTECT VIII on BAY 94-9027 and PATHFINDER 2 on N8-GP could be used for an indirect comparison. Matching-adjusted indirect comparison (MAIC) and simulated treatment comparison were performed. Results: No significant differences (unadjusted and adjusted) were observed in the mean annualized bleeding rate (ABR) for any bleed and proportion of patients with zero bleeds when comparing BAY 94-9027 to N8-GP. The adjusted treatment difference [incidence rate ratio (IRR)] in terms of ABR was 1.11 (95% CI, 0.85–1.44). The odds ratio (OR) of any bleed, measuring the relative effect of BAY 94-9027 versus N8-GP on the proportion of patients with zero bleeds, was 1.03 (95% CI, 0.60–1.77). FVIII consumption was significantly lower in BAY 94-9027 [mean adjusted difference=−1292.57 IU/kg/year (95% CI, ‒ 2152.44 to ‒432.70)]; a 26.7% reduction in consumption of BAY-94-9027. The results of the sensitivity analyses were similar to the main analysis for mean ABRs, percentages of patients with zero bleeds, and significant reduction in rFVIII consumption. For patients on BAY 94-9027 every-5-days and every-7-days, no differences versus every-4-days N8-GP were observed for the mean ABR for any bleed [IRR=0.90 (95% CI, 0.68‒1.20)] and proportion of patients with zero bleeds [OR=1.06 (95% CI, 0.56‒2.02)]. Conclusion: BAY 94-9027 prophylaxis demonstrated 26.7% lower annual consumption when compared to N8-GP with similar efficacy in terms of ABR and percentage of patients with zero bleeds.
