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1.
Cost-Effectiveness of Thrombopoietin Mimetics in Patients with Thrombocytopenia: A Systematic Review
Van Remoortel H, Scheers H, Avau B, Georgsen J, Nahirniak S, Shehata N, Stanworth SJ, De Buck E, Compernolle V, Vandekerckhove P
PharmacoEconomics. 2023
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Editor's Choice
Abstract
OBJECTIVES Thrombopoietin (TPO) mimetics are a potential alternative to platelet transfusion to minimize blood loss in patients with thrombocytopenia. This systematic review aimed to evaluate the cost-effectiveness of TPO mimetics, compared with not using TPO mimetics, in adult patients with thrombocytopenia. METHODS Eight databases and registries were searched for full economic evaluations (EEs) and randomized controlled trials (RCTs). Incremental cost-effectiveness ratios (ICERs) were synthesized as cost per quality-adjusted life year gained (QALY) or as cost per health outcome (e.g. bleeding event avoided). Included studies were critically appraised using the Philips reporting checklist. RESULTS Eighteen evaluations from nine different countries were included, evaluating the cost-effectiveness of TPO mimetics compared with no TPO, watch-and-rescue therapy, the standard of care, rituximab, splenectomy or platelet transfusion. ICERs varied from a dominant strategy (i.e. cost-saving and more effective), to an incremental cost per QALY/health outcome of EUR 25,000-50,000, EUR 75,000-750,000 and EUR > 1 million, to a dominated strategy (cost-increasing and less effective). Few evaluations (n = 2, 10%) addressed the four principal types of uncertainty (methodological, structural, heterogeneity and parameter). Parameter uncertainty was most frequently reported (80%), followed by heterogeneity (45%), structural uncertainty (43%) and methodological uncertainty (28%). CONCLUSIONS Cost-effectiveness of TPO mimetics in adult patients with thrombocytopenia ranged from a dominant strategy to a significant incremental cost per QALY/health outcome or a strategy that is clinically inferior and has increased costs. Future validation and tackling the uncertainty of these models with country-specific cost data and up-to-date efficacy and safety data are needed to increase the generalizability.
PICO Summary
Population
Adult patients with thrombocytopenia (18 full economic evaluations).
Intervention
Thrombopoietin mimetics.
Comparison
No thrombopoietin mimetics, watch-and-rescue therapy, standard of care, rituximab, splenectomy or platelet transfusion.
Outcome
Incremental cost-effectiveness ratios varied from a dominant strategy (cost-saving and more effective), to an incremental cost per quality-adjusted life year gained/health outcome of EUR 25,000-50,000, EUR 75,000-750,000 and EUR > 1 million, to a dominated strategy (cost-increasing and less effective). Few evaluations (n= 2, 10%) addressed the four principal types of uncertainty (methodological, structural, heterogeneity and parameter). Parameter uncertainty was most frequently reported (80%), followed by heterogeneity (45%), structural uncertainty (43%) and methodological uncertainty (28%).
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Prophylactic and therapeutic strategies for intraoperative bleeding in women with von Willebrand disease and heavy menstrual bleeding: A systematic review
Eising, H. P., Punt, M. C., Leemans, J. C., Bongers, M. Y.
Blood reviews. 2023;:101131
Abstract
BACKGROUND Optimal peri-operative management for women with Von Willebrand disease (VWD) and heavy menstrual bleeding (HMB) remains undetermined. AIM AND METHODS To evaluate (pre)operative management in relation to (post)operative bleeding after endometrial ablation (EA) and hysterectomy in VWD women with HMB by performing a database search between 1994 and 2023. RESULTS Eleven cohort studies and 1 case-report were included, of overall 'low' quality, describing 691 operative procedures. Prophylaxis (Desmopressin, clotting factor concentrates or tranexamic acid) to prevent bleeding was described in 100% (30/30) of EA procedures and in 4% (24/661) of hysterectomies. Bleeding complications despite prophylaxis were described in 13% (3/24) of hysterectomies vs 0% (0/30) in EA. CONCLUSION VWD women often seem to experience bleeding complications during hysterectomy and all women with VWD received preprocedural hemostatic agents during EA, indicating potential under- and overdosing of current prophylactic strategies. Prospective studies are needed to determine the optimal (pre)operative strategy for gynecological surgical procedures in women with VWD.
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Sclerotherapy for Hereditary Hemorrhagic Telangiectasia-Related Epistaxis: A Systematic Review
Thiele B, Abdel-Aty Y, Marks L, Lal D, Marino M
The Annals of otology, rhinology, and laryngology. 2022;:34894221078075
Abstract
OBJECTIVES Hereditary hemorrhagic telangiectasia (HHT) is a common inherited condition characterized by mucosal telangiectasias, recurrent epistaxis, and arteriovenous malformations. HHT results in detriment to quality of life. Morbidity and mortality result from severe anemia. Conventional interventions for HHT-related epistaxis include nasal packing, diathermy, lasers, coblation, microdebridement, bevacizumab (topical and systemic), as well as septodermoplasty and nasal closure. Sclerotherapy has been recently described in the literature as a novel approach to HHT-related epistaxis. We hypothesize that sclerotherapy is an effective treatment for HHT-related epistaxis and improves upon the current standard of care for this disease. METHODS A systematic review was conducted to study sclerotherapy for treating HHT-related epistaxis. Ovid MEDLINE, Ovid EMBASE, Scopus, and Web of Science were searched. Articles were evaluated and excluded according to PRISMA guidelines and reviewed by 2 authors. Reported variables included number of injections, months of follow up, changes in Epistaxis Severity Score, previous treatments used to control epistaxis, and post-injection side effects. RESULTS Seven studies with a total of 196 patients met inclusion criteria. Three studies reported significant improvement as measured by the Epistaxis Severity Score scale. One reported improvement through subjective patient surveys and others used the Bergler-Sadick scale to measure frequency and intensity of epistaxis. All studies reported improvement in HHT-related epistaxis. The lack of uniform reporting measures however precluded formal meta-analysis. CONCLUSIONS Based on limited data, sclerotherapy appears to be effective for treating HHT-related epistaxis and offers promise for treating this recalcitrant condition. However, larger, prospective, multi-centered studies using universally validated instruments for epistaxis are needed to definitively evaluate outcomes from sclerotherapy.
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Total Hip Arthroplasty in Patients With Sickle Cell Disease: A Comprehensive Systematic Review
Fassihi SC, Lee R, Quan T, Tran AA, Stake SN, Unger AS
J Arthroplasty. 2020
Abstract
BACKGROUND Total hip arthroplasty (THA) is a common treatment for end-stage osteonecrosis of the hip in patients with sickle cell disease (SCD). This patient population presents unique challenges in the perioperative period. This systematic review aims to investigate the existing literature on the outcomes, complications, and survivorship of primary THA in SCD patients. METHODS A systematic search using Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines was performed of PubMed, MEDLINE, EMBASE, and Cochrane databases for clinical studies on THA in SCD patients. Studies on primary THA in SCD patients with a mean follow-up greater than 90 days were included. RESULTS Sixteen studies containing 5193 SCD patients met criteria for inclusion. The Coleman Quality of Evidence score ranged from poor to moderate. SCD patients had a significant increase in Harris Hip Scores and Merle d'Aubigne Scores after undergoing THA. Compared to non-SCD patients, SCD patients had increased hospital length-of-stay, 30-day and 90-day readmission rates, and rates of medical complications, including pain crises, acute chest syndrome, cardiac complications, sepsis, and mortality. SCD patients also had increased rates of surgical complications, including wound complications, infection, periprosthetic fracture, and aseptic loosening. Overall, THA revision rates were higher in SCD patients relative to those with primary osteoarthritis. CONCLUSION THA remains an effective treatment modality for osteonecrosis of the hip in SCD patients. However, these patients are at increased risk of medical and surgical complications. Surgeons should be aware of the unique challenges in this patient population when counseling and managing these patients in the perioperative period.
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Telehealth Interventions for Improving Self-Management in Patients With Hemophilia: Scoping Review of Clinical Studies
Qian W, Lam TT, Lam HHW, Li CK, Cheung YT
Journal of Medical Internet Research. 2019;21(7):e12340
Abstract
BACKGROUND The introduction of home therapy for hemophilia has empowered patients and their families to manage the disease more independently. However, self-management of hemophilia is demanding and complex. The uses of innovative interventions delivered by telehealth routes such as social media and Web-based and mobile apps, may help monitor bleeding events and promote the appropriate use of clotting factors among patients with hemophilia. OBJECTIVE This scoping review aims to summarize the literature evaluating the effectiveness of telehealth interventions for improving health outcomes in patients with hemophilia and provides direction for future research. METHODS A search was conducted in Ovid MEDLINE, EMBASE, and PubMed databases for studies that (1) focused on patients with hemophilia A or B; (2) tested the use of remote telehealth interventions via the internet, wireless, satellite, telephone, and mobile phone media on patients and caregivers; and (3) reported on at least one of the following patient-/caregiver-focused outcomes related to empowering patients/caregivers to be active decision makers in the emotional, social, and medical management of the illness: quality of life, monitoring of bleeding episodes, joint damage or other measures of functional status, medication adherence, and patients' knowledge. Implementation outcomes (user metrics, cost saving, and accuracy of electronic records) were also evaluated. Reviews, commentaries, and case reports comprising ≤10 cases were excluded. RESULTS Sixteen articles fulfilled the inclusion criteria. The majority of the interventions (10/16, 62%) evaluated both implementation outcomes and patient-/caregiver-focused outcomes. User performance and accuracy and comprehensiveness of electronic records were also measured in most studies (4/16, 87%). The components of the interventions were rather homogenous and typically involved electronic logging and reminders for prophylactic infusions, reporting of spontaneous and traumatic bleeding events, monitoring of infusion product usage and home inventory, and real-time communication with health care professionals and hemophilia clinics. Telemedicine-supported education and information interventions seemed to be particularly effective among adolescent and young adult patients. Although the patients reported improvements in their health-related quality of life and perception of illness, telemonitoring devices did not appear to have a significant effect on quantifiable health outcomes such as joint health. Longitudinal studies seemed to suggest that the response and adherence rates to recording decreased over time. CONCLUSIONS Preliminary evidence from this review suggests that telehealth-delivered interventions could feasibly improve patients' adherence to medication use and promote independence in disease management. Given the complexity and resources involved in developing a mature and established system, support from a dedicated network of hemophilia specialists and data managers will be required to maintain the technology, improve adherence to prophylactic treatment and recording, and validate the electronic data locally.
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6.
Laser-Assisted Control of Epistaxis in Hereditary Hemorrhagic Telangiectasia: A Systematic Review
Abiri A, Goshtasbi K, Maducdoc M, Sahyouni R, Wang MB, Kuan EC
Lasers in surgery and medicine. 2019
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Abstract
BACKGROUND AND OBJECTIVES Hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu disease, causes recurrent mucous membrane hemorrhage, especially epistaxis. In this systematic review, we discuss the efficacies of the three most common laser photocoagulation treatments for HHT-related epistaxis. STUDY DESIGN/MATERIALS AND METHODS A systematic literature search was conducted in PubMed and MEDLINE from database inception to March 2019. Studies reporting epistaxis outcomes following argon, neodymium-doped yttrium aluminum garnet (Nd:YAG), and diode laser photocoagulation for HHT were included. chi(2) and Barnard's exact tests were utilized to detect differences in reduced epistaxis frequency and intensity rates. RESULTS Fifteen out of 157 published studies met our eligibility criteria, spanning a collective 362 patients. Argon, Nd:YAG, and diode laser therapy reduced epistaxis frequency in 90.4%, 88.9%, and 71.1% of patients, respectively, and reduced epistaxis intensity in 87.8%, 87.2%, and 71.1% of patients, respectively. Diode laser photocoagulation significantly underperformed in both outcome measurements when compared with argon (frequency: P = 0.005; intensity: P = 0.034) and Nd:YAG (frequency: P = 0.012; intensity: P = 0.041). There was no significant difference between argon and Nd:YAG in reducing HHT epistaxis frequency (P = 0.434) or intensity (P = 0.969). Categorizing HHT patients by clinical severity demonstrated a higher rate of improvement in the mild-moderate group compared with the severe group in both argon (P < 0.001) and Nd:YAG (P < 0.001) therapeutic methods. While no significant differences were found in rates of improved epistaxis outcomes between argon and Nd:YAG in mild-moderate HHT patients (frequency: P = 0.061; intensity: P = 0.061), Nd:YAG demonstrated greater rates of reduction in epistaxis frequency (P = 0.040) and intensity (P = 0.028) than argon among severe HHT patients. CONCLUSIONS HHT is a lifelong disease, plaguing patients with debilitating epistaxis. Intranasal laser photocoagulation of telangiectasias using argon or Nd:YAG laser therapy can yield improved epistaxis outcomes compared with diode laser photocoagulation. In severe cases of HHT, Nd:YAG laser therapy provides greater improvements in epistaxis outcomes than argon photocoagulation. Lasers Surg. Med. (c) 2019 Wiley Periodicals, Inc.
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Surgical Risk on Patients with Coagulopathies: Guidelines on Hemophiliac Patients for Oro-Maxillofacial Surgery
Laino L, Cicciu M, Fiorillo L, Crimi S, Bianchi A, Amoroso G, Monte IP, Herford AS, Cervino G
International journal of environmental research and public health. 2019;16(8)
Abstract
BACKGROUND Haemophilia is a disease of genetic origin, which causes a defect in blood coagulation. Under normal conditions, in the case of leakage from the blood vessels, the blood forms a clot that reduces or blocks the bleeding. This process involves the activation of several plasma proteins in a cascade-like species. Two of these proteins, produced in the liver, factor VIII and factor IX, are deficient or present a functional defect in people with haemophilia. Because of this deficit, the haemophiliacs easily suffer external and internal bleeding. Surgical treatment of these patients is to be observed, and often their treatment is delayed due to unclear guidelines and risks in treating these patients. The aim is to provide clear guidelines in the case of surgical treatment of these patients. METHODS In this study, we have considered all the guidelines that refer to the gold-maxillofacial surgery, focusing on the literature of the last 10 years. RESULTS Surely, this collection of guidelines will favor the choice of the clinician towards safer and predictable protocols. This study does not want to create a guideline but evaluates the literature of the last 10 years, and highlights the latest for the treatment of these patients., with the aim of informing the pathology and at the same time making the surgical maneuvers safer. CONCLUSIONS Despite the research of literature has produced few results, it was nevertheless possible to draw up a guideline thanks to additional information extrapolated from textbooks and other scientific articles. According to the guidelines, it is possible to proceed to the treatment of these patients, if with appropriate therapy in a safe and risk-free manner.
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Diagnosis and treatment of hereditary hemorrhagic telangiectasia
Grigg C, Anderson D, Earnshaw J
The Ochsner Journal. 2017;17((2)):157-161.
Abstract
BACKGROUND Hereditary hemorrhagic telangiectasia (HHT) is a rare genetic disorder known for its debilitating symptoms. More than 90% of patients with HHT experience epistaxis, and they average up to 18 bleeds per month. We review the current literature on the pathophysiology, clinical presentation, and management of HHT. METHODS We searched MEDLINE, EMBASE, and PubMed and identified 19 articles published since 2000 with current information on HHT. RESULTS HHT is a disease more commonly associated with significant morbidity rather than mortality. The morbidity of the disease and decreased quality of life are the result of the recurrent and potentially severe epistaxis that the majority of patients with HHT experience. During active epistaxis, the effective emergency techniques of locally applied pressure, nasal packing anteriorly and/or posteriorly, and cauterization are effective. Medical treatment with antiestrogen therapy has shown promising results, but further research is needed to determine the long-term side effects and the limitations of lifelong therapy. Research directed toward bleeding reduction and prevention has yet to have a breakthrough. Although initial reports suggest that intranasal bevacizumab is an effective agent, further research is required. CONCLUSION Interventional treatments in life-threatening and/or severe circumstances will continue to be used because of their effectiveness. Research into the pathophysiology of HHT has led to the development of potential therapies that prevent and decrease the severity of epistaxis, but the current evidence is insufficient to ascertain best practice. At present, appropriate management of acute epistaxis coupled with early diagnosis and referral to an ear, nose, and throat specialist should be the mainstay of treatment.
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9.
Splenectomy for people with thalassaemia major or intermedia
Easow MM, Sharma A, Aravindakshan R
The Cochrane Database of Systematic Reviews. 2016;((6)):CD010517.
Abstract
BACKGROUND Thalassaemia is a genetic disease of the haemoglobin protein in red blood cells. It is classified into thalassaemia minor, intermedia and major, depending on the severity of the disease and the genetic defect. Thalassaemia major and intermedia require frequent blood transfusions to compensate for the lack of well-functioning red blood cells, although this need is significantly less in thalassaemia intermedia.Damaged or defective red blood cells are normally eliminated in the spleen. In people with thalassaemia there is a large quantity of defective red blood cells which results in an enlarged hyperfunctioning spleen (splenomegaly). Removal of the spleen may thus prolong red blood cell survival by reducing the amount of red blood cells removed from circulation and may ultimately result in the reduced need for blood transfusions. OBJECTIVES To assess the efficacy and safety of splenectomy in people with beta-thalassaemia major or intermedia. SEARCH METHODS We searched the Cochrane Cystic Fibrosis and Genetic Disorders Review Group's Haemoglobinopathies Trials Register, compiled from searches of electronic databases and the handsearching of journals and conference abstract books. We also searched the reference lists of relevant articles and reviews.Date of the most recent search: 25 April 2016. SELECTION CRITERIA We included randomised controlled studies and quasi-randomised controlled studies of people of any age with thalassaemia major or intermedia, evaluating splenectomy in comparison to conservative treatment (transfusion therapy and iron chelation) or other forms of splenectomy compared to each other (laparoscopic, open, radio-frequency). DATA COLLECTION AND ANALYSIS Two authors independently selected and extracted data from the single included study using a customised data extraction form and assessed the risk of bias. MAIN RESULTS One study, including 28 participants was included in the review; the results were described, primarily, in a narrative manner. The study assessed the feasibility of splenectomy using laparoscopy in comparison to open surgery. Given the lack of detail regarding the study methods beyond randomisation, the overall risk of bias for this study was unclear. The study was carried out over a period of 3.5 years, with each participant followed up only until discharge (less than one week after the intervention); it did not assess the majority of the outcomes outlined in this review (including two of the three primary outcomes, frequency of transfusion and quality of life). A total of three serious post-operative adverse events (the review's third primary outcome) were reported in the laparoscopic splenectomy group (one case of atelectasis and two cases of bleeding), compared to two events of atelectasis in the open surgery group; however, there were no significant differences between the groups for either atelectasis, risk ratio 0.50 (95% confidence interval 0.05 to 4.90) or for bleeding, risk ratio 5.00 (95% confidence interval 0.26 to 95.61). In addition, the study also reported three serious cases of intra-operative bleeding in the laparoscopic group which mandated conversion to open surgery, although the difference between groups was not statistically significant, risk ratio 7.00 (95% confidence interval 0.39 to 124.14). These effect estimates are based on very small numbers and hence are unreliable and imprecise. From this small study, there appeared to be an advantage for the laparoscopic approach, in terms of post-operative hospital stay, although the group difference was not large (median difference of 1.5 days, P = 0.03). AUTHORS' CONCLUSIONS The review was unable to find good quality evidence, in the form of randomised controlled studies, regarding the efficacy of splenectomy for treating thalassaemia major or intermedia. The single included study provided little information about the efficacy of splenectomy, and compared open surgery and laparoscopic methods. Further studies need to evaluate the long-term effectiveness of splenectomy and the comparative advantag