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%).
2.
Reported Outcomes in Perinatal Iron Deficiency Anemia Trials: A Systematic Review
Malinowski AK, D'Souza R, Khan KS, Shehata N, Malinowski M, Daru J
Gynecologic and obstetric investigation. 2019;:1-18
Abstract
BACKGROUND/AIMS: Iron deficiency (ID) and iron deficiency anemia (IDA) are global health concerns associated with adverse perinatal effects. Despite efforts taken at the international level, there is no consensus on unified prevention/treatment strategies, largely stemming from inconsistencies of outcome reporting. Our objective was to comprehensively assess outcome reporting perinatal iron intervention trials as Phase 1 of core outcome set (COS) development to inform future research. METHODS Systematic search in MEDLINE, EMBASE, Cochrane Databases, and CINAHL (January 2000 - April 2016), with inclusion of trials involving pregnant or postpartum women with ID/IDA confirmed before recruitment. Articles were independently screened and selected by 2 reviewers; data were extracted in duplicate. Quality was assessed using published scoring systems. Outcome definitions and measurement methods were tabulated. RESULTS Of 7,046 citations, 33 randomized controlled trials were included. Sixty-nine reported outcomes were categorized into 8 domains. High methodological quality characterized 25 (76%) studies. Reporting quality was low in 16 (49%), moderate in 13 (39%), and high in 4 (12%) studies. Variation was greatest for outcome definition, timing of assessment and measurement methods. CONCLUSION This review identifies a comprehensive long-list of outcomes reported of perinatal iron interventions for ID/IDA. Beyond highlighting existing variation in outcome reporting, it provides a foundation for development of a COS for future trials.
3.
Efficacy and safety of erythropoietin and iron therapy to reduce red blood cell transfusion in surgical patients: a systematic review and meta-analysis
Kei T, Mistry N, Curley G, Pavenski K, Shehata N, Tanzini RM, Gauthier MF, Thorpe K, Schweizer TA, Ward S, et al
Canadian journal of anaesthesia = Journal canadien d'anesthesie. 2019
Abstract
PURPOSE Iron restricted anemia is prevalent in surgical patients and is associated with an increased risk of allogeneic red blood cell (RBC) transfusion and adverse events. Treatment of anemia includes oral and intravenous iron and erythropoiesis stimulating agents (ESAs). More recent studies have focused on the use of intravenous iron as the primary approach to treating anemia. Nevertheless, the optimal treatment strategy for anemia remains to be established. Our primary objective was to evaluate the efficacy and safety of ESA and iron therapy relative to iron therapy alone in reducing RBC transfusion in surgical patients. SOURCE We searched the Cochrane Library, MEDLINE, EMBASE, and ClinicalTrials.gov from inception to May 2018. We included randomized-controlled trials in which adult surgical patients received an ESA and iron, vs iron alone, prior to cardiac and non-cardiac surgery. Our primary outcome was RBC transfusion rate. Secondary outcomes included hemoglobin concentration (post-treatment and postoperatively), number of RBC units transfused, mortality, stroke, myocardial infarction (MI), renal dysfunction, pulmonary embolism (PE), and deep vein thrombosis (DVT). PRINCIPAL FINDINGS In total, 25 studies (4,719 participants) were included. Erythropoiesis stimulating agents and iron therapy reduced RBC transfusion relative to iron therapy (relative risk [RR] 0.57; 95% confidence interval [CI], 0.46 to 0.71) without any change in mortality (RR 1.31; 95% CI, 0.80 to 2.16), stroke (RR 1.91; 95% CI, 0.63 to 5.76), MI (RR 1.12; 95% CI, 0.50 to 2.50), renal dysfunction (RR 0.96; 95% CI, 0.72 to 1.26), PE (RR 0.92; 95% CI, 0.15 to 5.83), or DVT (RR 1.48; 95% CI, 0.95 to 2.31). CONCLUSION Administration of ESA and iron therapy reduced the risk for RBC transfusion compared with iron therapy alone in patients undergoing cardiac and non-cardiac surgery. Nevertheless, publication bias and heterogeneity reduces the confidence of the finding. Although the analysis was probably under-powered for some outcomes, no difference in the incidence of serious adverse events was observed with ESA and iron compared with iron alone. Further large prospective trials are required to confirm these findings.
4.
Efficacy and Safety of Erythropoietin and Iron Therapy to Reduce Red Blood Cell Transfusion in Surgical Patients: A Systematic Review and Meta-Analysis
Kei T, Curley G, Pavenski K, Ward S, Shehata N, Tanzini R, Gauthier M-F, Mistry N, Mazer CD, Hare G
Transfusion. 2017;57((53)):114A.. cp-124
5.
The use of erythropoiesis-stimulating agents in patients with non-myeloid hematological malignancies: a systematic review
Shehata N, Walker I, Meyer R, Haynes AE, Imrie K, The Cancer Care Ontario Hematology Disease Site Group
Annals of Hematology. 2008;87((12):):961-73.
Abstract
The effectiveness of erythropoiesis-stimulating agents (ESAs) for the treatment of anemia in patients with non-myeloid hematological malignancies needs to be assessed as the response to their administration is not uniform and their cost is high. We conducted a systematic review (SR) of the literature to identify reports of the effect of ESAs on survival, quality of life (QOL), transfusion requirements, and anemia. The entries to MEDLINE, EMBASE, and the Cochrane Library databases, and abstracts published in the proceedings of the annual meetings of the American Society of Clinical Oncology and the American Society of Hematology were searched. Seventeen reports and five abstracts of randomized trials fulfilled prospective criteria for inclusion. Five trials reported on survival; three failed to detect differences between groups and two demonstrated inferior survival in patients allocated to an ESA. Seven trials and three abstracts reported on QOL with four articles and three abstracts describing improvements in patients allocated to erythropoietin. However, important methodologic limitations were identified in these reports. Seven randomized controlled trials reported a reduction in the proportion of patients transfused. The absolute risk reduction in transfusions ranged from 15% to 24%. This is the only SR that assesses the use of erythropoiesis-stimulating agents specifically in patients with hematological malignancies. We conclude that available data evaluating ESAs in patients with hematologic malignancies demonstrate that these agents reduce transfusion requirements. Limitations of these data preclude conclusions that these agents improve QOL. More data are required to confirm the inferior survival associated with ESAs.