Cost-effectiveness of eltrombopag vs intravenous immunoglobulin for the perioperative management of immune thrombocytopenia.

Eltrombopag has been shown to be noninferior to intravenous immunoglobulin (IVIG) for improving perioperative platelet counts in patients with immune thrombocytopenia (ITP) in a randomized trial; thus, cost is an important factor for treatment and policy decisions. We used patient-level data from the trial to conduct a cost-effectiveness analysis comparing perioperative eltrombopag 50 mg daily starting dose, with IVIG 1 or 2 g/kg (according to local practice) from a Canadian public health care payer's perspective over the observation period, from preoperative day 21 to postoperative day 28. Resource utilization data were obtained from the trial data (eltrombopag, n = 38; IVIG, n = 36), and unit costs were collected from the Ontario Schedule of Benefits, Ontario Drug Formulary, and secondary sources. All costs were adjusted to 2020 Canadian dollars. We calculated the incremental cost per patient for all patients randomized. Uncertainty was addressed using nonparametric bootstrapping. The use of perioperative eltrombopag for patients with ITP resulted in a cost-saving of $413 Canadian per patient. Compared with IVIG, the probability of eltrombopag being cost effective was 70% even with no willingness to pay. In a sensitivity analysis based on IVIG dose, we found that with the higher dose of IVIG (2 g/kg), eltrombopag saved $2,714 per patient, whereas with the lower dose of IVIG (1 g/kg), eltrombopag had a higher mean cost of $562 per patient. In summary, based on data from the randomized trial that demonstrated noninferiority, the use of eltrombopag for the management of ITP in the perioperative setting was less costly than IVIG.

Clinical Studies of Ex Vivo Expansion to Accelerate Engraftment After Umbilical Cord Blood Transplantation: A Systematic Review.

Cell dose limits greater use of umbilical cord blood (UCB) in hematopoietic cell transplantation. The clinical benefits of ex vivo expansion need clarity to understand its potential impact. A systematic search of studies addressing UCB ex vivo expansion was conducted. Fifteen clinical studies (349 transplanted patients) and 13 registered trials were identified. The co-infusion of an expanded unit and a second unmanipulated unit (8 studies), the fractional expansion of 12% to 60% of a single unit (5 studies), and the infusion of a single expanded unit (2 studies) were reported. More recently, published studies and 12 of 13 ongoing trials involve the use of novel small molecules in addition to traditional cytokine cocktails. Higher total cell number was closely associated with faster neutrophil engraftment. Compared with historical controls, neutrophil engraftment was significantly accelerated in more recent studies using small molecules or mesenchymal stromal cells (MSC) co-culture, and in some cases, platelet recovery was also statistically improved. Recent studies using nicotinamide and StemRegenin-1 reported long-term chimerism of the expanded unit. No significant improvement in survival or other transplant-related outcomes was demonstrated for any of the strategies. Ex vivo expansion of UCB can accelerate initial neutrophil engraftment after transplant. More recent studies suggest that long-term engraftment of ex vivo expanded cord blood units is achievable. Results of larger randomized controlled trials are needed to understand the impact on patient outcomes and health care costs.

BEST-TEST2: assessment of hematology trainee knowledge of transfusion medicine.

BACKGROUND: As transfusion is a common therapy and key component in every hematologist's practice, hematology training programs should dedicate significant time and effort to delivering high-quality transfusion medicine education to their trainees. The current state of hematology trainee knowledge of transfusion medicine is not known. STUDY DESIGN AND METHODS: A validated assessment tool developed by the Biomedical Excellence for Safer Transfusion (BEST) Collaborative was used to assess prior transfusion medicine education, attitudes, perceived ability, and transfusion medicine knowledge of hematology trainees. RESULTS: A total of 149 hematology trainees at 17 international sites were assessed. The overall mean exam score was 61.6% (standard deviation, 13.4%; range, 30%-100%) with no correlation in exam scores with postgraduate year or previous transfusion medicine education in medical school or internal medicine residency. However, better scores correlated with 3 or more hours of transfusion medicine education (p = 0.0003) and perceived higher-quality education during hematology training (p = 0.03). Hematology trainees at US sites, where hematology is often combined with oncology training, had statistically lower scores than trainees at non-US sites (56.2% vs. 67.4%; p < 0.0001). In terms of topic areas, although 93% of participants had obtained consent for transfusion, the lowest scores were on transfusion reaction-related questions. CONCLUSION: Given the overall poor performance, this study serves as an impetus for all hematology training programs to reevaluate the quality and quantity of transfusion medicine training and can assist in the development of targeted curricula.

Internal medicine resident knowledge of transfusion medicine: results from the BEST-TEST international education needs assessment.

BACKGROUND: Blood transfusion is the most common hospital procedure performed in the United States. While inadequate physician transfusion medicine knowledge may lead to inappropriate practice, such an educational deficit has not been investigated on an international scale using a validated assessment tool. Identifying specific deficiencies is critical for developing curricula to improve patient care. STUDY DESIGN AND METHODS: Rasch analysis, a method used in high-stakes testing, was used to validate an assessment tool consisting of a 23-question survey and a 20-question examination. The assessment tool was administered to internal medicine residents to determine prior training, attitudes, perceived ability, and actual knowledge related to transfusion medicine. RESULTS: A total of 474 residents at 23 programs in nine countries completed the examination. The overall mean score of correct responses was 45.7% (site range, 32%-56%). The mean score for Postgraduate Year (PGY)1 (43.9%) was significantly lower than for PGY3 (47.1%) and PGY4 (50.6%) residents. Although 89% of residents had participated in obtaining informed consent from a patient for transfusion, residents scored poorly (<25% correct) on questions related to transfusion reactions. The majority of residents (65%) would find additional transfusion medicine training "very" or "extremely" helpful. CONCLUSION: Internationally, internal medicine residents have poor transfusion medicine knowledge and would welcome additional training. The especially limited knowledge of transfusion reactions suggests an initial area for focused training. This study not only represents the largest international assessment of transfusion medicine knowledge, but also serves as a model for rigorous, collaborative research in medical education.

The societal unit cost of allogenic red blood cells and red blood cell transfusion in Canada.

BACKGROUND: There is a dearth of information about the cost of allogenic red blood cells (RBCs) and RBC transfusion in Canada in the aftermath of the Canadian blood system reorganization and the introduction of various safety measures. The unit cost of allogenic RBCs and RBC transfusion in Canada in 1994 was estimated at 152.17 US dollars. The objective of this study was to determine the unit cost of allogenic RBC transfusion in Canada from a societal perspective. STUDY DESIGN AND METHODS: A cost-structure analysis using the cost information from 2001 through 2002 was used. Costs of blood collection, production, distribution, delivery (hospital transfusion service processing and patient administration), transfusion reaction management, and opportunity cost of donor's time were included in the analysis. Canadian Blood Services and Héma-Québec supplied the data for collection, production, and distribution stages. Delivery and transfusion reaction costs were collected from eight hospitals across six Canadian provinces. In-patient costs were assessed for the intensive care unit, emergency, general medicine ward, and operating room. RESULTS: The aggregate mean societal unit cost of RBCs transfused on an inpatient basis in 2002 was 264.81 US dollars (95% confidence interval [CI], 256.29 dollars-275.65 dollars). The mean cost of blood collection, production, and distribution was 202.74 US dollars (95% CI, 199.63 dollars-204.31 dollars), the mean opportunity cost of donor time was 18.21 US dollars (95% CI, 17.11 dollars-21.63 dollars), the mean cost of hospital transfusion service processing was 16.65 US dollars (95% CI, 13.50 dollars-19.79 dollars), of RBC transfusion was 26.92 US dollars (95% CI, 25.33 dollars-28.52 dollars), and of transfusion reaction management was 0.29 US dollars(95% CI, 0.22 dollars-0.36 dollars). There were substantial variations in hospital transfusion service processing and RBC transfusion costs across hospitals. CONCLUSION: The societal unit cost of RBC transfusion has doubled since 1994 to 1995. Further increases in unit costs would be expected as additional safety measures are introduced. This will have important financial implications for treating patient populations that require a high level of RBC transfusions.

Does a perception of increased blood safety mean increased blood transfusion? An assessment of the risk compensation theory in Canada.

BACKGROUND: The risk compensation theory is a widely used concept in transport economics to analyze driver risk behaviour. This article explores the feasibility of applying the theory in blood transfusion to raise important questions regarding the increased blood safety measures and their possible effects on blood usage (e.g., the appropriateness in transfusion). Further, it presents the findings of a pilot survey of physicians in Canada. DISCUSSION: While studies have attempted to define transfusion appropriateness, this article argues that if the risk compensation theory holds true for transfusion practice, physicians may actually be transfusing more. This may increase the possibility of contracting other unknown risks, such as the variant Creutzfeldt-Jakob Disease (vCJD), as well as increasing the risk of non-infectious transfusion risks, such as transfusion reactions. SUMMARY: A much larger study involving psychosocial assessment of physician decision making process to fully assess physician behaviour within the context of risk compensation theory and transfusion practice in Canada is needed to further explore this area.