Burden of illness among patients with severe aplastic anemia who have had insufficient response to immunosuppressive therapy: a multicenter retrospective chart review study.

This study assessed treatment patterns and healthcare resource utilization (HRU) of patients with severe aplastic anemia (SAA) with insufficient response to immunosuppressive therapy (IST). A retrospective chart review was conducted at Dana-Farber Cancer Institute (DFCI), United States, and Hôpital Saint-Louis (HSL), France. Eligible patients were ≥ 18 years old, diagnosed with acquired SAA between January 1, 2006, and July 31, 2016, had insufficient response to IST, and had ≥ 12 months of follow-up post-diagnosis. Overall survival (OS) was estimated using the Kaplan-Meier method. Among the 40 patients, mean age at diagnosis was 44 years and 53% were women. Median follow-up time after SAA diagnosis was 48.3 months. Ninety-five percent of patients received antithymocyte globulin (ATG) as primary therapy prior to hematopoietic stem cell transplant (HSCT). Most common secondary SAA therapies prior to HSCT were eltrombopag (28%) and androgens (15%). Seventy-five percent of patients received HSCT. Prior to HSCT, patients received an average of 2.7 red blood cell (RBC) and 3.3 platelet transfusions per month; patients had 0.9 hospitalizations, 0.4 emergency room visits, and 12.8 office visits per year. Five-year OS was 75%, with infection as the primary cause of death. Additionally, this study provides information on the subgroup of patients receiving eltrombopag which was the most common secondary therapy. This study quantified transfusion and HRU burden associated with SAA and demonstrated high 5-year survival in a recently treated cohort.

Healthcare resource use and direct costs in severe aplastic anemia (SAA) patients before and after treatment with eltrombopag.

Purpose: This study evaluated healthcare resource utilization (HCRU), and direct costs among severe aplastic anemia (SAA) patients treated with eltrombopag (EPAG) using US claims data.Methods: This retrospective, real-world claims database study identified SAA patients aged ≥2 years treated with EPAG who initiated any SAA treatment between 1 July 2014 and 31 December 2017 (identification period) using the Truven MarketScan databases. A subset of 82 patients treated with EPAG during the identification period were evaluated for all-cause and SAA-related HCRU and direct costs as well as blood transfusion 1 month before EPAG initiation (baseline) and at Month 6 after EPAG initiation (follow-up period).Results: The average patient age was 50.8 (SD = 20.6) years old, predominantly female (n = 43, 52.4%), and had a mean CCI at baseline of 1.1 (SD = 1.7). Hospitalizations, and ER, office, and outpatient visits were significantly lower at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (p < .05 for all four all-cause HCRU and SAA-related hospitalizations). An almost two-fold decrease in reliance on biweekly blood transfusions was observed: 1.0 at weeks 1-2 to 0.5 at Month 6 after EPAG initiation. Although prescription costs (mean [SD]) were significantly higher at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (difference of $11,045 USD [SD = $18,801]), these increases were offset by savings in direct costs. Overall, a mean reduction in total all-cause costs of $29,391 USD [SD = $137,770] was reported at Month 6 after EPAG initiation due to substantial reductions in hospitalization ($40,060 USD [SD = $123,198]) and outpatient visits ($2,043 USD [SD = $25,264]).Conclusion: All-cause and SAA-related HCRU were reduced following EPAG treatment. Prescription costs were higher following treatment; however, these costs were generally offset by reductions in direct costs. These results provide real-world evidence around the role of EPAG in SAA treatment.

Healthcare costs and resource utilization in patients with severe aplastic anemia in the US.

Purpose: This study aimed to evaluate the healthcare resource utilization (HCRU) and costs for patients with severe aplastic anemia (SAA) using US claims data. Methods: This retrospective, observational database study analyzed claims data from the Truven MarketScan databases. SAA patients aged ≥2 years identified between 2014 and 2017 who were continuously enrolled for 6 months before their first SAA treatment or blood transfusion, with a ≥6-month follow-up, were included. Baseline demographics and comorbidities were evaluated. Monthly all-cause and SAA-related HCRU and direct costs in the follow-up period were analyzed and differences were presented for all patients and across age groups. Results: With an average follow-up period of 21.5 months, 939 patients were included in the study. Monthly all-cause and SAA-related HCRU [mean (SD)] were 1.65 days (2.61 days) and 0.18 days (0.70 days) for length of stay, 0.18 (0.23) and 0.01 (0.04) for hospital admissions, 0.25 (0.30) and 0.02 (0.07) for ER visits, 2.24 (1.40) and 0.46 (0.99) for office visits, and 2.90 (2.64) and 0.55 (1.31) for outpatient visits, respectively. On average, SAA patients received 0.15 (0.57) blood transfusions per month. Mean monthly all-cause direct costs were $28,280 USD ($36,127) [US dollars, mean (SD)]. Direct costs related to admissions were $11,433 USD (SD $25,040), followed by $624 USD ($1,703) for ER visits, $528 USD ($694) for office visits, $7,615 USD ($13,273) for outpatient visits, and $5,998 USD ($11,461) for pharmacy expenses. Monthly SAA-related direct costs averaged $7,884 USD (SD $16,254); of these costs, $1,608 USD ($7,774) were from admissions, $47 USD ($257) from ER visits, $127 USD ($374) from office visits, $1,462 USD ($4,994) from outpatient visits, and $4,451 USD ($10,552) from pharmacy expenses. Conclusion: SAA is associated with high economic burden, with costs comparable to blood malignancies, implying that US health plans should consider appropriately managing SAA while constraining the total healthcare costs when making formulary decisions.

Patient features and survival of pediatric aplastic anemia in the USA: a large institution experience.

BACKGROUND: We performed the first epidemiologic investigation to examine association of demographics and clinical characteristics at diagnosis, as well as health care expense coverage, with survival of US children with aplastic anemia (AA). METHODS: We obtained electronic medical record data of 1140 children aged 0-19 years diagnosed with AA followed at a pediatric health system between 2004 and 2014. Kaplan-Meier curve and Cox proportional hazards regressions were used. RESULTS: Self-pay patients had a mortality risk five times higher than that of those insured by publicly funded insurance (hazards ratio, 95% CI: 6.0, 3.7-9.8). Other features associated with higher mortality risk include pancytopenia (hazards ratio, referent: 4.2, constitutional AA); underweight (2.0, normal-weight); platelet count <50 × 109/l (1.3, ≥50 × 109/l); male sex (1.3, female); and ages at diagnosis 6-11, 11-16 and 16-19 years (1.6, 1.9, 2.3, 1-3 years), respectively. CONCLUSIONS: Self-pay was the strongest prognostic factor for pediatric AA mortality. Older age, pancytopenia, underweight, male sex and lower platelet count were also associated with increased risk of mortality. These findings may be useful for providers, researchers and policymakers to ensure effective health care delivery to this population and to motivate future etiologic research and establishment of a surveillance registry.

Modelling cost effectiveness of horse antithymocyte globulin for treating severe aplastic anaemia in Germany.

Acquired severe aplastic anaemia (AA) is a serious condition caused by immune-triggered bone marrow failure. For patients not eligible for bone marrow transplantation, treatment of choice is immunosuppression by a combined treatment with antithymocyte globulin (ATG) and cyclosporine. The debate on treatment optimization in AA is focused on conflicting data regarding ATG preparations from horse (h-ATG) versus rabbit (r-ATG), recently favouring h-ATG. H-ATG has been withdrawn from the European market in 2007. Reimbursement for imported preparations from outside Europe is frequently denied in negotiations with statutory health insurance companies. This raises the question of whether h-ATG is cost effective and a sensible investment with regard to healthcare budgets as well as patient health. We modelled the cost effectiveness of r-ATG versus h-ATG based on a recent randomized trial and cost data provided by the hospital pharmacy of Jena University Hospital. We calculated the amount of life years gained and the average incremental costs per life year gained when comparing h-ATG and r-ATG. Our calculations revealed average incremental costs per life year gained of 11,033.80 for the examined patient population treated with h-ATG when compared to r-ATG. Assuming a cost effectiveness threshold of 25,000-35,000 per life year gained, our calculations demonstrate cost effectiveness of h-ATG as compared to r-ATG.

[rHU-EPO in cancer patients. Target or wider use?]. / rHu-EPO chez les patients cancéreux. Utilisation ciblée ou plus large?

THE DEBATE: Although some believe that rHu-EPO should not be widely used in malignant affections, others think that because of the varied impact of these anaemia, its wider use should be recommended. FOR A TARGET USE: Various observations (influence of the degree of extension of the myelomas to the skeleton and response to specific treatment in the case of myeloma, whatever the haemoglobin concentration, degree of prevention of rHu-EPO chemo-induced anaemia) are in favour of its use in selected patients. FOR A WIDER USE: The benefits of treatment with rHu-EPO are not limited to the symptomatology of anaemia but extend to its potential complications in the most fragile patients. Other than the risks of infection, the heavy costs of transfusions must also be taken into account.

New strategies for prophylactic platelet transfusion in patients with hematologic diseases.

There is an increasing demand for platelet transfusions due to intensive chemotherapy and blood stem cell or bone marrow transplantation for the treatment of hematologic and oncologic diseases. There has been a long-lasting debate over whether the traditional threshold for prophylactic platelet transfusion of 20,000/microl is really necessary to prevent hemorrhagic complications. During the last 10 years several studies with more than 1,000 patients together have proven the safety of a platelet transfusion trigger of 10,000/microl or even lower when patients are clinically stable without active bleeding. This experience has been mostly gathered in patients with acute leukemia. But this stringent platelet transfusion policy can be used also after blood stem cell and bone marrow transplantation. In stable patients with aplastic anemia and myelodysplasia, prophylactic transfusions should be replaced in most patients by a therapeutic transfusion strategy. Such restrictive platelet transfusion strategies decrease the risk of infectious disease transmission, immunization, and febrile transfusion reactions. Besides reduced hospital visits and a shorter hospital stay for the patients, the costs for platelet transfusions are lowered by 20%-30% compared with traditional transfusion strategies. The decision to administer platelet transfusions should incorporate individual clinical characteristics of the patients and not simply be a reflexive reaction to the platelet count. Further clinical studies are needed to answer the still open question of whether patients with acute leukemia should also be transfused therapeutically rather than prophylactically when they are in stable condition without signs of active bleeding.

Antilymphocyte globulin and high-dose methylprednisolone improve survival in patients with aplastic anaemia without additional financial costs.

The cost-benefit ratio in the treatment of aplastic anaemia with antilymphocyte globulin (ALG) combined with high-dose methylprednisolone (HDMP) was retrospectively compared with supportive palliative treatment alone. Over a 4-year period financial cost, response rate, survival and performance status was documented in 26 consecutive patients receiving this regimen. Outcome was favourable in 69% (group 1; N = 18) and in 13 comprehensive expenditure data were available. In the remainder (group 2; N = 8) treatment failed and they were considered to be the equivalent of a no-treatment population. Here hospital charges were analysed both with and without inclusion of ALG and HDMP. Over the 1-year study period, no significant difference in the median expenditure was found between group 1 at R19 281 (range R35 657-13 379) and group 2 at R18 522 (range R22 449-16 951). The median number of admissions for group 1 was one and for group 2 three, requiring a median of 19 and 20 days of hospitalisation respectively. At the end of the 1-year study period 19 of 26 patients were alive (73%), 69% having responded. At this time, 16 of the 18 patients in group 1 had returned to their previous occupations, but this had not been possible for any of the 8 in group 2. In suitably selected patients who do not have an allogenic bone marrow transplant option the expenditure on ALG and HDMP is both medically and economically sound and the combination can be recommended as a valuable treatment option.

Cost of allogeneic bone marrow transplantation in four diseases.

The cost of bone-marrow transplantation is compared in 4 diseases: acute myelogenous leukaemia, severe combined immunodeficiency, severe aplastic anaemia and chronic granulocytic leukaemia. Hospital cost components directly related to the clinical protocols applied are valorized. Results confirm the well-known fact that bone-marrow transplantation is a costly technique. The unit cost of a transplantation can vary from 1 to 2 between departments for the sole reason that patients treated are not suffering from the same illness. For one disease, the unit cost may vary from 1 to 2.7 when post-graft complications arise. Furthermore, in the health-care sector, as well as in every other economic sector, costs do not remain stable: they vary in time most especially when treatment protocols evolve. This type of cost information is the basis for management control systems without which physicians, hospital managers and health-care authorities cannot communicate effectively. In countries where health care is largely financed by the community, what is at stake is the future of advanced technologies in medicine.