Do anemia treatments improve quality of life and physical function in patients with myelodysplastic syndromes (MDS)? A systematic review.

Anemia is common in Myelodysplastic Syndromes (MDS). Different anemia treatments have been tested in clinical studies, but the full impact on patients' health-related quality of life (HRQoL) and physical function is unknown. The main aim of this review was to assess whether improvements in anemia are associated with changes in HRQoL/physical function. Twenty-six full-text publications were identified, enrolling 2211 patients: nine randomized trials (RCTs), fourteen non-randomized studies of interventions and three cross-sectional studies. Interventions included: growth factors/erythropoiesis-stimulating agents (n = 14), red cell transfusion (n = 9), erythroid maturation agents (n = 1), or a combination (n = 2). Five RCTs reported no changes in HRQoL despite erythroid response to the intervention, raising the question of whether anemia treatment alone can effectively improve HRQoL. Many studies were considered at high risk of bias for assessing HRQoL. There is a pressing need for future clinical trials to better define the nature of the relationship between anemia and HRQoL/functional outcomes.

The future of myelodysplastic syndrome-patient priorities and outcomes that matter.

Background: Without a definitive curative option available to many patients, learning to live with myelodysplastic syndrome (MDS) and manage symptoms effectively becomes a priority in their care. Anaemia is an almost universal feature of MDS. Individuals suffer differently and better individualisation of care is needed. Most MDS patient information offers scant appreciation for disease heterogeneity, variable response to treatment and each patient's likely trajectory. Methods: We undertook a two-part, online workshop to discuss what matters most to people living with MDS. Patients generated questions about their condition which they felt should be addressed by research or change how their care is delivered. Patients voted on the importance of each topic, creating a "prioritised" list of issues. Results: Fourteen participants of varying age and experience took part raising 56 unique questions under the themes of: prognosis; end of life; treatment; supportive care; medical staff training; diagnosis and communication. These reflect the symptoms of MDS, improving quality of life (QoL) and communication. Discussion: Although haemoglobin (Hb) levels have correlation to QoL, it is widely reported that other factors are important in determining QoL and need for transfusions varies despite stable Hb levels. We showed that Hb level and the need for transfusions is not comparable between different patients and even non-comparable over time meaning that the maximal benefit and timing of transfusions cannot be determined from Hb alone. This workshop highlighted patient dissatisfaction with the "numbers-led" approach and the need for an alternative method to determine when to transfuse.

The costs and benefits of water fluoridation in NZ.

BACKGROUND: Implementing community water fluoridation involves costs, but these need to be considered against the likely benefits. We aimed to assess the cost-benefit and cost-effectiveness of water fluoridation in New Zealand (NZ) in terms of expenditure and quality-adjusted life years. METHODS: Based on published studies, we determined the risk reduction effects of fluoridation, we quantified its health benefits using standardised dental indexes, and we calculated financial savings from averted treatment. We analysed NZ water supplies to estimate the financial costs of fluoridation. We devised a method to represent dental caries experience in quality-adjusted life years. RESULTS: Over 20 years, the net discounted saving from adding fluoride to reticulated water supplies supplying populations over 500 would be NZ$1401 million, a nine times pay-off. Between 8800 and 13,700 quality-adjusted life years would be gained. While fluoridating reticulated water supplies for large communities is cost-effective, it is unlikely to be so with populations smaller than 500. CONCLUSIONS: Community water fluoridation remains highly cost-effective for all but very small communities. The health benefits-while (on average) small per person-add up to a substantial reduction in the national disease burden across all ethnic and socioeconomic groups.

Prevention and diagnosis of invasive fungal disease in high-risk patients within an integrative care pathway.

OBJECTIVES: The aim of this study was to assess the clinical utility of enhanced diagnostics on the management of invasive fungal disease in high risk patients within an integrated care pathway and to audit compliance and efficacy of antifungal prophylaxis. METHODS: A cohort of 549 high risk haematology and stem-cell transplant recipients was followed over a 5 year period. The routine standard of care involved the use of antimould prophylaxis and a neutropenic care pathway utilizing twice weekly antigen and PCR testing. RESULTS: Prophylaxis with itraconazole was poorly tolerated and therapeutic levels could not be maintained. Antigen testing and PCR showed good clinical utility in the management of invasive aspergilosis with high sensitivity (98%) and negative predictive value (99.6%) when both tests were used together, allowing a diagnosis IA to be excluded and obviating the need for empirical antifungal agents. When used serially, multiple positive PCR and antigen test results enabled accurate diagnosis of IA with a specificity of 95% and a positive likelihood ratio of 11. Biomarkers preceded clinical signs in 85% of proven and probable invasive disease. CONCLUSION: The combination of both tests showed optimum clinical utility for the diagnosis and management of IA in this high risk group.

Do pharmaceutical score cards give us the answers we seek?

Few countries can afford to fund all pharmaceuticals for all of their people all of the time, and the current international economic climate brings this into clearer focus. Various agencies have tried to solve the problem in different ways, varying from funding a restricted list that applies to the whole population, to funding most medicines but with a significant part charge, or as in the United States, funding for only selected groups and leaving others to fend for themselves other than in an emergency. For countries like New Zealand and Australia who have universal health coverage but restricted (and different) lists of funded pharmaceuticals, comparisons of those lists can occur, but are problematic. Comparisons need to be interpreted with caution as systems and policies vary between countries. That one country funds more new medicines than the other is one thing, but the more important questions are whether one country gets more health gains and more value for precious health dollars than the other.