Florian S. Gutzwiller

FERGIcor, a Randomized Controlled Trial on Ferric Carboxymaltose for Iron Deficiency Anemia in Inflammatory Bowel Disease

BACKGROUND & AIMS Iron deficiency anemia (IDA) is common in chronic diseases and intravenous iron is an effective and recommended treatment. However, dose calculations and inconvenient administration may affect compliance and efficacy. We compared the efficacy and safety of a novel fixed-dose ferric carboxymaltose regimen (FCM) with individually calculated iron sucrose (IS) doses in patients with inflammatory bowel disease (IBD) and IDA. METHODS This randomized, controlled, open-label, multicenter study included 485 patients with IDA (ferritin <100 μg/L, hemoglobin [Hb] 7-12 g/dL [female] or 7-13 g/dL [male]) and mild-to-moderate or quiescent IBD at 88 hospitals and clinics in 14 countries. Patients received either FCM in a maximum of 3 infusions of 1000 or 500 mg iron, or Ganzoni-calculated IS dosages in up to 11 infusions of 200 mg iron. Primary end point was Hb response (Hb increase ≥ 2 g/dL); secondary end points included anemia resolution and iron status normalization by week 12. RESULTS The results of 240 FCM-treated and 235 IS-treated patients were analyzed. More patients with FCM than IS achieved Hb response (150 [65.8%] vs 118 [53.6%]; 12.2% difference, P = .004) or Hb normalization (166 [72.8%] vs 136 [61.8%]; 11.0% difference, P = .015). Both treatments improved quality of life scores by week 12. Study drugs were well tolerated and drug-related adverse events were in line with drug-specific clinical experience. Deviations from scheduled total iron dosages were more frequent in the IS group. CONCLUSIONS The simpler FCM-based dosing regimen showed better efficacy and compliance, as well as a good safety profile, compared with the Ganzoni-calculated IS dose regimen.

Health economic assessment of ferric carboxymaltose in patients with iron deficiency and chronic heart failure based on the FAIR-HF trial: an analysis for the UK

The purpose of this study was to evaluate the cost‐effectiveness of iron repletion using intravenous (i.v.) ferric carboxymaltose (FCM) in chronic heart failure (CHF) patients with iron deficiency with or without anaemia. Cost‐effectiveness was studied from the perspective of the National Health Service in the UK.

Determinants of quality of life of patients with heart failure and iron deficiency treated with ferric carboxymaltose : FAIR-HF sub-analysis

BACKGROUND Heart failure (HF) is a burden to patients and health care systems. The objectives of HF treatment are to improve health related quality of life (HRQoL) and reduce mortality and morbidity. We aimed to evaluate determinants of health-related quality of life (HRQoL) in patients with iron deficiency and HF treated with intravenous (i.v.) iron substitution or placebo. METHODS A randomised, double-blind, placebo-controlled trial (n = 459) in iron-deficient chronic heart failure (CHF) patients with or without anaemia studied clinical and HRQoL benefits of i.v. iron substitution using ferric carboxymaltose (FCM) over a 24-week trial period. Multivariate analysis was carried out with various clinical variables as independent variables and HRQoL measures as dependent variables. RESULTS Mean change from baseline of European Quality of Life - 5 Dimensions (EQ-5D) (value set-based) utilities (on a 0 to 100 scale) at week 24 was 8.91 (i.v. iron) and 0.68 (placebo; p < 0.01). In a multivariate analysis excluding baseline HRQoL, a higher exercise tolerance and i.v. iron substitution positively influenced HRQoL, whereas impaired renal function and a history of stroke had a negative effect. The level of HRQoL was also influenced by country of residence. When baseline HRQoL was factored in, the multivariate model remained stable. CONCLUSION In this study, i.v. iron substitution, exercise tolerance, stroke, country of residence and renal function influenced measures of HRQoL in patients with heart failure and iron deficiency.

A Cost-effectiveness Analysis of Ferric Carboxymaltose in Patients With Iron Deficiency and Chronic Heart Failure in Spain

INTRODUCTION AND OBJECTIVES Treatment with ferric carboxymaltose improves symptoms, functional capacity, and quality of life in patients with chronic heart failure and iron deficiency. The aim of this study was to assess the cost-effectiveness of ferric carboxymaltose treatment vs no treatment in these patients. METHODS We used an economic model based on the Spanish National Health System, with a time horizon of 24 weeks. Patient characteristics and ferric carboxymaltose effectiveness (quality-adjusted life years) were taken from the Ferinject® Assessment in patients with IRon deficiency and chronic Heart Failure trial. Health care resource use and unit costs were taken either from Spanish sources, or from the above mentioned trial. RESULTS In the base case analysis, patients treated with and without ferric carboxymaltose treatment acquired 0.335 and 0.298 quality-adjusted life years, respectively, representing a gain of 0.037 quality-adjusted life years for each treated patient. The cost per patient was €824.17 and €597.59, respectively, resulting in an additional cost of €226.58 for each treated patient. The cost of gaining 1 quality adjusted life year with ferric carboxymaltose was €6123.78. Sensitivity analyses confirmed the robustness of the model. The probability of ferric carboxymaltose being cost-effective (< €30 000 per quality-adjusted life year) and dominant (more effective and lower cost than no treatment) was 93.0% and 6.6%, respectively. CONCLUSIONS Treatment with ferric carboxymaltose in patients with chronic heart failure and iron deficiency, with or without anemia, is cost-effective in Spain.

Methoden zur Bestimmung von Nutzen bzw. Wert medizinischer Leistungen und ihre ethischen Implikationen

1 Institut für Pharmazeutische Medizin (ECPM), Universität Basel, Schweiz 2 Institut für Sozialund Präventivmedizin, Universität Zürich, Schweiz 3 Institut für Biomedizinische Ethik, Universität Zürich, Schweiz Der Ausbau der Gesundheitssysteme führte in allen Industrieländern zu einer Verbesserung der Gesundheitsversorgung, aber auch zu einem deutlichen Kostenanstieg. Verfügbare Mittel müssen deshalb effizient eingesetzt und der Nutzen von medizinischen Leistungen muss bestimmt und bewertet werden, damit Entscheide über deren Einsatz transparent und fundiert gefällt werden können. Methoden der Nutzenbestimmung und -bewertung, die im Gesundheitswesen zum Einsatz kommen, können in klinisch-epidemiologische, gesundheitsökonomische, rein ökonomische und integrative Methoden (health technology assessment, HTA) unterteilt werden. Alle diese Methoden haben ethische Implikationen, die in der Anwendung zu berücksichtigen sind. Angesichts der aktuellen Kostenentwicklung sollten die Potentiale eines modernen und breit abgestützten HTA verstärkt genutzt werden.