BackgroundRandomized controlled trials are the standard for health technology assessment, but when they are infeasible or unethical, single-arm trials (SATs) are submitted.ObjectivesThis study examined when SATs were accepted for added benefit by the Institute for Quality and Efficiency in Health Care (IQWiG) and/or the Federal Joint Committee (G-BA) in Germany.MethodsWe identified health technology assessments via the AMNOG-Monitor database through December 2024, with additional details from G-BA documents. We compared the SATs and other evidence for added benefit decisions (granted/not granted), stratified by orphan drug status, special marketing authorization, approved indication (chronic hepatitis C/others), and population (adults/children). Added benefit claims by manufacturers, IQWiG recommendations, and G-BA appraisals were compared.ResultsAmong 1738 G-BA decisions, 85.8% (1491/1738) of the subpopulations were fully assessed by IQWiG, with 13.5% (202/1491) based on SATs. Among the 247 orphan drugs assessed by the G-BA, 37.7% (93/247) were SAT-based. Overall, SAT-based assessments demonstrated an added benefit in 12.2% (36/295) of cases. This included 13.4% (27/202) of full assessments and 9.7% (9/93) of orphan drug assessments. IQWiG accepted only 18.5% (5/27) of the SATs endorsed by the G-BA. Statistical tests revealed significant differences between manufacturers' claims, IQWiG recommendations, and G-BA appraisals. SATs were most frequently accepted for chronic hepatitis C treatments (mostly with non-standard marketing authorization) and paediatric indications. The G-BA cited reasons such as dramatic effects, rare diseases, a lack of alternatives, or fewer side effects, although justifications were often unclear.ConclusionAcceptance rates for SATs remain low, and criteria for added benefit are not always explicitly defined. To enable benefit assessments when randomised controlled trials are infeasible or unethical, clear and binding criteria developed in collaboration with the G-BA are essential.

Single-arm trial (SAT) data are increasingly reviewed by regulatory and Health Technology Assessment (HTA) agencies. External comparators (ECs) are used to contextualize SAT data. We analyzed SAT-based submissions using Real World Data (RWD) as ECs to evaluate the role in respective regulatory and HTA assessments.

ZusammenfassungZiel: Das Prostatakarzinom (PCa) ist die zweithaufigste krebsbedingte Todesursache des Mannes. Untersuchungsziel ist es, anhand von Krankenkassendaten (Sekundardaten) die Pravalenz des PCa mit und ohne weitere Neubildungen (Fern- und Nahmetastasen, Zweittumoren) zu ermitteln und beim fortgeschrittenen PCa Angaben zur Chemo-, Hormon- und Schmerztherapie zu machen.Methodik: Basis war eine dynamische Krankenkassenkohorte der Jahre 2007-2010. Die Stichprobe wurde anhand der KM6-Statistik fur die Gesetzliche Krankenversicherung (GKV) altersadjustiert hochgerechnet. PCa-Patienten wurden mittels ICD-10-DiagnoseC61, eine zusatzliche sekundare Neubildung uber die ICD-10 C77, C78 oder C79 identifiziert. Fur 2010 wurde der Anteil an PCa-Patienten mit weiteren onkologischen Erkrankungen, bei Patienten mit sekundaren Neubildungen Art und Haufigkeit der abgerechneten Chemo-, Hormon- und Schmerztherapie uber ATC-Codes, Pharmazentralnummern (PZN) und Operationen- und Prozeduren-Schlussel (OPS) ermittelt. Nach Art der Schmerz- und/oder Anamietherapie wurden die Patienten als asymptomatisch, mild-symptomatisch oder symptomatisch klassifiziert.Ergebnisse: Die Pravalenz des PCa stieg in den Jahren 2007-2010 von 1,26% auf 1,65% und die des regionar- und fernmetastasierten PCa (mPCa) von 0,12% auf 0,18%. 35,7% der mPCa-Patienten erhielten 2010 keine Hormon- oder Chemotherapie, 37,9% nur eine Hormontherapie und 26,4% eine Chemotherapie. Anhand der Verordnungen von Analgetika und Antianamika wurden 34,4% als asymptomatisch, 30,3% als mild-symptomatisch und 35,3% als symptomatisch eingestuft. Weitere onkologische Erkrankungen wurden bei 23,5% der PCa- und 36,0% der mPCa-Patienten nachgewiesen. Bei den 10,9% PCa-Patienten mit Kodierung von Metastasen entfielen 0,1% auf regionare pelvine Metastasen.Schlussfolgerung: Die Pravalenz des PCa auf Basis der vorliegenden Krankenkassendaten entspricht ungefahr der bisher berichteten. Der Anteil an Patienten mit Chemotherapie erscheint mit 26,4% zu niedrig vor dem Hintergrund, dass 35,3% der Patienten als symptomatisch eingestuft wurden. Der hohe Anteil von Patienten mit weiteren onkologischen Erkrankungen und der niedrige Anteil an regionaren Lymphknotenmetastasen konnten ein Hinweis darauf sein, dass Metastasen nicht eindeutig diagnostiziert und/oder kodiert werden.AbstractBackground: Prostate cancer (PCa) is the second most frequent cancer-related cause of death in men. Using statutory health insurance (SHI) data, this study aims to determine the prevalence of both non-metastatic and advanced/metastatic (with distant or regional metastasis) prostate cancer (mPCa) as well as of patients with secondary tumors, and to provide information on chemo, hormone, and pain therapy for advanced prostate cancer.Methods: The database consisted of a dynamic cohort from health insurers for the years 2007-2010. Age-adjusted extrapolation by means of the KM6 statistic for SHI was used to determine PCa prevalence. The ICD-10 code C61 was used to identify patients with PCa; ICD-10 C77, C78 or C79 for additional neoplasms. For 2010, the study determined the proportion of PCa patients with further oncological conditions and type and frequency of chemo, hormone, and pain therapy for patients with mPCa, using anatomical therapeutic chemical classification system (ATC) codes, package codes (PZN) as well as the classification codes for surgical interventions and procedures (OPS). Based on their pain and/or anemia therapy, patients were classified as asymptomatic, mildly symptomatic or symptomatic.Results: Prevalence of PCa and mPCa rose in the years 2007-2010 from 1.26% to 1.65% and 0.12% to 0.18%, respectively. In 2010, 35.7% of the mPCa patients did not receive hormone or anti-hormone therapy. 37.9% received hormone or anti-hormone therapy only and 26.4% chemotherapy. Based on analgesics and anti-anemia prescriptions, 34.4% were classified as asymptomatic, 30.3% as mildly symptomatic and 35.3% as symptomatic. Other oncological conditions were found in 23.5% and 36.0% of patients with PCa and mPCa, respectively. Among the 10.9% of patients, in whom some kind of metastasis was coded, only 0.1% had a code for regional pelvic metastases.Conclusion: The prevalence of prostate cancer found in the SHI data is roughly consistent with the previously reported. Given the fact that 35.3% of the patients were classified as symptomatic, a chemotherapy rate of 26.4% appears too low. The high percentage of other oncological conditions and the low percentage of regional lymph node metastases could indicate that metastases are not clearly diagnosed and/or not explicitly coded.

Background: The objective of this study is to determine the number and costs of patients with HIV-diagnoses and their dependence on age and gender by evaluating German statutory health insurance data.Methods: We analyzed various databases of several million customers of various statutory sickness funds operating independently nationwide and extrapolated the results to the overall population.Results: The number of HIV-positive patients varied considerably between the particular statutory sickness funds analyzed. The prevalence of HIV diagnoses in the year 2008 was significantly higher than reported by the governmental Robert Koch-Institute. After elimination of a coding error in the administration software of German ophthalmologists the number of HIV-diagnosis decreased from 2008 to 2010. In the year 2011 no further decline was notable. At that time 60.7 % of documented HIV-positive patients had prescriptions for antiretroviral medications. 38.7 % patients without prescriptions for ARM had a diagnosis by the B23.8 ICD-code only. Unexpectedly for Germany women had a higher proportion of B23.8 diagnoses than men (56.2 % with B23.8 versus 38.4 % all other HIV-codes).Conclusions: False coded HIV-diagnosis presumably triggered financing the statutory sickness funds in Germany. Even after the decline of false coded diagnoses, our results show a high prevalence of HIV diagnoses. The disproportional high numbers of ICD-10 B23.8 for women in the year 2011 are notes for remaining false coded B23.8 diagnoses in the statutory health care system.

The aim of this study was to investigate the number of persons who obtain medical attention for Hepatitis C Virus (HCV). In addition, we wanted to get an overview of the supply situation (frequency and length of therapy or lab controls) for patients with HCV infection.Data obtained from statutory insurance companies were analyzed.The prevalence of HCV diagnosis was 0.19 % within 3 years. Following a positive HCV-test in 19 % of patients the diagnosis of acute HCV was documented. In 2008, 9.3 % of patients were treated with pegylated Interferon alpha with or without Ribavirin (11.9 % for chronic and 3.6 % for acute HCV). A general practitioner initiated in about 5.1 % and a specialist in 21.7 % of cases a therapy within a year. 37.0 % of patients treated by a general practitioner and 57.0 % of patients treated by specialists received prescriptions in the third quarter after initiating the therapy. Not all patients had a documented test for viral load prior to therapy.50 % of HCV-patients visited a physician in a 3 year time period while an average of 9 % received a therapy within a year. For the coding of acute HCV diagnosis, duration of therapy and the necessary viral load test prior therapy, the analyses showed deviations from the guideline and differences between general practitioners and specialists. For future therapies, patients with HCV-infection should be treated in specialized centers so that these therapies may develop their efficiency in the health system.

To determine the incidence and prevalence of patients diagnosed with MW (with or without other diagnoses of cancer) and to determine rate and type of chemotherapy and care settings using sickness data. Data from 1,771,217 beneficiaries in 2012 were analysed. A patient was incident for WM if no diagnosis was coded in 2010. WM patients were identified by ICD-10 C88.0, oncological co-diagnoses by ICD-10, chemotherapy by Anatomical Therapeutic Chemical (ATC) Code L01*, pharmacy number (PZN) 9999092 and/or operating and procedure code (OPS) 8-54*. 108 patients with a diagnosis C88.0 could be identified (prevalence 0.0060%). 22 (20.4%) of them had their first diagnosis in 2012 (incident). Overall gender ratio was 56.5% male and 43.5% female. The highest share of patients was found in the age class 70-79 (41.7%). Additional oncological diagnoses were found in 67 (62.0%) patients diagnosed with WM. Top 3 reported oncological disorders were: 61 (41.5%) had neoplasms with unknown behaviour (D37-48), 40 (27.2%) had a melanoma (C43-44) and 10 (6.8%) had malignant neoplasms of male genital organs (C60-63). The outpatient diagnosis rate for WM was 84.3%, inpatient rate 3.7% and in- and outpatient rate 11.1%. 10 of the 108 patients (9.3%) started chemotherapy in 2012 (30.0% out- and inpatient and 70.0% outpatient). 3 of the incident patients (n=22) started chemotherapy (13.6%) and 7 of the prevalent (n=86, 8.1%). 6 patients received first-line (1 monotherapy) and 4 second-line treatment. Prescribed compounds were identified by ATC (outpatient only): 75.9% rituximab, 48.3% bendamustine, 20.7% chlorambucil, 17.2% cyclophosphamide, 6.9% fludarabine and 6.9% vincristine (data suggesting combinations). The yearly chemotherapy rate was 9.3%. The majority of the patients received no chemotherapy (n=98, 90.7%). Most of the patients were diagnosed and treated in the outpatient sector. Oncological disorders were relatively high (62.0%).

No national registries for chronic lymphocytic leukaemia (CLL) exist in Germany. The objective of this analysis was to examine the number of patients with CLL diagnosed (with or without other diagnoses of cancer) and to characterize the types of treatment being utilized and care settings using sickness funds claim data. This analysis evaluates data from 1,771,225 beneficiaries in 2012 from different statutory sickness funds. Patients with CLL were identified by ICD-10 C91.1*, oncological co-diagnoses by ICD-10 C00-79; D37-48 and chemotherapy by Anatomical Therapeutic Chemical (ATC) Code L01*, pharmacy number (PZN) 9999092 and/or operating and procedure code (OPS) 854*. In total, 1,405 patients with a diagnosis C91.1* (CLL) were identified (prevalence 79/100,000) with 60.2% were male and 39.8% female. Overall, 32.2% of patients with CLL had a co-diagnosis with another cancer; 25.1% melanoma (C43-44), 21.3% had neoplasms with unknown behaviour (D37-48), 11.8% had malignant male GU neoplasms (90% prostate cancer) (C60-63), 10.7% had malignant neoplasms of digestive organs (C15-26) and 8,4% showed malignant neoplasms of ill-defined, secondary and unspecified sites (C76-80). The outpatient diagnosis rate for CLL was 94.9%, inpatient rate 0.6% and in-and outpatient rate 4.6%. Overall, 266 of 1,405 pts (18.9%) (175 men [65.8%], 91 women [34.2%] ) received chemotherapy in 2012 (ATC Code L01* 74.1%, PZN 9999092 23.0%, OPS 854* 2.9%). Most patients received outpatient treatment (94.0%), with 5.3% of patients received both out- and inpatient treatment and 0.7% inpatient treatment. The most commonly used treatments were rituximab (26.7%), bendamustine (20.2%), chlorambucil (11.1%), cyclophosphamide (7.7%), fludarabine (6.2%) and other treatments (28.1%). The majority of patients being diagnosed with CLL did not require treatment within a time period of a year. Approximately 1/3 of patients had a second malignancy, predominantly skin cancer. Treatment was primarily composed of chemotherapy or chemoimmunotherapy.

Using sickness fund claims data, we sought to determine osteoarthritis rate, drug compound class, pain therapy prevalence and type of medical specialists providing treatment. A group of company health-sickness funds (approx. 2.1 million insured patients in 2010; 2.5 million insured patients in 2011) was used. Osteoarthritis was identified based on ICD-10 diagnoses (M16.0-9, M17.0-5, M17.9, M19.05, M19.25, M19.85, M19.95), then linked to prescriptions using the ATC codes: M01A (nonsteroidal anti-inflammatory drugs, NSAIDS), N02B (analgesics and antipyretics), and N02A (opioids). Furthermore, we determined which groups of medical specialists prescribed the drugs. Osteoarthritis was diagnosed in 7.8% (in 2010) and in 7.1% (in 2011) of patients. In one year, 65.4% of patients received a prescription for at least one drug from the analysed ATC codes: 81.4% of patients received at least one NSAID, 36.4% an analgesic and antipyretic, and 27.4% an opioid. For M01A, diclofenac (54%) was most frequently prescribed; the proportion of coxibs was 6%. For N02B, 99% of prescriptions were for metamizol; 1% for paracetamol. For N02A, most prescriptions were for tramadol (29%) or tilidin (28%). General practitioners most frequently prescribed these drugs (42.2% [M01A] /46.2% [N02B] /45.9% [N02A] ). In Germany in 2010-2011, OA prevalence was 7-8%, and associated with analgesic prescriptions for the majority of evaluated patients. Diclofenac (NSAIDs, metamizol (analgesics and antipyretics), and tramadol or tilidin (opioids) were most frequently prescribed in each group. General practitioners were the most frequent painkiller prescribers.

Hintergrund: Die Prävalenz der HIV-Infektion in Deutschland wird durch das staatliche Robert Koch-Institut geschätzt. Die vorliegende Untersuchung hat das Ziel, die Anzahl Personen und Kosten mit HIV-Diagnose in Krankenkassendaten zu ermitteln und Aussagen über deren Alter und Geschlecht zu treffen. Ferner soll eine Abschätzung der möglichen Auswirkungen potenziell fehlerhafter Kodierungen auf den morbiditätsorientierten Risikostrukturausgleich (Morbi-RSA) für HIV/AIDS erfolgen. Methode: Es wurden Daten verschiedener Datenbanken mit mehreren Millionen Versicherten aus den Jahren 2008 – 2011 ausgewertet und auf die Gesamtpopulation hochgerechnet. Ergebnisse: Die Anzahl Patienten mit HIV-Diagnose variierte zwischen den Krankenkassen erheblich. Es wurde eine auffällig hohe Fallzahl von Patienten mit HIV-Diagnose im Jahr 2008 festgestellt, die maßgeblich auf die Fehlkodierung einer HIV-Diagnose (ICD-10 B23.8) bei Augenärzten durch einen Softwarefehler zurückzuführen ist. Nach Elimination des Fehlers nahmen die HIV-Diagnosen bis 2010 deutlich ab. In der Stichprobe des Jahres 2011 war kein weiterer Rückgang mehr feststellbar. Zu diesem Zeitpunkt waren bei 60,7 % der Patienten mit HIV-Diagnose eingelöste Verordnungen antiretroviraler Medikamente (ARM) dokumentiert. In der Gruppe der Patienten mit HIV-Diagnose ohne ARM erfolgte die Diagnose HIV-Krankheit bei 38,7 % der Patienten allein durch den ICD-Code B23.8. Von dieser Diagnose waren statistisch signifikant häufiger Frauen betroffen (56,2 % mit B23.8). Schlussfolgerung: In der Vergangenheit hat es Fehlkodierungen einer HIV-Diagnose gegeben, die mutmaßlich auch Fehlallokationen über den Morbi-RSA an die Krankenkassen ausgelöst haben. Die auch über das Jahr 2010 fortbestehende, in Deutschland unerwartete Häufung der ICD-10 B23.8 bei Frauen spricht dafür, dass auch nach dem Jahr 2010 noch HIV-Fehlkodierungen im GKV-System vorhanden waren.

Among patients with acute myeloid leukemia (AML), the DACO-016 randomized study showed reduction in mortality for DACOGEN®(decitabine, DAC) compared with treatment choice (TC): at primary analysis the Hazard Ratio (HR) was 0.85 (95% CI: 0.69- 1.04; stratified log-rank p=0.108). With two interim analyses, 2-sided alpha was adjusted to 0.0462. With one year additional follow-up the HR reached 0.82 (nominal p=0.037). These data, together with significant outcomes in secondary endpoints and a positive benefit-risk resulted in approval of DACOGEN in the EU, however not in the US. With the primary analysis only showing a strong trend, the French Haute Autorité de Santé negated a mortality benefit. Though pre-specified, the log-rank test could be considered not optimal to assess the observed survival difference because of the non-proportional hazard nature of the survival curves. We applied the Wilcoxon test as a sensitivity analysis. Patients (age ≥ 65 years, ineligible for chemotherapy) were randomized to DAC (N=242) or TC (N=243). For testing the observed treatment effect, Wilcoxon-test is considered more powerful in the context of non-proportional hazard curves compared to the log-rank test, as the former assigns more weight to earlier events. A total of 108 (44.4%) patients in the TC arm and 91 (37.6%) patients in the DAC arm selectively crossed over to subsequent disease modifying therapies at progression, which might impact the survival beyond the median with resultant converging curves (and disproportional hazards). The Wilcoxon-test stratified by baseline age, cytogenetic-risk and ECOG performance status showed a significant improvement in OS with DAC (7.7 [6.2; 9.2] months) versus TC (5.0 [4.3; 6.3] months) (p=0.0456). Wilcoxon-test indicated significant increase in survival for DAC vs TC in patients with AML compared to log-rank test at primary analysis.

Drug Prescribing for Patients with Chronic Kidney Disease in General Practice: a Cross-Sectional Study

Drug Prescribing for Patients with Chronic Kidney Disease in General Practice: a Cross-Sectional Study

There is no central registry for patients with multiple myeloma in Germany and data from regional registries can be biased. Since data from sick funds are recently available, these were used to compare prevalence, co-morbidities and treatment rates of multiple myeloma patients with data from cancer registries. Three German statutory health insurances (SHI) with 1.780.000 beneficiaries were analyzed for patients diagnosed with multiple myeloma (MM) (ICD-10 C90.0) in 2008-2010. Treatment was identified for outpatients by prescriptions with ATC Codes L01x (antineoplastic agents) and H02A (corticosteroids). Co-morbidities were expressed by Hierarchical Condition Categories. A total of 746 patients had a confirmed diagnosis of multiple myeloma, 53% female and 47% male. The highest incidence for myeloma was between 65 and 80 years. 35% were hospitalized with no information on treatment. Of the outpatients, 10% received a treatment identified by ATC Code L01x (preparations of parenteral chemotherapy not included). Most prominently elevated co-morbidities of MM patients compared to all other patients aged 55 -85 years were anemia (41% vs. 7%), osteoporosis (41% vs. 18%), renal failure (31% vs. 7%), depression (23% vs. 11%) and infectious diseases (38% vs. 19 %). A higher rate of patients received dexamethasone rather than prednisone (41% vs. 28%). The most frequently used antineoplastic agents identifiable by ATC were lenalidomide (16%), followed by melphalan (6%) and bortezomib (5%). The age- and gender adjusted and to the German population extrapolated prevalence of 34.000 patients was higher than reported prevalence numbers of 13.500 patients (5-year prevalence with 41% relative 5 year survival rate). The age distribution is consistent but the gender distribution was slightly different from reported numbers1. Patient shares for melphalan and bortezomib are likely to be underreported because parenteral preparations were not identifiable. Treatment judged for outpatients revealed a higher share of patients who received 2nd line treatment for multiple myeloma rather than a 1st line therapy.

To determine the number of patients with diagnosed acute myeloid leukaemia (AML) including chemotherapy rates, types and care settings using sick-fund data. Data provided by 4 statutory sick funds (SHI) and 2 data providers were analyzed for 2010. AML diagnosis was identified by ICD-10 C92.0 and other types of cancer by ICD-10 C00-79; D00-09; D37-48. Prevalence was extrapolated to the German population by correcting for a representative age- and gender distribution. The median proportion of AML patients > 60 years was 61.1% or 6,590 patients (SHI A: 63.0% = 4,938; B: 50.9% = 5,858; C: 68.9% = 8,975). The proportion of AML patients > 65 years (sick fund D) was 47.6% or 4,400 patients. Gender ratio was 57% male and 43% female. On average, 2 additional oncological disorders were found in 77% of AML patients. 50% had an unspecified leukaemia (C95.90), 25% had CML (C92.1*) and 20% MDS (D46.9). Among patients >65 years the rate of treatment was 60.9% (Anatomical Therapeutic Chemical (ATC) Code L01* (40%), pharmacy number (PZN) 9999092 (19%), operating and procedure code (OPS) 854* (26%)). 16.4% received both out- and inpatient treatment, 34.5% received outpatient treatment and 10.0% inpatient treatment. Identification of comppunds was possible when ATC codes were reported. 63.6% received imatinib and/or hydroxycarbamide, 11.4% tioguanine, 9.1% mercaptopurine, 9.1% cytarabine and 6.8% received dasatinib. The outpatient treatment rate in patients > 60 years diagnosed with AML only was 34.3% (6,2% mercaptopurine and idarubicine, 28.1% PZN 9999092). Prevalence was higher than previously reported incidence numbers of 3,100 AML patients (SHI, all age groups). The yearly outpatient treatment rate was relatively low with 34.3% in patients diagnosed with AML alone. Other oncological disorders are likely to be diagnosed before AML diagnosis could be confirmed. Treatment rates increased with additional oncological disorders with the use of substances not approved for AML.

In a national audit of elective orthopedic surgery conducted in the US, 30% of patients were found to have hemoglobin levels <13 g/dL at preadmission testing. Preoperative anaemia has been associated with increased mortality and morbidity after surgery, exposure to allogeneic blood transfusion therapy and increased rates of postoperative infection leading to a longer length of stay. Because of the risks associated with allogeneic blood transfusions the patient has to be offered an autologeous measure due to the German law if the risk for getting an allogeneic blood transfusion is >10%. However, one of these measures, the autologeous blood donation, can exaggerate the anaemia and can increase the overall transfusion rates (allogeneic and/or autologeous). Since autologeous procedures are not always appropriate for anaemic patients together with an expected shortage of blood and because preoperative anaemia is associated with perioperative risks of blood transfusion, as well as increased perioperative morbidity and mortality, a standardized approach for the detection, evaluation, and management of anaemia in this setting was identified as an unmet medical need. A panel of multidisciplinary physicians was convened by the Society for Blood Management to develop a clinical care pathway for anaemia management in the elective surgical patient for whom blood transfusion is a probability. In these guidelines elective surgical patients should have a Hgb level determination as close to 28 days before the scheduled surgical procedure. The patient's target Hgb before elective surgery should be within the normal range (normal female >= 2 g/dL, normal male >= 13 g/dL). Laboratory testing should take place to further evaluate for nutritional deficiencies, chronic renal insufficiency, and/or chronic inflammatory disease. Nutritional deficiencies should be treated and erythropoiesis-stimulating agent (ESA) therapy should be used for anaemic patients in whom nutritional deficiencies have been ruled out and/or corrected.

Etwa ein Drittel der Patienten, die sich einer elektiven Hüft- oder Kniegelenkendoprothesenoperation unterziehen, weist einen Hämoglobinwert unter 13 g/dl (Männer) bzw. unter 12 g/dl (Frauen) auf, was nach der gültigen WHO-Definition einer Anämie entspricht. Diese ist mit einer erhöhten postoperativen Morbidität- und Mortalität und mit häufigen Transfusionen verbunden. Transfundierte Patienten haben ebenfalls ein erhöhtes Risiko für Mortalität und Infektionen und verweilen länger im Krankenhaus. Entsprechend dem Transfusionsgesetz und den Richtlinien der Bundesärztekammer sind Patienten mit einer Transfusionswahrscheinlichkeit von mindestens 10% auf die Möglichkeit autologer Hämotherapieverfahren hinzuweisen. Die Eigenblutspende als ein gängiges autologes Verfahren zur Vermeidung allogener Transfusionen wird in Deutschland in vielen Kliniken angewendet, kann jedoch unter bestimmten Bedingungen eine präoperative Anämie noch verstärken. Deswegen, aber auch weil die Ressource Fremdblut zunehmend knapper wird, besteht die Notwendigkeit neuer Standards in der Diagnostik und Therapie der präoperativen Anämie. In aktuellen internationalen Leitlinien wird empfohlen, den Hämoglobinwert spätestens 28 Tage vor dem Eingriff zu bestimmen und entsprechend der Definition der WHO bei Männern auf > 13 g/dl und bei Frauen auf > 12 g/dl anzuheben. Als Ursache für eine Anämie sollten eine chronische Niereninsuffizienz, eine maligne Erkrankung und eine chronische Entzündung abgeklärt und ein Mangel an Eisen, Vitamin B12 und/oder Folsäure ausgeglichen werden. Für Patienten, bei denen keine Mangelerkrankung vorliegt oder eine Substitutionstherapie keine ausreichende Therapie der Anämie erzielt, wird vorgeschlagen, eine Therapie mit erythropoesestimulierenden Substanzen durchzuführen.

In a national audit of elective orthopedic surgery conducted in the US, 30% of patients were found to have hemoglobin (Hgb) levels < 13 g/dl at preadmission testing. Preoperative anemia has been associated with increased mortality and morbidity after surgery, increased allogeneic blood transfusion therapy and increased rates of postoperative infection leading to a longer length of hospital stay. Because of the risks associated with allogeneic blood transfusions according to German law patients have to be offered the option of autologous transfusion if the risk associated with allogeneic blood transfusion is > 10%. However, one of these measures, the autologous blood donation, can exaggerate anemia and can increase the overall transfusion rates (allogeneic and autologous). As autologous procedures (autologous blood donation and cell salvage) are not always appropriate for anemic patients together with an expected shortage of blood and because preoperative anemia is associated with perioperative risks of blood transfusion, a standardized approach for the detection, evaluation and management of anemia in this setting was identified as an unmet medical need. A panel of multidisciplinary physicians was convened by the Society for Blood Management to develop a clinical care pathway for anemia management in elective surgery patients for whom blood transfusion is an option. In these guidelines elective surgery patients should have Hgb level determination at the latest 28 days before the scheduled surgical procedure. The patient target Hgb before elective surgery should be within the normal range (normal female >= 120 g/l, normal male >= 130 g/l). Laboratory testing should take place to further determine nutritional deficiencies, chronic renal insufficiency and/or chronic inflammatory diseases. Nutritional deficiencies should be treated and erythropoiesis-stimulating agent (ESA) therapy should be used for anemic patients in whom nutritional deficiencies have been ruled out and/or corrected.

Transfusion of allogeneic blood still is common in orthopedic surgery albeit associated with higher morbidity and mortality. This analysis evaluates from the perspective of a German hospital the potential cost savings of Epoetin alfa compared to predonated autologous blood transfusions or to no-blood-conservation-strategy during elective hip and knee replacement surgery by reducing allogeneic blood transfusions and their associated infectious adverse events. Individual patients (n = 10,000) were created based on data from controlled trials, the German DRG institute (InEK) and various publications and entered into a stochastic model (Monte-Carlo) one of three treatment arms: Epoetin alfa, preoperative autologous donation and no-blood-conservation-strategy. The model is focused on the costs and events of the procedure and follow-up. The model was validated by an independent external consultant. Clinical and economical variables were obtained from clinical trial databases, the German DRG System, patient records and medical publications- in particular cost per transfusion (allogeneic red blood cells: € 320/unit and autologous red blood cells: € 280/unit), pneumonia treatment (€ 5,000), and length of stay (€ 300/day). Probabilistic sensitivity analyses were performed to determine which, if any, factors had an influence on the model's clinical and cost outcomes. At acquisition costs of € 375/40,000 IE Epoetin alfa is cost saving compared to autologous blood donation, and at € 185/40,000 IE compared to no-blood-conservation-strategy. The results were most sensitive to the cost of Epoetin alfa, blood units and hospital days. Upcoming shortages and increasing prices of red blood cells will make Epoetin alfa an attractive blood conservation strategy for anemic patients at reasonable costs, due the reduction in allogeneic blood transfusions and their associated infectious adverse events.