IntroductionSipuleucel-T is a novel active cellular immunotherapy for the treatment of asymptomatic or minimally symptomatic metastatic castrate-resistant prostate cancer (mCRPC). It is assumed to be associated with less adverse events than conventional docetaxel-based chemotherapy.Material and methodsA systematic review of literature published between January, 1 1966 and February, 6 2012 was performed to assess the efficacy and safety of sipuleucel-T in patients with mCRPC. Databases were searched: Medline, EMBASE, Cochrane, CancerLit as well as ASCO and ESCO websites.ResultsThree randomized clinical trials with a total of 737 participants fulfilled established criteria. The overall survival of patients who received sipuleucel-T in comparison to the control group was significantly longer with a hazard ratio (HR) of 0.73 (95% CI: 0.61-0.88; p = 0.001). Time to disease progression was not prolonged using sipuleucel-T compared to placebo, HR = 0.89 (95% CI: 0.75-1.05; p = 0.18). Relative benefit (RB) of serum PSA level reduction of at least 50% for sipuleucel-T compared to placebo did not meet statistical significance, RB = 1.97 (95% CI: 0.48-8.14; p = 0.38). The safety population consisted of 729 patients with mCRPC. Compared to the control group, the pooled relative risks (RR) of all adverse events – RR = 1.03 (95% CI: 1.00-1.05; p = 0.06), grade 3 to 5 adverse events – RR = 0.98 (95% CI: 0.79-1.22; p = 0.86) and cerebrovascular events – RR = 1.93 (95% CI: 0.73-5.09; p = 0.18) were not significantly higher for men treated with sipuleucel-T.ConclusionsThe use of sipuleucel-T prolonged the overall survival among men with mCRPC. No effect on time to disease progression was observed and the safety profile was acceptable.
A b s t r a c t IIn nt tr ro od du uc ct ti io on n: : Administration of human C1 esterase inhibitor (Berinert ® P) from target import is the most widespread treatment strategy for patients with hereditary angioedema (HAE). However, a therapeutic health program including Ruconest ® (conestat alfa) could shorten a patient's expectancy for a life-saving treatment. A Ai im m: : To evaluate the cost-utility of Ruconest ® (conestat alfa) financed from public funds within the newly introduced therapeutic health program compared with Berinert ® P (human C1 esterase inhibitor) in the treatment of acute angioedema attacks in adults with HAE. M Ma at te er ri ia al l a an nd d m me et th ho od ds s: : The cost-utility analysis from the Polish healthcare payer's perspective was performed for 1 year (2012). The costs and health outcomes were simulated for three pairs of eligible HAE patient groups (active treatment and corresponding placebo). The incremental costs of each intervention compared with placebo were listed together (direct or indirect comparisons between options were impossible due to limited clinical data available). R Re es su ul lt ts s: : The incremental cost-utility ratios (ICURs) for the evaluated interventions compared with placebo were as follows: EUR 15,226 per QALY (Ruconest ® ) and EUR 27,786 per QALY (Berinert ® P). The probability of cost-utility (ICUR < EUR 24,279 per QALY) assessed for Ruconest ® administered in the case of acute angioedema attack was 61% and 41% for Berinert ® P. C Co on nc cl lu us si io on ns s: : The administration of Ruconest ® in acute life-threatening angioedema attacks is economically justified from the Polish healthcare payer's perspective, results in lower costs and is characterized by higher cost-utility probability compared with Berinert ® P. K Ke ey y w wo or rd ds s: : acute angioedema attacks, conestat alfa, cost-utility analysis, hereditary angioedema, human C1 esterase inhibitor.
Introduction: Hereditary angioedema (HAE) is a genetic disease caused by C1-esterase inhibitor deficiency, characterized by recurrent attacks of intense, massive, localized subcutaneous oedema that can involve all parts of the body. The aim of this study is a comparison of the clinical effectiveness of conestat alfa, human C1 esterase inhibitor (C1INH) and icatibant in the treatment of acute angioedema attacks in adults with HAE. Materials and methods: A systematic review of literature published up to May 2012 was performed to assess the efficacy and safety of conestat alfa, C1INH and icatibant in the treatment of acute angioedema attacks in adults with HAE. Databases were searched at MEDLINE (PubMed), EMBASE and Cochrane. The general search structure was designed as a combination of keywords or synonyms: (hereditary angioedema) AND (conestat alfa OR human C1 esterase inhibitor concentrate or synonyms OR icatibant). Only randomized clinical studies were selected. Results: Systematic review yielded no clinical trials directly comparing the therapeutic options mentioned. Two randomized clinical trials were found which compared each of the following: conestat alfa, C1INH and icatibant with placebo. Based on the gathered evidence it was demonstrated that taking any of the medicinal substances mentioned in the treatment of acute angioedema attack results in a shorter time to the start of symptom relief and the time to reduce symptoms, the probability of treatment response after 4 hours is increased and the safety profile is comparable to placebo. Conclusions: Due to the significant heterogeneity of the identified trials, the scientific evidence available was insufficient to identify the most effective therapeutic option in the treatment of acute oedemas in HAE.
S u m m a r y B Ba ac ck kg gr ro ou un nd d: : Anakinra is an interleukin-1 receptor antagonist (IL-1Ra) that blocks the biological activity of IL-1, including inflammation. Anakinra in combination with methotrexate (MTX) is indicated for reduction in signs and symptoms in active rheumatoid arthritis, in adults who have failed one or more disease-modifying antirheumatic drugs (DMARDs). M Ma at te er ri ia al l a an nd d m me et th ho od ds s: : According to the rules of evidence-based medicine popularized by The Cochrane Collaboration, a systematic review of medical literature has been performed in order to assess clinical efficacy and safety of the therapy. The review yielded three randomized clinical studies which compared the administration of anakinra in combination with MTX (or other DMARDs) to placebo with MTX. R Re es su ul lt ts s: : Moderate clinical efficacy defined as ACR20, ACR50 and ACR70 response and safety profile of anakinra in combination with MTX or other DMARDs was proven in most studies mentioned and confirmed by the results of the meta-analysis performed. C Co on nc cl lu us si io on ns s: : Injection site reactions were the most common adverse events frequently leading to withdrawal. S t r e s z c z e n i eW Ws st tę ęp p: : Anakinra jest rekombinowanym ludzkim antagonistą receptora interleukiny 1 (IL-1Ra), który prowadzi do zahamowania aktywności biologicznej IL-1, m.in. działania prozapalnego. Anakinra w skojarzeniu z metotreksatem (MTX) jest zalecana w leczeniu przedmiotowych i podmiotowych objawów reumatoidalnego zapalenia stawów (RZS) u dorosłych pacjentów, u których zastosowanie jednego lub więcej leków modyfikujących przebieg choroby (LMPCh) nie przyniosło pożądanych efektów. M Ma at te er ri ia ał ł i i m me et to od dy y: : W celu oceny skuteczności klinicznej tej opcji terapeutycznej, zgodnie z zasadami medycyny opartej na dowodach naukowych (evidence based medicine -EBM) zalecanymi przez organizację The Cochrane Collaboration oraz polską Agencję Oceny Technologii Medycznych (AOTM), dokonano systematycznego przeglądu literatury medycznej, w wyniku którego odnaleziono łącznie trzy badania kliniczne z randomizacją dotyczące porównania anakinry stosowanej z MTX (lub innymi LMPCh) względem placebo podawanego z MTX. W Wy yn ni ik ki i: : Umiarkowana skuteczność kliniczna wg kryteriów ACR20, ACR50, ACR70 i bezpieczeństwo stosowania anakinry w terapii skojarzonej u pacjentów z RZS została potwierdzona w większości odnalezionych badań oraz dodatkowo na podstawie wyników metaanaliz przeprowadzonych z wykorzystaniem wyników przeglądu systematycznego. W Wn ni io os sk ki i: : Najczęstszym występującym działaniem niepożądanym, któ-re w wielu przypadkach było przyczyną rezygnacji z dalszego leczenia, było pojawienie się reakcji alergicznej w miejscu wstrzyknięcia leku.A Ad dr re es ss s f fo or r c co or rr re es sp po on nd de en nc ce e: : dr n. med. Paweł Kawalec, Zakład Gospodarki Lekiem, Instytut Zdrowia Publicznego, Wydział Nauk o Zdrowiu, Uniwersytet Jagielloński, ul. Grzegórzecka 20, ...
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