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La eficacia de una nueva intervención se establece generalmente a través de ensayos clínicos (EC) con asignación aleatoria (AA). Sin embargo, entre otros tantos desafíos metodológicos, el especificar la hipótesis de un EC con AA, sigue siendo un problema complejo de resolver para los investigadores clínicos. En este manuscrito discutimos las características de tres variantes de los EC con AA: EC de superioridad (ECS), EC de no-inferioridad (ECNI), y EC de equivalencia (ECE). Estos tres tipos de EC tienen supuestos diferentes sobre los efectos de una intervención, por lo que plantear hipótesis y definir objetivos requiere conocer algunos supuestos subyacentes a estos EC, incluso hasta elementos relacionados con la estimación del tamaño de muestra para cada cual. El objetivo de este manuscrito fue describir las diferencias metodológicas entre ECS, ECNI y ECE.
Efficacy and effectivity of new interventions are generally established through randomized clinical trials (RCTs). However, among many other methodological challenges, specifying the hypothesis of a RCT remains complex problem for clinical researchers. In this manuscript we discuss the characteristics of three variants of RCTs: superiority RCT (SRCT), non-inferiority RCT (NIRCT), and equivalence RCT (ERCT). These three types of RCT have different assumptions about the effects of an intervention, so setting hypotheses and defining objectives requires knowing some assumptions underlying these RCTs, including elements related to the estimation of the sample size for each one. The aim of this manuscript was to describe methodological differences between SRCT, NIRCT and ERCT.
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Ensayos Clínicos como Asunto , Proyectos de Investigación , Ensayos Clínicos Controlados no Aleatorios como Asunto , Estudios de Equivalencia como AsuntoRESUMEN
Objective:To compare the effect of prognosis between drug-coated balloon (DCB) and drug eluting stent (DES) interventional therapy in patients with coronary heart disease.Methods:The clinical data of 346 coronary heart disease patients underwent interventional therapy because of small vessel lesion, in-stent restenosis and bifurcation lesion in Xuanwu Hospital, Capital Medical University from December 2018 to July 2021 were retrospectively analyzed. Among them, 179 patients were treated with DES (DES group), including small vessel lesion 81 cases, in-stent restenosis 35 cases, and bifurcation lesion 63 cases; 167 patients were treated with DCB (DCB group), including small vessel lesion 69 cases, in-stent restenosis 62 cases, and bifurcation lesion 36 cases. The major adverse cardiac and cerebrovascular event (MACCE) within 1 year after discharge was compared between two groups. Non-inferiority was analyzed, and non-inferiority margin was set to be 4%.Results:There was no statistical difference in the incidence of MACCE between DCB group and DES group: 3.59% (6/167) vs. 7.26% (13/179), P>0.05. There were no statistical differences in the incidences of MACCE in patients with small vessel lesion, in-stent restenosis and bifurcation lesion between DCB group and DES group: 1.45% (1/69) vs. 4.94% (4/81), 8.06% (5/62) vs. 14.29% (5/35) and 0 vs. 6.35% (4/63); P>0.05. Non-inferiority analysis result showed that DCB was non-inferior to DES on the prognosis in patients with coronary heart disease (95% CI - 8.41% to 1.07%). Conclusions:Non-inferiority of DCB versus DES is shown in coronary heart disease patients with small vessel lesion, in-stent restenosis and bifurcation lesion.
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ObjectiveTo evaluate the efficacy and safety of topical Bisaitong （鼻塞通） in treating moderate-to-severe allergic rhinitis （AR）. MethodsA randomized， positive-controlled， non-inferiority clinical trial design was adopted. Totally， 108 cases of moderate-to-severe AR were randomly divided into Bisaitong group and mometasone furoate group，with 54 cases in each group. The Bisaitong group was treated with Bisaitong smeared at the nasal cavity twice a day， and the mometasone furoate group received inhalation of mometasone furoate nasal spray 100 μg in each nostril， once a day. Both groups were treated for 4 weeks and followed up after additional 4 weeks. Both groups were compared on the rhinoconjunctivitis quality of life questionnaire （RQLQ）， rhinoconjunctivitis total symptom score （RTSS）， visual analogue score （VAS） of sneezing， runny nose， nasal itching， nasal congestion degree， days of AR episodes at enrollment， after 2- and 4-week， and at follow-up. The peripheral blood eosinophil （EOS） count and percentage （EOS%）， serum eosinophil cationic protein （ECP）， serum dust mite， dermatophagoides farinae， and cockroach allergen-specific IgE （sIgE） levels were compared between groups at enrollment and after 4-week treatment. Drug overuse rate was calculated， and the safety was evaluated. The analysis of all efficacy outcomes was based on both full analysis set （FAS） and per-protocol set （PPS）. ResultsThe lower limit of the 95% confidence interval for the differences in RQLQ scores were greater than -0.6 measured after 2- and 4-week treatment and at follow-up compared to that measured at the enrollment in both groups， indicating of the Bisaitong group being non-inferior to the mometasone furoate group. There was no statistically significant difference between groups on RTSS score， VAS scores of sneezing， runny nose， nasal itching， nasal congestion degree and days of episodes at all timepoints （P＞0.05）， but each outcome changed significantly over time in both groups （P＜0.01）. The differences between groups in EOS count， EOS%， ECP levels， serum dust mite， dermatophagoides farinae， cockroach sIgE levels， and drug overuse rate were not statistically significant at enrollment and after 4-week treatment （P＞0.05）. Adverse events occurred in eight cases （15.10%） in the Bisaitong group and five cases （9.30%） in the mometasone furoate group， showing no significant difference between groups （P＞0.05）. ConclusionTopical Bisaitong is non-inferior to mometasone furoate nasal spray in the treatment of moderate to severe AR in terms of clinical symptom relief，reduction in the episodes， improvement of quality of life， and sound safety.
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Objective:To evaluate the performance, efficacy and safety of a novel portable endoscopy system for upper gastrointestinal examination.Methods:A multicentered, open-label, randomized, non-inferiority controlled study was conducted in 3 clinical research centers from June 2019 to June 2020, and a total of 90 outpatients admitted to Department of Gastroenterology were randomly assigned to the trial group ( n=44) undergoing portable endoscopy and the control group ( n=46) undergoing Olympus endoscopy. The examination success rate, image quality, performance, overall operation satisfaction rate, biopsy success rate and adverse events of the two groups were compared. Results:The examination success rates of the trial group and the control group were 97.73% (43/44) and 100.00% (46/46) respectively with a difference of -2.27% (95% CI: -6.68%-2.13%), higher than the set non-inferiority margin of -10%. Rates of good and excellent image quality were 100% in both groups, and the difference of 0 was higher than the set non-inferiority margin of -10%. There was no significant difference in the rate of good and excellent performance of the operating system between the two groups [97.67% (42/43) VS 100.00% (46/46), P=0.483]. There was significant difference in the overall satisfactory rate of the operation between the two groups [86.05% (37/43) VS 100.00% (46/46), P=0.011]. A total of 9 cases underwent endoscopic biopsy, including 5 cases in the trial group and 4 cases in the control group. The biopsy channels in both groups were smooth and the biopsy were successfully completed. There was no significant difference in adverse event rate between the two groups [25.00% (11/44) VS 10.87%(5/46), χ2=3.07, P=0.080]. All adverse events disappeared in 48 hours, and no severe adverse events or device defect events occurred. Conclusion:The novel portable endoscopic system is comparable to Olympus endoscopic system in terms of the operating performance, the image quality and safety. Therefore, this system is safe and effective for upper gastrointestinal examination.
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Objectives@#Vitex negundo is an endemic shrub in the Philippines which has been clinically tested for the symptomatic treatment of cough in syrup and tablet formats. However, the effectiveness and safety of the capsule have not been formally documented in a clinical trial setting. Therefore, in compliance with the Philippine FDA directive, this study compared the efficacy and safety of the capsule and tablet formats after three days of treatment among Filipinos with acute uncomplicated cough. @*Methods@#This is a Phase 3b randomized, open-label, parallel-group non-inferiority study with 335 subjects using improvement based on Global Rating of Change Scale scores as primary efficacy endpoint and several secondary endpoints. Descriptive and inferential analyses were performed. The Farrington-Manning Method of Z-test with -10% non-inferiority margin was used for the primary outcome. Appropriate inferential tests were used for the secondary outcomes. @*Results@#Of 335 enrolled subjects, 170 were randomized to the capsule group and 165 to the tablet group with comparable baseline characteristics. The proportion of success based on the Global Rating of Change Scale rated by patients was 95.71% and 91.19% for the capsule and tablet groups, respectively. Based on doctors’ ratings, they were 96.93% and 94.34%, respectively. In addition, the Farrington-Manning Method of Z-test revealed the capsule was not inferior to the tablet based on patients’ and doctors’ ratings (90% Confidence Intervals: -0.0086 to 0.0988 and -0.0228 to 0.0747, respectively). The intention-to-treat analysis also showed non-inferiority, indicating robust results. Significant and similar improvements in cough severity and quality of life were observed in both groups based on Cough Severity Diary scores and Leicester Cough Questionnaire for acute cough, respectively. There were also improvements in the Forced Expiratory Volume at 1 second [FEV1] (capsule group) and Peak Expiratory Flow Rate [PEFR] (both groups), but these were not clinically significant. The safety profiles were also comparable (p= 0.4437) with 1.23% and 2.52% incidence of adverse events, respectively, all of which were mild and assessed as not related to the drug. @*Conclusion@#In terms of efficacy, Ascof® Forte capsule was non-inferior to Ascof® Forte tablet in treating acute uncomplicated cough among Filipinos based on Global Rating of Change Scale scores as rated by patients and doctors. Both treatments showed significant and similar improvements in cough severity and quality of life. They were also comparable in safety with few adverse events in both groups, all mild and assessed unrelated to drug intake.
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CápsulasRESUMEN
Objective:To explore the effect of kangaroo care for mothers and fathers on preterm infants' heart rate, blood oxygen, body temperature and so on, and to explore the influence of kangaroo care on the anxiety of the implementers.Methods:Totally 132 cases of preterm infants and their parents from September 2017 to September 2018 in Shengjing Hospital of China Medical University were randomly divided into mother group (67 cases) and father group (65 cases) by envelope method. The intervention lasted for two hours. State Anxiety Inventory(SAI) was used to evaluate the anxiety status of the implementers before and after the intervention, and the heart rate, oxygen saturation and body temperature of preterm infants were recorded 15 minutes before and at the end of the intervention.Results:Before the intervention, the SAI scores of the two groups were 35.00(30.00, 45.00) in the mother group and 33.00(30.00, 43.00) in the father group, with no significant difference ( Z value was -0.645, P>0.05); after the intervention, the SAI score of the mother group was 29.00(23.00, 32.00) and that of the father group was 25.00(21.50, 28.00), with significant difference ( Z value was -3.518, P<0.01). Before and after the intervention, the changes of heart rate ( δ1=- 4.25), blood oxygen saturation ( δ2=1.45), body temperature ( δ3=0.20) of preterm infants before and after the intervention were tested by non inferiority test, and the difference was statistically significant ( t values were -6.370, 5.343, 12.612, P<0.01). Conclusions:The effect of kangaroo care on preterm infants' heart rate, blood oxygen and body temperature are no less than that of mothers. Compared with mother, kangaroo care can reduce father's anxiety better.
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The appropriate sample size estimation is very important in the design of clinical trials. However, insufficient or inappropriate sample size estimation is still a prominent problem in the currently published acupuncture and moxibustion clinical trials. At present, the superiority test, non-inferiority test and equivalence test have been widely used in acupuncture and moxibustion clinical trials. This article focuses on the application, calculation methods and PASS11 software using of these three hypothesis test types. In view of the problems in the estimation of sample size in acupuncture and moxibustion clinical trials, the particularity of sample size estimation in acupuncture and moxibustion is summarized from the aspects of parameter setting, ratio of intervention group and control group, and multi-group comparison, in order to guide acupuncture clinical researchers to correctly estimate sample size when conducting clinical trials.
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Acupuntura , Terapia por Acupuntura , Ensayos Clínicos como Asunto , Moxibustión , Tamaño de la MuestraRESUMEN
In the era of diseases with highly efficacious treatments, the publication of randomized noninferiority clinical trials is increasingly frequent. However, users of medical literature are less familiar with this type of studies. The aim of this article is to give an introduction to the critical assessment of noninferiority clinical trials, through the solving of a therapeutic dilemma, which will be addressed through the analysis of a recently published trial of this type.
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Humanos , Femenino , Adulto , Publicaciones/estadística & datos numéricos , Proyectos de Investigación , Ensayos Clínicos Controlados Aleatorios como Asunto/métodos , Encuestas y Cuestionarios , Mortalidad , Ensayos Clínicos Controlados como Asunto/normas , Mejoramiento de la CalidadRESUMEN
Objective To evaluate the noninferiority of domestic electomyographic (EMG) evoked potential instrument utility on avoiding iatrogenic injury of nerve roots compare to the imported ones,and understand the methods,accuracy and practicability of domestic machine on monitoring nerve root at the fracture fixation process.Methods Domestic of Neurocare-C machine and Nicolet Viking select8 concurrent monitoring 30 cases of bone fracture fixation,including 20 cases of spinal fixation and 10 cases of fractures fixation of limbs and pelvic.The trend of positive change,the rate and amplitude variation of electromyographic response between two instruments were compared.Results The nerve root function could be accurately judged by EMG monitoring during operation.Intraoperative EMG could be detected by both domestic and imported instruments occurred in 5 patients,3 cases were internal fixation of vertebral fracture,1 case was humeral fractures fixation,1 case was pelvic fracture.The rate of electromyographic response of both domestic and imported instruments were 16.67％.There was no significant difference in amplitude variation of electromyographic response of quadriceps femoris,biceps femoris,rectus femoris and tibialis anterior between two instruments (P ＞ 0.05).Conclusion Both domestic and imported EMG evoked potentials can accurately and timely respond to nerve root function,avoid nerve root injury,and ensure the safety of fracture fixation surgery.
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Objective To investigate the efficacy and safety of domestic exenatide injection versus imported exenatide injection in type 2 diabetic patients with inadequate glycemic control on monotherapy or combination therapy of metformin and insulin secretagogues. Methods A multicenter, randomized, parallel-controlled, and non-inferiority trial was carried out. A total of 240 subjects were randomized at a 1:1 ratio to add domestic exenatide injection (trial group) or imported exenatide injection (control group) on the background therapies. The primary endpoint of efficacy was HbA1C change from baseline to week 16. The secondary endpoints of efficacy were the proportion of HbA1C0.05). The changes in FPG, 2hPG, 7P-SMBG and body weight from baseline to week 16 were comparable between the two groups (all P>0.05). Moreover, the incidences of hypoglycemia and adverse events were similar between the two groups (both P>0.05). Conclusion In type 2 diabetic patients inadequately controlled by monotherapy or combination therapy of metformin and insulin secretagogues, the efficacy of cotreatment with domestic exenatide injection is not inferior to that of imported product ones, with a similar safety profile.
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ABSTRACT INTRODUCTION: Allergic rhinitis is considered the most prevalent respiratory disease in Brazil and worldwide, with great impact on quality of life, affecting social life, sleep, and also performance at school and at work. OBJECTIVE: To compare the efficacy and safety of two formulations containing mometasone furoate in the treatment of mild, moderate, or severe persistent allergic rhinitis after four weeks of treatment. METHODS: Phase III, randomized, non-inferiority, national, open study comparing mometasone furoate in two presentations (control drug and investigational drug). The primary endpoint was the percentage of patients with reduction of at least 0.55 in nasal index score (NIS) after four weeks of treatment. Secondary outcomes included total nasal index score score after four and 12 weeks of treatment; individual scores for symptoms of nasal obstruction, rhinorrhea, sneezing, and nasal pruritus; as well as score for pruritus, lacrimation, and ocular redness after four and 12 weeks of treatment. The study was registered at clinicaltrials.gov with the reference number NCT01372865. RESULTS: The efficacy primary analysis demonstrated non-inferiority of the investigational drug in relation to the control drug, since the upper limit of the confidence interval (CI) of 95% for the difference between the success rates after four weeks of treatment (12.6%) was below the non-inferiority margin provided during the determination of the sample size (13.7%). Adverse events were infrequent and with mild intensity in most cases. CONCLUSION: The efficacy and safety of investigational drug in the treatment of persistent allergic rhinitis were similar to the reference product, demonstrating its non-inferiority.
Resumo Introdução: A rinite alérgica é considerada a doença respiratória mais prevalente no Brasil e em todo o mundo, com grande impacto na qualidade de vida; além de, afetar a vida social, o sono e também o desempenho na escola e no trabalho. Objetivo: Comparar a eficácia e segurança de duas formulações contendo furoato de mometasona no tratamento da rinite alérgica persistente leve, moderada ou grave por um período de quatro semanas. Método: Trata-se de um estudo nacional aberto de fase III, randomizado, de não inferioridade de comparação do furoato de mometasona em duas apresentações (medicação de controle e fármaco sob investigação). O ponto final primário foi o percentual de pacientes com redução mínima de 0,55 no escore de índice nasal (EIN) após quatro semanas de tratamento. Os desfechos secundários foram: escore NIS total após 4 e 12 semanas de tratamento; escores individuais para sintomas de obstrução nasal, rinorréia, espirros e prurido nasal, bem como escores para prurido, lacrimejamento e hiperemia conjuntival após 4 e 12 semanas de tratamento. O estudo foi registrado em clinicaltrials.gov com o número de referência NCT01372865. Resultados: A análise de eficácia primária demonstrou não inferioridade do fármaco sob investigação em relação à medicação de controle, visto que o limite superior do intervalo de confiança (IC) de 95% para a diferença entre os percentuais de sucesso após quatro semanas de tratamento (12,6%) situava-se abaixo da margem de não inferioridade proporcionada durante a determinação do tamanho da amostra (13,7%). Eventos adversos foram pouco frequentes e de leve intensidade na maioria dos casos. Conclusão: A eficácia e a segurança de um fármaco experimental no tratamento da rinite alérgica persistente foram similares às do produto de referência, o que demonstrou sua não inferioridade.
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Humanos , Masculino , Femenino , Adolescente , Adulto , Persona de Mediana Edad , Anciano , Antialérgicos/uso terapéutico , Rinitis Alérgica/tratamiento farmacológico , Furoato de Mometasona/uso terapéutico , Índice de Severidad de la Enfermedad , Resultado del TratamientoRESUMEN
OBJECTIVES: To compare the efficacy and safety of two filgrastim formulations for controlling chemotherapy-induced neutropenia and to evaluate the non-inferiority of the test drug relative to the originator. METHODS: This phase III non-inferiority study had a randomized, multicenter, and open-label design. The patients were randomized at a ratio of 1:1 with a follow-up period of 6 weeks for each patient. In both study arms, filgrastim was administered subcutaneously at a daily dose of 5 mg/kg body weight. The primary endpoint was the rate of grade 4 neutropenia in the first treatment cycle. The secondary endpoints were the duration of grade 4 neutropenia, the generation of anti-filgrastim antibodies, and the rates of adverse events, laboratory abnormalities, febrile neutropenia, and neutropenia of any grade. RESULTS: The primary efficacy analysis demonstrated the non-inferiority of the test drug compared with the originator drug; the upper limit of the 90% confidence interval (CI) for the rate of neutropenia between the two groups (12.61%) was lower than the established margin of non-inferiority. The two treatments were similar with respect to the secondary endpoints and safety. CONCLUSION: The efficacy and safety profile of the test drug were similar to those of the originator product based on the rate of grade 4 neutropenia in the first treatment cycle. This study supports Anvisa’s approval of the first biosimilar drug manufactured by the Brazilian industry (Fiprima¯).
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Humanos , Femenino , Adulto , Persona de Mediana Edad , Biosimilares Farmacéuticos/uso terapéutico , Neoplasias de la Mama/tratamiento farmacológico , Filgrastim/uso terapéutico , Fármacos Hematológicos/uso terapéutico , Neutropenia/inducido químicamente , Neutropenia/prevención & control , Antineoplásicos/efectos adversos , Biosimilares Farmacéuticos/farmacocinética , Filgrastim/farmacocinética , Fármacos Hematológicos/farmacocinética , Recuento de Leucocitos , Valores de Referencia , Reproducibilidad de los Resultados , Índice de Severidad de la Enfermedad , Estadísticas no Paramétricas , Resultado del TratamientoRESUMEN
La sobreexpresión o amplificación del receptor HER2 seobserva en el 20% de pacientes con cáncer de mama y seasocia con un pronóstico adverso. El agente anti HER2más ampliamente utilizado en la clínica es el trastuzumab,anticuerpo monoclonal. El tratamiento adyuvante tieneuna duración de 12 meses y en cáncer de mama metastásicose continúa más allá de la progresión. La mayoría delos pacientes recibirá trastuzumab durante 1 año, muchosrecibirán 2 a 3 años, y algunos han recibido/recibirán más de8 años. Surge así un interés creciente por la vía subcutánea(SC) de administración: menos invasiva, menos costosay más cómoda. El estudio HannaH, ensayo de fase III,abierto y aleatorizado, utilizó una dosis fija subcutánea de600 mg de trastuzumab en combinación con quimioterapianeoadyuvante y la comparó con la vía de administraciónendovenosa (EV) aprobada. Se incluyeron pacientes concáncer de mama HER2 (+) operable, localmente avanzado einflamatorio. Los objetivos primarios fueron: concentraciónsérica mínima predosis del ciclo 8 y respuesta patológicacompleta. Se aleatorizaron 299 pacientes a trastuzumab EVy 297 a trastuzumab SC. La concentración sérica mínimamedia fue 57.8 μ/ml en el grupo EV y 78.7 en el grupo SC.El 40.7% de los pacientes en el grupo EV y el 45.4% en elgrupo SC logró respuesta patológica completa. TrastuzumabSC resultó no inferior para ambos objetivos primarios.La incidencia de eventos adversos grados 3 a 5 fue similar enambos grupos. La demostración de no-inferioridad sugiereque el trastuzumab SC ofrece una válida y más convenientealternativa al trastuzumab EV.
Overexpression or amplification of HER2 is found in 20%of patients with breast cancer and is associated with anunfavourable prognosis. Trastuzumab is the anti HER2agent most widely used in clinical practice. Adjuvanttreatment should be given during 12 months, and in themetastatic setting treatment should continue beyond progression.Most patients will receive treatment during oneyear, many will be treated during 2 to 3 years, and somepatients will remain in treatment for more than 8 years.In this context, a subcutaneous route of administrationbecomes an attractive option: less invasive, less costly,and more comfortable for patients. The HannaH study is a phase III, open, randomized clinical trial that used a fixedsubcutaneous dose of 600 mg of trastuzumab in combinationwith neoadjuvant chemotherapy and compared it tothe approved intravenous (IV) route of administration.Patients with HER2 (+) operable, locally advanced andinflammatory breast cancer were included in the study.Primary endpoints included: serum trough concentrationpre-dose cycle 8 and pathologic complete response. Twohundred and ninety-nine patients were randomized to IVtrastuzumab and 297 to SC trastuzumab. Mean serumtrough concentration was 57.8 μ/ml in the IV group and78.7 in the SC group. In the IV group 40.7% of patientsachieved pathologic complete response, and 45.4% in the SCgroup. The SC formulation resulted non-inferior for bothprimary objectives. Incidence of grade 3-5 adverse eventswas similar in both groups. The non-inferior results suggestthat SC trastuzumab is a valid and more convenientalternative to IV trastuzumab.
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Neoplasias de la Mama , Quimioterapia , Tejido Subcutáneo , Peso Corporal , Relación Dosis-Respuesta a DrogaRESUMEN
The sample size of non-inferiority,equivalence and superiority design in clinical trial was estimated by using PASS 11 software.The result was compared with that by using SAS to evaluate the practicability and accuracy of PASS 11 software for the purpose of providing reference for sample size estimation in clinical trial design.
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The sample size of non-inferiority,equivalence and superiority design in clinical trial was estimated by using PASS 11 software.The result was compared with that by using SAS to evaluate the practicability and accuracy of PASS 11 software for the purpose of providing reference for sample size estimation in clinical trial design.
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To evaluate the efficacy and safety of Choudongning (CDN)capsule in children with Tourette's syndrome of spleen deficiency and phlegm accumulation through a randomized double-blind three-arm controlled phase Ⅲ study in 588 patients from 8 hospitals. The included patients were randomly divided into test group, positive control group and placebo group at the ratio of 3∶1∶1. Patients in the test group orally took CDN capsules and simulated Tiapridal tablets; the patients in positive control group took Tiapridal tablets and simulated CDN capsules; whereas the patients in placebo group orally took the simulated agents of the above two drugs. The treatment course was 6 weeks for three groups. The global grading rates, YGTSS scores and its factor scores, the degree of social function damage, as well as traditional Chinese medicine syndrome efficacy were evaluated as the outcome measures on efficacy. The AEs/ADRs, vital signs and laboratory testing were observed as outcome measures on safety. The total effective rate of YGTSS was 75.92% in the test group, 72.65% in the positive control group, and 37.29% in the placebo group. Non inferiority test stands between the test group and the positive control group, and they were superior to placebo group in efficacy with statistical difference. Significant difference had also been found among the 3 groups in YGTSS tics score, motor tics score, vocal tics, degree of social function damage and traditional Chinese medicine syndrome efficacy. During the study, there were 5 (1.42%)ADRs in the test group, 10 (8.55%)in the positive control group and 3 (2.54%)in the placebo group. The incidence of ADRs in the test group was lower than that in the positive control group, with statistical difference. It is clear to say that CDN capsule can effectively treat the Tourette's syndrome of spleen deficiency and phlegm accumulation. Its efficacy is not inferior to the commonly used Tiapridal tablets, with even less adverse reactions, so it has clinical application value.
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In this double-blind,double-dummy,non-inferiority study,144 cases of newly diagnosed acute gouty arthritis were reassigned to Chuanhu anti-gout mixture group (CH,n =72) and colchicine group (Col,n =72).After treatment for 10 days,the total efficiency rates of CH and Col groups were 93.02％ and 95.35％,respectively,showing no difference between two groups.Three months later,the overall recurrence rates in CH and Col groups were 12.50％ and 13.89％ respectively (P＜0.05).The incidence of adverse reactions in colchicine group was significantly higher than CH group (34.72％ vs 2.78％,P ＜ 0.01).Blood uric acid,alanine aminotransferase,aspartate aminotransferase,and creatinine in CH group were decreased significantly compared with Col group (P＜0.05 or P＜0.01).The results suggest that the clinical efficacy of Chuanhu anti-gout mixture in the treatment of acute gouty arthritis was not inferior to colchicine,but with higher safety.
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Context: Currently, there is limited data on the prevention of chemotherapy-induced nausea and vomiting (CINV) in Indian patients. Aims: This post hoc study assessed the efficacy and safety of fosaprepitant compared with aprepitant for prevention of CINV in the Indian population. A subgroup analysis was performed from data collected in a phase 3 study of intravenous (IV) fosaprepitant or oral aprepitant, plus the 5-HT 3 antagonist ondansetron and the corticosteroid dexamethasone, in cisplatin-naοve patients with solid malignancies. Materials and Methods: Patients scheduled to receive cisplatin (≥70 mg/m 2 ) were administered a single IV dose of fosaprepitant dimeglumine (150 mg) on day 1 or a 3-day dosing regimen of oral aprepitant (day 1:125 mg, days 2 and 3:80 mg) with standard doses of ondansetron and dexamethasone. Patients recorded nausea and/or vomiting episodes and their use of rescue medication and were monitored for adverse events (AEs) and tolerability. Statistical Analysis Used: Differences in response rates between fosaprepitant and aprepitant were calculated using the Miettinen and Nurminen method. Results: In the Indian subpopulation (n = 372), efficacy was similar for patients in both the fosaprepitant or aprepitant groups; complete response in the overall, acute, and delayed phases and no vomiting in all phases were approximately 4 percentage points higher in the fosaprepitant group compared with the aprepitant group. Fosaprepitant was generally well-tolerated; common AEs were similar to oral aprepitant. Conclusions: IV fosaprepitant is as safe and effective as oral aprepitant in the Indian subpopulation and offers an alternative to the oral formulation.
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Adulto , Anciano , Pueblo Asiatico , Población Negra , Antieméticos/uso terapéutico , Cisplatino/efectos adversos , Grupos Raciales , Método Doble Ciego , Femenino , Humanos , Indígenas Norteamericanos , Masculino , Persona de Mediana Edad , Morfolinas/administración & dosificación , Morfolinas/uso terapéutico , Neoplasias/tratamiento farmacológico , Náusea/inducido químicamente , Náusea/tratamiento farmacológico , Náusea/prevención & control , Nativos de Hawái y Otras Islas del Pacífico , Vómitos/inducido químicamente , Vómitos/tratamiento farmacológico , Vómitos/prevención & controlRESUMEN
Noninferiority trials test whether a new experimental treatment is not unacceptably less efficacious than an active control treatment already in use. With continuous improvements in health technologies, standard care, and clinical outcomes, the incremental benefits of newly developed treatments may be only marginal over existing treatments. Sometimes assigning patients to a placebo is unethical. In such circumstances, there has been increasing emphasis on the use of noninferiority trial designs. Noninferiority trials are more complex to design, conduct, and interpret than typical superiority trials. This paper reviews the concept of noninferiority trials and discusses some important issues related to them.