2015
Selection of the treatment effect for sample size determination in a superiority clinical trial using a hybrid classical and Bayesian procedure
Ciarleglio MM, Arendt CD, Makuch RW, Peduzzi PN. Selection of the treatment effect for sample size determination in a superiority clinical trial using a hybrid classical and Bayesian procedure. Contemporary Clinical Trials 2015, 41: 160-171. PMID: 25583273, DOI: 10.1016/j.cct.2015.01.002.Peer-Reviewed Original Research
1998
Portal hypertension and variceal bleeding: An AASLD single topic symposium
Grace N, Groszmann R, Garcia‐Tsao G, Burroughs A, Pagliaro L, Makuch R, Bosch J, Stiegmann G, Henderson J, de Franchis R, Wagner J, Conn H, Rodes J. Portal hypertension and variceal bleeding: An AASLD single topic symposium. Hepatology 1998, 28: 868-880. PMID: 9731585, DOI: 10.1002/hep.510280339.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsClinical versus Statistical Considerations in the Design and Analysis of Clinical Research
Horwitz R, Singer B, Makuch R, Viscoli C. Clinical versus Statistical Considerations in the Design and Analysis of Clinical Research. Journal Of Clinical Epidemiology 1998, 51: 305-307. PMID: 9539886, DOI: 10.1016/s0895-4356(98)00006-7.Peer-Reviewed Original Research
1989
Issues in planning and interpreting active control equivalence studies
Makuch R, Johnson M. Issues in planning and interpreting active control equivalence studies. Journal Of Clinical Epidemiology 1989, 42: 503-511. PMID: 2661729, DOI: 10.1016/0895-4356(89)90146-7.Peer-Reviewed Original ResearchMeSH KeywordsAdultAspirinCerebrovascular DisordersClinical Trials as TopicHumansRandom AllocationResearch DesignSulfinpyrazoneTiclopidineConceptsActive control equivalence studiesEquivalence studyActive-control equivalence trialsPrevention of strokeNew drugsActive control drugActive control treatmentAlternative study designsCurrent therapiesPlaceboControl drugTherapeutic equivalenceEquivalence trialNew treatmentsMethodologic issuesDrug efficacyStudy designTreatment controlDrugsEfficacyEquivalence designTrialsTreatmentStudy validation
1988
A prospective randomized trial of HLA‐matched versus mismatched single‐donor platelet transfusions in cancer patients
Messerschmidt G, Makuch R, Appelbaum F, Ungerleider R, Abrams R, O'Donnell J, Holohan T, Fontana J, Wright D, Anagnou N, Shan T, Chesbro B, Deisseroth A. A prospective randomized trial of HLA‐matched versus mismatched single‐donor platelet transfusions in cancer patients. Cancer 1988, 62: 795-801. PMID: 3293762, DOI: 10.1002/1097-0142(19880815)62:4<795::aid-cncr2820620426>3.0.co;2-7.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAmbulatory CareAntibody FormationBlood DonorsBlood PlateletsBlood TransfusionChildChild, PreschoolClinical Trials as TopicFemaleHemorrhageHistocompatibility TestingHumansInfantMaleMiddle AgedNeoplasmsPlatelet TransfusionProspective StudiesRandom AllocationThrombocytopeniaTransfusion ReactionConceptsSingle-donor platelet transfusionsPlatelet transfusionsCancer patientsFebrile patientsPosttransfusion incrementsThrombocytopenic cancer patientsTransfusion of HLASevere bleeding episodesMulti-institution trialNonfebrile patientsIntensive chemotherapyBleeding episodesAntiplatelet antibodiesAntibody titersSignificant thrombocytopeniaHistocompatability antigensTransfusionPatientsMismatched groupHLAThrombocytopeniaPlateletsEpisodesSeparate episodesTrialsStatistical Methods for the Analysis of HIV-1 Core Polypeptide Antigen Data in Clinical Studies
Makuch R, Parks W. Statistical Methods for the Analysis of HIV-1 Core Polypeptide Antigen Data in Clinical Studies. AIDS Research And Human Retroviruses 1988, 4: 305-316. PMID: 3061416, DOI: 10.1089/aid.1988.4.305.Peer-Reviewed Original ResearchConceptsPlacebo-treated patientsInitiation of therapyPlacebo-controlled studyHIV-1 antigensDemonstrated clinical efficacyHIV-1 coreAntiretroviral effectClinical efficacyAIDS patientsAntiretroviral activityClinical studiesLymphocyte culturesVirus expressionSerum dataAntigen dataPatientsSurvival analysis methodsLaboratory dataAZTGroup differencesMost laboratory dataCulture dataStatistical methodsSignificant declineSerum
1986
A trial of combination chemotherapy followed by hormonal therapy for previously untreated metastatic carcinoma of the prostate.
Seifter E, Bunn P, Cohen M, Makuch R, Dunnick N, Javadpour N, Bensimon H, Eddy J, Minna J, Ihde D. A trial of combination chemotherapy followed by hormonal therapy for previously untreated metastatic carcinoma of the prostate. Journal Of Clinical Oncology 1986, 4: 1365-73. PMID: 2943877, DOI: 10.1200/jco.1986.4.9.1365.Peer-Reviewed Original ResearchMeSH KeywordsAdenocarcinomaAdultAgedAntineoplastic Combined Chemotherapy ProtocolsCisplatinClinical Trials as TopicCyclophosphamideDiethylstilbestrolDoxorubicinDrug Therapy, CombinationHumansMaleMedroxyprogesteroneMedroxyprogesterone AcetateMiddle AgedNeoplasm MetastasisOrchiectomyPilot ProjectsProstatic NeoplasmsConceptsHormonal therapyCombination chemotherapyStable diseasePartial responseHormonal manipulationResponse rateInitial hormonal therapySubsequent hormonal manipulationUntreated metastatic carcinomaObjective response rateIntensive combination chemotherapyNormal testosterone levelsMetastatic prostate cancerEfficacy of chemotherapyProgression of diseaseTime of deathSimilar chemotherapyEndocrine treatmentUntreated patientsObjective responseOverall survivalMetastatic carcinomaModest efficacySerum testosteroneProstate cancer
1985
The role of radiation therapy in the treatment of small cell lung cancer
Lichter A, Bunn P, Ihde D, Cohen M, Makuch R, Carney D, Johnston‐Early A, Minna J, Glatstein E. The role of radiation therapy in the treatment of small cell lung cancer. Cancer 1985, 55: 2163-2175. PMID: 2983875, DOI: 10.1002/1097-0142(19850501)55:9+<2163::aid-cncr2820551420>3.0.co;2-y.Peer-Reviewed Original ResearchMeSH KeywordsAntineoplastic Combined Chemotherapy ProtocolsBrain NeoplasmsCarcinoma, Small CellClinical Trials as TopicCombined Modality TherapyCyclophosphamideDoxorubicinFollow-Up StudiesHumansLomustineLung NeoplasmsMethotrexateProcarbazineProspective StudiesRadiotherapyRadiotherapy DosageRandom AllocationTime FactorsVincristineWhole-Body IrradiationConceptsSmall cell lung cancerLimited-stage small cell lung cancerProphylactic cranial irradiationCell lung cancerCranial irradiationThoracic irradiationLung cancerExtensive-stage small-cell lung cancerComplete response statusLimited-stage diseaseOptimal treatment approachCombination of chemotherapyMinimum of toxicityLong-term survivalSystemic irradiationSystemic chemotherapyAggressive therapyPartial responseCNS failureResponse statusTreatment protocolOngoing protocolRadiation therapyTreatment approachesPatientsApproaches to sample size estimation in the design of clinical trials—a review
Donner B, Makuch R. Approaches to sample size estimation in the design of clinical trials—a review. Statistics In Medicine 1985, 4: 247-247. PMID: 4023483, DOI: 10.1002/sim.4780040215.Peer-Reviewed Original Research
1983
Treatment of extensive stage small cell bronchogenic carcinoma Effects of variation in intensity of induction chemotherapy
Brower M, Ihde D, Johnston-Early A, Bunn P, Cohen M, Carney D, Makuch R, Matthews M, Radice P, Minna J. Treatment of extensive stage small cell bronchogenic carcinoma Effects of variation in intensity of induction chemotherapy. The American Journal Of Medicine 1983, 75: 993-1000. PMID: 6316784, DOI: 10.1016/0002-9343(83)90880-x.Peer-Reviewed Original ResearchConceptsHigh-intensity groupSmall cell bronchogenic carcinomaHigh-intensity therapyCell bronchogenic carcinomaInduction chemotherapyInduction courseInduction deathBronchogenic carcinomaDay 1Reversible congestive heart failureModerate-intensity therapyOverall median survivalCongestive heart failureSevere peripheral neuropathyModerate-intensity groupEvaluable patientsComplete remissionInduction therapyInfectious complicationsPartial remissionMedian survivalUntreated patientsComplete responseHeart failurePeripheral neuropathy
1982
Dose-response curves for predicting misonidazole-induced peripheral neuropathy.
Schwade J, Makuch R, Strong J, Glatstein E. Dose-response curves for predicting misonidazole-induced peripheral neuropathy. Journal Of The National Cancer Institute 1982, 66: 1743-50. PMID: 6288238.Peer-Reviewed Original ResearchConceptsPeripheral neuropathyLinear logistic regression analysisTotal cumulative doseLogistic regression analysisLogistic regression modelsDose-response curveCumulative AUCCumulative dosePlasma eliminationPlasma concentrationsNeuropathyLinear logistic regression modelPharmacokinetic parametersPharmacokinetic curveCancer treatmentToxic agentsRegression analysisAUCRegression modelsPatientsPharmacokineticsDoseMisonidazoleSample size requirements for comparing time-to-failure among k treatment groups
Makuch R, Simon R. Sample size requirements for comparing time-to-failure among k treatment groups. Journal Of Clinical Epidemiology 1982, 35: 861-867. PMID: 7142364, DOI: 10.1016/0021-9681(82)90051-0.Peer-Reviewed Original ResearchAdjusted survival curve estimation using covariates
Makuch R. Adjusted survival curve estimation using covariates. Journal Of Clinical Epidemiology 1982, 35: 437-443. PMID: 7042727, DOI: 10.1016/0021-9681(82)90058-3.Peer-Reviewed Original ResearchClinical Trials as TopicHumansL-Lactate DehydrogenaseMortalityRecurrenceSarcoma, EwingStatistics as TopicExperience of the National Cancer Institute (USA) in the treatment and biology of small cell lung cancer.
Minna J, Bunn P, Carney D, Cohen M, Cuttita F, Fosieck B, Gazdar A, Ihde D, Johnston-Early A, Matthews M, Makuch R, Oie H, Rosen S, Lichter A, Glatstein E. Experience of the National Cancer Institute (USA) in the treatment and biology of small cell lung cancer. Bulletin Du Cancer 1982, 69: 83-93. PMID: 6280795.Peer-Reviewed Original Research
1981
Adjuvant Immunotherapy or Chemotherapy for Malignant Melanoma: Preliminary Report of the National Cancer Institute Randomized Clinical Trial
Fisher R, Terry W, Hodes R, Rosenberg S, Makuch R, Gordon H, Fisher S. Adjuvant Immunotherapy or Chemotherapy for Malignant Melanoma: Preliminary Report of the National Cancer Institute Randomized Clinical Trial. Surgical Clinics Of North America 1981, 61: 1267-1277. PMID: 7031934, DOI: 10.1016/s0039-6109(16)42582-x.Peer-Reviewed Original ResearchMeSH KeywordsBCG VaccineCell LineClinical Trials as TopicFemaleHumansMaleMelanomaMitomycinsNeoplasm Recurrence, LocalNeuraminidaseRandom AllocationSemustineSkin NeoplasmsTime FactorsConceptsBacille Calmette-GuerinDisease-free intervalAdjuvant immunotherapyClinical trialsMalignant melanomaStage I malignant melanomaStage II melanomaSurvival of patientsNational Cancer InstituteOverall survivalPostsurgical patientsMethyl-CCNUCalmette-GuerinCancer InstituteMelanoma cellsMelanomaFurther treatmentPreliminary reportImmunotherapyChemotherapyPatientsTrialsSurvivalSignificant improvementPreoperative Cisplatin and Bleomycin Therapy in Head and Neck Squamous Carcinoma: Prognostic Factors for Tumor Response
Baker S, Makuch R, Wolf G. Preoperative Cisplatin and Bleomycin Therapy in Head and Neck Squamous Carcinoma: Prognostic Factors for Tumor Response. JAMA Otolaryngology - Head & Neck Surgery 1981, 107: 683-689. PMID: 6170283, DOI: 10.1001/archotol.1981.00790470031008.Peer-Reviewed Original ResearchMeSH KeywordsBleomycinCarcinoma, Squamous CellCisplatinClinical Trials as TopicDrug Therapy, CombinationHead and Neck NeoplasmsHumansLymphatic MetastasisPreoperative CarePrognosisConceptsPrimary tumorInduction chemotherapyPreoperative cisplatinPartial responseComplete responseRegional adenopathyResectable stage IIISquamous cell carcinomaNational Cancer Institute trialsNeck squamous carcinomaPretreatment patientsPrognostic factorsBleomycin therapyNeck nodesSquamous carcinomaTumor characteristicsCell carcinomaPrognostic indicatorTumor responseRegional nodesBleomycin sulfateOral cavityStage IIIResponse rateChemotherapyLaboratory parameters as an alternative to performance status in prognostic stratification of patients with small cell lung cancer.
Cohen M, Makuch R, Johnston-Early A, Ihde D, Bunn P, Fossieck B, Minna J. Laboratory parameters as an alternative to performance status in prognostic stratification of patients with small cell lung cancer. Journal Of The National Cancer Institute 1981, 65: 187-95. PMID: 6263468.Peer-Reviewed Original ResearchMeSH KeywordsCarcinoma, Small CellClinical Trials as TopicFemaleHemoglobinsHumansLung NeoplasmsMaleProbabilityPrognosisSerum AlbuminTime FactorsConceptsSmall cell lung cancerCell lung cancerLaboratory parametersLung cancerPerformance statusPrognostic factorsPrognostic indexSmall cell anaplastic lung cancerInfluential prognostic factorObjective prognostic indexPretreatment performance statusMajor prognostic factorSelf-limited eventAlpha-1 globulinCancer clinical trialsLaboratory resultsSignificant prognosticatorMedian followupAlbumin levelsMinimum followupPrognostic stratificationSurvival durationClinical trialsHigher hemoglobinTreatment response
1978
Thymosin in cancer patients: in vitro effects and correlations with clinical response to thymosin immunotherapy.
Chretien P, Lipson S, Makuch R, Kenady D, Cohen M, Minna J. Thymosin in cancer patients: in vitro effects and correlations with clinical response to thymosin immunotherapy. Journal Of The National Cancer Institute 1978, 62: 1787-90. PMID: 215307.Peer-Reviewed Original ResearchMeSH KeywordsCarcinoma, Small CellClinical Trials as TopicGlycoproteinsHumansImmunity, CellularIn Vitro TechniquesLeukocyte CountLung NeoplasmsThymosinThymus HormonesT-Lymphocytes