1996
Gene Therapy in the United States: A Five-Year Status Report
Ross G, Erickson R, Knorr D, Motulsky A, Parkman R, Samulski J, Straus S, Smith B. Gene Therapy in the United States: A Five-Year Status Report. Human Gene Therapy 1996, 7: 1781-1790. PMID: 8886849, DOI: 10.1089/hum.1996.7.14-1781.Peer-Reviewed Original ResearchConceptsInstitutional review boardLocal institutional review boardFDA approvalHuman gene transfer trialsPhase I/IIGene transfer studiesPhase II studyPhase III studyRecruitment of patientsGene therapyYears of ageGene transfer trialsFinal FDA approvalII studyIII studyTotal patientsMedian timeFirst patientAnatomic distributionExperimental therapiesUnexpected toxicitiesNineteen studiesPatientsAutopsy rateAcademic centers
1988
Phase I/II study of recombinant human granulocyte-macrophage colony-stimulating factor in aplastic anemia and myelodysplastic syndrome.
Antin J, Smith B, Holmes W, Rosenthal D. Phase I/II study of recombinant human granulocyte-macrophage colony-stimulating factor in aplastic anemia and myelodysplastic syndrome. Blood 1988, 72: 705-13. PMID: 3042046, DOI: 10.1182/blood.v72.2.705.bloodjournal722705.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedAnemia, AplasticBlood Cell CountBone MarrowCell DivisionColony-Stimulating FactorsDose-Response Relationship, DrugDrug EvaluationFemaleGranulocyte-Macrophage Colony-Stimulating FactorGrowth SubstancesHumansMaleMiddle AgedMyelodysplastic SyndromesRecombinant ProteinsReticulocytesConceptsPhase I/II studyRecombinant human granulocyte-macrophage colony-stimulating factorAplastic anemiaGranulocyte-macrophage colony-stimulating factorMyelodysplastic syndromeColony-stimulating factorHuman granulocyte-macrophage colony-stimulating factorII studyReticulocyte countErythrocyte transfusion requirementsLow-grade feverMyalgia/arthralgiaMicrograms/m2Four-hour intravenous infusionNumber of eosinophilsGM-CSF therapyImmature myeloid cellsLow back discomfortTransfusion requirementsBlood countIntravenous infusionPlatelet countGranulocyte countMonocyte countPatientsPhase I/II study of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in bone marrow failure.
Antin JH, Smith BR, Holmes W, David RN, Rosenthal S. Phase I/II study of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in bone marrow failure. Behring Institute Mitteilungen 1988, 149-53. PMID: 3071331.Peer-Reviewed Original ResearchConceptsPhase I/II studyMicrograms/m2Bone marrow failureII studyMarrow failureGM-CSFSevere bone marrow failureErythrocyte transfusion requirementsMyalgia/arthralgiaLow-grade feverRecombinant human granulocyte-macrophage colony-stimulating factorGranulocyte-macrophage colony-stimulating factorSevere marrow failureLow back discomfortColony-stimulating factorHuman granulocyte-macrophage colony-stimulating factorRecombinant human GM-CSFGrade feverTransfusion requirementsIntravenous infusionMyelodysplastic syndromePlatelet countAplastic anemiaGranulocyte countMonocyte countPhase I/II Study of Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor in Aplastic Anemia and Myelodysplastic Syndrome
Antin J, Smith B, Holmes W, Rosenthal D. Phase I/II Study of Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor in Aplastic Anemia and Myelodysplastic Syndrome. Blood 1988, 72: 705-713. DOI: 10.1182/blood.v72.2.705.705.Peer-Reviewed Original ResearchPhase I/II studyAplastic anemiaMyelodysplastic syndromeII studyReticulocyte countErythrocyte transfusion requirementsLow-grade feverMyalgia/arthralgiaMicrograms/m2Four-hour intravenous infusionRecombinant human granulocyte-macrophage colony-stimulating factorNumber of eosinophilsGM-CSF therapyImmature myeloid cellsGranulocyte-macrophage colony-stimulating factorRecombinant human granulocyte-macrophage colonyLow back discomfortColony-stimulating factorHuman granulocyte-macrophage colony-stimulating factorGranulocyte-macrophage colonyTransfusion requirementsIntravenous infusionPlatelet countBlood countGranulocyte count