2021
Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)
Tisdale J, Thompson A, Mapara M, Kwiatkowski J, Krishnamurti L, Aygun B, Kasow K, Rifkin-Zenenberg S, Schmidt M, Pierciey F, Whitney D, Rogers C, Nnamani M, Foos M, Miller A, Zhang X, Lynch J, Walters M, Kanter J, Bonner M. Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT). Blood 2021, 138: 561. DOI: 10.1182/blood-2021-147760.Peer-Reviewed Original ResearchAcute myeloid leukemiaVaso-occlusive eventsCurrent equity holderSevere vaso-occlusive eventsGroup AGroup CMonth 6Clinical outcomesBluebird BioClinical benefitBusulfan conditioningCell doseRisk of AMLAutologous stem cell transplantTricuspid regurgitant jet velocityAdvisory CommitteeDriver mutationsLentiviral vectorsAML driver mutationsOpioid withdrawal syndromeSerious adverse eventsRegurgitant jet velocityFavorable clinical outcomeConsultancy feesStem cell transplant
2020
Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Kanter J, Tisdale J, Mapara M, Kwiatkowski J, Krishnamurti L, Chen R, Gallagher M, Ding Y, Goyal S, Paramore C, Thompson A, Walters M. Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. Blood 2020, 136: 10. DOI: 10.1182/blood-2020-136193.Peer-Reviewed Original ResearchSickle cell diseaseAcute chest syndromeNumeric rating scalePatient-reported outcomesMonth 12Pain intensitySleep disturbance domainsBaseline scoresPhysical functionBluebird BioPopulation normsMeaningful improvementsMonth 6Pain intensity numeric rating scalePROMIS-57T-scorePatient-reported HRQoL outcomesUS general population normsAdvisory CommitteePain interference scoresGene therapyGroup C patientsCohort of patientsHealth-related qualityHistory of stroke