Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Walters M, Tisdale J, Mapara M, Krishnamurti L, Kwiatkowski J, Aygun B, Kasow K, Rifkin-Zenenberg S, Jaroscak J, Garbinsky D, Chirila C, Gallagher M, Zhang X, Ho P, Thompson A, Kanter J. Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. Blood 2021, 138: 7. DOI: 10.1182/blood-2021-146905.Peer-Reviewed Original ResearchPain intensity numeric rating scaleNumeric rating scaleVaso-occlusive eventsSickle cell diseasePatient-reported outcomesCurrent equity holderMonth 24Severe vaso-occlusive eventsBaseline scoresQuality of lifeBluebird BioPopulation normsException of anxietyComplete resolutionGroup CSmall sample size limits interpretationMean pain interference scoreSevere sickle cell diseaseUS general population normsAdvisory CommitteePatient-reported QOL outcomesMean pain interferencePain interference scoresPain intensity scoresGroup C patientsPolyclonality 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