2021
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 patients
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 strokeResolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Thompson A, Walters M, Mapara M, Kwiatkowski J, Krishnamurti L, Aygun B, Kasow K, Rifkin-Zenenberg S, Schmidt M, DelCarpini J, Pierciey F, Miller A, Gallagher M, Chen R, Goyal S, Kanter J, Tisdale J. Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. Blood 2020, 136: 16-17. DOI: 10.1182/blood-2020-134940.Peer-Reviewed Original ResearchAcute chest syndromeVaso-occlusive crisisSickle cell diseaseVaso-occlusive eventsPain intensity scoresGroup C patientsAdverse eventsPain intensityBluebird BioC patientsLast visitBusulfan conditioningHemolysis markersTotal HbIntensity scoresAcute vaso-occlusive painBackground Sickle cell diseasePathophysiology of SCDVaso-occlusive pain crisesPopulation normsPatient-reported pain intensityAdvisory CommitteeGene therapy×109/LAbnormal sickle hemoglobinLentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study
Walters M, Kanter J, Kwiatkowski J, Krishnamurti L, Mapara M, Schmidt M, Miller A, Pierciey F, Bonner M, Huang W, Ribeil J, Thompson A, Tisdale J. Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study. Transplantation And Cellular Therapy 2020, 26: s1-s2. DOI: 10.1016/j.bbmt.2019.12.136.Peer-Reviewed Original ResearchPeripheral blood mononuclear cellsGroup C patientsAdverse eventsC patientsMedian HbTotal HbPlerixafor mobilizationNon-hematologic gradeGene therapySerious adverse eventsBlood mononuclear cellsLentiviral vectorsStrong therapeutic benefitFebrile neutropeniaSevere SCDConclusions PatientsGraft failureInitial patientsLast visitMononuclear cellsBusulfan conditioningHemolysis markersAutologous CD34Colony-forming unit assaysTherapeutic benefit
2019
Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study
Kanter J, Tisdale J, Mapara M, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Huang W, Ribeil J, Thompson A, Walters M. Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study. Blood 2019, 134: 990. DOI: 10.1182/blood-2019-128894.Peer-Reviewed Original ResearchGroup C patientsRed blood cell transfusionBlood cell transfusionAdverse eventsC patientsMedian Hb levelHb levelsBluebird BioHematopoietic stem cellsCell transfusionLast visitBusulfan conditioningTotal hemoglobinHSC collectionGene therapyNon-hematologic gradeSerious adverse eventsLentiviral vectorsSickle cell diseaseStrong therapeutic effectVector copy numberCancer Research CenterAdditional patient dataAdvisory CommitteeSevere SCDS1633 UPDATED RESULTS FROM THE HGB‐206 STUDY IN PATIENTS WITH SEVERE SICKLE CELL DISEASE TREATED UNDER A REVISED PROTOCOL WITH LENTIGLOBIN GENE THERAPY USING PLERIXAFOR‐MOBILISED HAEMATOPOIETIC STEM CELLS
Kanter J, Thompson A, Mapara M, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Huang W, Ribeil J, Walters M, Tisdale J. S1633 UPDATED RESULTS FROM THE HGB‐206 STUDY IN PATIENTS WITH SEVERE SICKLE CELL DISEASE TREATED UNDER A REVISED PROTOCOL WITH LENTIGLOBIN GENE THERAPY USING PLERIXAFOR‐MOBILISED HAEMATOPOIETIC STEM CELLS. HemaSphere 2019, 3: 754-755. DOI: 10.1097/01.hs9.0000564780.30358.eb.Peer-Reviewed Original ResearchSickle cell diseaseSevere sickle cell diseaseGroup C patientsAdverse eventsHaematopoietic stem cellsLast visitGroup CDP infusionC patientsRBC transfusionMonth followHb levelsCell diseaseVeno-occlusive liver diseaseHSC collectionRed blood cell transfusionGene therapyX109/LBlood cell transfusionTotal HbVaso-occlusive eventsLentiviral vectorsInclusion of adolescentsStem cellsBusulfan levels
2018
Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
Tisdale J, Kanter J, Mapara M, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Shi W, Ribeil J, Asmal M, Thompson A, Walters M. Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study. Blood 2018, 132: 1026. DOI: 10.1182/blood-2018-99-113480.Peer-Reviewed Original ResearchSevere sickle cell diseaseSickle cell diseaseNon-cardiac chest painVaso-occlusive painAdverse eventsHb levelsBluebird BioHematopoietic stem cellsHSC collectionDP infusionChest painMyeloablative conditioningPlatelet engraftmentCell diseaseGroup CAdvisory CommitteeMedical directorsGene therapyMonths of transfusionNon-hematologic gradeGroup C patientsSerious adverse eventsSubstantial clinical benefitLentiviral vectors