2024
Deciding between Multiple Curative Options in Sickle Cell Disease: Cost-Effectiveness of Non-Myeloablative/Reduced Intensity Conditioning Haploidentical Allo-HSCT Vs Gene Therapy Vs Standard of Care in Adult Patients with Sickle Cell Disease
Chetlapalli K, Butt A, Ito S, Wang D, Calhoun C, Krishnamurti L, Pandya A, Goshua G. Deciding between Multiple Curative Options in Sickle Cell Disease: Cost-Effectiveness of Non-Myeloablative/Reduced Intensity Conditioning Haploidentical Allo-HSCT Vs Gene Therapy Vs Standard of Care in Adult Patients with Sickle Cell Disease. Blood 2024, 144: 601-601. DOI: 10.1182/blood-2024-203234.Peer-Reviewed Original ResearchChronic graft-versus-host diseaseSickle cell diseaseQuality-adjusted life yearsPost-transplant cyclophosphamideReduced intensity conditioningAllo-HSCTGene therapyCurative optionIncremental net monetary benefitProbabilistic sensitivity analysesMyeloablative conditioningHaploidentical donorsCell diseaseAdult patientsExpansion of donor poolUS health system perspectiveCenter for International Blood and Marrow Transplant ResearchGraft-versus-host diseaseEuropean Society for BloodSickle cell disease severityUS commercially insured patientsCost-effective therapeutic optionCost-effectiveness analysisAllo-HSCT outcomesDonor allo-HSCTPreservation of Pulmonary Function Following Hematopoietic Cell Transplant for Sickle Cell Disease: A STAR Study
Horan F, Bendiak G, Abraham A, Liu K, Gillespie S, Chellapandian D, Shah R, Bhatia M, Chaudhury S, Eckrich M, Jaroscak J, Kasow K, Krajewski J, Ngwube A, Horan J, Krishnamurti L, Shenoy S, Guilcher G, Stenger E. Preservation of Pulmonary Function Following Hematopoietic Cell Transplant for Sickle Cell Disease: A STAR Study. Transplantation And Cellular Therapy 2024, 30: s45-s46. DOI: 10.1016/j.jtct.2023.12.079.Peer-Reviewed Original ResearchHematopoietic cell transplantationPulmonary function testsPost-HCTYears post-HCTSickle cell diseaseAmerican Thoracic SocietyCell transplantationPulmonary dysfunctionPulmonary functionCurative hematopoietic cell transplantationPreservation of pulmonary functionCell diseaseGroup of SCD patientsPulmonary function test dataPost-HCT patientsProgressive pulmonary dysfunctionLung volume valuesSevere clinical phenotypeMyeloablative conditioningMedian followHLA matchingMedian ageNo significant differencePFT resultsPre-HCTA phase 2 trial of CD24Fc for prevention of graft-versus-host disease
Magenau J, Jaglowski S, Uberti J, Farag S, Riwes M, Pawarode A, Anand S, Ghosh M, Maciejewski J, Braun T, Devenport M, Lu S, Banerjee B, DaSilva C, Devine S, Zhang M, Burns L, Liu Y, Zheng P, Reddy P. A phase 2 trial of CD24Fc for prevention of graft-versus-host disease. Blood 2024, 143: 21-31. PMID: 37647633, PMCID: PMC10934299, DOI: 10.1182/blood.2023020250.Peer-Reviewed Original ResearchConceptsAcute graft-versus-host diseaseGraft-versus-host diseaseHematopoietic stem cell transplantationDose-limiting toxicityMUD hematopoietic stem cell transplantationGVHD-free survivalMultidose regimenMyeloablative conditioningHematologic malignanciesHuman leukocyte antigen-matched unrelated donorsPrevention of graft-versus-host diseaseAllogeneic hematopoietic stem cell transplantationCalcineurin inhibitor-based prophylaxisMatched ControlsDose-escalation phaseSingle-dose regimensStem cell transplantationPhase 2 trialSustained drug exposureAssociated with higher ratesDamage-associated molecular patternsExpansion cohortDouble-blindPlacebo-ControlledUnrelated donor
2023
Allogeneic stem cell transplant for treatment of mycosis fungoides and Sezary syndrome: a systematic review and meta-analysis
Goyal A, O’Leary D, Foss F. Allogeneic stem cell transplant for treatment of mycosis fungoides and Sezary syndrome: a systematic review and meta-analysis. Bone Marrow Transplantation 2023, 59: 41-51. PMID: 37853164, DOI: 10.1038/s41409-023-02122-0.Peer-Reviewed Original ResearchConceptsGraft-versus-host diseaseReduced-intensity conditioningDonor lymphocyte infusionAllo-HSCTStem cell transplantationMyeloablative conditioningSezary syndromeOverall survivalMycosis fungoidesCell transplantationRates of graft-versus-host diseaseTreated with donor lymphocyte infusionsChronic graft-versus-host diseaseAllogeneic hematopoietic stem cell transplantationMeta-analysisAdvanced-stage mycosis fungoidesAssociated with superior OSGraft-versus-lymphoma effectOutcomes of allo-HSCTAllogeneic stem cell transplantationHematopoietic stem cell transplantationTreatment of mycosis fungoidesGraft-versus-lymphomaNon-relapse mortalityProgression-free survivalFemale Reproductive Health Outcomes after Hematopoietic Cell Transplantation for Sickle Cell Disease: Is Reduced Intensity Better Than Myeloablative Conditioning?
Meacham L, George S, Veludhandi A, Pruett M, Haight A, Arnold S, Elchuri S, Stenger E, Krishnamurti L. Female Reproductive Health Outcomes after Hematopoietic Cell Transplantation for Sickle Cell Disease: Is Reduced Intensity Better Than Myeloablative Conditioning? Transplantation And Cellular Therapy 2023, 29: 531.e1-531.e4. PMID: 37169288, DOI: 10.1016/j.jtct.2023.05.004.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationReduced-intensity conditioningPremature ovarian insufficiencySickle cell diseaseNormal AMH levelsMyeloablative conditioningAnti-Müllerian hormoneOvarian outcomeAMH levelsCell transplantationCell diseaseRIC HCTFollicle-stimulating hormone levelsPediatric oncology patientsRisk of infertilityMIU/mLStudy 2 patientsReproductive health outcomesMelphalan regimenConditioning regimenGonadal damageOvarian damageConditioning regimensOvarian reserveRIC regimensSurvival of TP53-mutated acute myeloid leukemia patients receiving allogeneic stem cell transplantation after first induction or salvage therapy: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)
Badar T, Atallah E, Shallis R, Saliba A, Patel A, Bewersdorf J, Grenet J, Stahl M, Duvall A, Burkart M, Palmisiano N, Bradshaw D, Kubiak M, Dinner S, Goldberg A, Abaza Y, Murthy G, Kota V, Litzow M. Survival of TP53-mutated acute myeloid leukemia patients receiving allogeneic stem cell transplantation after first induction or salvage therapy: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND). Leukemia 2023, 37: 799-806. PMID: 36807649, DOI: 10.1038/s41375-023-01847-7.Peer-Reviewed Original ResearchConceptsEvent-free survivalAllo-HSCTOverall survivalChronic GVHDAllogeneic hematopoietic stem cell transplantAllogeneic stem cell transplantationMedian event-free survivalHematopoietic stem cell transplantAcute myeloid leukemia patientsMedian overall survivalPost allo-HSCTReduced intensity conditioningStem cell transplantStem cell transplantationLong-term outcomesMyeloid leukemia patientsMulti-center studyAcute graftComplete remissionHost diseaseSalvage therapyComplex cytogeneticsMyeloablative conditioningMedian ageCell transplantFeasibility and Toxicity of Full-Body Volumetric Modulated Arc Therapy Technique for High-Dose Total Body Irradiation
Keit E, Liveringhouse C, Figura N, Weygand J, Sandoval M, Garcia G, Peters J, Nieder M, Faramand R, Khimani F, Kim S, Robinson T, Johnstone P, Penagaricano J, Latifi K. Feasibility and Toxicity of Full-Body Volumetric Modulated Arc Therapy Technique for High-Dose Total Body Irradiation. Technology In Cancer Research & Treatment 2023, 22: 15330338231180779. PMID: 37287260, PMCID: PMC10272663, DOI: 10.1177/15330338231180779.Peer-Reviewed Original ResearchConceptsHigh-dose total body irradiationTotal body irradiationHelical tomotherapyBody irradiationDosimetric outcomesLower lung dosesMucosal sparing techniquesArc therapy (VMAT) techniqueMucosal sparingHead-first positionMyeloablative conditioningDaily fractionsLung dosesAcute leukemiaPrescription doseHT plansDose goalsFFS plansOAR sparingPatientsLow dosesArc therapyUnique protocolDosesVMAT plans
2022
Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance
Stenger E, Xiang Y, Wetzel M, Gillespie S, Chellapandian D, Shah R, Arnold S, Bhatia M, Chaudhury S, Eckrich M, Kanter J, Kasow K, Krajewski J, Nickel R, Ngwube A, Olson T, Rangarajan H, Wobma H, Guilcher G, Horan J, Krishnamurti L, Shenoy S, Abraham A. Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance. Transplantation And Cellular Therapy 2022, 29: 47.e1-47.e10. PMID: 36273784, DOI: 10.1016/j.jtct.2022.10.012.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseOrgan functionMultivariable analysisBrain magnetic resonance imagingCentral nervous system indicationsPost-HCT patientsRelated bone marrowSevere acute GVHDPredictors of dysfunctionLong-term survivalMagnetic resonance imagingSevere clinical phenotypeAcute GVHDChronic graftIntense conditioningHost diseaseMyeloablative conditioningNeurologic eventsOvert strokeRetrospective cohortMedian ageOrgan dysfunctionCardiac dysfunctionCell transplantation
2021
Non-Relapse Mortality in TP53-Mutated MDS/AML - a Multi-Center Collaborative Study
Byrne M, Kurian T, Patel D, Tamari R, Hong S, Abdelhakim H, Klein V, Rojas P, Madhavan R, Kent A, Logan A, Lee C, Husnain M, Manning B, Tschernia N, Dias A, Margalski D, Goldenson B, Byrne N, Chen H, Petrova-Drus K, Sengsayadeth S, Goodman A, Howard D, Wood W, Gill S, Jimenez A, Gutman J, Gowda L, Metheny L, Bhatnagar B, Hamilton B, Mishra A, Savona M. Non-Relapse Mortality in TP53-Mutated MDS/AML - a Multi-Center Collaborative Study. Blood 2021, 138: 2922. DOI: 10.1182/blood-2021-154151.Peer-Reviewed Original ResearchNon-relapse mortalityMDS/AMLChronic GVHDPost-HCT mortalityPost-HCT relapseOverall survivalRelapse/progressionShorter overall survivalBM chimerismComplex karyotypeImproved OSMyeloablative conditioningHigher KPSMultivariate analysisSecondary MDS/AMLAdvisory CommitteeLarge multi-institutional cohortMDS/AML patientsMulti-center collaborative studyLow NRM ratePost-HCT outcomesAllogeneic HCT recipientsSpeakers bureauYear overall survivalInferior overall survival
2020
Pre-transplant molecular minimal residual disease (MMRD) is associated with inferior outcomes in patients with acute myeloid leukemia undergoing allogeneic stem cell transplantation.
Husnain M, Komanduri K, Ramdial J, Lekakis L, Wang T, Goodman M, Pereira D, Beitinjaneh A, Carollo D, Ali R, Mohammed Y, Arshad J, Byrnes D, Saul E, Aguirre L, Jimenez A. Pre-transplant molecular minimal residual disease (MMRD) is associated with inferior outcomes in patients with acute myeloid leukemia undergoing allogeneic stem cell transplantation. Journal Of Clinical Oncology 2020, 38: 7547-7547. DOI: 10.1200/jco.2020.38.15_suppl.7547.Peer-Reviewed Original ResearchMolecular minimal residual diseaseAllogeneic stem cell transplantationMinimal residual diseaseStem cell transplantationPredictors of post-transplant outcomesPost-transplant outcomesResidual diseaseCell transplantationELN criteriaAssociated with inferior outcomesCohort of AML patientsAdverse risk patientsPoor-risk categoriesPost-transplant relapseTwo-year OSDiagnosis of AMLHigh-risk patientsAcute myeloid leukemiaCytogenetic/molecular dataDTA mutationsInferior LFSMorphologic remissionMRD statusMyeloablative conditioningTransplant variablesTargeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Leads to High Rates of Allogeneic Transplantation and Successful Engraftment in Older Patients with Active, Relapsed or Refractory (rel/ref) AML after Failure of Chemotherapy and Targeted Agents: Preliminary Midpoint Results from the Prospective, Randomized Phase 3 Sierra Trial
Gyurkocza B, Nath R, Stiff P, Agura E, Litzow M, Tomlinson B, Choe H, Abhyankar S, Seropian S, Chen G, Hari P, Al-Kadhimi Z, Foran J, Orozco J, van Besien K, Sabloff M, Kebriaei P, Abboud C, Levy M, Lazarus H, Giralt S, Berger M, Reddy V, Pagel J. Targeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Leads to High Rates of Allogeneic Transplantation and Successful Engraftment in Older Patients with Active, Relapsed or Refractory (rel/ref) AML after Failure of Chemotherapy and Targeted Agents: Preliminary Midpoint Results from the Prospective, Randomized Phase 3 Sierra Trial. Transplantation And Cellular Therapy 2020, 26: s32-s33. DOI: 10.1016/j.bbmt.2019.12.575.Peer-Reviewed Original ResearchHematopoietic cell transplantationSalvage therapyOlder patientsCC armConventional careCC patientsConventional hematopoietic cell transplantationGrade 3 febrile neutropeniaAllogeneic hematopoietic cell transplantationPre-treated patientsPhase 3 trialFailure of chemotherapyYears of ageFebrile neutropeniaInduction therapyActive diseaseMyeloablative conditioningRefractory AMLBackground PatientsBM blastsInfusion reactionsMethods PatientsTransplant candidatesAllogeneic transplantationCurative therapyIncidence, Risk Factors, and Outcomes of Patients Who Develop Mucosal Barrier Injury–Laboratory Confirmed Bloodstream Infections in the First 100 Days After Allogeneic Hematopoietic Stem Cell Transplant
Dandoy CE, Kim S, Chen M, Ahn KW, Ardura MI, Brown V, Chhabra S, Diaz MA, Dvorak C, Farhadfar N, Flagg A, Ganguly S, Hale GA, Hashmi SK, Hematti P, Martino R, Nishihori T, Nusrat R, Olsson RF, Rotz SJ, Sung AD, Perales MA, Lindemans CA, Komanduri KV, Riches ML. Incidence, Risk Factors, and Outcomes of Patients Who Develop Mucosal Barrier Injury–Laboratory Confirmed Bloodstream Infections in the First 100 Days After Allogeneic Hematopoietic Stem Cell Transplant. JAMA Network Open 2020, 3: e1918668. PMID: 31913492, PMCID: PMC6991246, DOI: 10.1001/jamanetworkopen.2019.18668.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantAllogeneic hematopoietic stem cell transplantMucosal barrier injury laboratoryMBI-LCBIOutcomes of patientsStem cell transplantBloodstream infectionsCumulative incidenceCell transplantRisk factorsDay 100First allogeneic hematopoietic stem cell transplantMarrow Transplant Research databaseHost disease (GVHD) prophylaxisLansky performance statusTransplant-related mortalityOne-year mortalityTranslocation of bacteriaCord blood graftsCause of deathChronic graftConsecutive pediatricHost diseaseMyeloablative conditioningAdult patients
2019
Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206
Mapara M, Tisdale J, Kanter J, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Shi W, Ribeil J, Asmal M, Thompson A, Walters M. Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206. Transplantation And Cellular Therapy 2019, 25: s64-s65. DOI: 10.1016/j.bbmt.2018.12.147.Peer-Reviewed Original ResearchSickle cell diseaseGrade 3 adverse eventsAdverse eventsHematopoietic stem cellsGrp BC patientsCell diseaseSevere sickle cell diseaseGene therapyNon-hematologic gradeVaso-occlusive painPhase 1 studyLentiviral vectorsAutologous hematopoietic stem cellsGrp CFebrile neutropeniaMyeloablative conditioningClinical effectsHb levelsLast visitBusulfan conditioningAutologous CD34Treatment characteristicsMethods AdultsPatients
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
2016
Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease
Kanter J, Walters M, Hsieh M, Krishnamurti L, Kwiatkowski J, Kamble R, von Kalle C, Kuypers F, Cavazzana M, Leboulch P, Joseney-Antoine M, Asmal M, Thompson A, Tisdale J. Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease. Blood 2016, 128: 1176. DOI: 10.1182/blood.v128.22.1176.1176.Peer-Reviewed Original ResearchSevere sickle cell diseaseSevere SCDBone marrow harvestAdverse eventsSickle cell diseaseCell doseBluebird BioReplication-competent lentivirusHematopoietic stem cellsMyeloablative conditioningPeripheral bloodCell diseaseGrade 3 adverse eventsAdvisory CommitteeMedical directorsPhase 1/2 clinical studyTransfusion-dependent β-thalassemiaSerious adverse eventsLong-term complicationsVector copy numberStart of conditioningSymptoms 1 yearIntegration site analysisGene therapyDP infusion
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