2024
Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease
Walters M, Eapen M, Liu Y, El Rassi F, Waller E, Levine J, Strouse J, Antin J, Parikh S, Bakshi N, Dampier C, Jaroscak J, Bergmann S, Wong T, Kota V, Pace B, Lekakis L, Lulla P, Nickel R, Kasow K, Popat U, Smith W, Yu L, DiFronzo N, Geller N, Kamani N, Klings E, Hassell K, Mendizabal A, Sullivan K, Neuberg D, Krishnamurti L. Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease. Blood Advances 2024 PMID: 39471440, DOI: 10.1182/bloodadvances.2024013926.Peer-Reviewed Original ResearchHematopoietic cell transplantationVaso-occlusive painStandard of careSickle cell diseaseCell transplantationCell diseaseSCD-related eventsHLA-matched siblingTransplant-related mortalityPeriod of follow-upSurvival 2 yearsIntent-to-treat principleComparison of survivalDisease-modifying therapiesDisease-related mortalityUnrelated donorPrimary endpointCurative potentialTreatment armsDonor searchFollow-upCompare outcomesEligibility confirmationFunctional outcomesSecondary outcomes
2022
Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials
Krishnamurti L, Neuberg D, Sullivan K, Smith S, Eapen M, Walters M. Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials. Transplantation And Cellular Therapy 2022, 29: 217-221. PMID: 36270432, PMCID: PMC10539686, DOI: 10.1016/j.jtct.2022.10.008.Peer-Reviewed Original ResearchConceptsBone marrow transplantationSickle cell diseaseMarrow transplantationNonmalignant diseasesCell diseaseAdult sickle cell diseaseMyeloablative bone marrow transplantationSingle-arm feasibility studyYoung adultsCellular therapy trialsMulticenter National InstituteRegimen-related toxicitySingle-arm studyFrequency of HLACoronavirus disease 2019 (COVID-19) pandemicAcademic medical centerDisease 2019 pandemicShort-term endpointsBMT centersBMT regimenCare armPain episodesPrimary endpointStandard careCurative therapy
2016
Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation
Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle J, Zecca M, Walters M, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier J, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood 2016, 129: 1548-1556. PMID: 27965196, PMCID: PMC5356458, DOI: 10.1182/blood-2016-10-745711.Peer-Reviewed Original ResearchConceptsEvent-free survivalSickle cell diseaseGraft failureCell transplantationHLA-identical sibling transplantationReduced-intensity conditioning regimensHLA-identical sibling transplantsHematopoietic stem cell transplantationMyeloablative conditioning regimenHematopoietic cell transplantationStem cell transplantationCox regression modelMarrow Transplant ResearchBenefit of transplantStem cell sourceSibling transplantationConditioning regimenPrimary endpointConditioning regimensMost patientsOverall survivalSibling transplantsCurative therapyInternational BloodMedian age
2013
A double-blind, randomized, multicenter phase 2 study of prasugrel versus placebo in adult patients with sickle cell disease
Wun T, Soulieres D, Frelinger A, Krishnamurti L, Novelli E, Kutlar A, Ataga K, Knupp C, McMahon L, Strouse J, Zhou C, Heath L, Nwachuku C, Jakubowski J, Riesmeyer J, Winters K. A double-blind, randomized, multicenter phase 2 study of prasugrel versus placebo in adult patients with sickle cell disease. Journal Of Hematology & Oncology 2013, 6: 17. PMID: 23414938, PMCID: PMC3585853, DOI: 10.1186/1756-8722-6-17.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAnemia, Sickle CellBiomarkers, TumorCell Adhesion MoleculesDouble-Blind MethodFemaleFollow-Up StudiesHumansMaleMicrofilament ProteinsMiddle AgedPainPhosphoproteinsPiperazinesPlatelet ActivationPlatelet Aggregation InhibitorsPrasugrel HydrochloridePrognosisPurinergic P2Y Receptor AntagonistsThiophenesYoung AdultConceptsSickle cell diseaseHemorrhagic eventsAdult patientsAntiplatelet agentsSCD patientsCell diseaseP-selectinMedical interventionsMulticenter phase 2 studyPlatelet surface P-selectinMethodsThe primary endpointPlatelet activation biomarkersSafety of prasugrelSerious hemorrhagic eventsPlacebo-controlled studyPhase 2 studyVivo platelet activationSoluble P-selectinSurface P-selectinThienopyridine antiplatelet agentPain ratePrasugrel armPrimary endpointActivation biomarkersStudy arms
2011
Non-Cardiopulmonary Factors Affecting the Six-Minute Walk Distance in Patients with Sickle Cell Disease: Results From the Walk-PHaSST Study
Goel R, Hassell K, Machado R, Barst R, Yovetich N, Kato G, Gordeuk V, Little J, Gibbs J, Schraufnagel D, Girgis R, Rosenzweig E, Morris C, Badesch D, Lanzkron S, Onyekwere O, Nouraie M, Castro O, Sachdev V, Waclawiw M, Woolson R, Goldsmith J, Gladwin M, Krishnamurti L. Non-Cardiopulmonary Factors Affecting the Six-Minute Walk Distance in Patients with Sickle Cell Disease: Results From the Walk-PHaSST Study. Blood 2011, 118: 1074. DOI: 10.1182/blood.v118.21.1074.1074.Peer-Reviewed Original ResearchTR jet velocitySickle cell diseaseSix-minute walk distanceAvascular necrosisMultivariable linear regressionPulmonary hypertensionCell diseasePrimary endpointWalk distanceChronic painSCD patientsSevere genotypeAmerican Thoracic Society guidelinesSix-minute walk testSelf-reported medical historyThoracic Society guidelinesMulti-center studySided p valueGreater predictive valueCardiopulmonary factorsOral sildenafilPain 2NT-proBNPExercise capacityWalk test