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
2019
A Phase II Trial to Compare Allogeneic Transplant Vs. Standard of Care for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 1503
Eapen M, Neuberg D, Mendizabal A, Stevenson K, Antin J, DiFronzo N, Rassi F, Lulla P, Waller E, Garrison J, Smith S, Sullivan K, Walters M, Krishnamurti L. A Phase II Trial to Compare Allogeneic Transplant Vs. Standard of Care for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 1503. Blood 2019, 134: 4592. DOI: 10.1182/blood-2019-126793.Peer-Reviewed Original ResearchSickle cell diseaseHematopoietic cell transplantationSevere sickle cell diseaseUnrelated donorsUnrelated donor hematopoietic cell transplantationDonor hematopoietic cell transplantationCurrent phase II trialMyeloablative conditioning regimenPhase II trialConditioning regimenII trialCurative treatmentCell transplantationTreatment optionsCurrent therapiesCell diseasePilot trialRare diseaseSuitable donorStudy designHLAAge 15Young adultsTrialsGold standard
2018
From trust to skepticism: An in-depth analysis across age groups of adults with sickle cell disease on their perspectives regarding hydroxyurea
Sinha C, Bakshi N, Ross D, Krishnamurti L. From trust to skepticism: An in-depth analysis across age groups of adults with sickle cell disease on their perspectives regarding hydroxyurea. PLOS ONE 2018, 13: e0199375. PMID: 29949647, PMCID: PMC6021071, DOI: 10.1371/journal.pone.0199375.Peer-Reviewed Original ResearchConceptsSickle cell diseaseGroup 1 participantsGroup 2 participantsCell diseaseSide effectsAdult SCD patientsPatient-related factorsPotential side effectsAdult patientsSCD patientsHU usePatients' attitudesAge 31Age groupsAge 18PatientsYoung adultsEfficacySignificant differencesAdultsEffective alternativeDiseaseRiskParticipantsQualitative interviews
2015
Development, Content Validity, and User Review of a Web-based Multidimensional Pain Diary for Adolescent and Young Adults With Sickle Cell Disease
Bakshi N, Stinson J, Ross D, Lukombo I, Mittal N, Joshi S, Belfer I, Krishnamurti L. Development, Content Validity, and User Review of a Web-based Multidimensional Pain Diary for Adolescent and Young Adults With Sickle Cell Disease. The Clinical Journal Of Pain 2015, 31: 580-590. PMID: 25565585, DOI: 10.1097/ajp.0000000000000195.Peer-Reviewed Original ResearchConceptsSickle cell diseasePain diaryHealth care utilizationElectronic pain diaryYoung adultsCare utilizationCell diseasePoor health-related qualityContent validityVaso-occlusive painDaily pain diarySeverity of painHealth-related qualityPain burdenModification of itemsPain experiencePainPatientsEnd-user reviewsElectronic diaryDiseaseAdultsAdolescentsPsychometric propertiesDiary