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
Shared Decision Making or Physician Advocate for a Particular Treatment Option: A Spectrum of Approaches to Decision Making about Disease Modifying Therapies in Sickle Cell Disease
Bakshi N, Sinha C, Ross D, Khemani K, Loewenstein G, Krishnamurti L. Shared Decision Making or Physician Advocate for a Particular Treatment Option: A Spectrum of Approaches to Decision Making about Disease Modifying Therapies in Sickle Cell Disease. Blood 2016, 128: 4757. DOI: 10.1182/blood.v128.22.4757.4757.Peer-Reviewed Original ResearchBone marrow transplantationSickle cell diseaseChronic blood transfusionsTherapeutic optionsTreatment optionsCurrent clinical conditionPhysician narrativesTherapeutic planCell diseasePatient valuesTreatment planAdult sickle cell diseaseDisease-modifying therapeutic optionsPatient’s current clinical conditionLong-term side effectsDecisional needsDisease-Modifying TherapiesMajority of patientsGroup of patientsPatient-related factorsAvailable treatment optionsGood clinical evidenceProgression of diseaseParticular treatment optionPatients' decision processes