2023
Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507
Kassim A, Walters M, Eapen M, Ritzau N, Smith M, Solh M, McKinney C, Nieder M, Ross M, Kent M, Abusin G, Mallhi K, Silva J, Shaughnessy P, Kanter J, Haines H, Farah R, Khaled Y, Abraham A, Bollard C, Cooke K, de La Fuente J, Hanna R, Horowitz M, Jordan L, Krishnamurti L, Leifere E, Mahadeo K, Shenoy S, Ritzau N, DeBaun M, Brodsky R. Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507. Blood 2023, 142: lba-4. DOI: 10.1182/blood-2023-192022.Peer-Reviewed Original ResearchPost-transplant cyclophosphamideEvent-free survivalBone marrow transplantSickle cell diseaseSevere sickle cell diseaseTotal body irradiationGraft failureAcute GVHDPost-BMTCell diseaseDay 100Multi-center phase II trialFirst year post-BMTHaploidentical bone marrow transplantVaso-occlusive pain episodesHaploidentical bone marrow transplantationHematopoietic stem cell transplantationChronic transfusion regimenDurable donor engraftmentHemoglobin SS diseaseAcute chest syndromePrimary graft failureSecondary graft failureEnd-organ toxicityPhase II trial
2020
Assessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease
Bakshi N, Katoch D, Sinha C, Ross D, Quarmyne M, Loewenstein G, Krishnamurti L. Assessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease. JAMA Network Open 2020, 3: e206742. PMID: 32469414, PMCID: PMC7260617, DOI: 10.1001/jamanetworkopen.2020.6742.Peer-Reviewed Original ResearchConceptsBone marrow transplantCaregivers of patientsSickle cell diseasePatient decision aidMarrow transplantMedian ageCurative treatmentCell diseaseGroup 2Group 1Caregiver attitudesNovel disease-modifying therapiesDisease-modifying therapiesSickle cell clinicAssessment of patientsFuture prospective studiesRandomized clinical trialsParent trialProspective studyTreatment optionsClinical trialsNeeds assessment phaseMAIN OUTCOMECell clinicPatients
2018
Ovarian Sertoli–Leydig tumor after bone marrow transplant for sickle cell disease
Phillips L, Krishnamurti L, Rytting H, Olson T. Ovarian Sertoli–Leydig tumor after bone marrow transplant for sickle cell disease. Pediatric Blood & Cancer 2018, 65: e27367. PMID: 30039911, DOI: 10.1002/pbc.27367.Peer-Reviewed Original Research
2016
A trial of unrelated donor marrow transplantation for children with severe sickle cell disease
Shenoy S, Eapen M, Panepinto J, Logan B, Wu J, Abraham A, Brochstein J, Chaudhury S, Godder K, Haight A, Kasow K, Leung K, Andreansky M, Bhatia M, Dalal J, Haines H, Jaroscak J, Lazarus H, Levine J, Krishnamurti L, Margolis D, Megason G, Yu L, Pulsipher M, Gersten I, DiFronzo N, Horowitz M, Walters M, Kamani N. A trial of unrelated donor marrow transplantation for children with severe sickle cell disease. Blood 2016, 128: 2561-2567. PMID: 27625358, PMCID: PMC5123194, DOI: 10.1182/blood-2016-05-715870.Peer-Reviewed Original ResearchConceptsEvent-free survivalIncidence rateAcute chest syndrome episodesPosterior reversible encephalopathy syndromeSevere sickle cell diseaseUnrelated donor marrow transplantationVaso-occlusive pain crisesAllogeneic bone marrow transplantEffective GVHD prophylaxisGVHD-related deathsHost disease (GVHD) prophylaxisShort-course methotrexateReversible encephalopathy syndromeUnrelated donor transplantsPhase 2 trialBone marrow transplantTranscranial Doppler velocitiesSickle cell diseaseAcute GVHDChronic GVHDEFS ratesEncephalopathy syndromeGVHD prophylaxisTransplant indicationConditioning regimen