2022
Decision-making about gene therapy in transfusion dependent thalassemia
Quarmyne M, Ross D, Sinha C, Bakshi N, Boudreaux J, Krishnamurti L. Decision-making about gene therapy in transfusion dependent thalassemia. BMC Pediatrics 2022, 22: 536. PMID: 36085025, PMCID: PMC9461218, DOI: 10.1186/s12887-022-03598-3.Peer-Reviewed Original ResearchConceptsTransfusion-dependent thalassemiaPatient/family knowledgeDependent thalassemiaTransfusion independenceTreatment optionsStudy participantsGene therapyFamily knowledgeElimination of transfusionsFrequency of transfusionMorbidity/mortalityStem cell transplantationLong-term outcomesPreferred treatment modalityPromising treatment optionBackgroundHematopoietic stem cell transplantationPatients/familiesMethodsParents of childrenCurative intentTransfusion reductionHost diseaseDonor HSCTParents of childrenCell transplantationMean age
2014
Feasibility of a Newborn Screening and Follow-up Programme for Sickle Cell Disease among South Gujarat (India) Tribal Populations
Italia Y, Krishnamurti L, Mehta V, Raicha B, Italia K, Mehta P, Ghosh K, Colah R. Feasibility of a Newborn Screening and Follow-up Programme for Sickle Cell Disease among South Gujarat (India) Tribal Populations. Journal Of Medical Screening 2014, 22: 1-7. PMID: 25341880, DOI: 10.1177/0969141314557372.Peer-Reviewed Original ResearchConceptsSickle cell diseaseSCD babiesCell diseaseNewborn screeningSevere clinical complicationsHigh fetal hemoglobinNewborn screening programsSickle cell disordersClinical presentationTribal populationClinical complicationsNatural courseScreening programNewborn babiesCell disordersXmn I polymorphismBabiesDiseaseHome careFetal hemoglobinHigh-performance liquid chromatographyRegular monitoringScreeningPopulationΑ-thalassaemia
2003
Availability of unrelated donors for hematopoietic stem cell transplantation for hemoglobinopathies
Krishnamurti L, Abel S, Maiers M, Flesch S. Availability of unrelated donors for hematopoietic stem cell transplantation for hemoglobinopathies. Bone Marrow Transplantation 2003, 31: 547-550. PMID: 12692619, DOI: 10.1038/sj.bmt.1703887.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationNational Marrow Donor ProgramStem cell transplantationUnrelated donorsHemoglobinopathy patientsCell transplantationThalassemia patientsURD HSCTLife-threatening blood diseaseSickle cell disease patientsUnrelated bone marrow donorsUmbilical cord blood unitsHLA-identical siblingsOnly curative optionCord blood unitsBone marrow donorsCurative optionPreparative regimensHLA matchDisease patientsPotential HLAMarrow donorsNMDP RegistryPatientsVolunteer donors