2018
Sickle cell disease
Kato G, Piel F, Reid C, Gaston M, Ohene-Frempong K, Krishnamurti L, Smith W, Panepinto J, Weatherall D, Costa F, Vichinsky E. Sickle cell disease. Nature Reviews Disease Primers 2018, 4: 18010. PMID: 29542687, DOI: 10.1038/nrdp.2018.10.Peer-Reviewed Original ResearchConceptsSickle cell diseaseCell diseaseHaematopoietic stem cell transplantationAcute pain eventsAcute chest syndromeHigh-burden settingsStem cell transplantationCommon acute complicationHemoglobin subunit βNewborn screening programsUniversal newborn screening (NBS) programsChest syndromeAcute complicationsChronic complicationsBlood transfusionCell transplantationPathophysiological mechanismsPain eventsScreening programEarly diagnosisSCD phenotypeImmune systemΒ-globin subunitDiseaseComplications
2016
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approaches
Arnold S, Bhatia M, Horan J, Krishnamurti L. Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approaches. British Journal Of Haematology 2016, 174: 515-525. PMID: 27255787, DOI: 10.1111/bjh.14167.Peer-Reviewed Original ResearchConceptsHaematopoietic stem cell transplantSickle cell diseaseCell diseaseAllogeneic haematopoietic stem cell transplantOnly available curative therapyHaematopoietic stem cell transplantationAlternative donor transplantsAvailable curative therapyCurrent maintenance therapiesTransplant-related mortalityDisease-free survivalIncidence of rejectionStem cell transplantStem cell transplantationHuman leucocyte antigenPool of donorsComplications persistHaploidentical donorsMaintenance therapyConditioning regimensCurative optionDonor transplantsSibling transplantsSupportive careCurative therapy