2024
Sickle cell disease in India: the journey and hope for the future.
Gupta K, Krishnamurti L, Jain D. Sickle cell disease in India: the journey and hope for the future. Hematology 2024, 2024: 1-9. PMID: 39644008, PMCID: PMC11665512, DOI: 10.1182/hematology.2024000678.Peer-Reviewed Original ResearchConceptsSickle cell diseaseHematopoietic stem cell transplantationCell diseaseSeverity of sickle cell diseaseStem cell transplantationAcute chest syndromeVaso-occlusive crisisSurvive into adulthoodChest syndromePenicillin prophylaxisCell transplantationRenal involvementHydroxyurea therapyGene therapyClinical featuresNovel therapiesA-thalassemiaFetal HbNewborn screeningAvascular necrosisTranscranial DopplerComprehensive care programTherapyYounger ageFatal disease
2019
End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings
Farrell A, Panepinto J, Desai A, Kassim A, Lebensburger J, Walters M, Bauer D, Blaylark R, DiMichele D, Gladwin M, Green N, Hassell K, Kato G, Klings E, Kohn D, Krishnamurti L, Little J, Makani J, Malik P, McGann P, Minniti C, Morris C, Odame I, Oneal P, Setse R, Sharma P, Shenoy S. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Advances 2019, 3: 4002-4020. PMID: 31809537, PMCID: PMC6963248, DOI: 10.1182/bloodadvances.2019000883.Peer-Reviewed Original ResearchConceptsLow-resource settingsEnd pointClinical trial end pointsTrial end pointsPatient-reported outcomesSickle cell disease clinical trialsPanel of cliniciansSickle cell diseaseMeasurement of cureClinical trialsNovel therapiesCell diseaseConsensus recommendationsGlobal burdenUS FoodBiomarker panelDrug AdministrationAvailable evidenceAmerican SocietyCureRelevant findingsLiterature reviewPainPatientsTherapy