2024
Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease
Walters M, Eapen M, Liu Y, El Rassi F, Waller E, Levine J, Strouse J, Antin J, Parikh S, Bakshi N, Dampier C, Jaroscak J, Bergmann S, Wong T, Kota V, Pace B, Lekakis L, Lulla P, Nickel R, Kasow K, Popat U, Smith W, Yu L, DiFronzo N, Geller N, Kamani N, Klings E, Hassell K, Mendizabal A, Sullivan K, Neuberg D, Krishnamurti L. Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease. Blood Advances 2024 PMID: 39471440, DOI: 10.1182/bloodadvances.2024013926.Peer-Reviewed Original ResearchHematopoietic cell transplantationVaso-occlusive painStandard of careSickle cell diseaseCell transplantationCell diseaseSCD-related eventsHLA-matched siblingTransplant-related mortalityPeriod of follow-upSurvival 2 yearsIntent-to-treat principleComparison of survivalDisease-modifying therapiesDisease-related mortalityUnrelated donorPrimary endpointCurative potentialTreatment armsDonor searchFollow-upCompare outcomesEligibility confirmationFunctional outcomesSecondary outcomes
2015
A multicenter randomized controlled trial of intravenous magnesium for sickle cell pain crisis in children
Brousseau D, Scott J, Badaki-Makun O, Darbari D, Chumpitazi C, Airewele G, Ellison A, Smith-Whitley K, Mahajan P, Sarnaik S, Casper T, Cook L, Dean J, Leonard J, Hulbert M, Powell E, Liem R, Hickey R, Krishnamurti L, Hillery C, Nimmer M, Panepinto J, Network F. A multicenter randomized controlled trial of intravenous magnesium for sickle cell pain crisis in children. Blood 2015, 126: 1651-1657. PMID: 26232172, PMCID: PMC4591790, DOI: 10.1182/blood-2015-05-647107.Peer-Reviewed Original ResearchConceptsSickle cell pain crisisLength of stayPediatric Emergency Care Applied Research NetworkPain crisisIntravenous magnesiumOpioid useNormal saline placeboStudy drug infusionPlacebo-controlled trialHealth-related qualityQuality of lifeMorphine equivalentsStudy drugSaline placeboSecondary outcomesStandard therapyHemoglobin SSPediatric patientsPrimary outcomeDrug infusionPain relieversPlaceboStayChildren 4Group demographics
2014
A pilot study of electronic directly observed therapy to improve hydroxyurea adherence in pediatric patients with sickle‐cell disease
Creary S, Gladwin M, Byrne M, Hildesheim M, Krishnamurti L. A pilot study of electronic directly observed therapy to improve hydroxyurea adherence in pediatric patients with sickle‐cell disease. Pediatric Blood & Cancer 2014, 61: 1068-1073. PMID: 24436121, DOI: 10.1002/pbc.24931.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAnemia, Sickle CellAntisickling AgentsCell PhoneChildChild, PreschoolDirectly Observed TherapyDrug Administration ScheduleElectronic MailFeedbackFemaleHealth SurveysHumansHydroxyureaMaleMedication AdherenceMicrocomputersPatient SatisfactionPilot ProjectsReimbursement, IncentiveReminder SystemsRewardText MessagingVideo RecordingYoung AdultConceptsSickle cell diseaseMedian medication possession ratioMean corpuscular volumeHU adherenceHydroxyurea adherencePilot studyHemoglobin F percentageMedication possession ratioParticipant satisfactionPossession ratioSecondary outcomesPediatric patientsPrimary outcomeStudy entryClinical outcomesHU useHU therapyOverall participant satisfactionSingle institutionCell diseaseReminder alertsCorpuscular volumeTherapyAdherenceOverall median
2013
An Analysis Of The Pediatric Sub-Group From The Phase 2 Study Of GMI 1070 – A Novel Agent For The Vaso-Occlusive Crisis Of Sickle Cell Anemia
McCavit T, Krishnamurti L, Hsu L, Quinn C, Odame I, Alvarez O, Driscoll C, Smith-Whitley K, Rhee S, Wun T, Telen M, Thackray H. An Analysis Of The Pediatric Sub-Group From The Phase 2 Study Of GMI 1070 – A Novel Agent For The Vaso-Occlusive Crisis Of Sickle Cell Anemia. Blood 2013, 122: 2206. DOI: 10.1182/blood.v122.21.2206.2206.Peer-Reviewed Original ResearchVaso-occlusive crisisSerious adverse eventsSickle cell anemiaAcute chest syndromeStudy drugPediatric subjectsAdult subjectsMaintenance dosesOpioid useSecondary outcomesMedian timePlacebo-controlled phase 2 trialCell anemiaRed blood cell transfusionPhase 3 clinical trialsStrong efficacy signalBlood cell transfusionPhase 2 studyPhase 2 trialKaplan-Meier methodVisual analog scaleInitial medical evaluationPan-selectin inhibitorAdvisory CommitteeMinimal safety concerns