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
2015
Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use
Telen M, Wun T, McCavit T, De Castro L, Krishnamurti L, Lanzkron S, Hsu L, Smith W, Rhee S, Magnani J, Thackray H. Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use. Blood 2015, 125: 2656-2664. PMID: 25733584, PMCID: PMC4408290, DOI: 10.1182/blood-2014-06-583351.Peer-Reviewed Original ResearchConceptsVaso-occlusive crisisSickle cell diseaseComposite primary end pointPrimary end pointPhase 2 studyEnd pointRandomized phase 2 studySCD vaso-occlusive crisisOpioid analgesic useSecondary end pointsActive treatment groupPhase 3 studyVaso-occlusive eventsAnalgesic usePlacebo groupProspective multicenterStudy drugAdverse eventsOpioid useSymptom reliefMedian timeSCD patientsCell diseaseTreatment groupsAnimal models
2011
Nitric Oxide for Inhalation in the Acute Treatment of Sickle Cell Pain Crisis: A Randomized Controlled Trial
Gladwin M, Kato G, Weiner D, Onyekwere O, Dampier C, Hsu L, Hagar R, Howard T, Nuss R, Okam M, Tremonti C, Berman B, Villella A, Krishnamurti L, Lanzkron S, Castro O, Gordeuk V, Coles W, Peters-Lawrence M, Nichols J, Hall M, Hildesheim M, Blackwelder W, Baldassarre J, Casella J, Investigators F. Nitric Oxide for Inhalation in the Acute Treatment of Sickle Cell Pain Crisis: A Randomized Controlled Trial. JAMA 2011, 305: 893-902. PMID: 21364138, PMCID: PMC3403835, DOI: 10.1001/jama.2011.235.Peer-Reviewed Original ResearchConceptsVaso-occlusive pain crisesSickle cell diseaseVisual analog pain scale scoreAcute chest syndromePrimary end pointPain scale scoresNitric oxideChest syndromePain crisisPainful crisesScale scoreSickle cell pain crisisSmall placebo-controlled trialsPlacebo-controlled clinical trialEnd pointNitric oxide gasCumulative opioid usageParenteral opioid useWhole blood nitritePlacebo-controlled trialSerious adverse eventsLength of hospitalizationSecondary outcome measuresEvidence of efficacyOpioid usage