2019
Phase 1b Study of the Epichaperome Inhibitor PU-H71 Administered Orally with Ruxolitinib Continuation for the Treatment of Patients with Myelofibrosis
Pemmaraju N, Gundabolu K, Pettit K, Talpaz M, Podoltsev N, Schiller G, Eisenberg P, Youssoufian H, Duggan S, Wallner B, Verstovsek S. Phase 1b Study of the Epichaperome Inhibitor PU-H71 Administered Orally with Ruxolitinib Continuation for the Treatment of Patients with Myelofibrosis. Blood 2019, 134: 4178. DOI: 10.1182/blood-2019-130310.Peer-Reviewed Original ResearchPhase 1b studyJazz PharmaceuticalsExpansion cohortPU-H71Daiichi SankyoMyeloproliferative Neoplasm Symptom Assessment Form Total Symptom ScoreInternational Working Group-Myeloproliferative Neoplasms ResearchPeripheral blood mononuclear cellsAdvisory CommitteeDose-expansion cohortsHigh-risk myelofibrosisPhase 2 doseSerum cytokine profilesBaseline platelet countDose-ranging studySpeakers bureauVentricular ejection fractionAST/ALTDose-escalation designTotal symptom scoreBlood mononuclear cellsDisease-related symptomsTreatment of patientsBone marrow histologyPost-PV myelofibrosis
2018
Impact of Leukapheresis and Time to Chemotherapy on Outcomes of Newly Diagnosed Patients (pts) with Acute Myeloid Leukemia (AML) Presenting with Hyperleukocytosis: An Analysis from a Large International Patient Cohort
Stahl M, Wei W, Montesinos P, Lengline E, Shallis R, Neukirchen J, Bhatt V, Sekeres M, Fathi A, Konig H, Luger S, Khan I, Roboz G, Cluzeau T, Martínez-Cuadron D, Raffoux E, Germing U, Umakanthan J, Mukherjee S, Brunner A, Miller A, McMahon C, Ritchie E, Rodríguez-Veiga R, Itzykson R, Boluda B, Rabian F, Tormo M, Cruz E, Rabinovich E, Yoo B, Podoltsev N, Gore S, Zeidan A. Impact of Leukapheresis and Time to Chemotherapy on Outcomes of Newly Diagnosed Patients (pts) with Acute Myeloid Leukemia (AML) Presenting with Hyperleukocytosis: An Analysis from a Large International Patient Cohort. Blood 2018, 132: 1428. DOI: 10.1182/blood-2018-99-112495.Peer-Reviewed Original ResearchWhite blood cell countInitiation of ICSAcute myeloid leukemiaUse of leukapheresisImpact of leukapheresisTumor lysis syndromeHours of presentationOdds of deathCytogenetic risk groupOverall survivalTime of presentationUnivariate analysisMultivariate analysisAstex PharmaceuticalsSpeakers bureauHazard ratioDaiichi SankyoRisk groupsEastern Cooperative Oncology Group performance statusJanssen PharmaceuticalsJazz PharmaceuticalsCleveland Clinic Taussig Cancer InstituteAdverse cytogenetic risk groupPoor cytogenetic risk groupCelgene CorporationCharacteristics, Treatment Patterns and Outcomes Among Newly Diagnosed Patients (pts) with Acute Myeloid Leukemia (AML) Who Present with Hyperleukocytosis: Findings from a Large International Patient Cohort
Stahl M, Wei W, Montesinos P, Lengline E, Shallis R, Neukirchen J, Bhatt V, Sekeres M, Fathi A, Konig H, Luger S, Khan I, Roboz G, Cluzeau T, Martínez-Cuadron D, Raffoux E, Germing U, Umakanthan J, Mukherjee S, Brunner A, Miller A, McMahon C, Ritchie E, Rodríguez-Veiga R, Itzykson R, Boluda B, Rabian F, Tormo M, Cruz E, Rabinovich E, Yoo B, Podoltsev N, Gore S, Zeidan A. Characteristics, Treatment Patterns and Outcomes Among Newly Diagnosed Patients (pts) with Acute Myeloid Leukemia (AML) Who Present with Hyperleukocytosis: Findings from a Large International Patient Cohort. Blood 2018, 132: 4040. DOI: 10.1182/blood-2018-99-112974.Peer-Reviewed Original ResearchWhite blood cell countMedian overall survivalAcute myeloid leukemiaHematopoietic stem cell transplantIntensive chemotherapyTumor lysis syndromeOverall survivalTime of presentationComplete remissionAstex PharmaceuticalsSpeakers bureauPartial remissionTreatment patternsDaiichi SankyoJazz PharmaceuticalsCleveland Clinic Taussig Cancer InstituteJanssen PharmaceuticalsMedian white blood cell countHigher white blood cell countCelgene CorporationLong-term clinical outcomesAdvisory CommitteeImpact of leukapheresisIncomplete count recoveryMDS International FoundationOutcomes of Patients with Newly-Diagnosed Acute Myeloid Leukemia and Hyperleukocytosis Who Did Not Undergo Intensive Chemotherapy: Results from a Large International Database
Shallis R, Stahl M, Wei W, Montesinos P, Lengline E, Neukirchen J, Bhatt V, Sekeres M, Fathi A, Konig H, Luger S, Khan I, Roboz G, Cluzeau T, Martínez-Cuadron D, Raffoux E, Germing U, Umakanthan J, Mukherjee S, Brunner A, Miller A, McMahon C, Ritchie E, Rodríguez-Veiga R, Itzykson R, Boluda B, Rabian F, Tormo M, Cruz E, Podoltsev N, Gore S, Zeidan A. Outcomes of Patients with Newly-Diagnosed Acute Myeloid Leukemia and Hyperleukocytosis Who Did Not Undergo Intensive Chemotherapy: Results from a Large International Database. Blood 2018, 132: 3999. DOI: 10.1182/blood-2018-99-119755.Peer-Reviewed Original ResearchTumor lysis syndromeIntensive chemotherapyIntensive care unitOverall survivalClinical evidenceAstex PharmaceuticalsSpeakers bureauDaiichi SankyoMedian OSWorse OSAML patientsPlatelet countEastern Cooperative Oncology Group performance statusJazz PharmaceuticalsCleveland Clinic Taussig Cancer InstituteJanssen PharmaceuticalsOne-year OS probabilityCelgene CorporationAdvisory CommitteeMDS International FoundationPoor-risk AMLBest supportive careLow-dose cytarabineMedian overall survivalOutcomes of patientsLong Term Results of a Randomized Phase 2 Dose-Response Study of Guadecitabine, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate or High Risk Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML)
Garcia-Manero G, Ritchie E, Walsh K, Savona M, Kantarjian H, Kropf P, O'Connell C, Tibes R, Daver N, Jabbour E, Lunin S, Rosenblat T, Yee K, Stock W, Griffiths E, Mace J, Podoltsev N, Berdeja J, Issa J, Naim S, Hao Y, Azab M, Roboz G. Long Term Results of a Randomized Phase 2 Dose-Response Study of Guadecitabine, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate or High Risk Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML). Blood 2018, 132: 231. DOI: 10.1182/blood-2018-99-110465.Peer-Reviewed Original ResearchMedian overall survivalOverall survivalDose groupMyelodysplastic syndromeTreatment-naïveChronic myelomonocytic leukemiaCMML patientsBristol-Myers SquibbHypomethylating agentAdverse eventsAstex PharmaceuticalsM2 groupMDS patientsMD cohortJazz PharmaceuticalsMarrow CRBM blastsHematological improvementMost patientsSpeakers bureauDaiichi SankyoHMA treatmentJanssen PharmaceuticalsTP53 mutationsCelgene Corporation
2016
The Use of Hypomethylating Agents (HMAs) in Patients with Relapsed and Refractory Acute Myeloid Leukemia (RR-AML): Clinical Outcomes and Their Predictors in a Large International Patient Cohort
Stahl M, Podoltsev N, DeVeaux M, Perreault S, Itzykson R, Ritchie E, Sekeres M, Fathi A, Komrokji R, Bhatt V, Al-Kali A, Cluzeau T, Santini V, Brunner A, Roboz G, Fenaux P, Litzow M, Vey N, Verma V, Germing U, Fernández P, Zelterman D, Kim T, Prebet T, Gore S, Zeidan A. The Use of Hypomethylating Agents (HMAs) in Patients with Relapsed and Refractory Acute Myeloid Leukemia (RR-AML): Clinical Outcomes and Their Predictors in a Large International Patient Cohort. Blood 2016, 128: 1063. DOI: 10.1182/blood.v128.22.1063.1063.Peer-Reviewed Original ResearchMultivariable logistic regression modelRR-AMLMinority of patientsOverall survivalPrior linesHypomethylating agentHMA therapyMedian numberLogistic regression modelsChromosome 7 abnormalitiesStable diseaseBlast percentageHematologic improvementProgressive diseaseHazard ratioClinical outcomesSpeakers bureauMultivariable Cox proportional hazards modelsPeripheral blood blast percentageRefractory acute myeloid leukemiaAllogeneic stem cell transplantationInternational Working Group criteriaWhite blood cell countCox proportional hazards modelBM blast percentage