2023
A multicenter phase Ib trial of the histone deacetylase inhibitor entinostat in combination with pembrolizumab in patients with myelodysplastic syndromes/neoplasms or acute myeloid leukemia refractory to hypomethylating agents
Bewersdorf J, Shallis R, Sharon E, Park S, Ramaswamy R, Roe C, Irish J, Caldwell A, Wei W, Yacoub A, Madanat Y, Zeidner J, Altman J, Odenike O, Yerrabothala S, Kovacsovics T, Podoltsev N, Halene S, Little R, Piekarz R, Gore S, Kim T, Zeidan A. A multicenter phase Ib trial of the histone deacetylase inhibitor entinostat in combination with pembrolizumab in patients with myelodysplastic syndromes/neoplasms or acute myeloid leukemia refractory to hypomethylating agents. Annals Of Hematology 2023, 103: 105-116. PMID: 38036712, DOI: 10.1007/s00277-023-05552-4.Peer-Reviewed Original ResearchConceptsDose-limiting toxicityAcute myeloid leukemiaMarrow complete remissionPhase Ib trialAdverse eventsIb trialDose escalationNCI Cancer Therapy Evaluation ProgramAcute myeloid leukemia refractoryHematologic adverse eventsProtocol-defined responseDose level 1Anti-PD1 therapyAnti-PD1 antibodyDose-escalation designLimited clinical efficacySystems immunology approachHistone deacetylase inhibitor entinostatLeukemia refractoryMCR patientsComplete remissionRespiratory failureSuppressor cellsEscalation designClinical efficacy
2022
Outcomes in Patients with FLT3-Mutated Relapsed/ Refractory Acute Myelogenous Leukemia Who Underwent Transplantation in the Phase 3 ADMIRAL Trial of Gilteritinib versus Salvage Chemotherapy
Perl A, Larson R, Podoltsev N, Strickland S, Wang E, Atallah E, Schiller G, Martinelli G, Neubauer A, Sierra J, Montesinos P, Recher C, Yoon S, Maeda Y, Hosono N, Onozawa M, Kato T, Kim H, Hasabou N, Nuthethi R, Tiu R, Levis M. Outcomes in Patients with FLT3-Mutated Relapsed/ Refractory Acute Myelogenous Leukemia Who Underwent Transplantation in the Phase 3 ADMIRAL Trial of Gilteritinib versus Salvage Chemotherapy. Transplantation And Cellular Therapy 2022, 29: 265.e1-265.e10. PMID: 36526260, PMCID: PMC10189888, DOI: 10.1016/j.jtct.2022.12.006.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationAcute myelogenous leukemiaPost-transplantation maintenance therapyLower relapse rateAdverse eventsADMIRAL trialMaintenance therapyPost-transplantation survivalGilteritinib armRelapse rateSalvage chemotherapyMyelogenous leukemiaRefractory acute myelogenous leukemiaTreatment-emergent adverse eventsFLT3 internal tandem duplication (ITD) mutationsCommon adverse eventsComposite complete remissionGlobal phase 3Incidence of gradePost-transplantation complicationsTransplantation-eligible patientsAlanine aminotransferase levelsHigher remission ratesOverall survival rateSurvival of patientsFollow-up of patients with R/R FLT3-mutation–positive AML treated with gilteritinib in the phase 3 ADMIRAL trial
Perl AE, Larson RA, Podoltsev NA, Strickland S, Wang ES, Atallah E, Schiller GJ, Martinelli G, Neubauer A, Sierra J, Montesinos P, Récher C, Yoon SS, Hosono N, Onozawa M, Chiba S, Kim HJ, Hasabou N, Lu Q, Tiu R, Levis MJ. Follow-up of patients with R/R FLT3-mutation–positive AML treated with gilteritinib in the phase 3 ADMIRAL trial. Blood 2022, 139: 3366-3375. PMID: 35081255, PMCID: PMC9197557, DOI: 10.1182/blood.2021011583.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaSalvage chemotherapyADMIRAL trialSC armOverall survivalFLT3 mutation-positive acute myeloid leukemiaPositive acute myeloid leukemiaComposite complete remissionStable safety profileAdverse event incidenceCommon adverse eventsLiver transaminase levelsMedian overall survivalSuperior overall survivalLong-term treatment effectsGilteritinib armComplete remissionMaintenance therapyAdverse eventsCumulative incidenceMedian survivalTransaminase levelsEvent incidenceMaintenance treatmentSafety profile
2021
Ruxolitinib discontinuation in polycythemia vera: Patient characteristics, outcomes, and salvage strategies from a large multi-institutional database
Tremblay D, Ronner L, Podoltsev N, Gotlib J, Heaney M, Kuykendall A, O'Connell C, Shammo JM, Fleischman A, Mesa R, Yacoub A, Hoffman R, Moshier E, Zubizarreta N, Mascarenhas J. Ruxolitinib discontinuation in polycythemia vera: Patient characteristics, outcomes, and salvage strategies from a large multi-institutional database. Leukemia Research 2021, 109: 106629. PMID: 34082375, DOI: 10.1016/j.leukres.2021.106629.Peer-Reviewed Original ResearchConceptsMulti-institutional databaseRuxolitinib discontinuationPolycythemia veraLarge multi-institutional databaseAvailable salvage therapiesDiscontinuation of ruxolitinibTreatment of patientsFurther therapeutic developmentLast followSalvage therapyCytoreductive therapyAdverse eventsPatient characteristicsThrombotic eventsTreatment initiationSimilar patientsDisease characteristicsDismal outcomeFavorable outcomeSalvage strategyPV patientsDiscontinuationInterferon αPatientsRuxolitinib
2020
Sequencing of novel agents in relapsed/refractory B‐cell acute lymphoblastic leukemia: Blinatumomab and inotuzumab ozogamicin may have comparable efficacy as first or second novel agent therapy in relapsed/refractory acute lymphoblastic leukemia
Badar T, Szabo A, Dinner S, Liedtke M, Burkart M, Shallis RM, Yurkiewicz IR, Kuo E, Khan MA, Balasubramanian S, Yang J, Hefazi M, Podoltsev N, Patel A, Curran E, Wang A, Arslan S, Aldoss I, Siebenaller C, Mattison RJ, Litzow MR, Wadleigh M, Advani AS, Atallah E. Sequencing of novel agents in relapsed/refractory B‐cell acute lymphoblastic leukemia: Blinatumomab and inotuzumab ozogamicin may have comparable efficacy as first or second novel agent therapy in relapsed/refractory acute lymphoblastic leukemia. Cancer 2020, 127: 1039-1048. PMID: 33259056, DOI: 10.1002/cncr.33340.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedAged, 80 and overAntibodies, BispecificAntineoplastic Agents, ImmunologicalDrug Administration ScheduleDrug Resistance, NeoplasmFemaleHematopoietic Stem Cell TransplantationHumansInotuzumab OzogamicinMaleMiddle AgedPrecursor Cell Lymphoblastic Leukemia-LymphomaRemission InductionRetrospective StudiesTreatment OutcomeWithholding TreatmentYoung AdultConceptsAcute lymphoblastic leukemiaRefractory B-cell acute lymphoblastic leukemiaB-cell acute lymphoblastic leukemiaMedian overall survivalINO groupNovel agentsOverall survivalLymphoblastic leukemiaComplete remissionInotuzumab ozogamicinNA therapyComparable efficacyRelapsed/refractory (r/r) B-cell acute lymphoblastic leukemiaAllogeneic hematopoietic stem cell transplantationCR/CRi rateIncomplete count recovery (CRi) ratesRefractory acute lymphoblastic leukemiaHematopoietic stem cell transplantationNovel agent therapyOutcomes of patientsStem cell transplantationBlinatumomab groupCRi rateTreatment discontinuationAdverse eventsPhase 1 dose escalation trial of volasertib in combination with decitabine in patients with acute myeloid leukemia
Cortes J, Podoltsev N, Kantarjian H, Borthakur G, Zeidan AM, Stahl M, Taube T, Fagan N, Rajeswari S, Uy GL. Phase 1 dose escalation trial of volasertib in combination with decitabine in patients with acute myeloid leukemia. International Journal Of Hematology 2020, 113: 92-99. PMID: 32951163, DOI: 10.1007/s12185-020-02994-8.Peer-Reviewed Original ResearchMeSH KeywordsAgedAntineoplastic Combined Chemotherapy ProtocolsCell Cycle ProteinsDecitabineDose-Response Relationship, DrugFebrile NeutropeniaFeeding and Eating DisordersFemaleGene ExpressionHumansLeukemia, Myeloid, AcuteMaleMolecular Targeted TherapyProtein Serine-Threonine KinasesProto-Oncogene ProteinsPteridinesTreatment OutcomeConceptsAcute myeloid leukemiaMyeloid leukemiaCommon treatment-emergent adverse eventsPhase 1 dose-escalation trialTreatment-emergent adverse eventsMTD of volasertibObjective response rateAdverse event profileDose-escalation trialPhase 1 trialAnti-leukemic activityPolo-like kinase 1Febrile neutropeniaEscalation trialAdverse eventsCell cycle kinase inhibitorsAML patientsEvent profilePoor prognosisResponse ratePatientsVolasertibDecitabineKinase inhibitorsNumerous cancersInterferon alpha therapy in essential thrombocythemia and polycythemia vera—a systematic review and meta-analysis
Bewersdorf JP, Giri S, Wang R, Podoltsev N, Williams RT, Tallman MS, Rampal RK, Zeidan AM, Stahl M. Interferon alpha therapy in essential thrombocythemia and polycythemia vera—a systematic review and meta-analysis. Leukemia 2020, 35: 1643-1660. PMID: 32868875, PMCID: PMC7917159, DOI: 10.1038/s41375-020-01020-4.Peer-Reviewed Original ResearchConceptsOverall response ratePEG-IFNPolycythemia veraEssential thrombocythemiaHematologic responsePV patientsResponse rateSystematic reviewMeta-regression analysis resultsSafe long-term treatmentPartial hematologic responseSafety of interferonInterferon-alpha therapyLong-term treatmentRandom-effects modelMeta-regression analysisWeb of ScienceTreatment discontinuationCochrane RegistryAdverse eventsComplete responsePartial responsePrimary outcomeThromboembolic complicationsClinical trialsInterferon Therapy in Myelofibrosis: Systematic Review and Meta-analysis
Bewersdorf JP, Giri S, Wang R, Podoltsev N, Williams RT, Rampal RK, Tallman MS, Zeidan AM, Stahl M. Interferon Therapy in Myelofibrosis: Systematic Review and Meta-analysis. Clinical Lymphoma Myeloma & Leukemia 2020, 20: e712-e723. PMID: 32669244, PMCID: PMC7541411, DOI: 10.1016/j.clml.2020.05.018.Peer-Reviewed Original ResearchConceptsOverall response rateTreatment of myelofibrosisPEG-IFNMF patientsResponse rateHematologic responseSystematic reviewFormulations of interferonPartial hematologic responseCochrane Central RegisterComplete hematologic responseRisk of progressionRole of interferonAcute myeloid leukemiaPhiladelphia chromosome-negative myeloproliferative neoplasmsRandom-effects modelBone marrow failureWeb of ScienceTreatment discontinuationCentral RegisterConstitutional symptomsHematologic improvementInterferon therapyAdverse eventsComplete responseReal-World Outcomes of Adult B-Cell Acute Lymphocytic Leukemia Patients Treated With Inotuzumab Ozogamicin
Badar T, Szabo A, Wadleigh M, Liedtke M, Arslan S, Siebenaller C, Aldoss I, Schultz E, Hefazi M, Litzow MR, Kuo E, Wang A, Curran E, Shallis RM, Podoltsev N, Balasubramanian S, Yang J, Mattison R, Burkart M, Dinner S, Advani A, Atallah E. Real-World Outcomes of Adult B-Cell Acute Lymphocytic Leukemia Patients Treated With Inotuzumab Ozogamicin. Clinical Lymphoma Myeloma & Leukemia 2020, 20: 556-560.e2. PMID: 32291234, DOI: 10.1016/j.clml.2020.03.004.Peer-Reviewed Original ResearchConceptsB-cell acute lymphocytic leukemia patientsAcute lymphocytic leukemia patientsAllo-HCTLymphocytic leukemia patientsInotuzumab ozogamicinOverall survivalLeukemia patientsAllogeneic hematopoietic stem cell transplantationMinimal residual disease negativityHematopoietic stem cell transplantationCommon grade 3Efficacy of iNOHigher adverse eventsMedian overall survivalMulticenter cohort analysisVeno-occlusive diseaseDuration of responseStem cell transplantationOverall response rateINO initiationComplete remissionMedian durationAdverse eventsMedian ageCumulative dose
2019
Real World Outcomes of Adult B-Cell Acute Lymphocytic Leukemia Patients Treated with Inotuzumab Ozogamicin
Badar T, Szabo A, Wadleigh M, Liedtke M, Arslan S, Siebenaller C, Aldoss I, Schultz E, Hefazi M, Litzow M, Kuo E, Wang A, Curran E, Shallis R, Podoltsev N, Balasubramanian S, Yang J, Mattison R, Burkart M, Dinner S, Advani A, Atallah E. Real World Outcomes of Adult B-Cell Acute Lymphocytic Leukemia Patients Treated with Inotuzumab Ozogamicin. Blood 2019, 134: 1302. DOI: 10.1182/blood-2019-124882.Peer-Reviewed Original ResearchMedian overall survivalDuration of responseAllo-HCTOverall survivalAcute lymphocytic leukemiaTyrosine kinase inhibitorsComplete remissionB cellsInotuzumab ozogamicinAdverse eventsRefractory B-cell acute lymphocytic leukaemiaJazz PharmaceuticalsResponse rateB-cell acute lymphocytic leukemiaMinimal residual disease negativityAllogeneic stem cell transplantationAdvisory CommitteeB-cell acute lymphocytic leukemia patientsEfficacy of iNOGenentech/RocheIncomplete count recoveryMedian response durationVeno-occlusive diseaseOutcomes of patientsKaplan-Meier methodThe VITAL Trial: Phase II Trial of Vosaroxin and Infusional Cytarabine for Frontline Treatment of acute Myeloid Leukemia
Strickland S, Podoltsev N, Mohan S, Zeidan A, Childress M, Ayers G, Byrne M, Gore S, Stuart R, Savona M. The VITAL Trial: Phase II Trial of Vosaroxin and Infusional Cytarabine for Frontline Treatment of acute Myeloid Leukemia. Blood 2019, 134: 180. DOI: 10.1182/blood-2019-131520.Peer-Reviewed Original ResearchIntermediate-dose cytarabineCR/CRiAdverse eventsOverall survivalInfusional cytarabineOral mucositisCardiac toxicityResponse assessmentCelgene CorporationAML ptsOral cryotherapyBoehringer IngelheimMedian ageCR rateDay 1Large randomized phase 3 trialsTwo-stage phase II studyClass anticancer quinolone derivativeCR/CRi rateRandomized phase 3 trialHematopoietic stem cell transplantAdvisory CommitteeDaiichi SankyoAcute cardiac toxicityIncomplete count recoveryPS1036 UPDATED RESULTS FROM A PHASE 1 STUDY OF GILTERITINIB IN COMBINATION WITH INDUCTION AND CONSOLIDATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED AML
Pratz K, Cherry M, Altman J, Cooper B, Cruz J, Jurcic J, Levis M, Lin T, Perl A, Podoltsev N, Schiller G, Liu C, Bahceci E. PS1036 UPDATED RESULTS FROM A PHASE 1 STUDY OF GILTERITINIB IN COMBINATION WITH INDUCTION AND CONSOLIDATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED AML. HemaSphere 2019, 3: 468. DOI: 10.1097/01.hs9.0000562440.81745.10.Peer-Reviewed Original ResearchPhase 1 studyMaintenance therapyAdverse eventsConsolidation chemotherapyExpansion doseFebrile neutropeniaSerious drug-related adverse eventsComposite complete remission rateSingle-agent maintenance therapyDrug-related adverse eventsWhite blood cell countMedian overall survivalSafety/tolerabilityComplete remission rateFront-line chemotherapyDose-limiting toxicitySmall intestinal obstructionBlood cell countCycles of inductionHigh response rateCRC ratesIdarubicin inductionDose expansionIntestinal obstructionOverall survivalGuadecitabine (SGI-110) in patients with intermediate or high-risk myelodysplastic syndromes: phase 2 results from a multicentre, open-label, randomised, phase 1/2 trial
Garcia-Manero G, Roboz G, Walsh K, Kantarjian H, Ritchie E, Kropf P, O'Connell C, Tibes R, Lunin S, Rosenblat T, Yee K, Stock W, Griffiths E, Mace J, Podoltsev N, Berdeja J, Jabbour E, Issa JJ, Hao Y, Keer HN, Azab M, Savona MR. Guadecitabine (SGI-110) in patients with intermediate or high-risk myelodysplastic syndromes: phase 2 results from a multicentre, open-label, randomised, phase 1/2 trial. The Lancet Haematology 2019, 6: e317-e327. PMID: 31060979, PMCID: PMC9012213, DOI: 10.1016/s2352-3026(19)30029-8.Peer-Reviewed Original ResearchConceptsHigh-risk myelodysplastic syndromeMyelodysplastic syndromeRefractory cohortAdverse eventsRefractory diseaseHypomethylating agentEastern Cooperative Oncology Group performance statusNorth American medical centersInternational Prognostic Scoring SystemCommon grade 3Phase 2 partWorse adverse eventsOpen-label studyProportion of patientsPrognostic scoring systemOverall responseChronic myelomonocytic leukemiaNew therapeutic optionsAmerican medical centersEligible patientsFebrile neutropaeniaIntravenous decitabinePrimary endpointRefractory patientsStudy drug
2018
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)
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 CorporationUpdated Results from a Phase 1 Study of Gilteritinib in Combination with Induction and Consolidation Chemotherapy in Subjects with Newly Diagnosed Acute Myeloid Leukemia (AML)
Pratz K, Cherry M, Altman J, Cooper B, Cruz J, Jurcic J, Levis M, Lin T, Perl A, Podoltsev N, Schiller G, Liu C, Bahceci E. Updated Results from a Phase 1 Study of Gilteritinib in Combination with Induction and Consolidation Chemotherapy in Subjects with Newly Diagnosed Acute Myeloid Leukemia (AML). Blood 2018, 132: 564. DOI: 10.1182/blood-2018-99-110975.Peer-Reviewed Original ResearchAcute myeloid leukemiaClinical trial workSingle-agent maintenance therapyDose-expansion cohortsSafety/tolerabilityFms-like tyrosine kinase 3Phase 1 studyMaintenance therapyAdverse eventsCRC ratesExpansion doseAstellas PharmaFebrile neutropeniaIntensive chemotherapyDay cohortDose escalationDay 1Serious drug-related adverse eventsDrug-related adverse eventsExperienced dose-limiting toxicityHigh-dose cytarabine consolidationMedian disease-free survivalRefractory acute myeloid leukemiaWhite blood cell countAdvisory Committee
2017
Guadecitabine (SGI-110) in treatment-naive patients with acute myeloid leukaemia: phase 2 results from a multicentre, randomised, phase 1/2 trial
Kantarjian HM, Roboz GJ, Kropf PL, Yee KWL, O'Connell CL, Tibes R, Walsh KJ, Podoltsev NA, Griffiths EA, Jabbour E, Garcia-Manero G, Rizzieri D, Stock W, Savona MR, Rosenblat TL, Berdeja JG, Ravandi F, Rock EP, Hao Y, Azab M, Issa JJ. Guadecitabine (SGI-110) in treatment-naive patients with acute myeloid leukaemia: phase 2 results from a multicentre, randomised, phase 1/2 trial. The Lancet Oncology 2017, 18: 1317-1326. PMID: 28844816, PMCID: PMC5925750, DOI: 10.1016/s1470-2045(17)30576-4.Peer-Reviewed Original ResearchMeSH KeywordsAdultAge FactorsAgedAged, 80 and overAzacitidineDisease-Free SurvivalDose-Response Relationship, DrugDrug Administration ScheduleHumansInfusions, IntravenousKaplan-Meier EstimateLeukemia, Myeloid, AcuteMaximum Tolerated DoseMiddle AgedNeoplasm InvasivenessNeoplasm StagingPatient SafetyPrognosisProspective StudiesRemission InductionRisk AssessmentSurvival AnalysisTreatment OutcomeConceptsAcute myeloid leukemiaComposite complete responseTreatment-naive patientsTreatment-naive acute myeloid leukaemiaAdverse eventsMyeloid leukemiaComplete responseFebrile neutropeniaIntensive chemotherapyMyelodysplastic syndromeTreatment cyclesCommon serious adverse eventsRefractory acute myeloid leukemiaPhase 2 resultsFrequent grade 3Worse adverse eventsSerious adverse eventsPhase 2 studyPhase 3 studyCohort of patientsPhase 1 studyComplete tumor responseStandard of careNumber of patientsEffective treatment scheduleOverall survival (OS) and stem cell transplant (SCT) in patients with FLT3 mutations treated with CPX-351 versus 7+3: Subgroup analysis of a phase III study of older adults with newly diagnosed, high-risk acute myeloid leukemia (AML).
Medeiros B, Hogge D, Newell L, Bixby D, Solomon S, Strickland S, Lin T, Erba H, Powell B, Podoltsev N, Ryan R, Chiarella M, Louie A, Lancet J. Overall survival (OS) and stem cell transplant (SCT) in patients with FLT3 mutations treated with CPX-351 versus 7+3: Subgroup analysis of a phase III study of older adults with newly diagnosed, high-risk acute myeloid leukemia (AML). Journal Of Clinical Oncology 2017, 35: e18507-e18507. DOI: 10.1200/jco.2017.35.15_suppl.e18507.Peer-Reviewed Original ResearchHigh-risk acute myeloid leukemiaAcute myeloid leukemiaCPX-351Median overall survivalOverall survivalComplete remissionFLT3 mutationsDe novo acute myeloid leukemiaNovo acute myeloid leukemiaSerious adverse eventsPhase III studyPhase III trialsOverall study populationOlder ptsNeutrophil recoveryIII trialsAdverse eventsIII studyCell transplantAML patientsSafety profileStudy treatmentSubgroup analysisAML subtypesMyeloid leukemia
2014
First Clinical Results of a Randomized Phase 2 Dose-Response Study of SGI-110, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate (Int) or High Risk (HR) Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML)
Garcia-Manero G, Ritchie E, Walsh K, Savona M, 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, Chung W, Naim S, Taverna P, Hao Y, Azab M, Kantarjian H, Roboz G. First Clinical Results of a Randomized Phase 2 Dose-Response Study of SGI-110, a Novel Subcutaneous (SC) Hypomethylating Agent (HMA), in 102 Patients with Intermediate (Int) or High Risk (HR) Myelodysplastic Syndromes (MDS) or Chronic Myelomonocytic Leukemia (CMML). Blood 2014, 124: 529. DOI: 10.1182/blood.v124.21.529.529.Peer-Reviewed Original ResearchTreatment-naïve patientsMyelodysplastic syndromeChronic myelomonocytic leukemiaTreatment armsCMML patientsHypomethylating agentClinical responseAdverse eventsAstex PharmaceuticalsSGI-110Marrow CRNaïve patientsTransfusion independenceHematological improvementMDS patientsHigh-risk myelodysplastic syndromePhase 1 clinical trialECOG PS 2Treatment-naïve groupRisk myelodysplastic syndromesBaseline patient characteristicsFirst clinical resultsDose-response studySignificant differencesMedian follow
2013
First Clinical Results Of a Randomized Phase 2 Study Of SGI-110, a Novel Subcutaneous (SQ) Hypomethylating Agent (HMA), In Adult Patients With Acute Myeloid Leukemia (AML)
Kantarjian H, Jabbour E, Yee K, Kropf P, O'Connell C, Stock W, Tibes R, Rizzieri D, Walsh K, Griffiths E, Roboz G, Savona M, Ervin T, Podoltsev N, Pemmaraju N, Daver N, Garcia-Manero G, Borthakur G, Wierda W, Ravandi F, Cortes J, Brandwein J, Odenike O, Feldman E, Chung W, Naim S, Choy G, Taverna P, Hao Y, Dimitrov G, Azab M, Issa J. First Clinical Results Of a Randomized Phase 2 Study Of SGI-110, a Novel Subcutaneous (SQ) Hypomethylating Agent (HMA), In Adult Patients With Acute Myeloid Leukemia (AML). Blood 2013, 122: 497. DOI: 10.1182/blood.v122.21.497.497.Peer-Reviewed Original ResearchAcute myeloid leukemiaPhase 2 studyTreatment of AMLRandomized phase 2 studyRefractory acute myeloid leukemiaOverall remission rateAdverse eventsComplete remissionRemission rateAstex PharmaceuticalsHypomethylating agentAML patientsSGI-110Primary endpointDose groupElderly acute myeloid leukemiaLINE-1 demethylationElderly AML patientsCommon adverse eventsComplete remission ratePhase 1 trialStem cell transplantPhase 3 investigationFirst clinical resultsECOG PS