2023
Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial
Zeidan A, Ando K, Rauzy O, Turgut M, Wang M, Cairoli R, Hou H, Kwong Y, Arnan M, Meers S, Pullarkat V, Santini V, Malek K, Kiertsman F, Niolat J, Ramos P, Menssen H, Fenaux P, Miyazaki Y, Platzbecker U. Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial. The Lancet Haematology 2023, 11: e38-e50. PMID: 38065203, DOI: 10.1016/s2352-3026(23)00333-2.Peer-Reviewed Original ResearchConceptsHigh-risk myelodysplastic syndromeProgression-free survivalComplete response rateMyelodysplastic syndromePlacebo groupPrimary endpointUntreated patientsAdverse eventsComplete responseResponse rateImmune-mediated adverse eventsMedian progression-free survivalRandomised phase 3 trialT-cell immunoglobulin domainFinal data cutoffTreatment-related deathsCommon adverse eventsFull analysis setMucin domain 3Phase 2 studyPhase 2 trialPhase 3 trialLeukaemic stem cellsFebrile neutropeniaData cutoff
2016
Phase 1 Study of Pomalidomide Given at the Time of Early Lymphocyte Recovery after Induction Timed Sequential Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndrome (HR-MDS)
Zeidner J, Montiel-Esparza R, Knaus H, Berglund S, Zeidan A, McCurdy S, Prince G, Gondek L, Ghiaur G, Showel M, DeZern A, Pratz K, Smith B, Levis M, Foster M, Jamieson K, Van Deventer H, Streicher H, Karp J, Luznik L, Gojo I. Phase 1 Study of Pomalidomide Given at the Time of Early Lymphocyte Recovery after Induction Timed Sequential Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndrome (HR-MDS). Blood 2016, 128: 2820. DOI: 10.1182/blood.v128.22.2820.2820.Peer-Reviewed Original ResearchEarly lymphocyte recoveryAcute myeloid leukemiaWhite blood cell countComplete remissionAdverse eventsT cellsLymphocyte recoveryDay 1Adverse-risk acute myeloid leukaemiaDose levelsALT increasePhase 1 dose-escalation studyAST/ALT increaseContinuous infusion days 1High-risk myelodysplastic syndromeCore-binding factor acute myeloid leukemiaTotal white blood cell countFlow cytometryFactor acute myeloid leukemiaFull recoveryGrade 3 hypoxiaIncomplete platelet recoveryInfusion days 1Common adverse eventsConventional chemotherapy regimens