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
2013
Management of High-Risk Myelodysplastic Syndrome
Zeidan A, Gore S. Management of High-Risk Myelodysplastic Syndrome. Hematologic Malignancies 2013, 189-210. DOI: 10.1007/978-3-642-36229-3_12.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeHR-MDSDNMTi therapyDNA methyltransferase inhibitorEmergence of resistanceTherapeutic optionsMyelodysplastic syndromeNovel agentsAllogeneic hematopoietic stem cell transplantationCare first-line therapyHematopoietic stem cell transplantationDuration of therapyFirst-line therapyComplex pathogenetic mechanismsStem cell transplantationLimited therapeutic optionsGood responseMechanism of actionAzacitidine therapyStable diseaseHematologic improvementIntensive chemotherapyMaintenance therapyUntreated patientsMedian survival