0000000000745763

AUTHOR

Argiris Symeonidis

showing 2 related works from this author

Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease

2018

Gaucher Disease type 1 (GD1) is a lysosomal disorder that affects many systems. Therapy improves the principal manifestations of the condition and, as a consequence, many patients show a modified phenotype which reflects manifestations of their disease that are refractory to treatment. More generally, it is increasingly recognised that information as to how a patient feels and functions [obtained by patient- reported outcome measurements (PROMs)] is critical to any comprehensive evaluation of treatment. A new set of management goals for GD1 in which both trends are reflected is needed. To this end, a modified Delphi procedure among 25 experts was performed. Based on a literature review and …

0301 basic medicinemedicine.medical_specialtyConsensusDelphi study; Gaucher disease; Management goals; PROMs; TherapyDelphi methodEarly detectionGaucher diseaseDiseasePROMs03 medical and health sciences0302 clinical medicineQuality of life (healthcare)HumansMedicineIntensive care medicineSet (psychology)Molecular BiologyComputingMilieux_MISCELLANEOUSManagement goalsbusiness.industryDisease ManagementType 1 Gaucher DiseaseExpert consensus[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/HematologyCell BiologyHematologySocial engagementDelphi study3. Good healthEurope030104 developmental biology030220 oncology & carcinogenesisQuality of LifePhysical therapyMolecular MedicineTherapybusinessBlood Cells, Molecules, and Diseases
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Pevonedistat (PEV) + Azacitidine (AZA) Versus AZA Alone As First-Line Treatment for Patients with Higher-Risk Myelodysplastic Syndromes (MDS)/Chronic…

2021

Abstract Background: Older patients with higher-risk MDS/CMML or AML with 20-30% marrow blasts typically receive single-agent hypomethylating agent (HMA) therapy. Median response duration and survival for these patients is poor, and many patients with MDS transform to secondary AML, which is associated with a particularly dismal prognosis. Novel HMA-based combination therapies that improve survival, delay transformation to AML, and increase depth and duration of response vs single-agent HMA therapy without additional toxicity or myelosuppression are needed. In a randomized, proof-of-concept phase 2 study PEV - a first-in-class, selective inhibitor of NEDD8-activating enzyme - in combination…

Oncologymedicine.medical_specialtybusiness.industryMyelodysplastic syndromesImmunologyAzacitidineChronic myelomonocytic leukemiaMyeloid leukemiaCell BiologyHematologymedicine.diseaseBiochemistryFirst line treatmentInternal medicinemedicinebusinessmedicine.drugBlood
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