How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms.
Carmelo GurnariZhuoer XieAmer M ZeidanPublished in: Clinical hematology international (2022)
Myelodysplastic neoplasms, formerly known as myelodysplastic syndromes (MDS), represent a group of clonal disorders characterized by a high degree of clinical and molecular heterogeneity, and an invariable tendency to progress to acute myeloid leukemia. MDS typically present in the elderly with cytopenias of different degrees and bone marrow dysplasia, the hallmarks of the disease. Allogeneic hematopoietic stem cell transplant is the sole curative approach to date. Nonetheless, given the disease's demographics, only a minority of patients can benefit from this procedure. Currently used prognostic schemes such as the Revised International Prognostic Scoring System (R-IPSS), and most recently the molecular IPSS (IPSS-M), guide clinical management by dividing MDS into two big categories: lower- and higher-risk cases, based on a cut-off score of 3.5. The main clinical problem of the lower-risk group is represented by the management of cytopenias, whereas the prevention of secondary leukemia progression is the goal for the latter. Herein, we discuss the non-transplant treatment of MDS, focusing on current practice and available therapeutic options, while also presenting new investigational agents potentially entering the MDS therapeutic arsenal in the near future.
Keyphrases
- bone marrow
- acute myeloid leukemia
- hematopoietic stem cell
- end stage renal disease
- mesenchymal stem cells
- allogeneic hematopoietic stem cell transplantation
- prognostic factors
- newly diagnosed
- ejection fraction
- stem cell transplantation
- primary care
- chronic kidney disease
- healthcare
- peritoneal dialysis
- minimally invasive
- randomized controlled trial
- middle aged
- clinical trial
- rectal cancer
- single cell
- case report
- low dose
- current status
- high dose
- patient reported