Evaluation of azacitidine in patients with transplant-ineligible myelodysplastic syndromes and acute myeloid leukemia with myelodysplasia-related changes in a Japanese clinical setting.
In conclusion, AZA could produce a hematologic response in ~53% of patients with MDS. Furthermore, IPSS-R may reflect MDS prognosis. Further studies are required to establish the criteria for identifying patients likely to obtain maximum benefit from AZA treatment. PMID: 31966063 [PubMed]
Authors: Gorshein E, Weber UM, Gore S Abstract Introduction: Myelodysplastic Syndrome (MDS) represents a group of cancers characterized by abnormal blood cell formation and maturation, leading to various degrees of cytopenias and potential transformation to acute myeloid leukemia. Deletion of the long arm of chromosome 5 (del(5q)) is the most common clonal chromosomal anomaly in MDS, yet the population in this disease subtype is quite heterogeneous. This manuscript analyzes literature on high-risk MDS with del(5q) abnormalities.Areas covered: The paper will review outcomes with lenalidomide among high-risk MDS pati...
Allogeneic stem cell transplantation (allo-SCT) is the only curative treatment option for many hematological diseases, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Outcomes of allo-SCT have improved with better supportive care, reduced intensity conditioning regimens, and use of alternative stem cell donor sources.  However, relapsed disease after allo-SCT remains a major cause of treatment failure, occurring in approximately 40% of AML cases, most of which occur within the first year.
The WHO 2016 AML-MRC designation applies to AML patients (pts) with a history of myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm, a MDS-related cytogenetic abnormality, or multilineage dysplasia in>50% of ≥2 cell lineages in the absence of NPM1 or biallelic CEBPA mutations. AML-MRC pts typically have a poor prognosis after induction chemotherapy. CPX-351 (Vyxeos®; daunorubicin and cytarabine liposome for injection), a dual-drug liposomal encapsulation of cytarabine [C] and daunorubicin [D] at a sy nergistic ratio, is approved by the FDA and EMA for the treatment of adults with newly diagnosed th...
For patients with refractory or high-risk hematologic malignancies, like acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and acute lymphoblastic leukemia (ALL), allogeneic hematopoietic stem cell transplant (Allo-HSCT) is a potentially curative approach. Morbidities and mortality associated with current conditioning regimens limit the use of this curative procedure. As a result, many eligible patients do not consider transplant and 2/3 of those transplanted are only able to tolerate a reduced intensity conditioning regimen, which is associated with increased relapse rates (Scott, J Clin Onc 2017).
Relapsed acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or myeloproliferative neoplasm (MPN) following an allogeneic stem cell transplant has a bleak prognosis with no standard for treatment identified. Current literature suggests combining hypomethylating agents (HMA) with a donor lymphocyte infusion (DLI) as salvage therapy to reduce disease burden and induce a graft versus leukemia (GVL) effect. At our institution, this salvage therapy option is being used for this population, peaking interest in our experience to date.
Therapy-related acute myeloid leukemia and myelodysplastic syndrome (t-AML/MDS) occur after hematopoietic cell transplantation (HCT). The incidence, risk factors and outcomes of t-AML/MDS among patients who underwent autologous or allogeneic HCT for a variety of disease have not been examined systemically. To address these questions, we conducted a retrospective cohort study using the Japanese national transplant registry database.
Relapse remains the leading cause of treatment failure for 30 –50% of patients with high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic blood or marrow transplantation (BMT). Safety and relapse risk reduction with an ex vivo-expanded, donor-derived, haploidentical (haplo) natural killer (NK)-cell infusion in conjun ction with haploBMT were reported by investigators at MD Anderson Cancer Center in a phase I study (Ciurea, Blood 2017). These data support investigation of CSTD002, haplo NK cells expanded ex vivo using plasma membrane nanoparticles bearing membrane-bound IL-21 and 4-1BBL.
Genetic abnormalities can predispose patients to develop acute myeloid leukemia (AML), aplastic anemia (AA) and myelodysplastic syndrome (MDS). These mutations are more often seen in pediatric patients so genetic screening is more routinely performed in that population. Recent algorithms propose screening in adolescent and young adults (AYAs) with AML/AA/MDS. However, this is not routine practice in community hospitals where most AYAs are treated. Discovery of familial genetic abnormalities can impact donor choice in patients undergoing allogeneic stem cell transplant and treatment/screening in family members.
Patients (pts) with newly diagnosed sAML may have previously received HMA therapy for an antecedent hematologic malignancy (eg, myelodysplastic syndrome [MDS]). Outcomes for pts with MDS who progress following HMA therapy are typically poor, with remission rates
Hematopoietic stem cell transplantation (HSCT) is often indicated and formal transplant referral guidelines exist for pts with AML and HR MDS. However, the proportion of transplant-eligible pts not referred is largely unknown. We therefore assessed HSCT referral patterns, including potential barriers to referral, in pts with ND MDS and AML enrolled in the Connect ® MDS/AML Disease Registry (NCT01688011), a large, US, multicenter, prospective observational cohort study of pts with ND AML (aged ≥ 55 years) or MDS (aged ≥ 18 years).