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Validation of a Predictive Model for Survival in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome Receiving Hematopoietic Stem Cell Transplantation

Background: Outcomes of allogeneic stem cell transplantation (AHSCT) vary based on both disease and patient characteristics. Our group developed a model to predict survival in patients with AML/MDS using 3 factors, 2 disease-related (cytogenetics, disease status at transplant) and 1 patient-related (HCT-CI) (Bachegowda et  al.Blood.2017). This model effectively stratified patients into 3 risk groups with very different survival. Here we aim to validate this model in a large cohort of AML/MDS patients receiving AHSCT with different donors.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research

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We report here indiscriminate killing of CD123+ normal and acute myeloid leukemia / myelodysplastic syndrome cells by SL-401, a diphtheria toxin interleukin-3 fusion protein. SL-401 induced cytotoxicity of CD123+ primary cells/blasts from acute myeloid leukemia and myelodysplastic syndrome patients but not CD123- lymphoid cells. Importantly, SL-401 was highly active even in cells expressing low levels of CD123, with minimal effect on modulation of the CD123 target in acute myeloid leukemia. SL-401 significantly prolonged survival of leukemic mice in acute myeloid leukemia patient-derived xenograft mouse models. In addition...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
This multicenter study evaluated a treosulfan-based regimen in children and young adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplant (HCT). Forty patients with median age 11 years (1-19) underwent allogeneic HCT for AML in first (n=18), second (n=11), third or greater remission (n=3); or MDS (n=8) using bone marrow (n=25), peripheral blood stem cells (n=5) or cord blood (n=9). The regimen consisted of body surface area (BSA)-based treosulfan 10 g/m2/day (BSA ≤ 0.5 m2), 12 g/m2/day (BSA> 0.5 – 1.0 m2), or 14 g/m2/day (BSA> 1.0 m2) on ...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
AbstractPrimary graft failure can be a cause of early morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT), as it leads to a high risk of severe infections and bleeding. Splenomegaly is associated with primary graft failure in patients of myelofibrosis, but the association between splenomegaly and outcomes after HSCT in patients with myeloid malignancies has not been previously evaluated. The aim of this study was to investigate the effect of spleen volume on engraftment kinetics in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). We enrolled 85 patients. The...
Source: Annals of Hematology - Category: Hematology Source Type: research
A 62-year-old man with acute myeloid leukemia, evolved from myelodysplastic syndrome after allogeneic bone marrow transplant, was admitted to the hospital because of worsening fatigue and altered level of consciousness. On arrival at the hospital, the patient was markedly fatigued and had signs of encephalopathy. He reported no fevers, chills, or diaphoresis but noted new urinary incontinence and nausea. His medical history was notable for a matched unrelated donor allogeneic hematopoietic stem cell transplant (HCT), performed 35 days previously, as well as hypertension and peripheral vascular disease.
Source: Mayo Clinic Proceedings - Category: Internal Medicine Authors: Tags: Residents' Clinic Source Type: research
Pediatric myelodysplastic syndromes (MDSs) are a heterogeneous group of clonal disorders with an annual incidence of 1 to 4 cases per million, accounting for less than 5% of childhood hematologic malignancies. MDSs in children often occur in the context of inherited bone marrow failure syndromes, which represent a peculiarity of myelodysplasia diagnosed in pediatric patients. Moreover, germ line syndromes predisposing individuals to develop MDS or acute myeloid leukemia have recently been identified, such as those caused by mutations in GATA2, ETV6, SRP72, and SAMD9/SAMD9-L. Refractory cytopenia of childhood (RCC) is the m...
Source: Blood - Category: Hematology Authors: Tags: Pediatric Hematology, Transplantation, How I Treat, Free Research Articles, Myeloid Neoplasia Source Type: research
Contributors : Hiroyoshi Kunimoto ; Cem Meydan ; Francine E Garrett-Bakelman ; Caroline Sheridan ; Tak Lee ; Yaseswini Neelamraju ; Ari Melnick ; Ross L LevineSeries Type : Methylation profiling by high throughput sequencingOrganism : Mus musculusRecent studies using next-generation sequencing technology have uncovered mutational landscapes of various myeloid malignancies (Cancer Genome Atlas Research Network, 2013; Yoshida et al., 2011). These genetic data revealed novel classes of mutations that commonly occur in patients with myeloid malignancies, including epigenetic regulators and spliceosomal genes. In addition, co-o...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Methylation profiling by high throughput sequencing Mus musculus Source Type: research
Contributors : Hiroyoshi Kunimoto ; Cem Meydan ; Francine E Garrett-Bakelman ; Caroline Sheridan ; Tak Lee ; Yaseswini Neelamraju ; Ari Melnick ; Ross L LevineSeries Type : Expression profiling by high throughput sequencingOrganism : Mus musculusRecent studies using next-generation sequencing technology have uncovered mutational landscapes of various myeloid malignancies (Cancer Genome Atlas Research Network, 2013; Yoshida et al., 2011). These genetic data revealed novel classes of mutations that commonly occur in patients with myeloid malignancies, including epigenetic regulators and spliceosomal genes. In addition, co-oc...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Mus musculus Source Type: research
Abstract Patient-derived xenotransplantation models of human myeloid diseases including acute myeloid leukemia, myelodysplastic syndromes and myeloproliferative neoplasms are essential for studying the disease's biology in pre-clinical studies. However, few studies have used these models for comparison purposes. Previous work has shown that acute myeloid leukemia blasts respond to human hematopoietic cytokines whereas myelodysplastic syndrome cells do not. We compared the engraftment of acute myeloid leukemia cells and myelodyplastic syndrome cells in NSG mice to NSG-S mice, which have transgene expression of huma...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
ConclusionDe novo myeloid sarcoma mostly presented isolated. Lesions were often localized at skin and lymph nodes. Genetic aberrations frequently involved coreā€binding factor rearrangements in de novo cases and a complex karyotype in secondary cases.This article is protected by copyright. All rights reserved.
Source: European Journal of Haematology - Category: Hematology Authors: Tags: Original Article Source Type: research
DISCUSSION: Treatment with chemotherapy plus G-CSF appears to provide better survival and treatment responses compared with chemotherapy alone, particularly for patients with previously untreated AML. ABBREVIATIONS: AML, acute myeloid leukemia; CI, confidence interval; CR, complete remission; DFS, disease-free survival; G-CSF, granulocyte colony-stimulating factor; GM-CSF, granulocyte macrophage colony-stimulating factor; HR, hazard ratio; MDS, myelodysplastic syndrome; OR, odds ratio; OS, overall survival; RCTs, randomized control trials; RR, relative risk. PMID: 29516766 [PubMed - as supplied by publisher]
Source: Hematology - Category: Hematology Tags: Hematology Source Type: research
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