Myelodysplastic Syndromes (MDS)
Dongyou Liu in Tumors and Cancers, 2017
This chapter presents a state of the art summary of myelodysplastic syndromes (MDS) to its definition, biology, epidemiology, disease mechanisms, clinical signs, diagnosis, treatment and prognosis. MDS are further divided into MDS with dysplasia, MDS with ring sideroblasts, MDS with excess blasts, MDS with isolated del, MDS unclassifiable, provisional entity—refractory cytopenia of childhood, and myeloid neoplasms with germline predisposition. Approximately 90% of MDS cases arise de novo without identifiable cause, and complex epigenetic, genetic, and immunologic mechanisms appear to underline MDS pathogenesis. MDS is a relatively common hematological malignancy, with an annual incidence of 5 per 100,000 in the general population, 30 per 100,000 in the age group of greater than 60 years, and 50 per 100,000 in the age group of greater than 80 years. Treatment options for MDS consist of hypomethylating agents, lenalidomide, and allogeneic hematopoietic stem cell transplantation.
Acute Myeloid Leukaemia
Tariq I. Mughal in Precision Haematological Cancer Medicine, 2018
This chapter briefly reviews the complex genetic landscape and treatment of patients with acute myeloid leukaemia (AML). Therapy-related AML is recognized as a subgroup of the World Health Organization 2016 classification of AML, classified as a therapy-related myeloid neoplasm, which also includes therapy-related myelodysplastic syndromes. The conventional treatment plan for most patients consists of remission induction followed by a post-remission, or 'consolidation' therapy. There can be no doubt of the additional prognostic impact of the refinements to the complex genetic and epigenetic landscape of AML. This is additive to the initial framework for risk stratification of AML, based on clinical features, biologics and cytogenetics. Allogeneic stem cell transplantation from an HLA-matched sibling donor has been standard treatment for patients with poor-risk AML in first complete remission since the 1980s. A substantial amount of high-quality genetic data has been garnered on patients with AML improving our understanding of the biology and informed a better genomic classification and prognostication methods.
Chronic myeloid luekaemia
John M Barrett, Jennifer G Treleaven in Clinical Practice of Stem-Cell Transplantation, 1999
The term ‘chronic myeloid leukaemia’ (CML) describes a specific form of leukaemia characterized by progressive splenomegaly, leukocytosis, anaemia, marrow hypercellularity and the finding in most cases of the Philadelphia (Ph) chromosome (Figure 4.1) in all dividing cells of the myeloid series and in some B-lymphocytes. Between 5 and 10% of patients with CML have variant features, such as typical CML without the Ph chromosome, haematologically atypical CML (also Ph-negative), juvenile chronic myeloid leukaemia and various forms of the myelodysplastic syndrome. It is likely that Ph-positive CML together with the rare Ph-negative BCR/ABL-positive CML patients are a relatively homogeneous group, and indications for and results of bonemarrow transplant (BMT) in this group cannot necessarily be extrapolated to other patients.
Following in the footsteps of acute myeloid leukemia: are we witnessing the start of a therapeutic revolution for higher-risk myelodysplastic syndromes?
Published in Leukemia & Lymphoma, 2020
Jan Philipp Bewersdorf, Amer M. Zeidan
For most patients with higher-risk myelodysplastic syndromes (HR-MDS) the hypomethylating agents (HMA) azacitidine and decitabine remain the mainstay of therapy. However, the prognosis mostly remains poor and aside from allogeneic hematopoietic stem cell transplantation no curative treatment options exist. Unlike acute myeloid leukemia, which has seen a dramatic expansion of available therapies recently, no new agents have been approved for MDS in the United States since 2006. However, various novel HMAs, HMA in combination with venetoclax, immune checkpoint inhibitors, and targeted therapies for genetically defined patient subgroups such as APR-246 or IDH inhibitors, have shown promising results in early stages of clinical testing. Furthermore, the wider availability of genetic testing is going to allow for a more individualized treatment of MDS patients. Herein, we review the current treatment approach for HR-MDS and discuss recent therapeutic advances and the implications of genetic testing on management of HR-MDS.
Treatment sequence of lenalidomide and hypomethylating agents and the impact on clinical outcomes for patients with myelodysplastic syndromes
Published in Leukemia & Lymphoma, 2019
Amer M. Zeidan, Andrew J. Klink, Michael McGuire, Bruce Feinberg
Lenalidomide and hypomethylating agents (HMAs) azacitidine and decitabine are approved for treating myelodysplastic syndromes (MDS), but optimal sequencing is unclear. Adults with MDS were identified from a US payer claims database (Inovalon MORE2 Registry) to compare outcomes with lenalidomide followed by HMA (LEN-HMA) or HMA followed by lenalidomide (HMA-LEN). There were 96 patients who received LEN-HMA and 89 who received HMA-LEN. LEN-HMA-treated patients had a longer time to second treatment discontinuation (29.0 vs. 19.0 months, p=.009; adjusted hazard ratio [HR] 0.52, 95% confidence interval [CI] 0.29–0.91, p=.023). LEN-HMA-treated patients had a longer median time to insurance disenrollment (22.4 vs. 16.1 months, p
Rheumatic manifestations and an epipharyngeal mass accompanied by myelodysplastic syndrome
Published in Modern Rheumatology, 2015
Mariko Inoue, Toshikazu Kano, Takashi Ozaki, Yuko Takahashi, Hiroyuki Yamashita, Hiroshi Kaneko, Akio Mimori
We herein report two cases of myelodysplastic syndrome with rheumatic manifestations. (Case 1) A 70-year-old man presented with fever, arthritis and bone pain and developed cranial nerve palsy caused by an epipharyngeal mass. Steroid therapy led to a prolonged remission of the febrile condition and mass lesion. (Case 2) An 82-year-old male was treated for intractable polyarthritis and fever with steroid therapy, and serious side effects resulted in lethal pneumonia. We herein describe the entire course of steroid therapy in these two cases. Various rheumatic manifestations in myelodyaplastic syndrome often require empirical steroid therapy. It was effective for the soft tissue mass in Case 1, in which indolent lymphoma could not be denied, and was only partially effective for Case 2 of the febrile and putatively benign conditions, suggesting heterogeneous nature of rheumatic complications in myelodysplastic syndrome.
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